Page last updated: 2024-10-21

1-anilino-8-naphthalenesulfonate and Inborn Urea Cycle Disorder

1-anilino-8-naphthalenesulfonate has been researched along with Inborn Urea Cycle Disorder in 2 studies

1-anilino-8-naphthalenesulfonate: RN given refers to parent cpd
8-anilinonaphthalene-1-sulfonic acid : A naphthalenesulfonic acid that is naphthalene-1-sulfonic acid substituted by a phenylamino group at position 8.

Research Excerpts

ExcerptRelevanceReference
" Twenty-four healthy adults underwent single-dose administration of GPB and NaPBA and eight healthy adults and 24 cirrhotic subjects underwent single-day and multiple-day dosing of GPB, with metabolites measured in blood and urine."2.75Pharmacology and safety of glycerol phenylbutyrate in healthy adults and adults with cirrhosis. ( Dickinson, K; Gargosky, S; Lowe, ME; Martinez, A; McGuire, BM; Mokhtarani, M; Monteleone, J; Scharschmidt, BF; Syplyviy, VA; Xiao, X; Zupanets, IA, 2010)
" Covariate analyses indicated that age did not influence the PK parameters, with body surface area (BSA) being the most significant covariate, reinforcing current BSA based dosing recommendations as seen in older patients."1.48Pharmacokinetics of glycerol phenylbutyrate in pediatric patients 2 months to 2 years of age with urea cycle disorders. ( Berry, SA; Diaz, GA; Dong, M; Ficicioglu, C; Harding, CO; Holt, RJ; Lichter-Konecki, U; Longo, N; McCandless, SE; Perdok, R; Robinson, B; Smith, WE; Vinks, AA; Vockley, J; Zori, R, 2018)

Research

Studies (2)

TimeframeStudies, this research(%)All Research%
pre-19900 (0.00)18.7374
1990's0 (0.00)18.2507
2000's0 (0.00)29.6817
2010's2 (100.00)24.3611
2020's0 (0.00)2.80

Authors

AuthorsStudies
Berry, SA1
Vockley, J1
Vinks, AA1
Dong, M1
Diaz, GA1
McCandless, SE1
Smith, WE1
Harding, CO1
Zori, R1
Ficicioglu, C1
Lichter-Konecki, U1
Perdok, R1
Robinson, B1
Holt, RJ1
Longo, N1
McGuire, BM1
Zupanets, IA1
Lowe, ME1
Xiao, X1
Syplyviy, VA1
Monteleone, J1
Gargosky, S1
Dickinson, K1
Martinez, A1
Mokhtarani, M1
Scharschmidt, BF1

Clinical Trials (3)

Trial Overview

TrialPhaseEnrollmentStudy TypeStart DateStatus
An Open Label Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate (GPB; RAVICTI®) in Pediatric Subjects Under Two Years of Age With Urea Cycle Disorders (UCDs)[NCT02246218]Phase 427 participants (Actual)Interventional2014-12-31Completed
[NCT00986895]Phase 132 participants (Actual)Interventional2006-09-30Completed
A Randomized, Crossover, Open-label Phase 1 Study of Glyceryl Tri-(4-phenylbutyrate) (GT4P)[NCT00977600]Phase 124 participants (Actual)Interventional2005-03-31Completed
[information is prepared from clinicaltrials.gov, extracted Sep-2024]

Trial Outcomes

Percentage of Participants With Successful Transition to RAVICTI With Controlled Ammonia (i.e. No Clinical Symptoms and Ammonia < 100 μmol/L): Cohort of 0 Months to <2 Months Participants

"The percentage of participants with successful transition is based on Investigator response to the question, Has transition to 100% RAVICTI been successful with controlled ammonia? For participants < 2 months of age, after a minimum of 24 hours of ammonia monitoring following the first full dose of RAVICTI alone, the participant was effectively transitioned when following conditions were met: no signs and symptoms of hyperammonemia; ammonia level less than 100 μmol/L (without normalization of ammonia, ie, without conversion of values from local laboratories with varying normal ranges to standardized values); and eligible for discharge per Investigator judgment." (NCT02246218)
Timeframe: Up to Day 4

Interventionpercentage of participants (Number)
RAVICTI: Age 0 to < 2 Months100

Percentage of Participants With Successful Transition to RAVICTI With Controlled Ammonia (i.e. No Clinical Symptoms and Ammonia < 100 μmol/L): Cohort of 2 Months to <2 Years Participants

"The percentage of participants with successful transition is based on Investigator response to the question, Has transition to 100% RAVICTI been successful with controlled ammonia? For participants 2 months of age and older, after a minimum of 24 hours of ammonia monitoring following the first full dose of RAVICTI alone, the participant was effectively transitioned when following conditions were met: no signs and symptoms of hyperammonemia; ammonia level less than 100 μmol/L (without normalization of ammonia, ie, without conversion of values from local laboratories with varying normal ranges to standardized values); and eligible for discharge per Investigator judgment." (NCT02246218)
Timeframe: Up to Day 4

Interventionpercentage of participants (Number)
RAVICTI: Age 2 Months to < 2 Years100

Plasma PAA AUC(0-last) on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants

(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionμg*hr/mL (Mean)
RAVICTI: Age 0 to < 2 Months1321.18

Plasma PAA AUC(0-last) on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants

(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionμg*hr/mL (Mean)
RAVICTI: Age 2 Months to < 2 Years246.126

Plasma PAA Cmax on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants

(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionμg/mL (Mean)
RAVICTI: Age 0 to < 2 Months115.3

Plasma PAA Cmin on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants

(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionμg/mL (Mean)
RAVICTI: Age 0 to < 2 Months98.98

Plasma PAA Cmin on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants

(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionμg/mL (Mean)
RAVICTI: Age 2 Months to < 2 Years4.197

Plasma PAA Tmax on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants

(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionhours (Mean)
RAVICTI: Age 0 to < 2 Months9.85

Plasma PAA Tmax on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants

(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionhours (Mean)
RAVICTI: Age 2 Months to < 2 Years7.422

Plasma PAGN AUC(0-last) on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants

(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionμg*hr/mL (Mean)
RAVICTI: Age 0 to < 2 Months1384.12

Plasma PAGN AUC(0-last) on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants

(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionμg*hr/mL (Mean)
RAVICTI: Age 2 Months to < 2 Years583.835

Plasma PAGN Cmax on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants

(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionμg/mL (Mean)
RAVICTI: Age 0 to < 2 Months102.1

Plasma PAGN Cmin on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants

(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionμg/mL (Mean)
RAVICTI: Age 0 to < 2 Months69.39

Plasma PAGN Cmin on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants

(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionμg/mL (Mean)
RAVICTI: Age 2 Months to < 2 Years20.62

Plasma PAGN Tmax on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants

(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionhours (Mean)
RAVICTI: Age 0 to < 2 Months11.72

Plasma PAGN Tmax on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants

(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionhours (Mean)
RAVICTI: Age 2 Months to < 2 Years6.573

Plasma PBA Area Under the Curve From Time Zero to the Time of Last Quantifiable Concentration (AUC[0-last]) on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants

(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionμg*hr/mL (Mean)
RAVICTI: Age 2 Months to < 2 Years280.936

Plasma PBA AUC(0-last) on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants

(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionμg*hr/mL (Mean)
RAVICTI: Age 0 to < 2 Months374.53

Plasma PBA Cmax on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants

(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionμg/mL (Mean)
RAVICTI: Age 0 to < 2 Months46.2

Plasma PBA Cmin on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants

(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionμg/mL (Mean)
RAVICTI: Age 0 to < 2 Months4.8

Plasma PBA Minimum Plasma Concentration (Cmin) on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants

(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionμg/mL (Mean)
RAVICTI: Age 2 Months to < 2 Years1.697

Plasma PBA Time to Cmax (Tmax) on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants

(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionhours (Mean)
RAVICTI: Age 2 Months to < 2 Years8.383

Plasma PBA Tmax on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants

(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionhours (Mean)
RAVICTI: Age 0 to < 2 Months9.39

Plasma Phenylacetate/Phenylacetic Acid (PAA) Cmax on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants

(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionμg/mL (Mean)
RAVICTI: Age 2 Months to < 2 Years36.52

Plasma Phenylacetylglutamine (PAGN) Cmax on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants

(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionμg/mL (Mean)
RAVICTI: Age 2 Months to < 2 Years62.45

Plasma Phenylbutyrate/Phenylbutyric Acid (PBA) Maximum Plasma Concentration (Cmax) on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants

(NCT02246218)
Timeframe: Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionμg/mL (Mean)
RAVICTI: Age 2 Months to < 2 Years42.44

Rate of HACs: Cohort of 0 Months to <2 Months Participants

HAC is defined as having signs and symptoms consistent with hyperammonemia (such as but not limited to frequent vomiting, nausea, headache, lethargy, irritability, combativeness, and/or somnolence) associated with high blood ammonia and requiring medical intervention. Rate of HACs per 6 months during the safety extension was calculated as sum of (number of HAC) / sum of (days during first 6 months starting on Day 8 or number days on RAVICTI, whichever is less) across all participants in the corresponding group. (NCT02246218)
Timeframe: Day 8 through up to Month 6

InterventionHACs per half-year of patient exposure (Number)
RAVICTI: Age 0 to < 2 Months0.003

Rate of Hyperammonemic Crises (HACs): Cohort of 2 Months to <2 Years Participants

HAC is defined having signs and symptoms consistent with hyperammonemia (such as but not limited to frequent vomiting, nausea, headache, lethargy, irritability, combativeness, and/or somnolence) associated with high blood ammonia and requiring medical intervention. Rate of HACs per 6 months during the safety extension is calculated as sum of (number of HAC) / sum of (days during first 6 months starting on Day 8 or number days on RAVICTI, whichever is less) across all participants in the corresponding group. (NCT02246218)
Timeframe: Day 8 through up to Month 6

InterventionHACs per half-year of patient exposure (Number)
RAVICTI: Age 2 Months to < 2 Years0.005

Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants

(NCT02246218)
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24

Interventionμmol/L (Mean)
BaselineDay 7 change from BaselineMonth 2 change from BaselineMonth 3 change from BaselineMonth 4 change from BaselineMonth 5 change from BaselineMonth 6 change from BaselineMonth 9 change from BaselineMonth 12 change from BaselineMonth 15 change from BaselineMonth 18 change from BaselineMonth 24 change from Baseline
RAVICTI: Age 0 to < 2 Months84.9726.8125.1650.0518.7757.4343.6533.418.7525.752.5016.10

Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants

(NCT02246218)
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24

Interventionµmol/L (Mean)
BaselineDay 7 change from BaselineMonth 2 change from BaselineMonth 3 change from BaselineMonth 4 change from BaselineMonth 5 change from BaselineMonth 6 change from BaselineMonth 9 change from BaselineMonth 12 change from BaselineMonth 15 change from BaselineMonth 24 change from Baseline
RAVICTI: Age 2 Months to < 2 Years122.43-54.507.80-16.33-13.000.25-2.2030.8022.2039.0048.00

Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 0 Months to <2 Months Participants

(NCT02246218)
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24

Interventionμmol/L (Mean)
BaselineDay 7 change from BaselineMonth 2 change from BaselineMonth 3 change from BaselineMonth 4 change from BaselineMonth 5 change from BaselineMonth 6 change from BaselineMonth 9 change from BaselineMonth 12 change from BaselineMonth 15 change from BaselineMonth 18 change from BaselineMonth 24 change from Baseline
RAVICTI: Age 0 to < 2 Months508.8321.04-27.62-15.09-113.98-99.82-138.16-56.08-181.50-103.75-184.00-219.93

Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 2 Months to <2 Years Participants

(NCT02246218)
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24

Interventionµmol/L (Mean)
BaselineDay 7 change from BaselineMonth 2 change from BaselineMonth 3 change from BaselineMonth 4 change from BaselineMonth 5 change from BaselineMonth 6 change from BaselineMonth 9 change from BaselineMonth 12 change from BaselineMonth 15 change from BaselineMonth 24 change from Baseline
RAVICTI: Age 2 Months to < 2 Years750.43-184.33-174.60-374.00-252.75-370.25-113.20-446.53-450.50-149.00195.00

Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 0 Months to <2 Months Participants

(NCT02246218)
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24

Interventionμmol/L (Mean)
BaselineDay 7 change from BaselineMonth 2 change from BaselineMonth 3 change from BaselineMonth 4 change from BaselineMonth 5 change from BaselineMonth 6 change from BaselineMonth 9 change from BaselineMonth 12 change from BaselineMonth 15 change from BaselineMonth 18 change from BaselineMonth 24 change from Baseline
RAVICTI: Age 0 to < 2 Months142.68-49.09-1.62-20.46-67.32-75.45-35.94-73.09-178.50-139.501.00-55.31

Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 2 Months to <2 Years Participants

(NCT02246218)
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24

Interventionµmol/L (Mean)
BaselineDay 7 change from BaselineMonth 2 change from BaselineMonth 3 change from BaselineMonth 4 change from BaselineMonth 5 change from BaselineMonth 6 change from BaselineMonth 9 change from BaselineMonth 12 change from BaselineMonth 15 change from BaselineMonth 24 change from Baseline
RAVICTI: Age 2 Months to < 2 Years54.862.674.20-25.67-20.25-20.00-16.40-6.73-13.33-18.001.50

Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 0 Months to <2 Months Participants

(NCT02246218)
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24

Interventionμmol/L (Mean)
BaselineDay 7 change from BaselineMonth 2 change from BaselineMonth 3 change from BaselineMonth 4 change from BaselineMonth 5 change from BaselineMonth 6 change from BaselineMonth 9 change from BaselineMonth 12 change from BaselineMonth 15 change from BaselineMonth 18 change from BaselineMonth 24 change from Baseline
RAVICTI: Age 0 to < 2 Months133.67-81.91-60.80-51.66-82.82-118.55-11.85-115.09-249.50-195.756.00-82.54

Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 2 Months to <2 Years Participants

(NCT02246218)
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24

Interventionµmol/L (Mean)
BaselineDay 7 change from BaselineMonth 2 change from BaselineMonth 3 change from BaselineMonth 4 change from BaselineMonth 5 change from BaselineMonth 6 change from BaselineMonth 9 change from BaselineMonth 12 change from BaselineMonth 15 change from BaselineMonth 24 change from Baseline
RAVICTI: Age 2 Months to < 2 Years90.86-0.839.80-33.00-31.25-39.50-25.40-19.13-34.37-40.00-1.50

Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants

(NCT02246218)
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24

Interventionμmol/L (Mean)
BaselineDay 7 change from BaselineMonth 2 change from BaselineMonth 3 change from BaselineMonth 4 change from BaselineMonth 5 change from BaselineMonth 6 change from BaselineMonth 9 change from BaselineMonth 12 change from BaselineMonth 15 change from BaselineMonth 18 change from BaselineMonth 24 change from Baseline
RAVICTI: Age 0 to < 2 Months593.8047.85-2.4634.96-95.21-42.39-94.51-22.66-172.75-78.00-181.50-203.83

Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants

(NCT02246218)
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24

Interventionµmol/L (Mean)
BaselineDay 7 change from BaselineMonth 2 change from BaselineMonth 3 change from BaselineMonth 4 change from BaselineMonth 5 change from BaselineMonth 6 change from BaselineMonth 9 change from BaselineMonth 12 change from BaselineMonth 15 change from BaselineMonth 24 change from Baseline
RAVICTI: Age 2 Months to < 2 Years872.86-238.83-166.80-390.33-265.75-370.00-115.40-415.73-428.30-110.00243.00

Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 0 Months to <2 Months Participants

(NCT02246218)
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24

Interventionμmol/L (Mean)
BaselineDay 7 change from BaselineMonth 2 change from BaselineMonth 3 change from BaselineMonth 4 change from BaselineMonth 5 change from BaselineMonth 6 change from BaselineMonth 9 change from BaselineMonth 12 change from BaselineMonth 15 change from BaselineMonth 18 change from BaselineMonth 24 change from Baseline
RAVICTI: Age 0 to < 2 Months181.49-63.96-39.04-23.86-74.41-98.672.64-90.40-238.25-137.0038.00-72.78

Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 2 Months to <2 Years Participants

(NCT02246218)
Timeframe: Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24

Interventionµmol/L (Mean)
BaselineDay 7 change from BaselineMonth 2 change from BaselineMonth 3 change from BaselineMonth 4 change from BaselineMonth 5 change from BaselineMonth 6 change from BaselineMonth 9 change from BaselineMonth 12 change from BaselineMonth 15 change from BaselineMonth 24 change from Baseline
RAVICTI: Age 2 Months to < 2 Years171.434.0040.60-27.33-31.50-56.00-21.60-11.90-48.87-46.005.00

Assessment of Growth and Development: Baseline and Change From Baseline in BMI Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants

To assess any effect of study drug treatment on growth, Z-scores were calculated to express the deviation from a reference population for BMI. The Z-scores are based on the World Health Organization's Child Growth Standards charts. Negative Z-scores indicate lower than typical for age and gender while positive scores indicate higher than typical for age and gender. (NCT02246218)
Timeframe: Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24

Interventionz-score (Mean)
BaselineMonth 1 change from BaselineMonth 2 change from BaselineMonth 3 change from BaselineMonth 4 change from BaselineMonth 5 change from BaselineMonth 6 change from BaselineMonth 9 change from BaselineMonth 12 change from BaselineMonth 15 change from BaselineMonth 18 change from BaselineMonth 24 change from Baseline
RAVICTI: Age 0 to < 2 Months-0.0544-0.2158-0.2598-0.1617-0.02640.08280.01360.46140.66460.68300.33080.7743

Assessment of Growth and Development: Baseline and Change From Baseline in Body Mass Index (BMI) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants

To assess any effect of study drug treatment on growth, Z-scores were calculated to express the deviation from a reference population for BMI. The Z-scores are based on the World Health Organization's Child Growth Standards charts. Negative Z-scores indicate lower than typical for age and gender while positive scores indicate higher than typical for age and gender. (NCT02246218)
Timeframe: Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24

Interventionz-score (Mean)
BaselineMonth 1 change from BaselineMonth 2 change from BaselineMonth 3 change from BaselineMonth 4 change from BaselineMonth 5 change from BaselineMonth 6 change from BaselineMonth 9 change from BaselineMonth 12 change from BaselineMonth 15 change from BaselineMonth 18 change from BaselineMonth 24 change from Baseline
RAVICTI: Age 2 Months to < 2 Years0.8107-0.2385-0.02490.18150.44340.14840.24970.64070.4164-0.2997-0.20380.5581

Assessment of Growth and Development: Baseline and Change From Baseline in Body Surface Area (BSA) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants

To assess any effect of study drug treatment on growth, Z-scores were calculated to express the deviation from a reference population for BSA. The Z-scores are based on weight-for-length charts. Negative Z-scores indicate lower than typical for age and gender while positive scores indicate higher than typical for age and gender. (NCT02246218)
Timeframe: Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24

Interventionz-score (Mean)
BaselineMonth 1 change from BaselineMonth 2 change from BaselineMonth 3 change from BaselineMonth 4 change from BaselineMonth 5 change from BaselineMonth 6 change from BaselineMonth 9 change from BaselineMonth 12 change from BaselineMonth 15 change from BaselineMonth 18 change from BaselineMonth 24 change from Baseline
RAVICTI: Age 2 Months to < 2 Years0.7143-0.2105-0.07040.10650.33650.10430.18420.48750.2944-0.3661-0.22140.4310

Assessment of Growth and Development: Baseline and Change From Baseline in BSA Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants

To assess any effect of study drug treatment on growth, Z-scores were calculated to express the deviation from a reference population for BSA. The Z-scores are based on weight-for-length charts. Negative Z-scores indicate lower than typical for age and gender while positive scores indicate higher than typical for age and gender. (NCT02246218)
Timeframe: Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24

Interventionz-score (Mean)
BaselineMonth 1 change from BaselineMonth 2 change from BaselineMonth 3 change from BaselineMonth 4 change from BaselineMonth 5 change from BaselineMonth 6 change from BaselineMonth 9 change from BaselineMonth 12 change from BaselineMonth 15 change from BaselineMonth 18 change from BaselineMonth 24 change from Baseline
RAVICTI: Age 0 to < 2 Months-0.19800.23360.20060.26840.23720.18100.29020.16790.13080.15950.10500.7341

Assessment of Urinary PAA Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants

(NCT02246218)
Timeframe: Hour 0 and between 0.5 and 1.5 hours, 1.5 and 2.5 hours, 4 and 6 hours, 7.5 and 8.5 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionμg/mL (Mean)
Hour 00.5 to 1.5 hours1.5 to 2.5 hours4 to 6 hours7.5 to 8.5 hours12 to 24 hours
RAVICTI: Age 0 to < 2 Months11.146.262.534.622.835.2

Assessment of Urinary PAA Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants

(NCT02246218)
Timeframe: Hour 0 and between 0.5 and 1.5 hours, 1.5 and 2.5 hours, 4 and 6 hours, 7.5 and 8.5 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionμg/mL (Mean)
Hour 00.5 to 1.5 hours1.5 to 2.5 hours4 to 6 hours7.5 to 8.5 hours12 to 24 hours
RAVICTI: Age 2 Months to < 2 Years18.786.507.292.604.484.31

Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants

(NCT02246218)
Timeframe: Day 7, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 15, End of Trial (up to Month 15)

Interventionμg/mL (Mean)
Day 7Month 1Month 2Month 3Month 4Month 5Month 6Month 9Month 12Month 15End of trial
RAVICTI: Age 0 to < 2 Months23.714.612.314.46.413.25.511.86.04.911.6

Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants

(NCT02246218)
Timeframe: Day 7, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 15, Month 18, End of Trial (up to Month 18)

Interventionμg/mL (Mean)
Day 7Month 1Month 2Month 3Month 4Month 5Month 6Month 9Month 15Month 18End of trial
RAVICTI: Age 2 Months to < 2 Years5.824.443.694.657.143.271.594.102.041.647.0

Assessment of Urinary PAGN Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants

(NCT02246218)
Timeframe: Hour 0 and between 0.5 and 1 hour, 1.5 and 2.5 hours, 4 and 6 hours, 7.5 and 8.5 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionμg/mL (Mean)
Hour 00.5 to 1.5 hours1.5 to 2.5 hours4 to 6 hours7.5 to 8.5 hours12 to 24 hours
RAVICTI: Age 0 to < 2 Months3530.431828174622603530.434404

Assessment of Urinary PAGN Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants

(NCT02246218)
Timeframe: Hour 0 and between 0.5 and 1 hour, 1.5 and 2.5 hours, 4 and 6 hours, 7.5 and 8.5 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC

Interventionμg/mL (Mean)
Hour 00.5 to 1.5 hours1.5 to 2.5 hours4 to 6 hours7.5 to 8.5 hours12 to 24 hours
RAVICTI: Age 2 Months to < 2 Years327341403145520239507561

Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants

(NCT02246218)
Timeframe: Day 7, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, End of Trial (up to Month 15)

Interventionμg/mL (Mean)
Day 7Month 1Month 2Month 3Month 4Month 5Month 6Month 9Month 12Month 15End of trial
RAVICTI: Age 0 to < 2 Months46434517411670372826697358837006584739156939

Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants

(NCT02246218)
Timeframe: Day 7, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, End of Trial (up to Month 18)

Interventionμg/mL (Mean)
Day 7Month 1Month 2Month 3Month 4Month 5Month 6Month 9Month 12Month 15Month 18End of trial
RAVICTI: Age 2 Months to < 2 Years885962747386114562141661295347935725806400525025333

Number of Participants With TEAEs, Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 0 Months to <2 Months Participants

An AE is any untoward medical occurrence, whether or not the event is considered related to the study drug. A serious AE is any AE that: results in death; is life threatening; requires hospitalization or prolongation of existing hospitalization; results in disability/incapacity; is a congenital anomaly/birth defect; is an important medical event. TEAEs are defined as AEs with an onset date on or after the first dose of study medication until study discontinuation. The Investigator assessed the causal relationship of each TEAE to the study drug as not related, possibly related, or probably related. (NCT02246218)
Timeframe: From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 10.67 [6.142] months).

InterventionParticipants (Count of Participants)
≥ 1 TEAE≥ 1 Related TEAE≥ 1 Serious TEAE≥ 1 Serious Related TEAEFatal Outcome TEAE≥ 1 TEAE Leading to Study Discontinuation
RAVICTI: Age 0 to < 2 Months161011001

Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 2 Months to <2 Years Participants

An adverse event (AE) is any untoward medical occurrence, whether or not the event is considered related to the study drug. A serious AE is any AE that: results in death; is life threatening; requires hospitalization or prolongation of existing hospitalization; results in disability/incapacity; is a congenital anomaly/birth defect; is an important medical event. TEAEs are defined as AEs with an onset date on or after the first dose of study medication until study discontinuation. The Investigator assessed the causal relationship of each TEAE to the study drug as not related, possibly related, or probably related. (NCT02246218)
Timeframe: From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months).

InterventionParticipants (Count of Participants)
≥ 1 TEAE≥ 1 Related TEAE≥ 1 Serious TEAE≥ 1 Serious Related TEAEFatal Outcome TEAE≥ 1 TEAE Leading to Study Discontinuation
RAVICTI: Age 2 Months to < 2 Years1046011

Trials

1 trial available for 1-anilino-8-naphthalenesulfonate and Inborn Urea Cycle Disorder

ArticleYear
Pharmacology and safety of glycerol phenylbutyrate in healthy adults and adults with cirrhosis.
    Hepatology (Baltimore, Md.), 2010, Volume: 51, Issue:6

    Topics: Adult; Cross-Over Studies; Female; Glutamine; Humans; Hydrolysis; Lipase; Liver Cirrhosis; Male; Mon

2010
Pharmacology and safety of glycerol phenylbutyrate in healthy adults and adults with cirrhosis.
    Hepatology (Baltimore, Md.), 2010, Volume: 51, Issue:6

    Topics: Adult; Cross-Over Studies; Female; Glutamine; Humans; Hydrolysis; Lipase; Liver Cirrhosis; Male; Mon

2010
Pharmacology and safety of glycerol phenylbutyrate in healthy adults and adults with cirrhosis.
    Hepatology (Baltimore, Md.), 2010, Volume: 51, Issue:6

    Topics: Adult; Cross-Over Studies; Female; Glutamine; Humans; Hydrolysis; Lipase; Liver Cirrhosis; Male; Mon

2010
Pharmacology and safety of glycerol phenylbutyrate in healthy adults and adults with cirrhosis.
    Hepatology (Baltimore, Md.), 2010, Volume: 51, Issue:6

    Topics: Adult; Cross-Over Studies; Female; Glutamine; Humans; Hydrolysis; Lipase; Liver Cirrhosis; Male; Mon

2010

Other Studies

1 other study available for 1-anilino-8-naphthalenesulfonate and Inborn Urea Cycle Disorder

ArticleYear
Pharmacokinetics of glycerol phenylbutyrate in pediatric patients 2 months to 2 years of age with urea cycle disorders.
    Molecular genetics and metabolism, 2018, Volume: 125, Issue:3

    Topics: Child; Child, Preschool; Female; Glutamine; Glycerol; Humans; Infant; Lipase; Male; Nitrogen; Phenyl

2018