Compounds > eliglustat
Page last updated: 2024-11-13

eliglustat

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Description

eliglustat: a potent inhibitor of glucosylceramide synthase [Medical Subject Headings (MeSH), National Library of Medicine, extracted Dec-2023]

eliglustat : A carboxamide obtained by formal condensation of the carboxy group of octanoic acid with the primary amino group of (1R,2R)-2-amino-1-(2,3-dihydro-1,4-benzodioxin-6-yl)-3-(pyrrolidin-1-yl)propan-1-ol. A ceramide glucosyltransferase inhibitor used (as its tartrate salt) for treatment of Gaucher's disease. [Chemical Entities of Biological Interest (ChEBI), Hastings J, Owen G, Dekker A, Ennis M, Kale N, Muthukrishnan V, Turner S, Swainston N, Mendes P, Steinbeck C. (2016). ChEBI in 2016: Improved services and an expanding collection of metabolites. Nucleic Acids Res]

Cross-References

ID SourceID
PubMed CID23652731
CHEMBL ID2110588
CHEBI ID82752
SCHEMBL ID421755
MeSH IDM0592238

Synonyms (56)

Synonym
genz-112638
eliglustat (usan/inn)
491833-29-5
D09893
CHEMBL2110588
chebi:82752 ,
genz-99067
dr40j4wa67 ,
unii-dr40j4wa67
n-((1r,2r)-2-(2,3-dihydro-1,4-benzodioxin-6-yl)-2-hydroxy-1-(pyrrolidin-1-ylmethyl)ethyl)octanamide
genz 99067
eliglustat
eliglustat [usan:inn]
octanamide, n-((1r,2r)-2-(2,3-dihydro-1,4-benzodioxin-6-yl)-2-hydroxy-1-(1-pyrrolidinylmethyl)ethyl)-
S7852
eliglustat [inn]
eliglustat [who-dd]
eliglustat [mi]
eliglustat [vandf]
n-((1r,2r)-2-(2,3-dihydro-1,4-benzodioxin-6-yl)-2-hydroxy-1-(pyrrolidin-1- ylmethyl)ethyl)octanamide
octanamide, n-((1r,2r)-2-(2,3-dihydro-1,4-benzodioxin-6-yl)-2-hydroxy-1-(1- pyrrolidinylmethyl)ethyl)-
eliglustat [usan]
SCHEMBL421755
AKOS022185521
gtpl7536
n-[(1r,2r)-1-(2,3-dihydro-1,4-benzodioxin-7-yl)-1-hydroxy-3-pyrrolidin-1-ylpropan-2-yl]octanamide
n-[(1r,2r)-1-(2,3-dihydro-1,4-benzodioxin-6-yl)-1-hydroxy-3-(pyrrolidin-1-yl)propan-2-yl]octanamide
mfcd19443735
AC-35323
DB09039
n-[(1r,2r)-1-(2,3-dihydro-1,4-benzodioxin-6-yl)-1-hydroxy-3-(1-pyrrolidinyl)-2-propanyl]octanamide
HY-14885
CS-5424
n-((1r,2r)-1-(2,3-dihydrobenzo[b][1,4]dioxin-6-yl)-1-hydroxy-3-(pyrrolidin-1-yl)propan-2-yl)octanamide
n-[(1r,2r)-1-(2,3-dihydro-1,4-benzodioxin-6-yl)-1-hydroxy-3-(1- pyrrolidinyl)-2-propanyl]octanamide;eliglustat tartrate
eliglustat(genz-99067)
n-[(1r,2r)-1-(2,3-dihydro-1,4-benzodioxin-6-yl)-1-hydroxy-3-pyrrolidin-1-ylpropan-2-yl]octanamide
eliglustat; genz 99067
EX-A2301
BS-17368
Q21011224
1000191-50-3
DTXSID20964175
n-[(1r,2r)-2-(2,3-dihydro-1,4-benzodioxin-6-yl)-2-hydroxy-1-(1-pyrrolidinylmethyl)ethyl]octanamide
CCG-101413
NCGC00509905-01
F53213
eliglustat free base
491833-29-5 (free base)
GLXC-90436
octanamide, n-[(1r,2r)-2-(2,3-dihydro-1,4-benzodioxin-6-yl)-2-hydroxy-1-(1-pyrrolidinylmethyl)ethyl]-; n-[(1r,2r)-2-(2,3-dihydro-1,4-benzodioxin-6-yl)-2-hydroxy-1-(1-pyrrolidinylmethyl)ethyl]octanamide; cerdelga; eliglustat; genz 99067
EN300-259910
n-((1r,2r)-1-(2,3-dihydro-1,4-benzodioxin-6-yl)-1-hydroxy-3-(pyrrolidin-1-yl)propan-2-yl)octanamide
a16ax10
eliglustatum
n-((1r,2r)-1-(2,3-dihydro-1,4-benzodioxin-6-yl)-1-hydroxy-3-(1-pyrrolidinyl)-2-propanyl)octanamide

Research Excerpts

Overview

Eliglustat is an oral substrate reduction therapy drug and has been approved as a first-line treatment for adults with Gaucher disease type 1. Eliglustat (EG) is a specific and potent inhibitor of glucosylceramide synthase, first step of glycosphingolipid biosynthesis, actually used for the treatment of Gaucher's disease.

ExcerptReferenceRelevance
"Eliglustat is a first-line oral therapy for adult patients with GD1."( Patient reported outcomes of patients with Gaucher disease type 1 treated with eliglustat in real-world settings: The ELIPRO study.
Belmatoug, N; Berger, MG; Cador-Rousseau, B; Camou, F; Coutinho, A; Gaches, F; Lagadec, A, 2023)		1.86
"Eliglustat is an oral substrate reduction therapy drug and has been approved as a first-line treatment for adults with Gaucher disease type 1 (GD 1). "( Liquid chromatography-tandem mass spectrometric method for the quantification of eliglustat in rat plasma and the application in a pre-clinical study.
Chen, J; Chen, X; Shao, Y; Ye, X; Zhu, H, 2020)		2.23
"Eliglustat is a first-line oral therapy for adults with Gaucher disease type 1 (GD1) who have extensive (EM), intermediate (IM), or poor (PM) CYP2D6 metabolizer phenotypes. "( Impact of hepatic and renal impairment on the pharmacokinetics and tolerability of eliglustat therapy for Gaucher disease type 1.
Chen, J; Gaemers, SJM; Hou, AW; Kanamaluru, V; Li, J; Peterschmitt, MJ; Rudin, D; Turpault, S; Xue, Y, 2020)		2.23
"Eliglustat is an oral substrate reduction therapy indicated for patients with Gaucher disease type 1. "( Effect of eliglustat on the pharmacokinetics of digoxin, metoprolol, and oral contraceptives and absorption of eliglustat when coadministered with acid-reducing agents.
Ibrahim, J; Peterschmitt, MJ; Puga, AC; Ross, L; Thibault, N; Turpault, S; Vu, L; Xue, Y, 2020)		2.4
"Eliglustat is a ceramide glucosyltransferase inhibitor work as first line oral therapy for adults with Gaucher disease type 1 (a rare disease) at present. "( A novel method for preparing Eligulstat through chiral resolution.
Chu, W; Du, J; Feng, W; Ma, C; Zhang, M, 2020)		2
"Eliglustat is a first-line oral treatment for adults with Gaucher disease type 1 who have cytochrome P450 (CYP) 2D6 extensive, intermediate, or poor metabolizer phenotypes. "( How a concentration-effect analysis of data from the eliglustat thorough electrocardiographic study was used to support dosing recommendations.
Cox, GF; Maison-Blanche, P; Msihid, J; Ortemann-Renon, C; Peterschmitt, MJ; Puga, AC; Ross, L; Ruskin, JN, )		1.82
"Eliglustat (EG) is a specific and potent inhibitor of glucosylceramide synthase, first step of glycosphingolipid biosynthesis, actually used for the treatment of Gaucher's disease."( Eliglustat prevents Shiga toxin 2 cytotoxic effects in human renal tubular epithelial cells.
Amaral, MM; Balestracci, A; Fischer Sigel, LK; Ibarra, C; Sánchez, DS; Silberstein, C, 2022)		2.89
"Eliglustat is a first-line oral therapy for adults with Gaucher disease type 1 (GD1) with compatible CYP2D6-metabolizer phenotypes (>90% of patients). "( Once- versus twice-daily dosing of eliglustat in adults with Gaucher disease type 1: The Phase 3, randomized, double-blind EDGE trial.
Charrow, J; Fraga, C; Gaemers, SJM; Gu, X; Ida, H; Jouvin, MH; Li, J; Longo, N; Lukina, E; Nonino, A; Peterschmitt, MJ; Wu, Y; Xue, Y, 2018)		2.2
"Eliglustat is a first-line oral therapy for adults with Gaucher disease type 1 (GD1) and poor, intermediate or extensive CYP2D6-metabolizer phenotypes (>90% of patients). "( Outcomes after 8 years of eliglustat therapy for Gaucher disease type 1: Final results from the Phase 2 trial.
Avila Arreguin, E; Dragosky, M; Foster, MC; Gaemers, SJM; Lau, H; Lukina, E; Peterschmitt, MJ; Rosenbaum, H; Watman, N; Zimran, A, 2019)		2.26
"Eliglustat is a first-line oral treatment for adults with Gaucher disease type 1 who have an extensive, intermediate or poor CYP2D6 metabolizer phenotype (> 90% of patients). "( Long-term adverse event profile from four completed trials of oral eliglustat in adults with Gaucher disease type 1.
Foster, MC; Freisens, S; Gaemers, SJM; Lewis, G; Peterschmitt, MJ; Underhill, LH, 2019)		2.19
"Eliglustat is an investigational oral substrate reduction therapy for Gaucher disease type 1 (GD1). "( Skeletal improvement in patients with Gaucher disease type 1: a phase 2 trial of oral eliglustat.
Aguzzi, R; Arreguin, EA; Dragosky, M; Kamath, RS; Lukina, E; Norfleet, AM; Pastores, GM; Peterschmitt, MJ; Puga, AC; Rosenbaum, H; Rosenthal, DI; Watman, N; Zimran, A, 2014)		2.07
"Eliglustat is an investigational, oral substrate reduction therapy for Gaucher disease type 1 (GD1). "( Eliglustat, an investigational oral therapy for Gaucher disease type 1: Phase 2 trial results after 4 years of treatment.
Angell, J; Arreguin, EA; Dragosky, M; Lukina, E; Pastores, GM; Peterschmitt, JM; Puga, AC; Rosenbaum, H; Ross, L; Watman, N; Zimran, A, 2014)		3.29
"Eliglustat is a novel glucosylceramide synthase inhibitor for long-term oral treatment of type 1 Gaucher disease (GD1), an inherited metabolic disorder. "( Carcinogenicity testing of eliglustat in mice and rats.
Chevalier, G; Dagher, R; Forster, R; Gervais, F; Thirion-Delalande, C; Watzinger, M, 2015)		2.16
"Eliglustat is a potent, selective inhibitor of glucosylceramide synthase, the rate-limiting enzyme in the synthesis of certain glycosphingolipids, and thus, reduces the rate of biosynthesis of glycosphingolipids to counteract the catabolic defect (i.e."( Eliglustat: A Review in Gaucher Disease Type 1.
Scott, LJ, 2015)		2.58
"Eliglustat is an oral substrate reduction therapy approved in the European Union and the United States as a first-line treatment for adults with type 1 Gaucher disease who have compatible CYP2D6 metabolism phenotypes."( Management and monitoring recommendations for the use of eliglustat in adults with type 1 Gaucher disease in Europe.
Belmatoug, N; Cox, TM; Di Rocco, M; Fraga, C; Giraldo, P; Hughes, D; Lukina, E; Maison-Blanche, P; Merkel, M; Niederau, C; Plӧckinger, U; Richter, J; Stulnig, TM; Vom Dahl, S, 2017)		1.42
"Eliglustat is a selective, potent inhibitor of glucosylceramide synthase, the enzyme responsible for biosynthesis of glucosylceramides which accumulate in Gaucher disease. "( Management and monitoring recommendations for the use of eliglustat in adults with type 1 Gaucher disease in Europe.
Belmatoug, N; Cox, TM; Di Rocco, M; Fraga, C; Giraldo, P; Hughes, D; Lukina, E; Maison-Blanche, P; Merkel, M; Niederau, C; Plӧckinger, U; Richter, J; Stulnig, TM; Vom Dahl, S, 2017)		2.14
"Eliglustat tartrate is an investigational oral substrate reduction therapy for Gaucher disease type 1 that is pharmacologically distinct from intravenous enzyme replacement therapy. "( Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study.
Arreguin, EA; Dragosky, M; Iastrebner, M; Kamath, RS; Kaper, M; Lukina, E; Pastores, GM; Peterschmitt, MJ; Phillips, M; Puga, AC; Rosenbaum, H; Rosenthal, DI; Singh, T; Watman, N, 2010)		2.02

Effects

ExcerptReferenceRelevance
"Eliglustat treatment has been started in Austria in patients recruited to a clinical trial, followed by its long-term extension and prescription treatment overall covering up to 10 years."( Long-term eliglustat treatment of Gaucher patients over up to 10 years in Vienna.
Stulnig, TM, 2022)		1.85

Treatment

Eliglustat treatment duration ranged from 3.3 to 6.5 years. Eliglustat treatment compliance was good and sustained, along with overall health state, fatigue and pain levels.

ExcerptReferenceRelevance
"Eliglustat treatment has been started in Austria in patients recruited to a clinical trial, followed by its long-term extension and prescription treatment overall covering up to 10 years."( Long-term eliglustat treatment of Gaucher patients over up to 10 years in Vienna.
Stulnig, TM, 2022)		1.85
"Mean eliglustat treatment duration ranged from 3.3 to 6.5 years."( Long-term effects of eliglustat on skeletal manifestations in clinical trials of patients with Gaucher disease type 1.
Charrow, J; Cox, TM; Foster, MC; Lukina, E; Mistry, PK; Peterschmitt, MJ, 2023)		1.68
"Eliglustat treatment compliance was good and sustained, along with overall health state, fatigue and pain levels, which was consistent with overall patients' clinical status. "( Patient reported outcomes of patients with Gaucher disease type 1 treated with eliglustat in real-world settings: The ELIPRO study.
Belmatoug, N; Berger, MG; Cador-Rousseau, B; Camou, F; Coutinho, A; Gaches, F; Lagadec, A, 2023)		2.58
"Eliglustat treatment was also associated with improvement in bone marrow burden score, bone mineral density, and established biomarkers of Gaucher disease, including reduction of the bioactive lipid, glucosylsphingosine."( Outcomes after 18 months of eliglustat therapy in treatment-naïve adults with Gaucher disease type 1: The phase 3 ENGAGE trial.
Assouline, S; Balwani, M; Baris, H; Ben Turkia, H; Danda, S; Gaemers, SJM; Ghosn, M; Hadjiev, E; Lukina, E; Mehta, A; Mistry, PK; Ortega, A; Packman, S; Pastores, G; Petakov, M; Peterschmitt, MJ; Shankar, SP; Tayag, R, 2017)		1.47

Toxicity

This pooled analysis of treatment-emergent adverse events combines data from four completed eliglustat clinical trials involving 393 Gaucher disease type 1 patients. All but one of these events were mild or moderate.

ExcerptReferenceRelevance
" No serious adverse events occurred."( Safety, tolerability, and pharmacokinetics of eliglustat tartrate (Genz-112638) after single doses, multiple doses, and food in healthy volunteers.
Blankstein, L; Bonate, PL; Burke, A; Harris, JA; Kramer, WG; Mathews, D; Peterschmitt, MJ; Puga, AC; Smith, SE, 2011)		0.63
" Clinicians have requested more information about frequency, timing, and duration of adverse events associated with eliglustat."( A pooled analysis of adverse events in 393 adults with Gaucher disease type 1 from four clinical trials of oral eliglustat: Evaluation of frequency, timing, and duration.
Cox, GF; Gaemers, SJM; Ibrahim, J; MacDougall, J; Patel, P; Peterschmitt, MJ; Underhill, LH, 2018)		0.9
" Clinicians and patients want to better understand which adverse events are most commonly associated with eliglustat, as well as their severity, frequency, and duration."( Long-term adverse event profile from four completed trials of oral eliglustat in adults with Gaucher disease type 1.
Foster, MC; Freisens, S; Gaemers, SJM; Lewis, G; Peterschmitt, MJ; Underhill, LH, 2019)		0.96
"This pooled analysis of treatment-emergent adverse events combines data from four completed eliglustat clinical trials involving 393 Gaucher disease type 1 patients."( Long-term adverse event profile from four completed trials of oral eliglustat in adults with Gaucher disease type 1.
Foster, MC; Freisens, S; Gaemers, SJM; Lewis, G; Peterschmitt, MJ; Underhill, LH, 2019)		0.97
"3%) withdrew from their respective trial due to one or more adverse events reported as eliglustat treatment-related; all but one of these events were mild or moderate."( Long-term adverse event profile from four completed trials of oral eliglustat in adults with Gaucher disease type 1.
Foster, MC; Freisens, S; Gaemers, SJM; Lewis, G; Peterschmitt, MJ; Underhill, LH, 2019)		0.97
"This final pooled analysis of treatment-emergent adverse events reinforces the favorable safety profile of eliglustat."( Long-term adverse event profile from four completed trials of oral eliglustat in adults with Gaucher disease type 1.
Foster, MC; Freisens, S; Gaemers, SJM; Lewis, G; Peterschmitt, MJ; Underhill, LH, 2019)		0.96
"There were no drug-related serious adverse effects and no drug-related cardiac events."( Effectiveness and Safety of Eliglustat Treatment in Gaucher Disease: Real-life Unicentric Experience.
Calafiore, V; Di Raimondo, F; Duminuco, A; Fazio, M; Giuffrida, G; Grasso, S; Gullo, L; Markovic, U; Riccobene, C, 2023)		1.2

Compound-Compound Interactions

ExcerptReferenceRelevance
" Based on in vitro data, clinical trials were conducted to assess the potential for drug-drug interactions between eliglustat and digoxin (P-glycoprotein substrate), metoprolol (sensitive CYP2D6 substrate), a combined oral contraceptive (CYP3A substrate), and acid-reducing agents."( Effect of eliglustat on the pharmacokinetics of digoxin, metoprolol, and oral contraceptives and absorption of eliglustat when coadministered with acid-reducing agents.
Ibrahim, J; Peterschmitt, MJ; Puga, AC; Ross, L; Thibault, N; Turpault, S; Vu, L; Xue, Y, 2020)		1.17

Bioavailability

ExcerptReferenceRelevance
" Pharmacological approaches that increase nitric oxide bioavailability or decrease reactive oxygen species completely normalized the elevated VWF secretion in GLA deficient cells."( α-galactosidase A deficiency promotes von Willebrand factor secretion in models of Fabry disease.
Bodary, PF; Desch, KC; Kaissarian, NM; Kang, JJ; Kelly, RJ; Shayman, JA; Shu, L, 2019)		0.51

Dosage Studied

In a phase Ib clinical trial in healthy volunteers, plasma glucocerebroside concentrations were decreased after dosing with eliglustat tartrate. In phase II clinical trials in patients with type 1 (non-neuronopathic) Gaucher disease, spleen and liver volumes were diminished.

ExcerptRelevanceReference
" In a phase Ib clinical trial in healthy volunteers, plasma glucocerebroside concentrations were decreased after dosing with eliglustat tartrate, and in phase II clinical trials in patients with type 1 (non-neuronopathic) Gaucher disease, spleen and liver volumes were diminished."( Eliglustat tartrate, an orally active glucocerebroside synthase inhibitor for the potential treatment of Gaucher disease and other lysosomal storage diseases.
Cox, TM, 2010)		2.01
" The randomized, double-blind EDGE trial (NCT01074944, Sanofi Genzyme) evaluated once-daily eliglustat dosing compared with the approved twice-daily regimen at the same total daily dose in adults with GD1."( Once- versus twice-daily dosing of eliglustat in adults with Gaucher disease type 1: The Phase 3, randomized, double-blind EDGE trial.
Charrow, J; Fraga, C; Gaemers, SJM; Gu, X; Ida, H; Jouvin, MH; Li, J; Longo, N; Lukina, E; Nonino, A; Peterschmitt, MJ; Wu, Y; Xue, Y, 2018)		0.98
" These predictions, together with other exposure-related factors, contributed to the CYP2D6 phenotype-based dosing recommendations for eliglustat, including dose adjustments and contraindications when co-administered with drugs metabolized by the CYP2D6 and CYP3A pathways."( How a concentration-effect analysis of data from the eliglustat thorough electrocardiographic study was used to support dosing recommendations.
Cox, GF; Maison-Blanche, P; Msihid, J; Ortemann-Renon, C; Peterschmitt, MJ; Puga, AC; Ross, L; Ruskin, JN, )		0.58
[information is derived through text-mining from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Roles (1)

RoleDescription
EC 2.4.1.80 (ceramide glucosyltransferase) inhibitorAn EC 2.4.1.* (hexosyltransferase) inhibitor that interferes with the activity of ceramide glucosyltransferase (EC 2.4.1.80).
[role information is derived from Chemical Entities of Biological Interest (ChEBI), Hastings J, Owen G, Dekker A, Ennis M, Kale N, Muthukrishnan V, Turner S, Swainston N, Mendes P, Steinbeck C. (2016). ChEBI in 2016: Improved services and an expanding collection of metabolites. Nucleic Acids Res]

Drug Classes (4)

ClassDescription
benzodioxineAny organic heterobicyclic compound containing ortho-fused benzene and dioxine rings.
N-alkylpyrrolidine
secondary alcoholA secondary alcohol is a compound in which a hydroxy group, -OH, is attached to a saturated carbon atom which has two other carbon atoms attached to it.
carboxamideAn amide of a carboxylic acid, having the structure RC(=O)NR2. The term is used as a suffix in systematic name formation to denote the -C(=O)NH2 group including its carbon atom.
[compound class information is derived from Chemical Entities of Biological Interest (ChEBI), Hastings J, Owen G, Dekker A, Ennis M, Kale N, Muthukrishnan V, Turner S, Swainston N, Mendes P, Steinbeck C. (2016). ChEBI in 2016: Improved services and an expanding collection of metabolites. Nucleic Acids Res]

Protein Targets (1)

Potency Measurements

ProteinTaxonomyMeasurementAverage (µ)Min (ref.)Avg (ref.)Max (ref.)Bioassay(s)
Spike glycoproteinSevere acute respiratory syndrome-related coronavirusPotency39.81070.009610.525035.4813AID1479145
[prepared from compound, protein, and bioassay information from National Library of Medicine (NLM), extracted Dec-2023]

Ceullar Components (1)

Processvia Protein(s)Taxonomy
virion membraneSpike glycoproteinSevere acute respiratory syndrome-related coronavirus
[Information is prepared from geneontology information from the June-17-2024 release]

Bioassays (4)

Assay IDTitleYearJournalArticle
AID1864871Inhibition of full length NTMT1 (1 to 222 residues) (unknown origin) expressed in Escherichia coli BL21 (DE3) assessed as SAH production at 100 uM by SAHH-coupled fluorescence assay2022Journal of medicinal chemistry, 09-22, Volume: 65, Issue:18
Venglustat Inhibits Protein N-Terminal Methyltransferase 1 in a Substrate-Competitive Manner.
AID1826294Uncompetitive inhibition of GCS (unknown origin) using 1 to 70 uM C8-ceramide and 0.1 to 200 uM UDP-glucose as substrate incubated for 1 hr by RapidFire mass spectrometry2022Journal of medicinal chemistry, 03-10, Volume: 65, Issue:5
Discovery of Brain-Penetrant Glucosylceramide Synthase Inhibitors with a Novel Pharmacophore.
AID1864868Inhibition of human NTMT1 in human HCT116 cells assessed as reduction in me3-RCC1 level up to 30 uM incubated for 72 hrs by western blot analysis2022Journal of medicinal chemistry, 09-22, Volume: 65, Issue:18
Venglustat Inhibits Protein N-Terminal Methyltransferase 1 in a Substrate-Competitive Manner.
AID1864869Inhibition of human NTMT1 in human HCT116 cells assessed as reduction in me3-SET level up to 30 uM incubated for 72 hrs by western blot analysis2022Journal of medicinal chemistry, 09-22, Volume: 65, Issue:18
Venglustat Inhibits Protein N-Terminal Methyltransferase 1 in a Substrate-Competitive Manner.
[information is prepared from bioassay data collected from National Library of Medicine (NLM), extracted Dec-2023]

Research

Studies (67)

TimeframeStudies, This Drug (%)All Drugs %
pre-19900 (0.00)18.7374
1990's0 (0.00)18.2507
2000's1 (1.49)29.6817
2010's46 (68.66)24.3611
2020's20 (29.85)2.80
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Market Indicators

Research Demand Index: 47.61

According to the monthly volume, diversity, and competition of internet searches for this compound, as well the volume and growth of publications, there is estimated to be strong demand-to-supply ratio for research on this compound.

MetricThis Compound (vs All)
Research Demand Index47.61 (24.57)
Research Supply Index4.45 (2.92)
Research Growth Index5.45 (4.65)
Search Engine Demand Index71.19 (26.88)
Search Engine Supply Index2.00 (0.95)

This Compound (47.61)

All Compounds (24.57)

Study Types

Publication TypeThis drug (%)All Drugs (%)
Trials17 (25.00%)5.53%
Reviews12 (17.65%)6.00%
Case Studies1 (1.47%)4.05%
Observational0 (0.00%)0.25%
Other38 (55.88%)84.16%
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Clinical Trials (15)

Trial Overview

TrialPhaseEnrollmentStudy TypeStart DateStatus
A Single-blind, Randomized, Unbalanced Crossover Design With 5 Vehicles, 5 Periods, and 5 Sequences, Repeated-doses (With no Ingestion) Study to Assess the Palatability of Eliglustat Prototype Liquid Formulations in Healthy Subjects [NCT02422654]Phase 18 participants (Actual)Interventional2015-04-30Completed
A Single-site, Open-label, Fixed-sequence Phase 1 Study Evaluating the Effect of Eliglustat (Genz-112638) on the Pharmacokinetics and Safety and Tolerability of Digoxin in Healthy Adult Subjects [NCT01357811]Phase 128 participants (Actual)Interventional2011-08-31Completed
An Open-Label Study to Evaluate the Absolute Bioavailability of Genz-112638 and the Absorption, Metabolism, and Excretion of [14C]-Genz-112638 in Male Subjects [NCT06143904]Phase 110 participants (Actual)Interventional2009-06-03Completed
Open Label Interventional Multicenter Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Successfully Completed the Phase 2 or Phase 3 Studies [NCT02536755]Phase 331 participants (Actual)Interventional2015-10-27Completed
A Phase 2, Open-Label, Multi-Center Study Evaluating the Efficacy, Safety and Pharmacokinetics of Genz-112638 in Gaucher Type 1 Patients [NCT00358150]Phase 226 participants (Actual)Interventional2006-06-30Completed
An Open-label Pharmacokinetic and Tolerability Study of Eliglustat Tartrate Given as a Single Dose in Subjects With Mild and Moderate Hepatic Impairment, and in Matched Subjects With Normal Hepatic Function [NCT02536911]Phase 124 participants (Actual)Interventional2015-09-30Completed
An Open-label Two-stage Pharmacokinetic and Tolerability Study of Eliglustat Tartrate Given as a Single Dose in Subjects With Mild, Moderate and Severe Renal Impairment, and in Matched Subjects With Normal Renal Function [NCT02536937]Phase 132 participants (Actual)Interventional2015-09-30Completed
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1 (ENGAGE) [NCT00891202]Phase 340 participants (Actual)Interventional2009-11-30Completed
A Phase 3, Randomized, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Once Daily Versus Twice Daily Dosing of Genz-112638 in Patients With Gaucher Disease Type 1 Who Have Demonstrated Clinical Sta [NCT01074944]Phase 3170 participants (Actual)Interventional2010-06-30Completed
A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1 Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE) [NCT00943111]Phase 3160 participants (Actual)Interventional2009-09-30Completed
A Single-site, Open-label, Fixed-sequence Phase 1 Study Evaluating the Effect of Eliglustat (Genz-112638) on the Pharmacokinetics, Safety and Tolerability of Metoprolol in Healthy Adult Subjects. [NCT01659944]Phase 114 participants (Actual)Interventional2012-05-31Completed
Phase ⅠStudy of GSL Synthetase Inhibitor in Combination With Immune Checkpoint Inhibitor in Treating Patients With Advanced Relapsed or Refractory Hematological Malignancies and Previously Treated Solid Tumors [NCT04944888]Phase 131 participants (Actual)Interventional2021-07-01Completed
Evaluation of the Safety in the Combination Usage of Cerdelga and Cerezyme in Type III Gaucher Disease Patients and the Efficacy on Soft Tissue Diseases. [NCT03519646]4 participants (Actual)Interventional2018-04-23Completed
A Pilot, Phase 1, Single-site, Single-dose, Randomized, Open-label, Two-treatment, Two-sequence, Four-period Replicated Crossover Study Evaluating the Within-subject Pharmacokinetic Variability and Relative Bioavailability of the Phase 3 and Common Blend [NCT01452542]Phase 122 participants (Actual)Interventional2011-10-31Completed
Open Label, Two Cohort (With and Without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients With Gaucher Disease Type 1 and Type 3 [NCT03485677]Phase 357 participants (Actual)Interventional2018-04-11Active, not recruiting
[information is prepared from clinicaltrials.gov, extracted Sep-2024]

Trial Outcomes

TrialOutcome
NCT00358150 (17) [back to overview]Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study
NCT00358150 (17) [back to overview]Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study
NCT00358150 (17) [back to overview]Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study
NCT00358150 (17) [back to overview]Percentage of Participants Demonstrating A Meaningful Clinical Response
NCT00358150 (17) [back to overview]Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study
NCT00358150 (17) [back to overview]Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS)
NCT00358150 (17) [back to overview]Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study
NCT00358150 (17) [back to overview]Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study
NCT00358150 (17) [back to overview]Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study
NCT00358150 (17) [back to overview]Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study
NCT00358150 (17) [back to overview]Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study
NCT00358150 (17) [back to overview]Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study
NCT00358150 (17) [back to overview]Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study
NCT00358150 (17) [back to overview]Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study
NCT00358150 (17) [back to overview]Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study
NCT00358150 (17) [back to overview]Percent Change From Baseline in Biomarker (Tartrate-Resistant Acid Phosphatase [TRAP]) Level at Year 1 and Year 2
NCT00358150 (17) [back to overview]Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study
NCT00891202 (9) [back to overview]LTTP: Absolute Change From Baseline in Hemoglobin Level at Week 234
NCT00891202 (9) [back to overview]LTTP: Percent Change From Baseline in Liver Volume (in MN) at Week 234
NCT00891202 (9) [back to overview]LTTP: Percent Change From Baseline in Platelet Counts at Week 234
NCT00891202 (9) [back to overview]LTTP: Percent Change From Baseline in Spleen Volume (in MN) at Week 234
NCT00891202 (9) [back to overview]PAP: Percent Change From Baseline in Spleen Volume (in Multiples of Normal [MN]) at Week 39 of the Primary Analysis Period With Eliglustat Tartrate Treatment as Compared to Placebo
NCT00891202 (9) [back to overview]PAP: Percent Change From Baseline in Platelet Counts at Week 39
NCT00891202 (9) [back to overview]PAP: Percent Change From Baseline in Liver Volume (in MN) at Week 39
NCT00891202 (9) [back to overview]PAP: Hemoglobin Level
NCT00891202 (9) [back to overview]PAP: Absolute Change From Baseline in Hemoglobin Level at Week 39
NCT00943111 (17) [back to overview]Absolute Change From Baseline in Hemoglobin Levels at Week 208
NCT00943111 (17) [back to overview]Absolute Change From Baseline in Hemoglobin Levels at Week 52
NCT00943111 (17) [back to overview]Percent Change From Baseline in Liver Volume (in MN) at Week 208
NCT00943111 (17) [back to overview]Percent Change From Baseline in Liver Volume (in MN) at Week 52
NCT00943111 (17) [back to overview]Percent Change From Baseline in Platelet Counts at Week 208
NCT00943111 (17) [back to overview]Percent Change From Baseline in Platelet Counts at Week 52
NCT00943111 (17) [back to overview]Percent Change From Baseline in Spleen Volume (in MN) at Week 208
NCT00943111 (17) [back to overview]Percent Change From Baseline in Spleen Volume (MN) at Week 52
NCT00943111 (17) [back to overview]Percentage of Participants Who Remained Stable for 52 Weeks During the Primary Analysis Period
NCT00943111 (17) [back to overview]Absolute Change From Baseline in Total T-Scores for Bone Mineral Density at Week 208
NCT00943111 (17) [back to overview]Absolute Change From Baseline in Total T-Scores for Bone Mineral Density at Week 52
NCT00943111 (17) [back to overview]Absolute Change From Baseline in Total Z-Scores for Bone Mineral Density at Week 208
NCT00943111 (17) [back to overview]Absolute Change From Baseline in Total Z-Scores for Bone Mineral Density at Week 52
NCT00943111 (17) [back to overview]Percentage of Participants Who Remained Stable Annually for 4 Years During the LTTP
NCT00943111 (17) [back to overview]Total T-Scores for Bone Mineral Density
NCT00943111 (17) [back to overview]Total Z-Scores for Bone Mineral Density
NCT00943111 (17) [back to overview]Hemoglobin Level
NCT01074944 (36) [back to overview]LIP: Mean Biomarker (MIP1-beta) Value at Baseline, Week 78
NCT01074944 (36) [back to overview]LTTP: Number of Participants With Mobility Status (MS) at Baseline, 1 Year and 2 Years
NCT01074944 (36) [back to overview]LTTP: Percentage of Participants Who Maintained a Stable Bone Criterion ,Hemoglobin Level, Platelet Count, Liver Volume and Spleen Volume at 1 Year and 2 Years
NCT01074944 (36) [back to overview]LTTP: Total Bone Marrow Burden Score (BMB) at Baseline, 1 Year, and 2 Years
NCT01074944 (36) [back to overview]LTTP: Total T-Scores for BMD at Baseline, 1 Year, and 2 Years
NCT01074944 (36) [back to overview]LTTP: Total Z-scores for BMD at Baseline, 1 Year, and 2 Years
NCT01074944 (36) [back to overview]PAP: Bone Mineral Density (BMD) at Baseline and Week 52
NCT01074944 (36) [back to overview]PAP: Mean Biomarker (Chitotriosidase) Value at Baseline, Weeks 26 and Week 52
NCT01074944 (36) [back to overview]PAP: Mean Hemoglobin (Hb) Level at Baseline, Weeks 26 and 52
NCT01074944 (36) [back to overview]PAP: Mean Biomarker Macrophage Inflammatory Protein-1 Beta (MIP1-beta) Value at Baseline, Weeks 26, 52
NCT01074944 (36) [back to overview]PAP: Mean Liver Volume at Baseline, Weeks 26, 52
NCT01074944 (36) [back to overview]PAP: Mean Platelet Count at Baseline, Weeks 26, 52
NCT01074944 (36) [back to overview]PAP: Mean Spleen Volume at Baseline, Weeks 26, 52
NCT01074944 (36) [back to overview]PAP: Number of Participants With Bone Crises at Baseline, Weeks 26 and 52
NCT01074944 (36) [back to overview]PAP: Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Weeks 26 and 52
NCT01074944 (36) [back to overview]PAP: Number of Participants With Mobility Status Asessments (MS) at Baseline, Weeks 26, and 52.
NCT01074944 (36) [back to overview]PAP: Total Bone Marrow Burden Score (BMB) at Baseline and Week 52
NCT01074944 (36) [back to overview]PAP: Total T-Scores for BMD at Baseline and Week 52
NCT01074944 (36) [back to overview]PAP: Total Z-scores for BMD at Baseline and Week 52
NCT01074944 (36) [back to overview]PAP: Percentage of Participants Who Remained Stable for 52 Weeks During the PAP
NCT01074944 (36) [back to overview]LIP: Mean Biomarker (Chitotriosidase) Value at Baseline, Weeks 26, 52, and 78
NCT01074944 (36) [back to overview]LIP: Mean Biomarker (GL-1 on DBS) Value at Baseline, Week 26, Week 52, and Week 78
NCT01074944 (36) [back to overview]LIP: Mean Hemoglobin (Hb) Level at Baseline, Weeks 26, 52 and 78
NCT01074944 (36) [back to overview]LIP: Mean Liver Volume at Baseline, Weeks 26, 52 and 78
NCT01074944 (36) [back to overview]LIP: Mean Platelet Count at Baseline, Weeks 26, 52 and 78
NCT01074944 (36) [back to overview]LIP: Mean Spleen Volume at Baseline, Weeks 26, 52 and 78
NCT01074944 (36) [back to overview]LIP: Number of Participants With Bone Crises Assessment at Baseline, Weeks 26, 52 and 78
NCT01074944 (36) [back to overview]LTTP: Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, 1 Year, and 2 Years
NCT01074944 (36) [back to overview]LIP: Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Weeks 26, 52 and 78
NCT01074944 (36) [back to overview]LIP: Number of Participants With Mobility Status (MS) at Baseline, Weeks 26, 52 and 78
NCT01074944 (36) [back to overview]LTTP: Bone Mineral Density (BMD) at Baseline, 1 Year, and 2 Years
NCT01074944 (36) [back to overview]PAP: Mean Biomarker (GL-1 on DBS) Value at Baseline, Week 26 and Week 52
NCT01074944 (36) [back to overview]LTTP: Mean Biomarker (Chitotriosidase) Value at Baseline, 1 Year, and 2 Years
NCT01074944 (36) [back to overview]LTTP: Mean Biomarker (GL-1 on DBS) Value at Baseline, 1 Year, and 2 Years
NCT01074944 (36) [back to overview]LTTP: Mean Biomarker (MIP1-beta) Value at Baseline, 1 Year, and 2 Years
NCT01074944 (36) [back to overview]LTTP: Number of Participants With Bone Crises Assessment at Baseline, 1 Year and 2 Years
NCT02536755 (37) [back to overview]Change From Current Study Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234
NCT02536755 (37) [back to overview]Change From Current Study Baseline in Bone Biomarker Level: Procollagen 1 N- Terminal Propeptide (P1NP) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234
NCT02536755 (37) [back to overview]Change From Current Study Baseline in Bone Biomarker Level: Type 1 Collagen C-Telopeptides (CTx) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234
NCT02536755 (37) [back to overview]Change From Current Study Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
NCT02536755 (37) [back to overview]Change From Current Study Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
NCT02536755 (37) [back to overview]Change From Current Study Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
NCT02536755 (37) [back to overview]Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234
NCT02536755 (37) [back to overview]Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208
NCT02536755 (37) [back to overview]Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208
NCT02536755 (37) [back to overview]Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208
NCT02536755 (37) [back to overview]Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234
NCT02536755 (37) [back to overview]Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234
NCT02536755 (37) [back to overview]Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
NCT02536755 (37) [back to overview]Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
NCT02536755 (37) [back to overview]Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
NCT02536755 (37) [back to overview]Change From Eliglustat Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
NCT02536755 (37) [back to overview]Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234
NCT02536755 (37) [back to overview]Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234
NCT02536755 (37) [back to overview]Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208
NCT02536755 (37) [back to overview]Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208
NCT02536755 (37) [back to overview]Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208
NCT02536755 (37) [back to overview]Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208
NCT02536755 (37) [back to overview]Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208
NCT02536755 (37) [back to overview]Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208
NCT02536755 (37) [back to overview]Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208
NCT02536755 (37) [back to overview]Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208
NCT02536755 (37) [back to overview]Observed Annual Incidence Rate for Spine and Femur Fracture at Week 52, 104, 156 and 208
NCT02536755 (37) [back to overview]Observed Annual Incidence Rate for Spine and Femur Infarcts at Week 52, 104, 156 and 208
NCT02536755 (37) [back to overview]Observed Annual Incidence Rate for Spine and Femur Osteonecrosis at Week 52, 104, 156 and 208
NCT02536755 (37) [back to overview]Observed Annual Incidence Rate for Spine Lytic Lesion at Week 104, and 208
NCT02536755 (37) [back to overview]Total Number of New or Worsening Fracture Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208
NCT02536755 (37) [back to overview]Total Number of New or Worsening Osteonecrosis Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208
NCT02536755 (37) [back to overview]Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
NCT02536755 (37) [back to overview]Total Number of New or Worsening Lytic Lesions Events for Spine at Study Baseline, Week 104, and 208
NCT02536755 (37) [back to overview]Change From Current Study Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208
NCT02536755 (37) [back to overview]Total Number of New or Worsening Infarcts Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208
NCT02536755 (37) [back to overview]Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study

Percent change in liver volume = ([liver volume at specified time points minus liver volume at baseline] divided by [liver volume at baseline]) multiplied by 100, where all volumes are in multiples of normal. (NCT00358150)
Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)

Interventionpercent change (Mean)
Change at Year 1 (n=22)Change at Year 2 (n=20)Change at Year 3 (n=19)Change at Year 4 (n=18)Change at Year 5 (n=19)Change at Year 6 (n=19)Change at Year 7 (n=19)Change at Year 8 (n=15)Change at Year 9 (n=4)Change at End of Study (n=19)
Eliglustat-16.9-23.9-26.8-28.0-31.2-28.4-36.2-31.1-22.0-36.4

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Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study

Percent change in platelet count = ([platelet count at specified time points minus platelet count at baseline] divided by [platelet count at baseline]) multiplied by 100. (NCT00358150)
Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)

Interventionpercent change (Mean)
Change at Year 1 (n=22)Change at Year 2 (n=20)Change at Year 3 (n=18)Change at Year 4 (n=19)Change at Year 5 (n=19)Change at Year 6 (n=17)Change at Year 7 (n=19)Change at Year 8 (n=16)Change at Year 9 (n=3)Change at End of Study (n=18)
Eliglustat41.381.587.995.190.9114.399.4109.845.4117.5

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Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study

Percent change in spleen volume = ([spleen volume at specified time points minus spleen volume at baseline] divided by [spleen volume at baseline]) multiplied by 100, where all volumes are in multiples of normal. (NCT00358150)
Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)

Interventionpercent change (Mean)
Change at Year 1 (n=22)Change at Year 2 (n=20)Change at Year 3 (n=19)Change at Year 4 (n=18)Change at Year 5 (n=19)Change at Year 6 (n=19)Change at Year 7 (n=19)Change at Year 8 (n=15)Change at Year 9 (n=4)Change at End of Study (n=19)
Eliglustat-38.5-52.4-59.1-62.5-63.9-66.2-67.8-67.9-52.6-68.6

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Percentage of Participants Demonstrating A Meaningful Clinical Response

A meaningful clinical response was defined as an improvement in at least 2 of the 3 main efficacy parameters: a) an increase in hemoglobin of greater than or equal to (>=) 0.5 gram/deciliter from baseline, b) an increase in platelets of >=15 percent (%) from baseline, c) reduction in total spleen volume of >= 15% from baseline. As hemoglobin, platelets, total spleen volume were abnormal at baseline, within each participant, only those parameters were used in the evaluation of meaningful clinical response which were abnormal at baseline. (NCT00358150)
Timeframe: Baseline, Year 1

Interventionpercentage of participants (Number)
Eliglustat77

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Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study

Absolute change = hemoglobin level at specified time points minus hemoglobin level at baseline. (NCT00358150)
Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)

Interventiong/dL (Mean)
Change at Year 1 (n=22)Change at Year 2 (n=20)Change at Year 3 (n=18)Change at Year 4 (n=19)Change at Year 5 (n=19)Change at Year 6 (n=18)Change at Year 7 (n=19)Change at Year 8 (n=16)Change at Year 9 (n=4)Change at End of Study (n=18)
Eliglustat1.702.132.472.272.092.012.072.081.002.01

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Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS)

Bone marrow infiltration assessments were designed to evaluate improvements in dark marrow using MRI. Each MRI assessment was performed for both femurs and consisted of reviewing 6 different zones (the femoral head, greater trochanter, intertrochanteric region, shaft, distal metaphysis, and condyles). MRI images recorded dark marrow for each zone as either present or not present at baseline. In this outcome, number of participants (for whom dark marrow was present at baseline) with improvement from baseline in dark marrow at each specified time point were reported. (NCT00358150)
Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (up to Year 9)

InterventionParticipants (Count of Participants)
Femoral Head: Improved: Year 1: (n=16)Femoral Head: Improved: Year 2: (n=14)Femoral Head: Improved: Year 3: (n=13)Femoral Head: Improved: Year 4: (n=13)Femoral Head: Improved: Year 5: (n=13)Femoral Head: Improved: Year 6: (n=12)Femoral Head: Improved: Year 7: (n=13)Femoral Head: Improved: Year 8: (n=13)Femoral Head: Improved: End of Study: (n=8)Greater Trochanter: Improved: Year 1 (n=8)Greater Trochanter: Improved: Year 2 (n=7)Greater Trochanter: Improved: Year 3 (n=6)Greater Trochanter: Improved: Year 4 (n=6)Greater Trochanter: Improved: Year 5 (n=7)Greater Trochanter: Improved: Year 6 (n=6)Greater Trochanter: Improved: Year 7 (n=7)Greater Trochanter: Improved: Year 8 (n=7)Greater Trochanter: Improved: End of Study (n=4)Intertrochanteric Regions: Improved: Year 1 (n=21)Intertrochanteric Regions: Improved: Year 2 (n=19)Intertrochanteric Regions: Improved: Year 3 (n=18)Intertrochanteric Regions: Improved: Year 4 (n=18)Intertrochanteric Regions: Improved: Year 5 (n=18)Intertrochanteric Regions: Improved: Year 6 (n=17)Intertrochanteric Regions: Improved: Year 7 (n=18)Intertrochanteric Regions: Improved: Year 8 (n=17)Intertrochanteric Regions: Improved: EOS (n=12)Shaft: Improved: Year 1 (n= 20)Shaft: Improved: Year 2 (n= 18)Shaft: Improved: Year 3 (n= 17)Shaft: Improved: Year 4 (n= 17)Shaft: Improved: Year 5 (n= 17)Shaft: Improved: Year 6 (n= 16)Shaft: Improved: Year 7 (n= 17)Shaft: Improved: Year 8 (n= 16)Shaft: Improved: End of Study (n=11)Distal Metaphysis: Improved: Year 1 (n=21)Distal Metaphysis: Improved: Year 2 (n=19)Distal Metaphysis: Improved: Year 3 (n=18)Distal Metaphysis: Improved: Year 4 (n=18)Distal Metaphysis: Improved: Year 5 (n=18)Distal Metaphysis: Improved: Year 6 (n=17)Distal Metaphysis: Improved: Year 7 (n=18)Distal Metaphysis: Improved: Year 8 (n=17)Distal Metaphysis: Improved: End of Study (n=12)Condyles: Improved: Year 1 (n=11)Condyles: Improved: Year 2 (n=9)Condyles: Improved: Year 3 (n=8)Condyles: Improved: Year 4 (n=8)Condyles: Improved: Year 5 (n=8)Condyles: Improved: Year 6 (n=7)Condyles: Improved: Year 7 (n=8)Condyles: Improved: Year 8 (n=8)Condyles: Improved: End of Study (n=5)
Eliglustat322245553323331110133356675133345554234378886311122222

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Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study

The SF-36 questionnaire, version 2, investigates the participant's health-related quality of life (HRQL). It is a 36-item questionnaire measuring 8 domains (physical functioning, role-physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health). Each domain score ranges from 0 (worst) to 100 (best), with higher scores reflecting best health-related quality of life. Two summary scale scores were computed from the 8 domain scores: the Physical Component Summary and the Mental Component Summary. Score range for both summary scale ranges from 0 (worst) to 100 (best), with higher scores reflecting best health-related quality of life. (NCT00358150)
Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)

Interventionunits on a scale (Mean)
Physical Functioning: Change at Year 1 (n=22)Physical Functioning: Change at Year 2 (n=20)Physical Functioning: Change at Year 3 (n=17)Physical Functioning: Change at Year 4 (n=19)Physical Functioning: Change at Year 5 (n=19)Physical Functioning: Change at Year 6 (n=19)Physical Functioning: Change at Year 7 (n=19)Physical Functioning: Change at Year 8 (n=16)Physical Functioning: Change at Year 9 (n=4)Physical Functioning:Change at End of Study (n=19)Role - Physical: Change at Year 1 (n=22)Role - Physical: Change at Year 2 (n=20)Role - Physical: Change at Year 3 (n=17)Role - Physical: Change at Year 4 (n=19)Role - Physical: Change at Year 5 (n=19)Role - Physical: Change at Year 6 (n=19)Role - Physical: Change at Year 7 (n=19)Role - Physical: Change at Year 8 (n=16)Role - Physical: Change at Year 9 (n=4)Role - Physical: Change at End of Study (n= 19)Bodily Pain: Change at Year 1 (n= 22)Bodily Pain: Change at Year 2 (n= 20)Bodily Pain: Change at Year 3 (n= 17)Bodily Pain: Change at Year 4 (n= 19)Bodily Pain: Change at Year 5 (n= 19)Bodily Pain: Change at Year 6 (n= 19)Bodily Pain: Change at Year 7 (n= 19)Bodily Pain: Change at Year 8 (n= 16)Bodily Pain: Change at Year 9 (n= 4)Bodily Pain: Change at End of Study (n= 19)General Health: Change at Year 1 (n= 22)General Health: Change at Year 2 (n= 20)General Health: Change at Year 3 (n= 17)General Health: Change at Year 4 (n= 19)General Health: Change at Year 5 (n= 19)General Health: Change at Year 6 (n= 19)General Health: Change at Year 7 (n= 19)General Health: Change at Year 8 (n= 16)General Health: Change at Year 9 (n= 4)General Health: Change at End of Study (n= 19)Vitality: Change at Year 1 (n= 22)Vitality: Change at Year 2 (n= 20)Vitality: Change at Year 3 (n= 17)Vitality: Change at Year 4 (n= 19)Vitality: Change at Year 5 (n= 19)Vitality: Change at Year 6 (n= 19)Vitality: Change at Year 7 (n= 19)Vitality: Change at Year 8 (n= 16)Vitality: Change at Year 9 (n= 4)Vitality: Change at End of Study (n= 19)Social Functioning: Change at Year 1 (n= 22)Social Functioning: Change at Year 2 (n= 20)Social Functioning: Change at Year 3 (n= 17)Social Functioning: Change at Year 4 (n= 19)Social Functioning: Change at Year 5 (n= 19)Social Functioning: Change at Year 6 (n= 19)Social Functioning: Change at Year 7 (n= 19)Social Functioning: Change at Year 8 (n= 16)Social Functioning: Change at Year 9 (n= 4)Social Functioning: Change at End of Study (n= 19)Role - Emotional: Change at Year 1 (n= 22)Role - Emotional: Change at Year 2 (n= 20)Role - Emotional: Change at Year 3 (n= 17)Role - Emotional: Change at Year 4 (n= 19)Role - Emotional: Change at Year 5 (n= 19)Role - Emotional: Change at Year 6 (n= 19)Role - Emotional: Change at Year 7 (n= 19)Role - Emotional: Change at Year 8 (n= 16)Role - Emotional: Change at Year 9 (n= 4)Role- Emotional: Change at End of study (n= 19)Mental Health: Change at Year 1 (n= 22)Mental Health: Change at Year 2 (n= 20)Mental Health: Change at Year 3 (n=17)Mental Health: Change at Year 4 (n= 19)Mental Health: Change at Year 5 (n= 19)Mental Health: Change at Year 6 (n= 19)Mental Health: Change at Year 7 (n= 19)Mental Health: Change at Year 8 (n= 16)Mental Health: Change at Year 9 (n= 4)Mental Health: Change at End of Study (n=19)Physical Component Summary:Change at Year 1(n= 22)Physical Component Summary:Change at Year 2(n= 20)Physical Component Summary:Change at Year 3(n= 17)Physical Component Summary:Change at Year 4(n= 19)Physical Component Summary:Change at Year 5(n= 19)Physical Component Summary:Change at Year 6(n= 19)Physical Component Summary:Change at Year 7(n= 19)Physical Component Summary:Change at Year 8(n= 16)Physical Component Summary: Change at Year 9 (n=4)Physical Component Summary: Change at EOS (n= 19)Mental Component Summary: Change at Year 1 (n= 22)Mental Component Summary: Change at Year 2 (n= 20)Mental Component Summary: Change at Year 3 (n= 17)Mental Component Summary: Change at Year 4 (n= 19)Mental Component Summary: Change at Year 5 (n= 19)Mental Component Summary: Change at Year 6 (n= 19)Mental Component Summary: Change at Year 7 (n= 19)Mental Component Summary: Change at Year 8 (n= 16)Mental Component Summary: Change at Year 9 (n= 4)Mental Component Summary: Change at EOS (n=19)
Eliglustat8.419.5012.6511.3210.7612.6311.5811.561.259.284.555.319.566.587.578.887.898.59-1.5610.53-0.774.404.24-2.530.37-2.321.741.441.006.1110.3611.3016.9415.5313.5812.8315.8918.1911.2515.005.687.7114.7110.2013.8212.507.5715.233.1315.13-0.5710.009.563.299.219.218.5511.723.139.21-3.034.582.453.952.193.070.445.21-10.421.320.231.757.654.216.845.536.057.663.758.683.583.815.173.693.753.884.574.361.984.70-1.032.043.452.113.503.031.944.59-0.523.73

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Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study

The FSS is an instrument consisting of 9 self-administered questions that measures the impact of severity of fatigue symptoms on everyday functioning, based on the recall over the past week. Score range for each question ranges from 1 (minimum) to 7 (maximum), where higher score indicates greater severity. FSS total score was calculated by averaging the results of all questions. Total FSS score ranges from 9 (minimum) to 63 (maximum), where higher scores indicates greater severity. (NCT00358150)
Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)

Interventionunits on a scale (Mean)
Change at Year 1 (n=17)Change at Year 2 (n=16)Change at Year 3 (n=14)Change at Year 4 (n=16)Change at Year 5 (n=16)Change at Year 6 (n=15)Change at Year 7 (n=16)Change at Year 8 (n=13)Change at Year 9 (n=4)Change at End of Study (n=16)
Eliglustat-0.56-0.63-1.37-1.41-1.26-1.21-0.97-1.22-1.20-1.16

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Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study

Images of the lumbar spine and femur were obtained by dual energy X-ray absorptiometry (DXA) to determine T-score for each bone area and total bone mineral density. T-scores compares participant's bone density with that of healthy young participant of same gender. The T-score bone density categories were: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5). (NCT00358150)
Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (up to Year 9)

InterventionT-Score (Mean)
Lumbar Spine T-Score: Baseline (n= 25)Lumbar Spine T-Score: Year 1 (n= 20)Lumbar Spine T-Score: Year 2 (n= 17)Lumbar Spine T-Score: Year 3 (n= 15)Lumbar Spine T-Score: Year 4 (n= 15)Lumbar Spine T-Score: Year 5 (n= 15)Lumbar Spine T-Score: Year 6 (n= 15)Lumbar Spine T-Score: Year 7 (n= 15)Lumbar Spine T-Score: Year 8 (n= 14)Lumbar Spine T-Score: Year 9 (n= 4)Lumbar Spine T-Score: End of Study (n= 6)Femur T-Scores: Baseline (n= 23)Femur T-Scores: Year 1 (n= 19)Femur T-Scores: Year 2 (n= 15)Femur T-Scores: Year 3 (n= 13)Femur T-Scores: Year 4 (n= 13)Femur T-Scores: Year 5 (n= 13)Femur T-Scores: Year 6 (n= 13)Femur T-Scores: Year 7 (n= 13)Femur T-Scores: Year 8 (n= 12)Femur T-Scores: Year 9 (n= 4)Femur T-Scores: End of Study (n= 6)
Eliglustat-1.85-1.43-0.93-1.09-0.88-0.79-0.69-0.59-0.590.00-0.35-0.47-0.24-0.090.160.130.140.190.120.330.330.18

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Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study

Images of the lumbar spine and femur were obtained by DXA to determine Z-score for each bone area and total bone mineral density. The Z-score bone density categories are: normal (score >-2) and below normal (score <=-2). (NCT00358150)
Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (up to Year 9)

InterventionZ-Score (Mean)
Lumbar Spine Z-Score: Baseline (n= 25)Lumbar Spine Z-Score: Year 1 (n= 20)Lumbar Spine Z-Score: Year 2 (n= 17)Lumbar Spine Z-Score: Year 3 (n= 15)Lumbar Spine Z-Score: Year 4 (n= 15)Lumbar Spine Z-Score: Year 5 (n= 15)Lumbar Spine Z-Score: Year 6 (n= 15)Lumbar Spine Z-Score: Year 7 (n= 15)Lumbar Spine Z-Score: Year 8 (n= 14)Lumbar Spine Z-Score: Year 9 (n= 4)Lumbar Spine Z-Score: End of Study (n= 6)Femur Z-Scores: Baseline (n= 23)Femur Z-Scores: Year 1 (n= 19)Femur Z-Scores: Year 2 (n= 15)Femur Z-Scores: Year 3 (n= 13)Femur Z-Scores: Year 4 (n= 13)Femur Z-Scores: Year 5 (n= 13)Femur Z-Scores: Year 6 (n= 13)Femur Z-Scores: Year 7 (n= 13)Femur Z-Scores: Year 8 (n= 12)Femur Z-Scores: Year 9 (n= 4)Femur Z-Scores: End of Study (n= 6)
Eliglustat-1.49-1.11-0.64-0.63-0.48-0.37-0.27-0.14-0.29-0.050.18-0.170.040.250.520.480.490.570.520.640.380.73

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Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study

Bone pain was assessed as a part of Gaucher disease assessment in participants. Participants were categorized as none (no bone pain), very mild bone pain, mild bone pain and moderate bone pain. In this outcome, number of participants with different levels of bone pain at specified time points were reported. (NCT00358150)
Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)

Interventionparticipants (Number)
None: Baseline (n=26)None: Year 1 (n=22)None: Year 2 (n=20)None: Year 3 (n=19)None: Year 4 (n=19)None: Year 5 (n=19)None: Year 6 (n=19)None: Year 7 (n=19)None: Year 8 (n=16)None: Year 9 (n=4)None: End of Study (n=19)Very Mild: Baseline (n=26)Very Mild: Year 1 (n=22)Very Mild: Year 2 (n=20)Very Mild: Year 3 (n=19)Very Mild: Year 4 (n=19)Very Mild: Year 5 (n=19)Very Mild: Year 6 (n=19)Very Mild: Year 7 (n=19)Very Mild: Year 8 (n=16)Very Mild: Year 9 (n=4)Very Mild: End of Study (n=19)Mild: Baseline (n= 26)Mild: Year 1 (n= 22)Mild: Year 2 (n= 20)Mild: Year 3 (n=19)Mild: Year 4 (n=19)Mild: Year 5 (n=19)Mild: Year 6 (n=19)Mild: Year 7 (n=19)Mild: Year 8 (n=16)Mild: Year 9 (n=4)Mild: End of Study (n= 19)Moderate: Baseline (n=26)Moderate: Year 1 (n=22)Moderate: Year 2 (n=20)Moderate: Year 3 (n=19)Moderate: Year 4 (n=19)Moderate: Year 5 (n=19)Moderate: Year 6 (n=19)Moderate: Year 7 (n=19)Moderate: Year 8 (n=16)Moderate: Year 9 (n=4)Moderate: End of Study (n=19)
Eliglustat231717141416171514418243341131011102121110000000000000

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Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study

Mobillity, i.e. ability to walk was assessed as a part of Gaucher disease assessment in participants.In this outcome, number of participants with their different mobility status (unrestricted mobility, walks with difficulty) at specified time points were reported. (NCT00358150)
Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)

Interventionparticipants (Number)
MS: Unrestricted Mobility: Baseline (n=26)MS: Unrestricted Mobility: Year 1 (n=22)MS: Unrestricted Mobility: Year 2 (n=20)MS: Unrestricted Mobility: Year 3 (n=19)MS: Unrestricted Mobility: Year 4 (n=19)MS: Unrestricted Mobility: Year 5 (n=19)MS: Unrestricted Mobility: Year 6 (n=19)MS: Unrestricted Mobility: Year 7 (n=19)MS: Unrestricted Mobility: Year 8 (n=16)MS: Unrestricted Mobility: Year 9 (n=4)MS: Unrestricted Mobility: End of Study (n=19)MS: Walks with Difficulty: Baseline (n=26)MS: Walks with Difficulty: Year 1 (n=22)MS: Walks with Difficulty: Year 2 (n=20)MS: Walks with Difficulty: Year 3 (n=19)MS: Walks with Difficulty: Year 4 (n=19)MS: Walks with Difficulty: Year 5 (n=19)MS: Walks with Difficulty: Year 6 (n=19)MS: Walks with Difficulty: Year 7 (n=19)MS: Walks with Difficulty: Year 8 (n=16)MS: Walks with Difficulty: Year 9 (n=4)MS: Walks with Difficulty: End of Study (n=19)
Eliglustat24222019191919191641820000000001

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Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study

Bone crisis was assessed as a part of Gaucher disease assessment in participants. Acute, excruciating episodic bone pain is characteristic of Gaucher bone crisis, which typically causes debilitation lasting several days or longer and requires treatment with immobilization, hydration, and opioid analgesics. Participants were categorized as 0= no bone crisis, 1= 1 bone crisis, and 2= 2 bone crises during the assessment period. In this outcome, number of participants with 0= no bone crises levels at specified time points were reported. (NCT00358150)
Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)

Interventionparticipants (Number)
Baseline (n=26)Year 1 (n=22)Year 2 (n=20)Year 3 (n=19)Year 4 (n=19)Year 5 (n=19)Year 6 (n=19)Year 7 (n=19)Year 8 (n=16)Year 9 (n=4)End of Study (n=19)
Eliglustat262220191919191916419

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Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study

(NCT00358150)
Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study (Up to Year 9)

Interventionpercent change (Mean)
Change at Year 1 (n=22)Change at Year 2 (n=20)Change at Year 3 (n=19)Change at Year 4 (n=18)Change at Year 5 (n=19)Change at Year 6 (n=17)Change at Year 7 (n=19)Change at Year 8 (n=16)Change at Year 9 (n=4)Change at End of Study (n=18)
Eliglustat-35.1-53.5-56.7-60.7-55.7-61.5-63.6-59.2-55.1-64.7

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Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study

(NCT00358150)
Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)

Interventionpercent change (Mean)
Change at Year 1 (n=20)Change at Year 2 (n=18)Change at Year 3 (n=17)Change at Year 4 (n=17)Change at Year 5 (n=17)Change at Year 6 (n=17)Change at Year 7 (n=17)Change at Year 8 (n=14)Change at Year 9 (n=4)Change at End of Study (n=17)
Eliglustat-49.9-73.5-76.4-79.2-76.7-74.1-75.5-72.5-61.2-69.8

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Percent Change From Baseline in Biomarker (Tartrate-Resistant Acid Phosphatase [TRAP]) Level at Year 1 and Year 2

(NCT00358150)
Timeframe: Baseline, Year 1, Year 2

Interventionpercent change (Mean)
Change at Year 1 (n=22)Change at Year 2 (n= 10)
Eliglustat-37.0-52.5

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Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study

(NCT00358150)
Timeframe: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study (Up to Year 9)

Interventionpercent change (Mean)
Change at Year 1 (n=21)Change at Year 2 (n=15)Change at Year 3 (n=18)Change at Year 4 (n=18)Change at Year 5 (n=18)Change at Year 6 (n=18)Change at Year 7 (n=18)Change at Year 8 (n=16)Change at Year 9 (n=4)Change at End of Study (n=18)
Eliglustat-49.0-72.1-68.6-80.2-89.4-82.7-78.5-79.8-88.6-85.8

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LTTP: Absolute Change From Baseline in Hemoglobin Level at Week 234

Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP. (NCT00891202)
Timeframe: PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234

Interventiong/dL (Mean)
LTTP: Eliglustat (Originally on Eliglustat)1.1
LTTP: Eliglustat (Originally on Placebo)1.9

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LTTP: Percent Change From Baseline in Liver Volume (in MN) at Week 234

Percent change in liver volume = ([liver volume at Week 234 minus liver volume at baseline] divided by [liver volume at baseline]) multiplied by 100, where all volumes are in MN. Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP. (NCT00891202)
Timeframe: PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234

Interventionpercent change (Mean)
LTTP: Eliglustat (Originally on Eliglustat)-24.3
LTTP: Eliglustat (Originally on Placebo)-22.4

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LTTP: Percent Change From Baseline in Platelet Counts at Week 234

Percent change in platelet count = ([platelet count at Week 234 minus platelet count at baseline] divided by [platelet count at baseline]) multiplied by 100. Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP. (NCT00891202)
Timeframe: PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234

Interventionpercent change (Mean)
LTTP: Eliglustat (Originally on Eliglustat)77.3
LTTP: Eliglustat (Originally on Placebo)100.1

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LTTP: Percent Change From Baseline in Spleen Volume (in MN) at Week 234

Percent change in spleen volume = ([spleen volume at Week 234 minus spleen volume at baseline] divided by [spleen volume at baseline]) multiplied by 100, where all volumes are in MN. Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP. (NCT00891202)
Timeframe: PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234

Interventionpercent change (Mean)
LTTP: Eliglustat (Originally on Eliglustat)-66.9
LTTP: Eliglustat (Originally on Placebo)-64.0

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PAP: Percent Change From Baseline in Spleen Volume (in Multiples of Normal [MN]) at Week 39 of the Primary Analysis Period With Eliglustat Tartrate Treatment as Compared to Placebo

Percent change in spleen volume = ([spleen volume at Week 39 minus spleen volume at baseline] divided by [spleen volume at baseline]) multiplied by 100, where all volumes are in MN. (NCT00891202)
Timeframe: PAP Baseline (Day 1), Week 39

Interventionpercent change (Least Squares Mean)
PAP: Eliglustat-27.77
PAP: Placebo2.26

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PAP: Percent Change From Baseline in Platelet Counts at Week 39

Percent change in platelet count = ([platelet count at Week 39 minus platelet count at baseline] divided by [platelet count at baseline]) multiplied by 100. (NCT00891202)
Timeframe: PAP Baseline (Day 1), Week 39

Interventionpercent change (Least Squares Mean)
PAP: Eliglustat32.00
PAP: Placebo-9.06

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PAP: Percent Change From Baseline in Liver Volume (in MN) at Week 39

Percent change in liver volume = ([liver volume at Week 39 minus liver volume at baseline] divided by [liver volume at baseline]) multiplied by 100, where all volumes are in MN. (NCT00891202)
Timeframe: PAP Baseline (Day 1), Week 39

Interventionpercent change (Least Squares Mean)
PAP: Eliglustat-5.20
PAP: Placebo1.44

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PAP: Hemoglobin Level

(NCT00891202)
Timeframe: PAP Baseline (Day 1)

Interventiongram per deciliter (g/dL) (Mean)
PAP: Eliglustat12.05
PAP: Placebo12.75

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PAP: Absolute Change From Baseline in Hemoglobin Level at Week 39

Absolute change = hemoglobin level at Week 39 minus hemoglobin level at baseline. (NCT00891202)
Timeframe: PAP Baseline (Day 1), Week 39

Interventiong/dL (Least Squares Mean)
PAP: Eliglustat0.69
PAP: Placebo-0.54

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Absolute Change From Baseline in Hemoglobin Levels at Week 208

Absolute change = hemoglobin level at Week 208 minus hemoglobin level at baseline. (NCT00943111)
Timeframe: Baseline, Week 208

Interventiong/dL (Mean)
Eliglustat: LTTP0.297

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Absolute Change From Baseline in Hemoglobin Levels at Week 52

Absolute change = hemoglobin level at Week 52 minus hemoglobin level at baseline. (NCT00943111)
Timeframe: Baseline, Week 52

Interventiong/dL (Least Squares Mean)
Eliglustat: PAP-0.22
Imiglucerase: PAP0.05

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Percent Change From Baseline in Liver Volume (in MN) at Week 208

Percent change in liver volume = ([liver volume at Week 208 minus liver volume at baseline] divided by [liver volume at baseline]) multiplied by 100, where all volumes are in multiples of normal. (NCT00943111)
Timeframe: Baseline, Week 208

Interventionpercent change (Mean)
Eliglustat: LTTP-2.345

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Percent Change From Baseline in Liver Volume (in MN) at Week 52

Percent change in liver volume = ([liver volume at Week 52 minus liver volume at baseline] divided by [liver volume at baseline]) multiplied by 100, where all volumes are in multiples of normal. (NCT00943111)
Timeframe: Baseline, Week 52

Interventionpercent change (Least Squares Mean)
Eliglustat: PAP1.99
Imiglucerase: PAP3.13

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Percent Change From Baseline in Platelet Counts at Week 208

Percent change in platelet counts = ([platelet count at Week 208 minus platelet count at baseline] divided by [platelet count at baseline]) multiplied by 100. (NCT00943111)
Timeframe: Baseline, Week 208

Interventionpercent change (Mean)
Eliglustat: LTTP6.990

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Percent Change From Baseline in Platelet Counts at Week 52

Percent change in platelet counts = ([platelet count at Week 52 minus platelet count at baseline] divided by [platelet count at baseline]) multiplied by 100. (NCT00943111)
Timeframe: Baseline, Week 52

Interventionpercent change (Least Squares Mean)
Eliglustat: PAP3.93
Imiglucerase: PAP2.63

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Percent Change From Baseline in Spleen Volume (in MN) at Week 208

Percent change in spleen volume = ([spleen volume at Week 208 minus spleen volume at baseline] divided by [spleen volume at baseline]) multiplied by 100, where all volumes are in MN. (NCT00943111)
Timeframe: Baseline, Week 208

Interventionpercent change (Mean)
Eliglustat: LTTP-14.768

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Percent Change From Baseline in Spleen Volume (MN) at Week 52

Percent change in spleen volume = ([spleen volume at Week 52 minus spleen volume at baseline] divided by [spleen volume at baseline]) multiplied by 100, where all volumes are in MN. (NCT00943111)
Timeframe: Baseline, Week 52

Interventionpercent change (Least Squares Mean)
Eliglustat: PAP-6.05
Imiglucerase: PAP-3.22

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Percentage of Participants Who Remained Stable for 52 Weeks During the Primary Analysis Period

For a participant to be classified as stable, the participant must have remained stable in hematological parameters (hemoglobin levels and platelet counts) and organ volumes (spleen, when applicable, and liver volumes in multiples of normal [MN]). Stable hematological parameters were defined as hemoglobin level did not decrease more than (>) 1.5 gram per deciliter (g/dL) from baseline and platelet count did not decrease >25% from baseline. Stable organ volumes were defined as spleen volume (in MN) did not increase >25% from baseline, if applicable, and liver volume (in MN) did not increase >20% from baseline. (NCT00943111)
Timeframe: Baseline up to Week 52

Interventionpercentage of participants (Number)
Eliglustat: PAP84.8
Imiglucerase: PAP93.6

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Absolute Change From Baseline in Total T-Scores for Bone Mineral Density at Week 208

Images of the spine and bilateral femur were obtained by DXA to determine T-score for each bone area and total bone mineral density. T-score compares participant's bone density with that of healthy young participant. The T-score bone density categories are: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5). Absolute change = T-score at Week 208 minus T-score at baseline. (NCT00943111)
Timeframe: Baseline, Week 208

InterventionT-Score (Mean)
Total SpineTotal Femur
Eliglustat: LTTP0.22-0.03

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Absolute Change From Baseline in Total T-Scores for Bone Mineral Density at Week 52

Images of the spine and bilateral femur were obtained by DXA to determine T-score for each bone area and total bone mineral density. T-score compares participant's bone density with that of healthy young participant. The T-score bone density categories are: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5). Absolute change = T-score at Week 52 minus T-score at baseline. (NCT00943111)
Timeframe: Baseline, Week 52

,
InterventionT-score (Least Squares Mean)
Change in Lumbar Spine T-Score (n=81, 38)Change in Femur T-Score (n=80, 37)
Eliglustat: PAP0.040.00
Imiglucerase: PAP0.03-0.03

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Absolute Change From Baseline in Total Z-Scores for Bone Mineral Density at Week 208

Images of the spine and bilateral femur were obtained by DXA to determine Z-score for each bone area and total bone mineral density. The Z-score bone density categories are: normal (score >-2) and below normal (score <=-2). Absolute change = Z-score at Week 208 minus Z-score at baseline. (NCT00943111)
Timeframe: Baseline, Week 208

InterventionZ-score (Mean)
Total SpineTotal Femur
Eliglustat: LTTP0.290.03

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Absolute Change From Baseline in Total Z-Scores for Bone Mineral Density at Week 52

Images of the spine and bilateral femur were obtained by DXA to determine Z-score for each bone area and total bone mineral density. The Z-score bone density categories are: normal (score >-2) and below normal (score <=-2). Absolute change = Z-score at Week 52 minus Z-score at baseline. (NCT00943111)
Timeframe: Baseline, Week 52

,
InterventionZ-score (Least Squares Mean)
Change in Lumbar Spine Z-Score (n=94, 45)Change in Femur Z-Score (n=93, 44)
Eliglustat: PAP0.060.03
Imiglucerase: PAP0.060.02

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Percentage of Participants Who Remained Stable Annually for 4 Years During the LTTP

For a participant to be classified as stable, the participant must have remained stable in hematological parameters (hemoglobin levels and platelet counts) and organ volumes (spleen, when applicable, and liver volumes in MN). Stable hematological parameters were defined as hemoglobin level did not decrease >1.5 g/dL from baseline and platelet count did not decrease >25% from baseline. Stable organ volumes were defined as spleen volume (in MN) did not increase >25% from baseline, if applicable, and liver volume did not increase >20% from baseline. (NCT00943111)
Timeframe: Week 52 up to week 208

Interventionpercentage of participants (Number)
Year 1 (n=127)Year 2 (n= 115)Year 3 (n= 92)Year 4 (n= 41)
Eliglustat: LTTP83.675.6560.5326.97

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Total T-Scores for Bone Mineral Density

Images of the spine and bilateral femur were obtained by dual energy X-Ray absorptiometry (DXA) to determine T-score for each bone area and total bone mineral density. T-score compares participant's bone density with that of healthy young participant. The T-score bone density categories are: normal (score greater than [>]-1), osteopenia (score -2.5 to less than or equal to [<=] -1), and osteoporosis (score <= -2.5). (NCT00943111)
Timeframe: Baseline

,
InterventionT-score (Mean)
Lumbar Spine T-Score (n=81, 38)Femur T-Score (n=80, 37)
Eliglustat: PAP-0.56-0.11
Imiglucerase: PAP-0.33-0.47

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Total Z-Scores for Bone Mineral Density

Images of the spine and bilateral femur were obtained by DXA to determine Z-score for each bone area and total bone mineral density. The Z-score bone density categories are: normal (score >-2) and below normal (score <=-2). (NCT00943111)
Timeframe: Baseline

,
InterventionZ-score (Mean)
Lumbar Spine Z-Score (n=94, 45)Femur Z-Score (n=93, 44)
Eliglustat: PAP-0.350.09
Imiglucerase: PAP-0.14-0.18

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Hemoglobin Level

(NCT00943111)
Timeframe: Baseline

Interventiongram per deciliter (g/dL) (Mean)
Eliglustat: PAP13.592
Imiglucerase: PAP13.797

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LIP: Mean Biomarker (MIP1-beta) Value at Baseline, Week 78

MIP1-beta biomarker was assayed from plasma. (NCT01074944)
Timeframe: Baseline and Week 78

Interventionpg/mL (Mean)
MIP-1beta: Baseline (n=170)MIP-1beta: Week 78 (n=41)
LIP: Eliglustat142.433132.180

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LTTP: Number of Participants With Mobility Status (MS) at Baseline, 1 Year and 2 Years

Mobility, i.e., ability to walk was assessed as a part of Gaucher disease assessment in participants. In this outcome, number of participants with their different mobility status along with the use of mobility aids (unrestricted mobility, walks with difficulty, walks with orthopaedic aid, requires wheelchair, bedridden) at specified time points were reported. (NCT01074944)
Timeframe: Baseline, 1 year, and 2 years

Interventionparticipants (Number)
MS, Unrestricted: Baseline (n=120)MS, Unrestricted: 1 year (n=104)MS, Unrestricted: 2 years (n=32)MS, Walks with Difficulty: Baseline (n=120)MS, Walks with Difficulty: 1 year (n=104)Ms, Walks with Difficulty: 2 years (n=32)MS, Walks with Orthopedic Aid: Baseline (n=120)MS, Walks with Orthopedic Aid: 1 year (n=104)MS, Walks with Orthopedic Aid: 2 years (n=32)MS, Required Wheelchair: Baseline (n=120)MS, Required Wheelchair: 1 year (n=104)MS, Required Wheelchair: 2 years (n=32)MS, Bedridden: Baseline (n=120)MS, Bedridden: 1 year (n=104)MS, Bedridden: 2 years (n=32)
LTTP: Eliglustat1119728742010222000

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LTTP: Percentage of Participants Who Maintained a Stable Bone Criterion ,Hemoglobin Level, Platelet Count, Liver Volume and Spleen Volume at 1 Year and 2 Years

Participant were considered as stable if they met the following criteria: hemoglobin level did not decrease >1.5 g/dL from baseline for PAP, platelet count does not decrease >25% below Baseline for PAP, liver volume does not increase >20% above Baseline for PAP, spleen volume does not increase >25% above Baseline for PAP. Baseline for PAP was defined as last available assessment prior to randomization. (NCT01074944)
Timeframe: 1 Year, 2 Years

Interventionpercentage of participants (Number)
Bone Criterion Stable at 1 year (n=104)Bone Criterion Stable at 2 years (n=32)Hemoglobin Level Stable at 1 year (n=104)Hemoglobin Level Stable at 2 years (n=32)Platelet Count Stable at 1 year (n=104)Platelet Count Stable at 2 years (n=32)Liver Volume Stable at 1 year (n=103)Liver Volume Stable at 2 years (n=31)Spleen Volume Stable at 1 year (n=72)Spleen Volume Stable at 2 years (n=20)
LTTP: Eliglustat92.384.492.381.393.384.493.283.995.895.0

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LTTP: Total Bone Marrow Burden Score (BMB) at Baseline, 1 Year, and 2 Years

BMB Score was measured using MRI, range from 0 (no abnormalities) to 8 points (severe disease) for the lumbar spine and from 0 (no abnormalities) to 8 points (severe disease) for the femurs. The total score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) -16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement. (NCT01074944)
Timeframe: Baseline, 1 year, and 2 years

InterventionBMB Score (Mean)
BMB Score: Baseline (n=115)BMB Score: 1 year (n=26)BMB Score: 2 years (n=17)
LTTP: Eliglustat8.1647.8538.059

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LTTP: Total T-Scores for BMD at Baseline, 1 Year, and 2 Years

Images of the spine and bilateral femur were obtained by DXA to determine T-score for each bone area and total bone mineral density. The T-score bone density categories were: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5). (NCT01074944)
Timeframe: Baseline, 1 year, and 2 years

InterventionT-score (Mean)
Lumbar Spine T-Score: Baseline (n=110)Lumbar Spine T-Score: 1 year (n=98)Lumbar Spine T-Score: 2 years (n=26)Left Femur T-Score: Baseline (n=103)Left Femur T-Score: 1 year (n=91)Left Femur T-Score: 2 years (n=22)Right Femur T-Score: Baseline (n=99)Right Femur T-Score: 1 year (n=87)Right Femur T-Score: 2 years (n=21)
LTTP: Eliglustat-0.674-0.718-0.750-0.421-0.382-0.682-0.461-0.500-1.005

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LTTP: Total Z-scores for BMD at Baseline, 1 Year, and 2 Years

Images of the spine and bilateral femur were obtained by DXA to determine Z-score for each bone area and total bone mineral density. The Z-score bone density categories are: normal (score >-2) and below normal (score <=-2). (NCT01074944)
Timeframe: Baseline, 1 year, and 2 years

InterventionZ-score (Mean)
Lumbar Spine Z-Score: Baseline (n=113)Lumbar Spine Z-Score: 1 year (n=101)Lumbar Spine Z-Score: 2 years (n=26)Left Femur Z-Score: Baseline (n=107)Left Femur Z-Score: 1 year (n=95)Left Femur Z-Score: 2 years (n=22)Right Femur Z-Score: Baseline (n=103)Right Femur Z-Score: 1 year (n=91)Right Femur Z-Score: 2 years (n=21)
LTTP: Eliglustat-0.460-0.512-0.385-0.164-0.132-0.264-0.214-0.262-0.605

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PAP: Bone Mineral Density (BMD) at Baseline and Week 52

BMD measurements of the spine and bilateral femur were acquired by dual-energy x-ray absorptiometry (DXA) scan. (NCT01074944)
Timeframe: Baseline, Week 52

,
Interventiong/cm^2 (Mean)
Lumbar Spine: Baseline (n=51, 55)Lumbar Spine: Week 52 (n=51, 55)Left Femur: Baseline (n=48, 47)Left Femur: Week 52 (n=48, 47)Right Femur: Baseline (n=48, 47)Right Femur: Week 52 (n=48, 47)
PAP, Eliglustat: Once Daily1.0731.0890.9790.9720.9710.967
PAP, Eliglustat: Twice Daily1.0811.0861.0000.9900.9960.981

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PAP: Mean Biomarker (Chitotriosidase) Value at Baseline, Weeks 26 and Week 52

Chitotriosidase biomarker was assayed from plasma. (NCT01074944)
Timeframe: Baseline, Week 26, Week 52

,
Interventionnmol/hr/mL (Mean)
Baseline (n=55, 59)Week 26 (n=52, 54)Week 52 (n=54, 55)
PAP, Eliglustat: Once Daily1523.71279.61076.6
PAP, Eliglustat: Twice Daily1554.91242.01170.1

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PAP: Mean Hemoglobin (Hb) Level at Baseline, Weeks 26 and 52

(NCT01074944)
Timeframe: Baseline, Week 26, Week 52

,
Interventiong/dL (Mean)
Baseline (n=56, 59)Week 26 (n=56, 57)Week 52 (n=56, 59)
PAP, Eliglustat: Once Daily13.64113.67713.605
PAP, Eliglustat: Twice Daily13.69113.94613.824

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PAP: Mean Biomarker Macrophage Inflammatory Protein-1 Beta (MIP1-beta) Value at Baseline, Weeks 26, 52

MIP1-beta biomarker was assayed from plasma. (NCT01074944)
Timeframe: Baseline, Week 26, Week 52

,
Interventionpg/mL (Mean)
Baseline (n=54, 58)Week 26 (n=52, 54)Week 52 (n=54, 55)
PAP, Eliglustat: Once Daily77.774.581.3
PAP, Eliglustat: Twice Daily118.8121.0117.9

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PAP: Mean Liver Volume at Baseline, Weeks 26, 52

(NCT01074944)
Timeframe: Baseline, Week 26 and Week 52

,
InterventionMN (Mean)
Baseline (n=56, 59)Week 26 (n=56, 59)Week 52 (n=56, 59)
PAP, Eliglustat: Once Daily0.9810.9870.970
PAP, Eliglustat: Twice Daily1.0401.0241.009

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PAP: Mean Platelet Count at Baseline, Weeks 26, 52

(NCT01074944)
Timeframe: Baseline, Week 26, Week 52

,
Interventionplatelets*10^9 /L (Mean)
Baseline (n=56, 59)Week 26 (n=56, 57)Week 52 (n=56, 59)
PAP, Eliglustat: Once Daily204.01195.75207.20
PAP, Eliglustat: Twice Daily171.09173.94176.10

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PAP: Mean Spleen Volume at Baseline, Weeks 26, 52

(NCT01074944)
Timeframe: Baseline, Week 26, Week 52

,
InterventionMN (Mean)
Baseline (n= 39, 45)Week 26 (n= 39, 45)Week 52 (n= 39, 45)
PAP, Eliglustat: Once Daily3.3093.0663.017
PAP, Eliglustat: Twice Daily3.7873.5043.394

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PAP: Number of Participants With Bone Crises at Baseline, Weeks 26 and 52

Bone crisis was assessed as a part of Gaucher disease assessment in participants. Acute, excruciating episodic bone pain is characteristic of Gaucher bone crisis, which typically causes debilitation lasting several days or longer and requires treatment with immobilization, hydration, and opioid analgesics. Participants were categorized as 0= no bone crisis, 1= 1 bone crisis, and 2= 2 bone crises during the assessment period. In this outcome, number of participants with different bone crises levels at specified time points were reported. (NCT01074944)
Timeframe: Baseline, Week 26, and Week 52

,
Interventionparticipants (Number)
Bone Crisis (0): Baseline (n=56, 59)Bone Crisis (0): Week 26 (n=55,57)Bone Crisis (0): Week 52 (n=56, 58)Bone Crisis (1): Baseline (n=56, 59)Bone Crisis (1): Week 26 (n=55, 57)Bone Crisis (1): Week 52 (n=56, 58)Bone Crisis (2): Baseline (n=56, 59)Bone Crisis (2): week 26 (n=55, 57)Bone Crisis (2): week 52 (n=56, 58)
PAP, Eliglustat: Once Daily555456100010
PAP, Eliglustat: Twice Daily575657211000

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PAP: Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Weeks 26 and 52

Bone pain was assessed as a part of Gaucher disease assessment in participants. Participants were categorized as none (no bone pain), very mild bone pain, mild bone pain, moderate bone pain, severe bone pain and extreme bone pain during the past 4 weeks. In this outcome, number of participants with different level of bone pain during the past 4 weeks at specified time points were reported. (NCT01074944)
Timeframe: Baseline, Week 26, and Week 52

,
Interventionparticipants (Number)
None: Baseline (n=56, 59)None: Week 26 (n=55, 57)None: Week 52 (n=56, 58)Very Mild: Baseline (n=56, 59)Very Mild: Week 26 (n=55, 57)Very Mild: Week 52 (n=56, 58)Mild: Baseline (n=56, 59)Mild: Week 26 (n=55, 57)Mild: Week 52 (n=56, 58)Moderate: Baseline (n=56, 59)Moderate: Week 26 (n=55, 57)Moderate: Week 52 (56, 58)Severe: Baseline (n=56, 59)Severe: Week 26 (n=55, 57)Severe: Week 52 (n=56, 58)Extreme: Baseline (n=56, 59)Extreme: Week 26 (n=55, 57)Extreme: Week 52 (n=56, 58)
PAP, Eliglustat: Once Daily424241233756456100000
PAP, Eliglustat: Twice Daily494945147332512102000

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PAP: Number of Participants With Mobility Status Asessments (MS) at Baseline, Weeks 26, and 52.

Mobility, i.e., ability to walk was assessed as a part of Gaucher disease assessment in participants. In this outcome, number of participants with their different mobility status along with the use of mobility aids (unrestricted mobility, walks with difficulty, walks with orthopaedic aid, requires wheelchair, bedridden) at specified time points were reported. (NCT01074944)
Timeframe: Baseline, Week 26 and Week 52

,
Interventionparticipants (Number)
MS, Unrestricted: Baseline (n=56, 59)MS, Unrestricted: Week 26 (n=55, 57)MS, Unrestricted: Week 52 (n=51, 58)MS, Walks with Difficulty: Baseline (n=56, 59)MS, Walks with Difficulty: Week 26 (n=55, 57)Ms, Walks with Difficulty: Week 52 (n=51, 58)MS, Walks with Orthopedic Aid: Baseline (n=56, 59)MS, Walks with Orthopedic Aid: Week 26 (n=55, 57)MS, Walks with Orthopedic Aid: Week 52 (n=51, 58)MS, Required Wheelchair: Baseline (n=56, 59)MS, Required Wheelchair: Week 26 (n=55, 57)MS, Required Wheelchair: Week 52 (n=51, 58)MS, Bedridden: Baseline (n=56, 59)MS, Bedridden: Week 26 (n=55, 57)MS, Bedridden: Week 52 (n=51, 58)
PAP, Eliglustat: Once Daily494650685110001000
PAP, Eliglustat: Twice Daily595657011000000000

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PAP: Total Bone Marrow Burden Score (BMB) at Baseline and Week 52

BMB Score was measured using magnetic resonance imaging (MRI), range from 0 (no abnormalities) to 8 points (severe disease) for the lumbar spine and from 0 (no abnormalities) to 8 points (severe disease) for the femurs. The total score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) -16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement. (NCT01074944)
Timeframe: Baseline, Week 52

,
InterventionBMB Score (Mean)
BMB Score: Baseline (n=52, 49)BMB Score: Week 52 (n=51, 48)
PAP, Eliglustat: Once Daily8.2767.971
PAP, Eliglustat: Twice Daily9.1368.705

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PAP: Total T-Scores for BMD at Baseline and Week 52

Images of the spine and bilateral femur were obtained by DXA to determine T-score for each bone area and total bone mineral density. The T-score bone density categories were: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5). (NCT01074944)
Timeframe: Baseline, Week 52

,
InterventionT-score (Mean)
Lumbar Spine T-Score: Baseline (n=49, 52)Lumbar Spine T-Score: Week 52 (n=49, 52)Left Femur T-Score: Baseline (n=46, 44)Left Femur T-Score: Week 52 (n=46, 44)Right Femur T-score: Baseline (n=46, 44)Right Femur T-score: Week 52 (n=46, 44)
PAP, Eliglustat: Once Daily-0.722-0.580-0.459-0.509-0.574-0.607
PAP, Eliglustat: Twice Daily-0.771-0.717-0.368-0.441-0.382-0.530

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PAP: Total Z-scores for BMD at Baseline and Week 52

Images of the spine and bilateral femur were obtained by DXA to determine Z-score for each bone area and total bone mineral density. The Z-score bone density categories are: normal (score >-2) and below normal (score <=-2). (NCT01074944)
Timeframe: Baseline, Week 52

,
InterventionZ-score (Mean)
Lumbar Spine Z-scores: Baseline (n=51, 55)Lumbar Spine Z-scores: Week 52 (n=51, 55)Left Femur Z-scores: Baseline (n= 48, 47)Left Femur Z-scores: Week 52 (n= 48, 47)Right Femur Z-scores: Baseline (n= 48, 47)Right Femur Z-scores: Week 52 (n= 48, 47)
PAP, Eliglustat: Once Daily-0.492-0.324-0.171-0.202-0.235-0.248
PAP, Eliglustat: Twice Daily-0.609-0.555-0.115-0.183-0.140-0.260

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PAP: Percentage of Participants Who Remained Stable for 52 Weeks During the PAP

Participants were considered as stable if they met all of the following criteria: 1) no more than 2 bone crisis during PAP (with no more than 1 bone crisis during either the first 6 months or the later 6 months of the period), and were free of other clinically symptomatic bone disease during the entire 52-week PAP; 2) hemoglobin level not decreased >1.5 g/dL from Baseline for PAP; 3) platelet count not decreased >25% from Baseline for PAP; 4) spleen volume (in multiples of normal [MN]) did not increase >25% from Baseline for PAP; 5) liver volume (in MN) did not increase >20% from Baseline for PAP. Baseline for PAP was defined as the last assessment prior to randomization. (NCT01074944)
Timeframe: PAP Baseline up to the end of PAP (Week 52)

Interventionpercentage of participants (Number)
PAP, Eliglustat: Once Daily80.4
PAP, Eliglustat: Twice Daily83.1

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LIP: Mean Biomarker (Chitotriosidase) Value at Baseline, Weeks 26, 52, and 78

Chitotriosidase biomarker was assayed from plasma. (NCT01074944)
Timeframe: Baseline, Week 26, Week 52 and Week 78

Interventionnmol/hr/mL (Mean)
Chitotriosidase: Baseline (n=170)Chitotriosidase: week 26 (n=157)Chitotriosidase: week 52 (n=72)Chitotriosidase: week 78 (n=41)
LIP: Eliglustat2437.921802.931755.701677.02

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LIP: Mean Biomarker (GL-1 on DBS) Value at Baseline, Week 26, Week 52, and Week 78

GL-1 on DBS biomarker was assayed from dried blood spot. (NCT01074944)
Timeframe: Baseline, Week 26, Week 52 and Week 78

Interventionmcg/mL (Mean)
GL-1 on DBS: Baseline (n=159)GL-1 on DBS: Week 26 (n=144)GL-1 on DBS: Week 52 (n=68)GL-1 on DBS: Week 78 (n=39)
LIP: Eliglustat4.3582.3402.2792.495

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LIP: Mean Hemoglobin (Hb) Level at Baseline, Weeks 26, 52 and 78

(NCT01074944)
Timeframe: Baseline, Week 26, Week, 52, and Week 78

Interventiong/dL (Mean)
Baseline (n=170)Week 26 (n=163)Week 52 (n=74)Week 78 (n=41)
LIP: Eliglustat13.43513.44313.43413.329

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LIP: Mean Liver Volume at Baseline, Weeks 26, 52 and 78

(NCT01074944)
Timeframe: Baseline, Week 26, Week 52, Week 78

InterventionMN (Mean)
Baseline (n=170)Week 26 (n=149)Week 52 (n=68)Week 78 (n=39)
LIP: Eliglustat1.0441.0401.0591.062

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LIP: Mean Platelet Count at Baseline, Weeks 26, 52 and 78

(NCT01074944)
Timeframe: Baseline, Week 26, Week 52, Week 78

Interventionplatelets*10^9 /L (Mean)
Baseline (n=170)Week 26 (n=163)Week 52 (n=74)Week 78 (n=41)
LIP: Eliglustat178.653180.021176.378168.720

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LIP: Mean Spleen Volume at Baseline, Weeks 26, 52 and 78

(NCT01074944)
Timeframe: Baseline, Week 26, Week 52, Week 78

InterventionMN (Mean)
Baseline (n=119)Week 26 (n=106)Week 52 (n=52)Week 78 (n=30)
LIP: Eliglustat4.4483.8404.0944.088

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LIP: Number of Participants With Bone Crises Assessment at Baseline, Weeks 26, 52 and 78

Bone crises was assessed as a part of Gaucher disease assessment in participants. Acute, excruciating episodic bone pain is characteristic of Gaucher bone crises, which typically causes debilitation lasting several days or longer and requires treatment with immobilization, hydration, and opioid analgesics. Participants were categorized as 0= no bone crisis, 1= 1 bone crisis, 2= 2 bone crises, 6= 6 bone crises, and 24= 24 bone crises during the assessment period. In this outcome, number of participants with different bone crises levels at specified time points were reported. (NCT01074944)
Timeframe: Baseline, Week 26, Week 52, Week 78

Interventionparticipants (Number)
Bone Crisis (0): Baseline (n=162)Bone Crisis (0): Week 26 (n=162)Bone Crisis (0): Week 52 (n=72)Bone Crisis (0): Week 78 (n=41)Bone Crisis (1): Baseline (n=162)Bone Crisis (1): Week 26 (n=162)Bone Crisis (1): Week 52 (n=72)Bone Crisis (1): Week 78 (n=41)Bone Crisis (2): Baseline (n=162)Bone Crisis (2): Week 26 (n=162)Bone Crisis (2): Week 52 (n=72)Bone Crisis (2): Week 78 (n=41)Bone Crisis (6): Baseline (n=162)Bone Crisis (6): Week 26 (n=162)Bone Crisis (6): Week 52 (n=72)Bone Crisis (6): Week 78 (n=41)Bone Crisis (24): Baseline (n=162)Bone Crisis (24): Week 26 (n=162)Bone Crisis (24): Week 52 (n=72)Bone Crisis (24): Week 78 (n=41)
LIP: Eliglustat15115972418300100010001000

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LTTP: Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, 1 Year, and 2 Years

Bone pain was assessed as a part of Gaucher disease assessment in participants. Participants were categorized as none (no bone pain), very mild bone pain, mild bone pain, moderate bone pain, severe bone pain and extreme bone pain. In this outcome, number of participants with different level of bone pain during the past 4 weeks at specified time points were reported. (NCT01074944)
Timeframe: Baseline, 1 year and 2 years

Interventionparticipants (Number)
None: Baseline (n=120)None: 1 year (n=104)None: 2 years (n=32)Very Mild: Baseline (n=120)Very Mild: 1 year (n=104)Very Mild: 2 years (n=32)Mild: Baseline (n=120)Mild: 1 year (n=104)Mild: 2 years (n=32)Moderate: Baseline (n=120)Moderate: 1 year (n=104)Moderate: 2 years (n=32)Severe: Baseline (n=120)Severe: 1 year (n=104)Severe: 2 years (n=32)Extreme: Baseline (n=120)Extreme: 1 year (n=104)Extreme: 2 years (n=32)
LTTP: Eliglustat91833212908100710200010

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LIP: Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Weeks 26, 52 and 78

Bone pain was assessed as a part of Gaucher disease assessment in participants. Participants were categorized as none (no bone pain), very mild bone pain, mild bone pain, moderate bone pain, severe bone pain and extreme bone pain. In this outcome, number of participants with different type of bone pain during the past 4 weeks at specified time points were reported. (NCT01074944)
Timeframe: Baseline, Week 26, Week 52, Week 78

Interventionparticipants (Number)
None: Baseline (n=163)None: Week 26 (n=161)None: Week 52 (n=72)None: Week 78 (n=41)Very Mild: Baseline (n=163)Very Mild: Week 26 (n=161)Very Mild: Week 52 (n=72)Very Mild: Week 78 (n=41)Mild: Baseline (n=163)Mild: Week 26 (n=161)Mild: Week 52 (n=72)Mild: Week 78 (n=41)Moderate: Baseline (n=163)Moderate: Week 26 (n=161)Moderate: Week 52 (n=72)Moderate: Week 78 (n=41)Severe: Baseline (n=163)Severe: Week 26 (n=161)Severe: Week 52 (n=72)Severe: Week 78 (n=41)Extreme: Baseline (n=163)Extreme: Week 26 (n=161)Extreme: Week 52 (n=72)Extreme: Week 78 (n=41)
LIP: Eliglustat11212562391714412210308103142000000

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LIP: Number of Participants With Mobility Status (MS) at Baseline, Weeks 26, 52 and 78

Mobility, i.e., ability to walk was assessed as a part of Gaucher disease assessment in participants. In this outcome, number of participants with their different mobility status along with the use of mobility aids (unrestricted mobility, walks with difficulty, walks with orthopaedic aid, requires wheelchair, bedridden) at specified time points were reported. (NCT01074944)
Timeframe: Baseline, Week 26, Week 52, Week 78

Interventionparticipants (Number)
MS,Unrestricted: Baseline (n=163)MS,Unrestricted: Week 26 (n=161)MS,Unrestricted: Week 52 (n=72)MS,Unrestricted: Week 78 (n= 41)MS, Walks With Difficulty: Baseline (n=163)MS, Walks With Difficulty: Week 26 (n=161)MS, Walks With Difficulty: Week 52 (n=72)MS, Walks With Difficulty: Week 78 (n=41)MS, Walks With Orthopedic Aid: Baseline (n=163)MS, Walks With Orthopedic Aid: Week 26 (n=161)MS, Walks With Orthopedic Aid: Week 52 (n=72)MS, Walks With Orthopedic Aid: Week 78 (n=41)MS, Required wheelchair: Baseline (n=163)MS, Required wheelchair: Week 26 (n=161)MS, Required wheelchair: Week 52 (n=72)MS, Required wheelchair: Week 78 (n=41)MS, Bedridden: Baseline (n=163)MS, Bedridden: Week 26 (n=161)MS, Bedridden: Week 52 (n=72)MS, Bedridden: Week 78 (n=41)
LIP: Eliglustat146153703912612310021100000

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LTTP: Bone Mineral Density (BMD) at Baseline, 1 Year, and 2 Years

BMD measurements of the spine and bilateral femur were acquired by DXA scan. (NCT01074944)
Timeframe: Baseline, 1 year, and 2 years

Interventiong/cm^2 (Mean)
Lumbar Spine: Baseline (n=113)Lumbar Spine: 1 year (n=101)Lumbar Spine: 2 years (n=26)Left Femur: Baseline (n=107)Left Femur: 1 year (n=95)Left Femur: 2 years (n=22)Right Femur: Baseline (n=103)Right Femur: 1 year (n=91)Right Femur: 2 years (n=21)
LTTP: Eliglustat1.0871.0831.0820.9860.9940.9500.9830.9790.908

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PAP: Mean Biomarker (GL-1 on DBS) Value at Baseline, Week 26 and Week 52

GL-1 on DBS biomarker was assayed from dried blood spot (DBS). (NCT01074944)
Timeframe: Baseline, Week 26 and week 52

,
Interventionmcg/mL (Mean)
GL-1 on DBS: Baseline (n=54, 55)GL-1 on DBS: week 26 (n=54, 54)GL-1 on DBS: week 52 (n=53, 55)
PAP, Eliglustat: Once Daily2.2572.4812.853
PAP, Eliglustat: Twice Daily2.4252.5632.707

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LTTP: Mean Biomarker (Chitotriosidase) Value at Baseline, 1 Year, and 2 Years

Chitotriosidase biomarker was assayed from plasma. (NCT01074944)
Timeframe: Baseline, 1 year, and 2 years

Interventionnmol/hr/mL (Mean)
Chitotriosidase: Baseline (n=118)Chitotriosidase: 1 year (n=97))Chitotriosidase: 2 years (n=31)
LTTP: Eliglustat1188.9831221.753598.161

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LTTP: Mean Biomarker (GL-1 on DBS) Value at Baseline, 1 Year, and 2 Years

GL-1 on DBS biomarker was assayed from dried blood spot. (NCT01074944)
Timeframe: Baseline, 1 year, and 2 years

Interventionmcg/mL (Mean)
GL-1 on DBS: Baseline (n=114)GL-1 on DBS: 1 year (n=98)GL-1 on DBS: 2 years (n=29)
LTTP: Eliglustat2.7252.5002.238

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LTTP: Mean Biomarker (MIP1-beta) Value at Baseline, 1 Year, and 2 Years

MIP1-beta biomarker was assayed from plasma. (NCT01074944)
Timeframe: Baseline, 1 year, and 2 years

Interventionpg/mL (Mean)
MIP-1beta: Baseline (n=114)MIP-1beta: 1 year (n=94)MIP-1beta: 2 years (n=31)
LTTP: Eliglustat97.85790.39868.445

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LTTP: Number of Participants With Bone Crises Assessment at Baseline, 1 Year and 2 Years

Bone crises was assessed as a part of Gaucher disease assessment in participants. Acute, excruciating episodic bone pain is characteristic of Gaucher bone crises, which typically causes debilitation lasting several days or longer and requires treatment with immobilization, hydration, and opioid analgesics. Participants were categorized as 0= no bone crises, 1= 1 bone crisis during the assessment period. In this outcome, number of participants with different bone crises levels at specified time points were reported. (NCT01074944)
Timeframe: Baseline, 1 year and 2 years

Interventionparticipants (Number)
Bone Crisis (0): Baseline (n=120)Bone Crisis (0): 1 year (n=104)Bone Crisis (0): 2 years (n=32)Bone Crisis (1): Baseline (n=120)Bone Crisis (1): 1 year (n=104)Bone Crisis (1): 2 years (n=32)
LTTP: Eliglustat11910432100

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Change From Current Study Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234

MIP-1β considered a biomarker of active bone disease, was assayed from plasma. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. (NCT02536755)
Timeframe: Study Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234

Interventionpicograms per milliliter (pg/mL) (Mean)
Study BaselineChange at Week 26Change at Week 52Change at Week 78Change at Week 104Change at Week 130Change at Week 156Change at Week 182Change at Week 208Change at Week 234
Eliglustat175.8296-36.0593-31.4692-16.6818-33.0217897.16801008.84001007.98801381.66526721.9000

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Change From Current Study Baseline in Bone Biomarker Level: Procollagen 1 N- Terminal Propeptide (P1NP) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234

P1NP, a marker of bone formation was assayed from plasma. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. (NCT02536755)
Timeframe: Study Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234

Interventionmicrograms per liter (mcg/L) (Mean)
Study BaselineChange at Week 26Change at Week 52Change at Week 78Change at Week 104Change at Week 130Change at Week 156Change at Week 182Change at Week 208Change at Week 234
Eliglustat44.3960-0.69472.09173.61331.63000.1675-1.90431.66320.13543.4467

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Change From Current Study Baseline in Bone Biomarker Level: Type 1 Collagen C-Telopeptides (CTx) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234

CTx, a marker of bone resorption was assayed from plasma. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. (NCT02536755)
Timeframe: Study Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234

Interventionmcg/L (Mean)
Study BaselineChange at Week 26Change at Week 52Change at Week 78Change at Week 104Change at Week 130Change at Week 156Change at Week 182Change at Week 208Change at Week 234
Eliglustat0.4540-0.0363-0.0034-0.00410.0000-0.0426-0.07380.0011-0.01760.1200

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Change From Current Study Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Chitotriosidase biomarker was assayed from plasma. Chitotriosidase biomarker levels for participants who were CYP2D6 non-Ultra Rapid Metabolizers (non-URM) was reported in this outcome measure. For this outcome measure, baseline refers to the study baseline, which was defined as status at study entry. (NCT02536755)
Timeframe: Study Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Interventionnmol/hr/mL (Mean)
Study BaselineChange at Week 26Change at Week 52Change at Week 78Change at Week 104Change at Week 130Change at Week 156Change at Week 182Change at Week 208Change at Week 234
Eliglustat4039.3333-411.2333-308.6897-492.4286-709.2143-712.4643-367.9286-582.1429-334.1538411.7692

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Change From Current Study Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Glucosylceramide (GL-1) biomarker was assayed from plasma. GL-1 biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. (NCT02536755)
Timeframe: Study Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Interventionmicrograms per milliliter (mcg/mL) (Mean)
Study BaselineChange at Week 26Change at Week 52Change at Week 78Change at Week 104Change at Week 130Change at Week 156Change at Week 182Change at Week 208Change at Week 234
Eliglustat5.3243-1.7770-1.7002-1.4000-2.0657-1.7405-1.6073-1.7304-2.04080.7691

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Change From Current Study Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Lyso-GL-1 biomarker was assayed from plasma. Lyso-GL-1 biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. (NCT02536755)
Timeframe: Study Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Interventionnanograms per milliliter (ng/mL) (Mean)
Study BaselineChange at Week 26Change at Week 52Change at Week 78Change at Week 104Change at Week 130Change at Week 156Change at Week 182Change at Week 208Change at Week 234
Eliglustat114.2429-43.8238-40.7500-30.6711-35.5721-33.6195-27.6795-32.3611-31.2882-45.5100

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Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234

The 36-Item Short-Form Health Survey (SF-36) is standardized survey evaluating 8 aspects of functional health and well-being. Physical Component Summary (PCS) with 4 sub-scales: physical function, role limitations due to physical problems, bodily pain, and general health perception; and Mental Component Summary (MCS) with 4 sub-scales: vitality, social function, role limitations due to emotional problems, and mental health. Summations of item scores of the same sub-scale give the sub-scale scores, which are transformed into a range from 0 to 100; 0= worst and 100=best outcome. Both PCS and MCS range from 0 to 100 with higher scores indicating better physical and mental health. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. (NCT02536755)
Timeframe: Study Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234

Interventionscore on a scale (Mean)
Study Baseline: PCSPCS: Week 26PCS: Change at Week 26PCS: Week 52PCS: Change at Week 52PCS: Week 78PCS: Change at Week 78PCS: Week 104PCS: Change at Week 104PCS: Week 130PCS: Change at Week 130PCS: Week 156PCS: Change at Week 156PCS: Week 182PCS: Change at Week 182PCS: Week 208PCS: Change at Week 208PCS: Week 234PCS: Change at Week 234Study Baseline: MCSMCS: Week 26MCS: Change at Week 26MCS: Week 52MCS: Change at Week 52MCS: Week 78MCS: Change at Week 78MCS: Week 104MCS: Change at Week 104MCS: Week 130MCS: Change at Week 130MCS: Week 156MCS: Change at Week 156MCS: Week 182MCS: Change at Week 182MCS: Week 208MCS: Change at Week 208MCS: Week 234MCS: Change at Week 234
Eliglustat52.879553.30520.425852.8240-0.055453.0769-0.543153.1225-0.497552.2996-1.320351.6359-1.984152.4094-1.210552.2733-1.346652.5847-1.028449.622351.74972.127450.15020.527951.0151-0.241250.0147-1.241551.61440.358251.50300.246852.15950.903349.2308-2.025447.7470-3.9989

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Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208

"BMD measurements of the spine and bilateral femur were acquired by DXA scan. The T-score bone density categories were: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5). Worst total femur at Baseline refers to the worst diseased left or right femur at baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry." (NCT02536755)
Timeframe: Study Baseline, Weeks 52, 104, 156 and 208

InterventionT-score (Mean)
Total Spine T-score: Study BaselineTotal Spine T-score: Week 52Total Spine T-score: Change at Week 52Total Spine T-score: Week 104Total Spine T-score: Change at Week 104Total Spine T-score: Week 156Total Spine T-score: Change at Week 156Total Spine T-score: Week 208Total Spine T-score: Change at Week 208Worst Total Femur T-score: Study BaselineWorst Total Femur T-score: Week 52Worst Total Femur T-score: Change at Week 52Worst Total Femur T-score: Week 104Worst Total Femur T-score: Change at Week 104Worst Total Femur T-score: Week 156Worst Total Femur T-score: Change at Week 156Worst Total Femur T-score: Week 208Worst Total Femur T-score: Change at Week 208
Eliglustat-0.4413-0.4362-0.0250-0.5566-0.0479-0.5152-0.0066-0.6238-0.10120.0119-0.01190.0273-0.1345-0.0245-0.10320.0179-0.1196-0.0073

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Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208

"BMD measurements of the spine and bilateral femur were acquired by DXA scan. The Z-score bone density categories were: normal (score >-2) and below normal (score <=-2). Worst total femur at Baseline refers to the worst diseased left or right femur at baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry." (NCT02536755)
Timeframe: Study Baseline, Weeks 52, 104, 156 and 208

InterventionZ-score (Mean)
Total Spine Z-score: Study BaselineTotal Spine Z-score: Week 52Total Spine Z-score: Change at Week 52Total Spine Z-score: Week 104Total Spine Z-score: Change at Week 104Total Spine Z-score: Week 156Total Spine Z-score: Change at Week 156Total Spine Z-score: Week 208Total Spine Z-score: Change at Week 208Worst Total Femur Z-score: Study BaselineWorst Total Femur Z-score: Week 52Worst Total Femur Z-score: Change at Week 52Worst Total Femur Z-score: Week 104Worst Total Femur Z-score: Change at Week 104Worst Total Femur Z-score: Week 156Worst Total Femur Z-score: Change at Week 156Worst Total Femur Z-score: Week 208Worst Total Femur Z-score: Change at Week 208
Eliglustat-0.3687-0.3338-0.0092-0.4555-0.0245-0.40340.0276-0.5035-0.05190.19350.21960.05040.09210.01140.15210.07570.14080.0746

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Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208

"Bone Mineral Density (BMD) measurements of the spine and bilateral femur were acquired by dual energy X-Ray absorptiometry (DXA) scan. Worst total femur at Baseline refers to the worst diseased left or right femur at Baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry." (NCT02536755)
Timeframe: Study Baseline, Weeks 52, 104, 156 and 208

Interventiongrams per centimeter square (g/cm^2) (Mean)
Total Spine BMD: Study BaselineTotal Spine BMD: Week 52Total Spine BMD: Change at Week 52Total Spine BMD: Week 104Total Spine BMD: Change at Week 104Total Spine BMD: Week 156Total Spine BMD: Change at Week 156Total Spine BMD: Week 208Total Spine BMD: Change at Week 208Worst Total Femur BMD: Study BaselineWorst Total Femur BMD: Week 52Worst Total Femur BMD: Change at Week 52Worst Total Femur BMD: Week 104Worst Total Femur BMD: Change at Week 104Worst Total Femur BMD: Week 156Worst Total Femur BMD: Change at Week 156Worst Total Femur BMD: Week 208Worst Total Femur BMD: Change at Week 208
Eliglustat1.14491.1476-0.00291.1326-0.00551.1373-0.00071.1236-0.01201.05671.05380.00351.0416-0.00301.04370.00211.0412-0.0015

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Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234

MIP-1β considered a biomarker of active bone disease, was assayed from plasma. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. (NCT02536755)
Timeframe: Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 of the current study

Interventionpg/mL (Mean)
Eliglustat BaselineChange at Week 26Change at Week 52Change at Week 78Change at Week 104Change at Week 130Change at Week 156Change at Week 182Change at Week 208Change at Week 234
Eliglustat: Participants From Phase 2, ENGAGE and EDGE265.3944-136.2167-153.7647-151.1857-152.8875-159.4750-141.7750-145.6750-149.8933-222.6857

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Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234

MIP-1β considered a biomarker of active bone disease, was assayed from plasma. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. (NCT02536755)
Timeframe: Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 of the current study

Interventionpg/mL (Mean)
Eliglustat BaselineChange at Week 26Change at Week 52Change at Week 78Change at Week 104Change at Week 130Change at Week 156Change at Week 182Change at Week 208
Eliglustat: Participants From ENCORE103.775066.775079.075037.700074.675053.625068.900046.650067.2500

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Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Chitotriosidase biomarker was assayed from plasma. Chitotriosidase biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT). (NCT02536755)
Timeframe: Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study

Interventionnmol/hr/mL (Mean)
Eliglustat BaselineChange at Week 26Change at Week 52Change at Week 78Change at Week 104Change at Week 130Change at Week 156Change at Week 182Change at Week 208Change at Week 234
Eliglustat: Participants From Phase 2, ENGAGE and EDGE9507.9630-6054.5185-6207.9615-6475.0800-6704.6000-6663.5200-6335.6000-6543.0000-6380.3913-7212.0769

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Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Glucosylceramide (GL-1) biomarker was assayed from plasma. GL-1 biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT). (NCT02536755)
Timeframe: Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study

Interventionmcg/mL (Mean)
Eliglustat BaselineChange at Week 26Change at Week 52Change at Week 78Change at Week 104Change at Week 130Change at Week 156Change at Week 182Change at Week 208Change at Week 234
Eliglustat: Participants From Phase 2, ENGAGE and EDGE11.8304-8.2095-8.0970-7.7498-8.6111-8.5354-8.1772-8.0321-8.5976-7.9463

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Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Glucosylceramide (GL-1) biomarker was assayed from plasma. GL-1 biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT). (NCT02536755)
Timeframe: Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study

Interventionmcg/mL (Mean)
Eliglustat BaselineChange at Week 26Change at Week 52Change at Week 78Change at Week 104Change at Week 130Change at Week 156Change at Week 182Change at Week 208
Eliglustat: Participants From ENCORE5.9667-2.2470-2.9880-3.1150-3.6920-3.2767-1.8643-3.9720-4.2390

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Change From Eliglustat Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Lyso-GL-1 biomarker was assayed from plasma. Lyso-GL-1 biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT). (NCT02536755)
Timeframe: Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study

Interventionng/mL (Mean)
Eliglustat BaselineChange at Week 26Change at Week 52Change at Week 78Change at Week 104Change at Week 130Change at Week 156Change at Week 182Change at Week 208Change at Week 234
Eliglustat: Participants From Phase 2, ENGAGE and EDGE612.2222-548.3111-537.1222-522.6833-516.3022-516.9889-511.6000-516.5111-495.3500-590.8100

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Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234

SF-36 is a standardized survey evaluating 8 aspects of functional health and well-being. PCS with 4 sub-scales: physical function, role limitations due to physical problems, pain, and general health perception; and MCS with 4 sub-scales: vitality, social function, role limitations due to emotional problems, and mental health. Summations of item scores of same sub-scale give the sub-scale scores, which are transformed into range from 0 to 100; 0= worst, and 100=best outcome. Both PCS and MCS range from 0 to 100, higher scores indicating better physical and mental health. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. (NCT02536755)
Timeframe: Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study

Interventionscore on a scale (Mean)
Study Baseline: PCSPCS: Week 26PCS: Change at Week 26PCS: Week 52PCS: Change at Week 52PCS: Week 78PCS: Change at Week 78PCS: Week 104PCS: Change at Week 104PCS: Week 130PCS: Change at Week 130PCS: Week 156PCS: Change at Week 156PCS: Week 182PCS: Change at Week 182PCS: Week 208PCS: Change at Week 208Study Baseline: MCSMCS: Week 26MCS: Change at Week 26MCS: Week 52MCS: Change at Week 52MCS: Week 78MCS: Change at Week 78MCS: Week 104MCS: Change at Week 104MCS: Week 130MCS: Change at Week 130MCS: Week 156MCS: Change at Week 156MCS: Week 182MCS: Change at Week 182MCS: Week 208MCS: Change at Week 208
Eliglustat: Participants From ENCORE42.781755.058912.277252.33799.556252.63639.854654.998312.216652.57149.789751.66718.885451.70478.922951.34008.558242.496045.05522.559247.17154.675650.07407.578041.0384-1.457645.48262.986647.51655.020548.04395.547944.18841.6924

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Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234

SF-36 is a standardized survey evaluating 8 aspects of functional health and well-being. PCS with 4 sub-scales: physical function, role limitations due to physical problems, pain, and general health perception; and MCS with 4 sub-scales: vitality, social function, role limitations due to emotional problems, and mental health. Summations of item scores of same sub-scale give the sub-scale scores, which are transformed into range from 0 to 100; 0= worst, and 100=best outcome. Both PCS and MCS range from 0 to 100, higher scores indicating better physical and mental health. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. (NCT02536755)
Timeframe: Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study

Interventionscore on a scale (Mean)
Study Baseline: PCSPCS: Week 26PCS: Change at Week 26PCS: Week 52PCS: Change at Week 52PCS: Week 78PCS: Change at Week 78PCS: Week 104PCS: Change at Week 104PCS: Week 130PCS: Change at Week 130PCS: Week 156PCS: Change at Week 156PCS: Week 182PCS: Change at Week 182PCS: Week 208PCS: Change at Week 208PCS: Week 234PCS: Change at Week 234Study Baseline: MCSMCS: Week 26MCS: Change at Week 26MCS: Week 52MCS: Change at Week 52MCS: Week 78MCS: Change at Week 78MCS: Week 104MCS: Change at Week 104MCS: Week 130MCS: Change at Week 130MCS: Week 156MCS: Change at Week 156MCS: Week 182MCS: Change at Week 182MCS: Week 208MCS: Change at Week 208MCS: Week 234MCS: Change at Week 234
Eliglustat: Participants From Phase 2, ENGAGE and EDGE43.057853.04549.447952.89619.058153.14749.636552.82249.093952.25618.936451.63098.158052.52229.558752.42269.288652.58478.725241.831252.74159.944150.59158.006051.16566.254351.45106.976452.59547.780752.14088.071452.81808.558250.03765.944947.74704.0884

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Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208

"BMD measurements of the spine and bilateral femur were acquired by DXA scan. The T-score bone density categories were: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5). Worst total femur at Baseline refers to the worst diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study." (NCT02536755)
Timeframe: Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study

InterventionT-score (Mean)
Total Spine T-score: Eliglustat BaselineTotal Spine T-score: Week 52Total Spine T-score: Week 104Total Spine T-score: Change at Week 104Total Spine T-score: Week 156Total Spine T-score: Change at Week 156Total Spine T-score: Week 208Total Spine T-score: Change at Week 208Worst Total Femur T-score: Eliglustat BaselineWorst Total Femur T-score: Week 52Worst Total Femur T-score: Change at Week 52Worst Total Femur T-score: Week 104Worst Total Femur T-score: Change at Week 104Worst Total Femur T-score: Week 156Worst Total Femur T-score: Change at Week 156Worst Total Femur T-score: Week 208Worst Total Femur T-score: Change at Week 208
Eliglustat: Participants From ENCORE-0.3000-0.7950-0.40500.5200-0.50250.4300-0.37000.73001.10000.3800-0.02000.8200-0.28000.9050-0.19501.3500-0.4500

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Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208

"BMD measurements of the spine and bilateral femur were acquired by DXA scan. The T-score bone density categories were: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5). Worst total femur at Baseline refers to the worst diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study." (NCT02536755)
Timeframe: Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study

InterventionT-score (Mean)
Total Spine T-score: Eliglustat BaselineTotal Spine T-score: Week 52Total Spine T-score: Change at Week 52Total Spine T-score: Week 104Total Spine T-score: Change at Week 104Total Spine T-score: Week 156Total Spine T-score: Change at Week 156Total Spine T-score: Week 208Total Spine T-score: Change at Week 208Worst Total Femur T-score: Eliglustat BaselineWorst Total Femur T-score: Week 52Worst Total Femur T-score: Change at Week 52Worst Total Femur T-score: Week 104Worst Total Femur T-score: Change at Week 104Worst Total Femur T-score: Week 156Worst Total Femur T-score: Change at Week 156Worst Total Femur T-score: Week 208Worst Total Femur T-score: Change at Week 208
Eliglustat: Participants From Phase 2, ENGAGE and EDGE-0.9381-0.40630.5126-0.58080.5215-0.51720.6150-0.65700.5544-0.01900.11420.0616-0.09500.0400-0.06150.0735-0.08670.1022

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Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208

"BMD measurements of the spine and bilateral femur were acquired by DXA scan. The Z-score bone density categories are: normal (score >-2) and below normal (score <=-2). Worst total femur at Baseline refers to the worst diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study." (NCT02536755)
Timeframe: Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study

,
InterventionZ-score (Mean)
Total Spine Z-score: Eliglustat BaselineTotal Spine Z-score: Week 52Total Spine Z-score: Change at Week 52Total Spine Z-score: Week 104Total Spine Z-score: Change at Week 104Total Spine Z-score: Week 156Total Spine Z-score: Change at Week 156Total Spine Z-score: Week 208Total Spine Z-score: Change at Week 208Worst Total Femur Z-score: Eliglustat BaselineWorst Total Femur Z-score: Week 52Worst Total Femur Z-score: Change at Week 52Worst Total Femur Z-score: Week 104Worst Total Femur Z-score: Change at Week 104Worst Total Femur Z-score: Week 156Worst Total Femur Z-score: Change at Week 156Worst Total Femur Z-score: Week 208Worst Total Femur Z-score: Change at Week 208
Eliglustat: Participants From ENCORE-0.5750-0.79500.0550-0.40500.1700-0.50250.0725-0.37000.09670.50000.11000.01000.3375-0.16250.3975-0.10250.2700-0.2633
Eliglustat: Participants From Phase 2, ENGAGE and EDGE-0.8826-0.29540.5030-0.46360.5186-0.38760.6276-0.52090.57160.08260.34750.18250.14290.15710.18760.20190.17580.2495

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Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208

Bone marrow burden (BMB) scores indicate the degree of bone marrow infiltration was measured using MRI, ranged from 0 (no abnormalities) to 8 points (severe disease) for the lumbar spine and from 0 (no abnormalities) to 8 points (severe disease) for the femurs. The total BMB score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) to 16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. (NCT02536755)
Timeframe: Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study

,
Interventionscore on a scale (Mean)
Total BMB Score: Eliglustat BaselineTotal BMB Score: Week 52Total BMB Score: Change at Week 52Total BMB Score: Week 104Total BMB Score: Change at Week 104Total BMB Score: Week 156Total BMB Score: Change at Week 156Total BMB Score: Week 208Total BMB Score: Change at Week 208
Eliglustat: Participants From ENCORE6.83338.83332.000010.08333.250010.66673.33339.77783.3333
Eliglustat: Participants From Phase 2, ENGAGE and EDGE10.42868.4533-1.71798.9420-0.18188.6250-1.24248.7879-1.0606

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Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208

"BMD measurements of the spine and bilateral femur were acquired by DXA scan. Worst total femur at Baseline refers to the worst diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study." (NCT02536755)
Timeframe: Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study

,
Interventiong/cm^2 (Mean)
Total Spine BMD: Eliglustat BaselineTotal Spine BMD: Week 52Total Spine BMD: Change at Week 52Total Spine BMD: Week 104Total Spine BMD: Change at Week 104Total Spine BMD: Week 156Total Spine BMD: Change at Week 156Total Spine BMD: Week 208Total Spine BMD: Change at Week 208Worst Total Femur BMD: Eliglustat BaselineWorst Total Femur BMD: Week 52Worst Total Femur BMD: Change at Week 52Worst Total Femur BMD: Week 104Worst Total Femur BMD: Change at Week 104Worst Total Femur BMD: Week 156Worst Total Femur BMD: Change at Week 156Worst Total Femur BMD: Week 208Worst Total Femur BMD: Change at Week 208
Eliglustat: Participants From ENCORE1.13501.12450.01951.16150.02651.14980.01481.17570.01901.16001.0853-0.00801.1260-0.03401.1328-0.02731.1430-0.0503
Eliglustat: Participants From Phase 2, ENGAGE and EDGE1.07571.14950.05821.12800.06001.13540.07121.11680.06161.03301.06430.01691.03460.01181.03800.01521.03040.0198

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Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208

Bone crisis was assessed as a part of Gaucher disease assessment in participants. Acute, excruciating episodic bone pain is characteristic of Gaucher bone crisis, which typically causes periosteal elevation, elevated white blood cell count, fever, or debilitation lasting several days or longer and requires treatment with immobilization of the affected area, and opioid analgesics. Participants were categorized as 0= no bone crisis, 1= 1 bone crisis, 2= 2 bone crisis and >=3 = more than 3 bone crisis during the assessment period. In this outcome measure, number of participants with different bone crises levels at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. (NCT02536755)
Timeframe: Study Baseline, Weeks 52, 104, 156 and 208

InterventionParticipants (Count of Participants)
Bone Crisis (0): Study BaselineBone Crisis (1): Study BaselineBone Crisis (2): Study BaselineBone Crisis (>=3): Study BaselineBone Crisis (0): Week 52Bone Crisis (1): Week 52Bone Crisis (2): Week 52Bone Crisis (>=3): Week 52Bone Crisis (0): Week 104Bone Crisis (1): Week 104Bone Crisis (2): Week 104Bone Crisis (>=3): Week 104Bone Crisis (0): Week 156Bone Crisis (1): Week 156Bone Crisis (2): Week 156Bone Crisis (>=3): Week 156Bone Crisis (0): Week 208Bone Crisis (1): Week 208Bone Crisis (2): Week 208Bone Crisis (>=3): Week 208
Eliglustat3100031000290002810029000

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Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208

Bone pain was assessed as a part of Gaucher disease assessment in participants. Participants were categorized as none (no bone pain), very mild bone pain, mild bone pain, moderate bone pain, severe bone pain and extreme bone pain during the past 4 weeks at each specified visit. In this outcome measure, number of participants with different level of bone pain during the past 4 weeks at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. (NCT02536755)
Timeframe: Study Baseline, Weeks 52, 104, 156 and 208

InterventionParticipants (Count of Participants)
None: Study BaselineVery Mild: Study BaselineMild: Study BaselineModerate: Study BaselineSevere: Study BaselineExtreme: Study BaselineNone: Week 52Very Mild: Week 52Mild: Week 52Moderate: Week 52Severe: Week 52Extreme: Week 52None: Week 104Very Mild: Week 104Mild: Week 104Moderate: Week 104Severe: Week 104Extreme: Week 104None: Week 156Very mild: Week 156Mild: Week 156Moderate: Week 156Severe: Week 156Extreme: Week 156None: Week 208Very Mild: Week 208Mild: Week 208Moderate: Week 208Severe: Week 208Extreme: Week 208
Eliglustat28111002910100281000029000002810000

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Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208

Mobility, i.e., ability to walk was assessed as a part of Gaucher disease assessment in participants. In this outcome measure, number of participants with their different mobility status along with the use of mobility aids (unrestricted mobility, walks with difficulty, walks with orthopaedic aid, requires wheelchair, bedridden) at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. (NCT02536755)
Timeframe: Study Baseline, Weeks 52, 104, 156 and 208

InterventionParticipants (Count of Participants)
Unrestricted mobility: Study BaselineWalks with difficulty: Study BaselineWalks with orthopedic aid: Study BaselineRequires wheelchair: Study BaselineBedridden: Study BaselineUnrestricted mobility: Week 52Walks with difficulty: Week 52Walks with orthopedic aid: Week 52Requires wheelchair: Week 52Bedridden: Week 52Unrestricted mobility: Week 104Walks with Difficulty: Week 104Walks with orthopedic aid: Week 104Requires wheelchair: Week 104Bedridden: Week 104Unrestricted mobility: Week 156Walks with difficulty: Week 156Walks with orthopedic aid: Week 156Requires wheelchair: Week 156Bedridden: Week 156Unrestricted mobility: Week 208Walks with difficulty: Week 208Walks with orthopedic aid: Week 208Requires wheelchair: Week 208Bedridden: Week 208
Eliglustat310000310000281000290000290000

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Observed Annual Incidence Rate for Spine and Femur Fracture at Week 52, 104, 156 and 208

Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Fracture was assessed by bone MRI and X-Ray for spine and by MRI for femur. (NCT02536755)
Timeframe: For 52 Weeks (i.e., 1 year),104 Weeks (i.e., 2 year), 156 Weeks (i.e., 3 year) and 208 Weeks (i.e., 4 years)

Interventionevents per participant-year (Number)
Spine Fracture (MRI): Week 52Spine Fracture (MRI): Week 104Spine Fracture(MRI): Week 156Spine Fracture (MRI): Week 208Spine Fracture (X-ray): Week 104Spine Fracture (X-ray): Week 208Femur Fracture (MRI): Week 52Femur Fracture (MRI): Week 104Femur Fracture (MRI): Week 156Femur Fracture (MRI): Week 208
Eliglustat0.13500.036600.01040.036900000

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Observed Annual Incidence Rate for Spine and Femur Infarcts at Week 52, 104, 156 and 208

Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Infarcts were assessed by bone MRI and X-Ray for spine and by MRI for femur. (NCT02536755)
Timeframe: For 52 Weeks (i.e., 1 year),104 Weeks (i.e., 2 year), 156 Weeks (i.e., 3 year) and 208 Weeks (i.e., 4 years)

Interventionevents per participant-year (Number)
Spine Infarcts (MRI): Week 52Spine Infarcts (MRI): Week 104Spine Infarcts (MRI): Week 156Spine Infarcts (MRI): Week 208Spine Infarcts (X-ray): Week 104Spine Infarcts (X-ray): Week 208Femur Infarcts (MRI): Week 52Femur Infarcts (MRI): Week 104Femur Infarcts (MRI): Week 156Femur Infarcts (MRI): Week 208
Eliglustat0000000.67520.24700.16640.1145

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Observed Annual Incidence Rate for Spine and Femur Osteonecrosis at Week 52, 104, 156 and 208

Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Osteonecrosis was assessed by bone MRI and X-Ray for spine and by MRI for femur. (NCT02536755)
Timeframe: For 52 Weeks (i.e., 1 year),104 Weeks (i.e., 2 year), 156 Weeks (i.e., 3 year) and 208 Weeks (i.e., 4 years)

Interventionevents per participant-year (Number)
Spine Osteonecrosis (MRI): Week 52Spine Osteonecrosis (MRI): Week 104Spine Osteonecrosis (MRI): Week 156Spine Osteonecrosis (MRI): Week 208Spine Osteonecrosis (X-ray): Week 104Spine Osteonecrosis (X-ray): Week 208Femur Osteonecrosis (MRI): Week 52Femur Osteonecrosis (MRI): Week 104Femur Osteonecrosis (MRI): Week 156Femur Osteonecrosis (MRI): Week 208
Eliglustat0000000.10130.03530.03840.0104

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Observed Annual Incidence Rate for Spine Lytic Lesion at Week 104, and 208

Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Lytic Lesions were assessed by bone X-Ray for spine. (NCT02536755)
Timeframe: For 104 Weeks (i.e., 2 year), and 208 Weeks (i.e., 4 years)

Interventionevents per participant-year (Number)
Spine Lytic Lesions (X-ray): Week 104Spine Lytic Lesions (X-ray): Week 208
Eliglustat00

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Total Number of New or Worsening Fracture Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208

Fracture was assessed by bone MRI and X-Ray for spine and by MRI for femur. Total number of new or worsening fracture events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. (NCT02536755)
Timeframe: Study Baseline, Weeks 52, 104, 156 and 208

Interventionevents (Mean)
Spine Fracture (MRI): Study BaselineSpine Fracture (MRI): Week 52Spine Fracture (MRI): Week 104Spine Fracture (MRI): Week 156Spine Fracture (MRI): Week 208Spine Fracture (X-ray): Study BaselineSpine Fracture (X-ray): Week 104Spine Fracture (X-ray): Week 208Femur Fracture (MRI): Study BaselineFemur Fracture (MRI): Week 52Femur Fracture (MRI): Week 104Femur Fracture (MRI): Week 156Femur Fracture (MRI): Week 208
Eliglustat0.00000.06900.00000.00000.04170.00000.07410.00000.00000.00000.00000.00000.0000

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Total Number of New or Worsening Osteonecrosis Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208

Osteonecrosis was assessed by bone MRI and X-Ray for spine and by MRI for femur. Total number of new or worsening osteonecrosis events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. (NCT02536755)
Timeframe: Study Baseline, Weeks 52, 104, 156 and 208

Interventionevents (Mean)
Spine Osteonecrosis (MRI): Study BaselineSpine Osteonecrosis (MRI): Week 52Spine Osteonecrosis (MRI): Week 104Spine Osteonecrosis (MRI): Week 156Spine Osteonecrosis (MRI): Week 208Spine Osteonecrosis (X-ray): Study BaselineSpine Osteonecrosis (X-ray): Week 104Spine Osteonecrosis (X-ray): Week 208Femur Osteonecrosis (MRI): Study BaselineFemur Osteonecrosis (MRI): Week 52Femur Osteonecrosis (MRI): Week 104Femur Osteonecrosis (MRI): Week 156Femur Osteonecrosis (MRI): Week 208
Eliglustat0.00000.00000.00000.00000.00000.00000.00000.00000.06900.03450.00000.03850.0000

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Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)

An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug and did not necessarily had to have a causal relationship with the treatment. A serious adverse event (SAE) was any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event. TEAEs were defined as AEs that developed, worsened or became serious during the treatment-emergent period (time from the first administration of the investigational medicinal product (IMP) to the last administration of the IMP + 5 days). (NCT02536755)
Timeframe: From the first administration of the IMP to the last administration of the IMP + 5 days (up to 4 years, or until commercial eliglustat was available to participants through reimbursement or through the compassionate use [expanded access] program)

InterventionParticipants (Count of Participants)
TEAEsTESAEs
Eliglustat194

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Total Number of New or Worsening Lytic Lesions Events for Spine at Study Baseline, Week 104, and 208

Lytic Lesions were assessed by bone X-Ray for spine. Total number of new or worsening lytic lesions events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. (NCT02536755)
Timeframe: Study Baseline, Weeks 104, and 208

Interventionevents (Mean)
Spine Lytic Lesions (X-ray): Study BaselineSpine Lytic Lesions (X-ray): Week 104Spine Lytic Lesions (X-ray): Week 208
Eliglustat0.03850.00000.0000

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Change From Current Study Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208

Bone marrow burden (BMB) scores indicate the degree of bone marrow infiltration. BMB score was measured using MRI (magnetic resonance imaging (MRI), ranged from 0 (no abnormalities) to 8 points (severe disease) for the lumbar spine and from 0 (no abnormalities) to 8 points (severe disease) for the femurs. The total BMB score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) to 16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. (NCT02536755)
Timeframe: Study Baseline, Weeks 52, 104, 156 and 208

Interventionscore on a scale (Mean)
Total BMB Score: Study BaselineTotal BMB Score: Week 52Total BMB Score: Change at Week 52Total BMB Score: Week 104Total BMB Score: Change at Week 104Total BMB Score: Week 156Total BMB Score: Change at Week 156Total BMB Score: Week 208Total BMB Score: Change at Week 208
Eliglustat8.66678.5057-0.02569.11111.26398.78210.71018.90670.8939

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Total Number of New or Worsening Infarcts Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208

Infarcts were assessed by bone MRI and X-Ray for spine and by MRI for femur. Total number of new or worsening infarcts events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry. (NCT02536755)
Timeframe: Study Baseline, Weeks 52, 104, 156 and 208

Interventionevents (Mean)
Spine Infarcts (MRI): Study BaselineSpine Infarcts (MRI): Week 52Spine Infarcts (MRI): Week 104Spine Infarcts (MRI): Week 156Spine Infarcts (MRI): Week 208Spine Infarcts (X-ray): Study BaselineSpine Infarcts (X-ray): Week 104Spine Infarcts (X-ray): Week 208Femur Infarcts (MRI): Study BaselineFemur Infarcts (MRI): Week 52Femur Infarcts (MRI): Week 104Femur Infarcts (MRI): Week 156Femur Infarcts (MRI): Week 208
Eliglustat0.00000.00000.00000.00000.00000.00000.00000.00000.60000.20690.00000.00000.0417

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Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234

Chitotriosidase biomarker was assayed from plasma. Chitotriosidase biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT). (NCT02536755)
Timeframe: Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study

Interventionnmol/hr/mL (Mean)
Eliglustat BaselineChange at Week 26Change at Week 52Change at Week 78Change at Week 104Change at Week 130Change at Week 156Change at Week 182Change at Week 208
Eliglustat: Participants From ENCORE2844.5000-122.5000-405.5000-1233.0000-1249.0000-981.5000-1181.0000-933.0000-1160.5000

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SubstanceRelationship StrengthStudiesTrialsClassesRoles
amiodarone
Amiodarone: An antianginal and class III antiarrhythmic drug. It increases the duration of ventricular and atrial muscle action by inhibiting POTASSIUM CHANNELS and VOLTAGE-GATED SODIUM CHANNELS. There is a resulting decrease in heart rate and in vascular resistance.. amiodarone : A member of the class of 1-benzofurans that is 1-benzofuran substituted by a butyl group at position 2 and a 4-[2-(diethylamino)ethoxy]-3,5-diiodobenzoyl group at position 3. It is a cardiovascular drug used for the treatment of cardiac dysrhythmias.		7.21101-benzofurans;
aromatic ketone;
organoiodine compound;
tertiary amino compound		cardiovascular drug
ether
Ether: A mobile, very volatile, highly flammable liquid used as an inhalation anesthetic and as a solvent for waxes, fats, oils, perfumes, alkaloids, and gums. It is mildly irritating to skin and mucous membranes.. ether : An organooxygen compound with formula ROR, where R is not hydrogen.. diethyl ether : An ether in which the oxygen atom is linked to two ethyl groups.		2.4110ether;
volatile organic compound		inhalation anaesthetic;
non-polar solvent;
refrigerant
flurbiprofen
Flurbiprofen: An anti-inflammatory analgesic and antipyretic of the phenylalkynoic acid series. It has been shown to reduce bone resorption in periodontal disease by inhibiting CARBONIC ANHYDRASE.. flurbiprofen : A monocarboxylic acid that is a 2-fluoro-[1,1'-biphenyl-4-yl] moiety linked to C-2 of propionic acid. A non-steroidal anti-inflammatory, analgesic and antipyretic, it is used as a pre-operative anti-miotic as well as orally for arthritis or dental pain.		3.2710fluorobiphenyl;
monocarboxylic acid		antipyretic;
EC 1.14.99.1 (prostaglandin-endoperoxide synthase) inhibitor;
non-narcotic analgesic;
non-steroidal anti-inflammatory drug
ketoconazole
1-acetyl-4-(4-{[2-(2,4-dichlorophenyl)-2-(1H-imidazol-1-ylmethyl)-1,3-dioxolan-4-yl]methoxy}phenyl)piperazine : A dioxolane that is 1,3-dioxolane which is substituted at positions 2, 2, and 4 by imidazol-1-ylmethyl, 2,4-dichlorophenyl, and [para-(4-acetylpiperazin-1-yl)phenoxy]methyl groups, respectively.		2.4110dichlorobenzene;
dioxolane;
ether;
imidazoles;
N-acylpiperazine;
N-arylpiperazine
metoprolol
Metoprolol: A selective adrenergic beta-1 blocking agent that is commonly used to treat ANGINA PECTORIS; HYPERTENSION; and CARDIAC ARRHYTHMIAS.. metoprolol : A propanolamine that is 1-(propan-2-ylamino)propan-2-ol substituted by a 4-(2-methoxyethyl)phenoxy group at position 1.		8.6411aromatic ether;
propanolamine;
secondary alcohol;
secondary amino compound		antihypertensive agent;
beta-adrenergic antagonist;
environmental contaminant;
geroprotector;
xenobiotic
thioridazine
Thioridazine: A phenothiazine antipsychotic used in the management of PHYCOSES, including SCHIZOPHRENIA.. thioridazine : A phenothiazine derivative having a methylsulfanyl subsitituent at the 2-position and a (1-methylpiperidin-2-yl)ethyl] group at the N-10 position.		2.1110phenothiazines;
piperidines		alpha-adrenergic antagonist;
dopaminergic antagonist;
EC 1.8.1.12 (trypanothione-disulfide reductase) inhibitor;
EC 3.4.21.26 (prolyl oligopeptidase) inhibitor;
first generation antipsychotic;
H1-receptor antagonist;
serotonergic antagonist
uridine diphosphate
Uridine Diphosphate: A uracil nucleotide containing a pyrophosphate group esterified to C5 of the sugar moiety.		2.1110pyrimidine ribonucleoside 5'-diphosphate;
uridine 5'-phosphate		Escherichia coli metabolite;
mouse metabolite
1-deoxynojirimycin
1-deoxy-nojirimycin: structure in first source. duvoglustat : An optically active form of 2-(hydroxymethyl)piperidine-3,4,5-triol having 2R,3R,4R,5S-configuration.		2.53202-(hydroxymethyl)piperidine-3,4,5-triol;
piperidine alkaloid		anti-HIV agent;
anti-obesity agent;
bacterial metabolite;
EC 3.2.1.20 (alpha-glucosidase) inhibitor;
hepatoprotective agent;
hypoglycemic agent;
plant metabolite
miglustat
miglustat: a glucosylceramide synthase inhibitor. miglustat : A hydroxypiperidine that is deoxynojirimycin in which the amino hydrogen is replaced by a butyl group.		2.1310piperidines;
tertiary amino compound		anti-HIV agent;
EC 2.4.1.80 (ceramide glucosyltransferase) inhibitor
migalastat
migalastat: a potent inhibitor of glycolipid biosynthesis		2.1310piperidines
bortezomib
[no description available]		2.2110amino acid amide;
L-phenylalanine derivative;
pyrazines		antineoplastic agent;
antiprotozoal drug;
protease inhibitor;
proteasome inhibitor
quinidine
Quinidine: An optical isomer of quinine, extracted from the bark of the CHINCHONA tree and similar plant species. This alkaloid dampens the excitability of cardiac and skeletal muscles by blocking sodium and potassium currents across cellular membranes. It prolongs cellular ACTION POTENTIALS, and decreases automaticity. Quinidine also blocks muscarinic and alpha-adrenergic neurotransmission.. quinidine : A cinchona alkaloid consisting of cinchonine with the hydrogen at the 6-position of the quinoline ring substituted by methoxy.		7.2110cinchona alkaloidalpha-adrenergic antagonist;
anti-arrhythmia drug;
antimalarial;
drug allergen;
EC 1.14.13.181 (13-deoxydaunorubicin hydroxylase) inhibitor;
EC 3.6.3.44 (xenobiotic-transporting ATPase) inhibitor;
muscarinic antagonist;
P450 inhibitor;
potassium channel blocker;
sodium channel blocker
sch-202676
SCH-202676: An allosteric modulator of both agonist and antagonist binding to G protein-coupled receptors; structure in first source		2.4110
digoxin
Digoxin: A cardiotonic glycoside obtained mainly from Digitalis lanata; it consists of three sugars and the aglycone DIGOXIGENIN. Digoxin has positive inotropic and negative chronotropic activity. It is used to control ventricular rate in ATRIAL FIBRILLATION and in the management of congestive heart failure with atrial fibrillation. Its use in congestive heart failure and sinus rhythm is less certain. The margin between toxic and therapeutic doses is small. (From Martindale, The Extra Pharmacopoeia, 30th ed, p666). digoxin : A cardenolide glycoside that is digitoxin beta-hydroxylated at C-12. A cardiac glycoside extracted from the foxglove plant, Digitalis lanata, it is used to control ventricular rate in atrial fibrillation and in the management of congestive heart failure with atrial fibrillation, but the margin between toxic and therapeutic doses is small.		8.6411cardenolide glycoside;
steroid saponin		anti-arrhythmia drug;
cardiotonic drug;
EC 3.6.3.9 (Na(+)/K(+)-transporting ATPase) inhibitor;
epitope
psychosine
[no description available]		2.9330glycosylsphingoidhuman metabolite
bosutinib
4-((2,4-dichloro-5-methoxyphenyl)amino)-6-methoxy-7-(3-(4-methyl-1-piperazinyl)propoxy)-3-quinolinecarbonitrile: a Src kinase inhibitor; structure in first source		2.2510aminoquinoline;
aromatic ether;
dichlorobenzene;
N-methylpiperazine;
nitrile;
tertiary amino compound		antineoplastic agent;
tyrosine kinase inhibitor
sphingosyl beta-glucoside
sphingosyl beta-glucoside: isolated from spleen in Gaucher's disease		3.240
cdw17 antigen
[no description available]		2.2110
cellulose
DEAE-Cellulose: Cellulose derivative used in chromatography, as ion-exchange material, and for various industrial applications.		2.1310glycoside
glycolipids
[no description available]		2.2510
globotriaosylceramide
globotriaosylceramide: receptor for Shigella. alpha-D-galactosyl-(1->4)-beta-D-galactosyl-(1->4)-beta-D-glucosyl-(1<->1)-ceramide : A glycotriaosylceramide having alpha-D-galactosyl-(1->4)-beta-D-galactosyl-(1->4)-beta-D-glucosyl component attached to the primary hydroxy function of a ceramide with undefined sphingoid base.		2.0510
g(m3) ganglioside
G(M3) Ganglioside: A ganglioside present in abnormally large amounts in the brain and liver due to a deficient biosynthetic enzyme, G(M3):UDP-N-acetylgalactosaminyltransferase. Deficiency of this enzyme prevents the formation of G(M2) ganglioside from G(M3) ganglioside and is the cause of an anabolic sphingolipidosis.. alpha-Neu5Ac-(2->3)-beta-D-Gal-(1->4)-beta-D-Glc-(1<->1')-Cer(d18:1/24:1(15Z)) : A sialotriaosylceramide consisting of beta-D-GalNAc-(1->4)-[alpha-Neu5Ac-(2->3)]-beta-D-Gal-(1->4)-beta-D-Glc attached to the primary hydroxy function of ceramide(d18:1/24:1(15Z)).		6.833alpha-N-acetylneuraminyl-(2->3)-beta-D-galactosyl-(1->4)-beta-D-glucosyl-(1<->1')-ceramide;
sialodiosylceramide;
sialotriaosylceramide		mouse metabolite
ConditionIndicatedRelationship StrengthStudiesTrials
Acid beta-Glucosidase Deficiency
[description not available]		018.9111661
Gaucher Disease
An autosomal recessive disorder caused by a deficiency of acid beta-glucosidase (GLUCOSYLCERAMIDASE) leading to intralysosomal accumulation of glycosylceramide mainly in cells of the MONONUCLEAR PHAGOCYTE SYSTEM. The characteristic Gaucher cells, glycosphingolipid-filled HISTIOCYTES, displace normal cells in BONE MARROW and visceral organs causing skeletal deterioration, hepatosplenomegaly, and organ dysfunction. There are several subtypes based on the presence and severity of neurological involvement.		125.25348183
Ache
[description not available]		02.610
Pain
An unpleasant sensation induced by noxious stimuli which are detected by NERVE ENDINGS of NOCICEPTIVE NEURONS.		02.610
Kidney Failure
A severe irreversible decline in the ability of kidneys to remove wastes, concentrate URINE, and maintain ELECTROLYTE BALANCE; BLOOD PRESSURE; and CALCIUM metabolism.		03.6411
Liver Dysfunction
[description not available]		03.6411
Liver Diseases
Pathological processes of the LIVER.		03.6411
Renal Insufficiency
Conditions in which the KIDNEYS perform below the normal level in the ability to remove wastes, concentrate URINE, and maintain ELECTROLYTE BALANCE; BLOOD PRESSURE; and CALCIUM metabolism. Renal insufficiency can be classified by the degree of kidney damage (as measured by the level of PROTEINURIA) and reduction in GLOMERULAR FILTRATION RATE.		03.6411
Benign Monoclonal Gammopathies
[description not available]		02.2510
Enlarged Spleen
[description not available]		05.7332
Cancer of Prostate
[description not available]		02.2510
Prostatic Neoplasms
Tumors or cancer of the PROSTATE.		02.2510
Low Bone Density
[description not available]		03.5911
Blood Diseases
[description not available]		03.5911
Enlarged Liver
[description not available]		03.5911
Bone Diseases, Metabolic
Diseases that affect the METABOLIC PROCESSES of BONE TISSUE.		03.5911
Hematologic Diseases
Disorders of the blood and blood forming tissues.		03.5911
alpha-Galactosidase A Deficiency
[description not available]		02.830
Disease Models, Animal
Naturally-occurring or experimentally-induced animal diseases with pathological processes analogous to human diseases.		03.0140
Fabry Disease
An X-linked inherited metabolic disease caused by a deficiency of lysosomal ALPHA-GALACTOSIDASE A. It is characterized by intralysosomal accumulation of globotriaosylceramide and other GLYCOSPHINGOLIPIDS in blood vessels throughout the body leading to multi-system complications including renal, cardiac, cerebrovascular, and skin disorders.		02.830
Carbohydrate Metabolism, Inborn Error
[description not available]		02.2110
Mucopolysaccharidosis
[description not available]		02.2110
Mucopolysaccharidoses
Group of lysosomal storage diseases each caused by an inherited deficiency of an enzyme involved in the degradation of glycosaminoglycans (mucopolysaccharides). The diseases are progressive and often display a wide spectrum of clinical severity within one enzyme deficiency.		02.2110
Bone Demineralization, Pathologic
Decrease, loss, or removal of the mineral constituents of bones. Temporary loss of bone mineral content is especially associated with space flight, weightlessness, and extended immobilization. OSTEOPOROSIS is permanent, includes reduction of total bone mass, and is associated with increased rate of fractures. CALCIFICATION, PHYSIOLOGIC is the process of bone remineralizing. (From Dorland, 27th ed; Stedman, 25th ed; Nicogossian, Space Physiology and Medicine, 2d ed, pp327-33)		09.7199
Bone Fractures
[description not available]		09.7199
Fractures, Bone
Breaks in bones.		09.7199
B-Cell Lymphoma
[description not available]		02.1110
Lymphoma, B-Cell
A group of heterogeneous lymphoid tumors generally expressing one or more B-cell antigens or representing malignant transformations of B-lymphocytes.		02.1110
Body Weight
The mass or quantity of heaviness of an individual. It is expressed by units of pounds or kilograms.		02.4820
Benign Neoplasms
[description not available]		02.1110
Neoplasms
New abnormal growth of tissue. Malignant neoplasms show a greater degree of anaplasia and have the properties of invasion and metastasis, compared to benign neoplasms.		02.1110
Orphan Diseases
Rare diseases that have not been well studied.		02.1310
Lysosomal Enzyme Disorders
[description not available]		02.1310
Cherry Red Spot Myoclonus Syndrome
[description not available]		02.1310
Liver Steatosis
[description not available]		02.0510
Compensatory Hyperinsulinemia
A GLUCOSE-induced HYPERINSULINEMIA, a marker of insulin-resistant state. It is a mechanism to compensate for reduced sensitivity to insulin.		02.0510
Fatty Liver
Lipid infiltration of the hepatic parenchymal cells resulting in a yellow-colored liver. The abnormal lipid accumulation is usually in the form of TRIGLYCERIDES, either as a single large droplet or multiple small droplets. Fatty liver is caused by an imbalance in the metabolism of FATTY ACIDS.		02.0510
Hyperinsulinism
A syndrome with excessively high INSULIN levels in the BLOOD. It may cause HYPOGLYCEMIA. Etiology of hyperinsulinism varies, including hypersecretion of a beta cell tumor (INSULINOMA); autoantibodies against insulin (INSULIN ANTIBODIES); defective insulin receptor (INSULIN RESISTANCE); or overuse of exogenous insulin or HYPOGLYCEMIC AGENTS.		02.0510
Obesity
A status with BODY WEIGHT that is grossly above the recommended standards, usually due to accumulation of excess FATS in the body. The standards may vary with age, sex, genetic or cultural background. In the BODY MASS INDEX, a BMI greater than 30.0 kg/m2 is considered obese, and a BMI greater than 40.0 kg/m2 is considered morbidly obese (MORBID OBESITY).		02.0510
Dizzyness
[description not available]		07.4944
Emesis
[description not available]		07.4944
Dizziness
An imprecise term which may refer to a sense of spatial disorientation, motion of the environment, or lightheadedness.		07.4944
Nausea
An unpleasant sensation in the stomach usually accompanied by the urge to vomit. Common causes are early pregnancy, sea and motion sickness, emotional stress, intense pain, food poisoning, and various enteroviruses.		07.4944
Vomiting
The forcible expulsion of the contents of the STOMACH through the MOUTH.		07.4944