Page last updated: 2024-12-07

rufinamide

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Description

rufinamide: for treatment of Lennox-Gastaut syndrome; structure in first source [Medical Subject Headings (MeSH), National Library of Medicine, extracted Dec-2023]

Cross-References

ID SourceID
PubMed CID129228
CHEMBL ID1201754
CHEBI ID134966
SCHEMBL ID230448
MeSH IDM0213280

Synonyms (109)

Synonym
AC-1429
c10h8f2n4o
xilep
inovelon
e-2080 ,
syn-111
rufinamide
banzel
ruf-331
cgp-33101
rufinamide, >=98% (hplc), powder
rufinamide (jan/usp/inn)
D05775
banzel (tn)
106308-44-5
smr000857122
MLS001332514
MLS001332513
NCGC00165883-01
NCGC00165883-02
CHEBI:134966
NCGC00165883-03
e2080
ruf 331
CHEMBL1201754
cgp 33101
AKOS005145897
170939-95-4
FT-0656828
1-[(2,6-difluorophenyl)methyl]triazole-4-carboxamide
1-[(2,6-difluorophenyl)methyl]-4-triazolecarboxamide
1-[[2,6-bis(fluoranyl)phenyl]methyl]-1,2,3-triazole-4-carboxamide
A801414
HMS3262O14
1h-1,2,3-triazole-4-carboxamide, 1-[(2,6-difluorophenyl)methyl]-
dtxsid1046506 ,
dtxcid9026506
tox21_112267
cas-106308-44-5
1-(2,6-difluorobenzyl)-1h-1,2,3-triazole-4-carboxamide
1-[(2,6-difluorophenyl)methyl]-1h-1,2,3-triazole-4-carboxamide
HMS2232M19
1h-1,2,3-triazole-4-carboxamide, 1-((2,6-difluorophenyl)methyl)-
syn111
unii-wfw942pr79
cgp 33,101
wfw942pr79 ,
cgp33101
rufinamide [usan:inn:ban]
e 2080
LP00796
rufinamide [ema epar]
rufinamide [mart.]
rufinamide [usp-rs]
rufinamide [orange book]
rufinamide [who-dd]
rufinamide [mi]
rufinamide [inn]
rufinamide [usp monograph]
rufinamide [jan]
rufinamide [usan]
rufinamide [vandf]
S1256
HMS3371A06
SCHEMBL230448
DB06201
CCG-222100
HY-A0042
CS-1455
NCGC00165883-04
tox21_112267_1
gtpl7470
AB00918347-05
tox21_500796
NCGC00261481-01
1-(2,6-difluorobenzyl)-1h-1,2,3-triazol-4-carboxamide
F0001-2404
tox21 112267
HB1039
R0143
AB00918347_06
mfcd00865314
J-001568
sr-01000842156
SR-01000842156-4
rufinamide, united states pharmacopeia (usp) reference standard
HMS3651O05
SW219770-1
1-[(2,6-difluorophenyl)methyl]-1h-1,2,3-triazole-4 carboxamide
FT-0674479
BCP21828
Q408565
Z1532717444
rufinamide (banzel)
AS-13861
EN300-120881
SB18904
SDCCGSBI-0633757.P001
NCGC00165883-11
HMS3884G07
C71253
bdbm50515492
rufinamida
syn11
n03af03
rufinamide (usp-rs)
rufinamide (mart.)
rufinamide (usp monograph)
rufinamidum

Research Excerpts

Overview

Rufinamide is a triazole derivative that is structurally dissimilar to other marketed antiepileptic drugs. It has been assumed a marketing authorization, by the European Union and FDA, for use as a complementary therapy for seizures associated with Lennox-Gastaut syndrome.

ExcerptReferenceRelevance
"Rufinamide (RFM) is a clinically utilized antiepileptic drug that, as a triazole derivative, has a unique structure. "( Rufinamide, a Triazole-Derived Antiepileptic Drug, Stimulates Ca
Huang, CW; Lai, MC; Wu, SN, 2022
)
3.61
"Rufinamide is an effective and well-tolerated drug for long-term treatment in pediatric patients with intractable epilepsy. "( Rufinamide efficacy and association with phenotype and genotype in children with intractable epilepsy: A retrospective single center study.
Bozarth, XL; Oesch, G, 2020
)
3.44
"Rufinamide is a triazole derivative that is structurally dissimilar to other marketed antiepileptic drugs, has been assumed a marketing authorization, by the European Union and FDA, for use as a complementary therapy for seizures associated with Lennox-Gastaut syndrome."( Determination of Rufinamide in the Presence of 1-[(2,6-Difluorophenyl)Methyl]-1H-1,2,3-Triazole-4 Carboxylic Acid Using RP-HPLC and Derivative Ratio Methods as Stability Indicating Assays to Be Applied on Dosage Form.
Hashem, HMA; Hassib, ST; Mahrouse, MA; Mostafa, EA, 2020
)
2.34
"Rufinamide is an antiseizure medication that acts through sodium channels and is found to be efficacious in patients with Lennox Gastaut syndrome (LGS). "( Efficacy and safety of rufinamide as adjunctive therapy in patients with Lennox Gastaut syndrome: A systematic review and Meta-analysis.
Dawman, L; Panda, P; Panda, PK; Sharawat, IK, 2021
)
2.37
"Rufinamide (R) is a triazole derivative approved for the management of partial seizures and seizures associated with Lennox-Gastaut Syndrome, in November 2007. "( Rufinamide: Crystal structure elucidation and solid state characterization.
Chavan, RB; Lodagekar, A; Mittapalli, S; Nangia, A; Salunke, N; Shastri, NR; Thipparaboina, R, 2018
)
3.37
"Rufinamide is a novel antiepileptic drug and commonly used in the treatment of Lennox-Gastaut syndrome. "( Rufinamide, an antiepileptic drug, improves cognition and increases neurogenesis in the aged gerbil hippocampal dentate gyrus via increasing expressions of IGF-1, IGF-1R and p-CREB.
Ahn, JH; Chen, BH; Hwang, IK; Kang, IJ; Kim, DW; Kim, H; Kim, YM; Lee, CH; Lee, JC; Lee, TK; Park, JH; Song, M; Won, MH; Yan, BC, 2018
)
3.37
"Rufinamide (RUF) is a structurally unique anti-epileptic drug, used in the treatment of seizure disorders such as Lennox-Gastaut syndrome. "( Effect of Rufinamide on the kainic acid-induced excitotoxic neuronal death in the mouse hippocampus.
Lee, CH; Park, JA, 2018
)
2.33
"Rufinamide is a new antiepileptic drug approved for use as adjunctive therapy of seizures associated with Lennox-Gastaut syndrome and it is also effective for refractory partial seizures."( Successful Treatment of Refractory Seizures With Rufinamide in Children With Schizencephaly: Report of 3 Cases.
Coppola, G; Delli Pizzi, A; Loiacono, G; Rossi, A; Tartaro, A; Verrotti, A, 2015
)
1.39
"Rufinamide (RFD) is a novel drug that was recently approved as an adjunctive treatment for Lennox-Gastaut syndrome. "( Effectiveness of Rufinamide in the Treatment of Idiopathic Generalized Epilepsy With Atypical Evolution: Case Report and Review of the Literature.
Albini, M; Casciato, S; Di Bonaventura, C; Fanella, M; Fattouch, J; Giallonardo, AT; Lapenta, L; Manfredi, M; Morano, A, 2016
)
2.22
"Rufinamide is a third-generation antiepileptic drug, approved recently as an orphan drug for the treatment of Lennox-Gastaut syndrome. "( Dose-dependent pharmacokinetics and brain penetration of rufinamide following intravenous and oral administration to rats.
Gáll, O; Gáll, Z; Kolcsár, M; Szilágyi, T; Vancea, S, 2015
)
2.1
"Rufinamide is a structurally novel, antiepileptic drug approved for the treatment of Lennox-Gastaut syndrome. "( Rufinamide Improves Functional and Behavioral Deficits via Blockade of Tetrodotoxin-Resistant Sodium Channels in Diabetic Neuropathy.
Kharatmal, SB; Sharma, SS; Singh, JN, 2015
)
3.3
"Rufinamide is a triazole derivative that is structurally unrelated to currently marketed antiepileptic medications for add-on treatment of seizures in the setting of Lennox-Gastaut syndrome in patients from the age of 4 years."( Pharmacokinetics and Tolerability of Rufinamide Following Single and Multiple Oral Doses and Effect of Food on Pharmacokinetics in Healthy Chinese Subjects.
Chen, H; He, X; Li, H; Li, W; Ni, Y; Xu, M; Zhou, Y, 2016
)
2.15
"Rufinamide is a novel antiepileptic drug used as adjunctive therapy in patients with Lennox-Gastaut syndrome and provides seizure control especially in tonic and atonic seizures. "( Aggravation of atonic seizures by rufinamide: A case report.
Aydın, A; Aydınlı, N; Bektaş, G; Çalışkan, M; Özmen, M; Pembegül Yıldız, E; Tatlı, B, 2016
)
2.16
"Rufinamide is a novel antiepileptic agent recently approved in the United States for adjunctive treatment of seizures associated with Lennox-Gastaut syndrome. "( Safety and tolerability of rufinamide in children with epilepsy: a pooled analysis of 7 clinical studies.
Conry, J; Krauss, G; LoPresti, A; Mann, A; Narurkar, M; Wheless, JW, 2009
)
2.09
"Rufinamide is an oral antiepileptic drug indicated for adjunctive therapy in treating generalized seizures associated with Lennox-Gastaut syndrome. "( Stability of extemporaneously prepared rufinamide oral suspensions.
Best, R; Hutchinson, DJ; Liou, Y; Zhao, F, 2010
)
2.07
"Rufinamide is a new antiepileptic agent that differs structurally from other antiepileptic drugs and is approved as adjunctive therapy for Lennox-Gastaut syndrome (LGS). "( Rufinamide: a new antiepileptic medication for the treatment of seizures associated with lennox-gastaut syndrome.
Wisniewski, CS, 2010
)
3.25
"Rufinamide is a new antiepileptic drug approved as adjunctive therapy to treat seizures in Lennox-Gastaut syndrome in those 4 years of age and older."( Rufinamide: a new antiepileptic drug treatment for Lennox-Gastaut syndrome.
Ferrie, CD, 2010
)
2.52
"Rufinamide is a new antiepileptic drug recently approved as adjunctive treatment for generalized seizures in Lennox-Gastaut syndrome. "( Experience with rufinamide in a pediatric population: a single center's experience.
Bergin, AM; Bourgeois, BF; Duffy, FH; Eksioglu, YZ; Gooty, VD; Kothare, SV; Libenson, M; Loddenkemper, T; Poduri, A; Rotenberg, A; Takeoka, M; Vendrame, M, 2010
)
2.15
"Rufinamide (RUF) is a new antiepileptic drug with efficacy in several types of seizures. "( Rapid assay of rufinamide in dried blood spots by a new liquid chromatography-tandem mass spectrometric method.
Falchi, M; Filippi, L; Guerrini, R; Innocenti, M; la Marca, G; Malvagia, S; Moneti, G; Pellacani, S; Rosati, A, 2011
)
2.16
"Rufinamide appears to be a well-tolerated and efficacious adjunctive therapeutic option for children with epileptic spasms. "( Rufinamide for the treatment of epileptic spasms.
Bergin, AM; Bourgeois, BF; Coulter, D; Duffy, FH; Kothare, SV; Libenson, MH; Loddenkemper, T; Olson, HE; Poduri, A; Rotenberg, A; Takeoka, M; Vendrame, M, 2011
)
3.25
"Rufinamide is a triazole derivative with broad-spectrum antiepileptic effects that is unrelated to any antiepileptic drug currently on the market. "( Rufinamide for pediatric patients with Lennox-Gastaut syndrome: a comprehensive overview.
Cerna, A; So, TY; Wier, HA, 2011
)
3.25
"Rufinamide (RUF) is an orphan drug for adjunctive treatment of seizures associated with Lennox-Gastaut syndrome in persons aged 4 years and older. "( Serum concentrations of rufinamide in children and adults with epilepsy: the influence of dose, age, and comedication.
Boor, R; Brandt, C; Jürgens, U; Korn-Merker, E; May, TW; Rambeck, B, 2011
)
2.12
"Rufinamide is a new orally active antiepileptic drug that has been found to be effective in the treatment of partial seizures and drop attacks associated with Lennox-Gastaut syndrome. "( Efficacy of rufinamide in drug-resistant epilepsy: a meta-analysis.
Ballone, E; Chiarelli, F; Curatolo, P; Loiacono, G; Mattei, PA; Verrotti, A, 2011
)
2.19
"Rufinamide (RUF) is a relatively new EMEA- and FDA-approved anticonvulsant licensed as an orphan drug for the adjunctive treatment of patients with Lennox-Gastaut syndrome."( Epilepsy with myoclonic absences - favourable response to add-on rufinamide treatment in 3 cases.
Häusler, M; Kluger, G; Nikanorova, M, 2011
)
1.33
"Rufinamide is a triazole derivative structurally unrelated to other antiepileptic drugs that is indicated for the adjunctive treatment of seizures associated with Lennox-Gastaut syndrome (LGS) in patients aged ≥4 years. "( Rufinamide from clinical trials to clinical practice in the United States and Europe.
Arzimanoglou, A; Brown, LW; Flamini, R; Harrison, M; Kerr, M; Kluger, G; Kothare, S; Narurkar, M; Philip, S; Resnick, T, 2011
)
3.25
"Rufinamide would appear to be a cost-effective alternative to topiramate."( Rufinamide: a pharmacoeconomic profile of its use as adjunctive therapy in Lennox-Gastaut syndrome.
McCormack, PL, 2012
)
2.54
"Rufinamide appears to be a safe and effective adjuvant treatment for many cases of intractable LGS."( Rufinamide as an adjuvant treatment in children with Lennox-Gastaut syndrome.
Byeon, JH; Eun, BL; Eun, SH; Kang, HC; Kim, HD; Kim, SH; Kwon, EJ; Lee, JS; Lee, YM, 2012
)
3.26
"Rufinamide is a new antiepileptic drug approved as add-on treatment in Lennox-Gastaut syndrome from the age of 4 years, and for the treatment of focal seizures in adults and adolescents. "( Rufinamide efficacy and safety as adjunctive treatment in children with focal drug resistant epilepsy: the first Italian prospective study.
Capuano, A; Curatolo, P; Cusmai, R; Fusco, L; Moavero, R; Specchio, N; Vigevano, F, 2012
)
3.26
"Rufinamide (RUF) is a novel antiepileptic drug considered as second-line therapy in the treatment of Lennox-Gastaut syndrome. "( Dramatic weight loss with rufinamide.
Crespel, A; Gelisse, P; Mourand, I, 2013
)
2.13
"Rufinamide is a third-generation antiepileptic drug, available since early 2010 in France. "( [Therapeutic drug monitoring of rufinamide].
Bentué-Ferrer, D; Tribut, O; Verdier, MC,
)
1.86
"Rufinamide is a novel antiepileptic drug (AED), which is known to be effective in the treatment of partial seizures and drop attacks in patients with Lennox-Gastaut syndrome (LGS). "( Effectiveness and tolerability of rufinamide in children and young adults with Lennox-Gastaut syndrome: a single center study in Korea.
Ko, TS; Lee, EH; Yum, MS, 2013
)
2.11
"Rufinamide is a structurally novel compound with anticonvulsant activity that is undergoing evaluation through the European Medicines Agency and the American Food and Drug Administration. "( Rufinamide: Pharmacology, clinical trials, and role in clinical practice.
Anderson, GD; Cheng-Hakimian, A; Miller, JW, 2006
)
3.22
"Rufinamide is a triazole derivative structurally unrelated to currently marketed antiepileptic drugs. "( Rufinamide.
Arroyo, S, 2007
)
3.23
"Rufinamide is a new antiepileptic drug that is effective in acute animal seizure models and also in the kindling model of epilepsy with a high protective index. "( Rufinamide.
Heaney, D; Walker, MC, 2007
)
3.23
"Rufinamide is a new, orally active antiepileptic drug (AED), which has been found to be effective in the treatment of partial seizures and drop attacks associated with the Lennox-Gastaut syndrome. "( Rufinamide: clinical pharmacokinetics and concentration-response relationships in patients with epilepsy.
Cloyd, J; Critchley, D; Fuseau, E; Perucca, E, 2008
)
3.23

Effects

Rufinamide has been used as a new antiepileptic drug in the treatment of drug-resistant epilepsy. It has shown promise as adjunctive treatment for Lennox-Gastaut syndrome and may have some role in localization related epilepsies.

ExcerptReferenceRelevance
"Rufinamide has been shown to be effective and generally well tolerated in children as young as one year and in adults."( Rufinamide for the treatment of Lennox-Gastaut syndrome: evidence from clinical trials and clinical practice.
Falip, M; McMurray, R; Santamarina, E; Striano, P, 2018
)
2.64
"Rufinamide has been used as a new antiepileptic drug in the treatment of drug-resistant epilepsy, in recent years."( Efficacy of rufinamide in childhood refractory epilepsy.
Aydınlı, N; Bektaş, G; Çalışkan, M; Hızlı, Z; Özmen, M; Tatlı, B; Ulak-Özkan, M; Yıldız, EP, 2018
)
1.58
"Rufinamide has shown some efficacy in epileptic encephalopathies other than LGS."( Current role of rufinamide in the treatment of childhood epilepsy: literature review and treatment guidelines.
Besag, F; Coppola, G; Curatolo, P; Cusmai, R; Dulac, O; Kluger, G; Moavero, R; Nabbout, R; Nikanorova, M; Pisani, F; Verrotti, A; von Stülpnagel, C, 2014
)
1.47
"Rufinamide has few clinically relevant drug interactions, although it does increase phenytoin serum concentrations, while valproate increases rufinamide serum concentrations."( Rufinamide: a new antiepileptic medication for the treatment of seizures associated with lennox-gastaut syndrome.
Wisniewski, CS, 2010
)
2.52
"Rufinamide has demonstrated statistically significant efficacy as an adjunctive therapy in adult patients with epilepsy and in patients with seizures associated with Lennox-Gastaut syndrome."( Rufinamide: CGP 33101, E 2080, RUF 331, Xilep.
, 2005
)
2.49
"Rufinamide has shown promise as adjunctive treatment for Lennox-Gastaut syndrome and may have some role in localization related epilepsies as well."( Rufinamide: a new anti-epileptic medication.
Anderson, GD; Cheng-Hakimian, A; Hakimian, S; Miller, JW, 2007
)
2.5
"Rufinamide has been shown, in this proof of principle trial, to be safe and effective in reducing seizure frequency in epileptic patients with no relevant influence on the metabolism of other AEDs."( Rufinamide: a double-blind, placebo-controlled proof of principle trial in patients with epilepsy.
Canger, R; Henriksen, O; Karolchyk, MA; Pålhagen, S; Rivière, ME; van Parys, JA, 2001
)
3.2

Treatment

Rufinamide treatments (3 and 10 mg/kg) significantly improved these functional and nociceptive deficits. Ruf inamide-treated subjects experienced a 20.4% median reduction in partial seizure frequency relative to baseline. placebo-treated patients had an increase of 1.6% (p = 0.02).

ExcerptReferenceRelevance
"Rufinamide treatments (3 and 10 mg/kg) significantly improved these functional and nociceptive deficits."( Rufinamide Improves Functional and Behavioral Deficits via Blockade of Tetrodotoxin-Resistant Sodium Channels in Diabetic Neuropathy.
Kharatmal, SB; Sharma, SS; Singh, JN, 2015
)
2.58
"Rufinamide-treated subjects experienced a 20.4% median reduction in partial seizure frequency relative to baseline, while placebo-treated subjects had an increase of 1.6% (p = 0.02)."( Rufinamide for the adjunctive treatment of partial seizures in adults and adolescents: a randomized placebo-controlled trial.
Arroyo, S; Brodie, MJ; Mann, A; Perdomo, C; Rosenfeld, WE; Sachdeo, R; Vazquez, B, 2009
)
2.52
"Rufinamide-treated patients were more than twice as likely to have had a ≥50% reduction in partial seizure frequency (32.5% vs."( A randomized, double-blind, placebo-controlled, parallel-group study of rufinamide as adjunctive therapy for refractory partial-onset seizures.
Bibbiani, F; Biton, V; Krauss, G; Mann, A; Narurkar, M; Perdomo, C; Vasquez-Santana, B, 2011
)
1.32
"Rufinamide treatment was stopped because of aggravation of seizures (30%), no effect (45%), and side effects (10%)."( Low long-term efficacy and tolerability of add-on rufinamide in patients with Dravet syndrome.
Boor, R; Coppola, G; Dahlin, M; Kluger, G; Lotte, J; Mueller, A; Staudt, M; Striano, P; von Stuelpnagel, C, 2011
)
1.34

Toxicity

Rufinamide can be effective in reducing focal seizure frequency in children with drug resistant epilepsy. Risk of CNS adverse events appears to be increased in patients exposed to the drug. Most common adverse effects were somnolence, vomiting and headache.

ExcerptReferenceRelevance
" The most common adverse effects observed in rufinamide-treated patients in the double-blind studies were somnolence, vomiting, and headache."( Safety and tolerability of rufinamide in children with epilepsy: a pooled analysis of 7 clinical studies.
Conry, J; Krauss, G; LoPresti, A; Mann, A; Narurkar, M; Wheless, JW, 2009
)
0.91
" Our experience suggests that Rufinamide can be effective in reducing focal seizure frequency in children with drug resistant epilepsy, and that it can be considered as a safe drug."( Rufinamide efficacy and safety as adjunctive treatment in children with focal drug resistant epilepsy: the first Italian prospective study.
Capuano, A; Curatolo, P; Cusmai, R; Fusco, L; Moavero, R; Specchio, N; Vigevano, F, 2012
)
2.11
" Adverse events were reported in 41% of the patients of which fatigue was most frequent (24%)."( Rufinamide in children with refractory epilepsy: pharmacokinetics, efficacy, and safety.
Dahlin, MG; Ohman, I, 2012
)
1.82
" Adverse reactions occurred in 37."( Efficacy and safety of rufinamide in children under four years of age with drug-resistant epilepsies.
Accorsi, P; Balestri, P; Coppola, G; Dontin, SD; Gobbi, G; Grosso, S; Parisi, P; Pruna, D; Verrotti, A, 2014
)
0.71
"The present study concludes that rufinamide may be a safe and effective drug for a broad range of seizures and epilepsy syndromes in infants and young children and represents a valid therapeutic option in this population."( Efficacy and safety of rufinamide in children under four years of age with drug-resistant epilepsies.
Accorsi, P; Balestri, P; Coppola, G; Dontin, SD; Gobbi, G; Grosso, S; Parisi, P; Pruna, D; Verrotti, A, 2014
)
0.99
" Most common adverse effects were sleepiness, vomiting, mood changes, nausea, and loss of appetite."( Safety and retention rate of rufinamide in 300 patients: a single pediatric epilepsy center experience.
Bergin, AM; Bolton, J; Bourgeois, BF; Harini, C; Kadish, NE; Kapur, K; Kothare, SV; Libenson, M; Loddenkemper, T; Olson, H; Peters, J; Poduri, A; Ramgopal, S; Rotenberg, A; Sánchez Fernández, I; Takeoka, M; Thome-Souza, S, 2014
)
0.69
" To date, there has been no attempt to evaluate systematically the risks of adverse events with rufinamide."( Exposure to rufinamide and risks of CNS adverse events in drug-resistant epilepsy: a meta-analysis of randomized, placebo-controlled trials.
Alsaad, AM; Koren, G, 2014
)
1
"We performed a quantitative risk analysis of central nervous system (CNS) adverse events of rufinamide from all randomized, double-blind, add-on, placebo-controlled trials."( Exposure to rufinamide and risks of CNS adverse events in drug-resistant epilepsy: a meta-analysis of randomized, placebo-controlled trials.
Alsaad, AM; Koren, G, 2014
)
1
"The risk of CNS adverse events appears to be increased in patients exposed to rufinamide as well as the treatment discontinuation rates."( Exposure to rufinamide and risks of CNS adverse events in drug-resistant epilepsy: a meta-analysis of randomized, placebo-controlled trials.
Alsaad, AM; Koren, G, 2014
)
1.01
"Interim safety results showed that treatment-emergent adverse events (TEAEs) were similar between the rufinamide (22 [88."( Safety and pharmacokinetic profile of rufinamide in pediatric patients aged less than 4 years with Lennox-Gastaut syndrome: An interim analysis from a multicenter, randomized, active-controlled, open-label study.
Arzimanoglou, A; Bibbiani, F; Critchley, D; Dhadda, S; Ferreira, JA; Hussein, Z; Kumar, D; Mendes, S; Satlin, A; Schuck, E; Williams, B, 2016
)
0.92
"Rufinamide was safe and well tolerated in these pediatric subjects."( Safety and pharmacokinetic profile of rufinamide in pediatric patients aged less than 4 years with Lennox-Gastaut syndrome: An interim analysis from a multicenter, randomized, active-controlled, open-label study.
Arzimanoglou, A; Bibbiani, F; Critchley, D; Dhadda, S; Ferreira, JA; Hussein, Z; Kumar, D; Mendes, S; Satlin, A; Schuck, E; Williams, B, 2016
)
2.15
" However, rufinamide may induce more tolerable (but not severe) adverse events."( The efficacy and safety of rufinamide in drug-resistant epilepsy: A meta-analysis of double-blind, randomized, placebo controlled trials.
Chen, Z; Xu, Z; Zhao, H, 2016
)
1.13
" Adverse events (AEs) were evaluated throughout both studies."( Long-term safety and seizure outcome in Japanese patients with Lennox-Gastaut syndrome receiving adjunctive rufinamide therapy: An open-label study following a randomized clinical trial.
Iyoda, K; Ohtsuka, Y; Shirasaka, Y; Takano, H; Takayama, R; Yoshinaga, H, 2016
)
0.65
" Adverse events were mild or moderate, except for transient seizure aggravation in three patients."( Long-term safety and seizure outcome in Japanese patients with Lennox-Gastaut syndrome receiving adjunctive rufinamide therapy: An open-label study following a randomized clinical trial.
Iyoda, K; Ohtsuka, Y; Shirasaka, Y; Takano, H; Takayama, R; Yoshinaga, H, 2016
)
0.65
" Rufinamide is safe and effective for treatment of different epilepsies including LGS and drug-resistant partial seizures."( [Effectiveness and safety of rufinamide at treatment of epilepsy with complications and drug-resistant epilepsy (according to meta-analysis data)].
Kislitsyn, YV; Mazin, PV; Mazina, NK; Sheshunov, IV,
)
1.33
" Eight patients had adverse effects such as somnolence (6 patients), sleep disturbance (1 patient), and appetite loss (4 patients) including weight loss in 2 patients."( Short-term efficacy and safety of rufinamide for Lennox-Gastaut syndrome.
Minagawa, K; Takayama, R; Watanabe, T, 2016
)
0.71
" Rufinamide tablets were safe and effective as an add-on treatment in Korean children and adolescents <20 years of age with LGS."( Rufinamide efficacy and safety in children aged 1-4 years with Lennox-Gastaut syndrome.
Kang, HC; Kim, HD; Kim, SH; Lee, JS, 2018
)
2.83
" Adverse events (AEs) were evaluated after 12 weeks of treatment."( Rufinamide efficacy and safety in children aged 1-4 years with Lennox-Gastaut syndrome.
Kang, HC; Kim, HD; Kim, SH; Lee, JS, 2018
)
1.92
" Primary safety/tolerability assessments included monitoring of treatment-emergent adverse events (TEAEs) and serious TEAEs."( Evaluation of long-term safety, tolerability, and behavioral outcomes with adjunctive rufinamide in pediatric patients (≥1 to <4 years old) with Lennox-Gastaut syndrome: Final results from randomized study 303.
Arzimanoglou, A; Bibbiani, F; Dhadda, S; Ferreira, J; Kumar, D; Olhaye, O; Satlin, A, 2019
)
0.74
" The number of patients with at least one treatment-emergent adverse effects was significantly higher in rufinamide treated patients (60."( Efficacy and safety of rufinamide as adjunctive therapy in patients with Lennox Gastaut syndrome: A systematic review and Meta-analysis.
Dawman, L; Panda, P; Panda, PK; Sharawat, IK, 2021
)
1.15
"Rufinamide is efficacious as adjunctive therapy in patients with LGS in terms of reduction in total seizure frequency and has mild adverse reaction."( Efficacy and safety of rufinamide as adjunctive therapy in patients with Lennox Gastaut syndrome: A systematic review and Meta-analysis.
Dawman, L; Panda, P; Panda, PK; Sharawat, IK, 2021
)
2.37
" The efficacy and safety were reported in terms of an at least 50% monthly seizure frequency reduction in drop seizures, dropout, and serious adverse events."( Efficacy and safety of antiseizure medication for Lennox-Gastaut syndrome: a systematic review and network meta-analysis.
Wang, C; Wang, J; Zhang, L, 2022
)
0.72

Pharmacokinetics

The objective of this study was to determine the pharmacokinetic properties and short-term adverse effect profile of single-dose oral rufinamide in healthy dogs. To evaluate the relationship between the pharmacokeretic (PK) parameters and therapeutic and adverse effects of rufInamide (RUF) in children with epileptic encephalopathies (EE) aged <4 years.

ExcerptReferenceRelevance
"To review the most important research published on the pharmacokinetic and pharmacodynamic properties of rufinamide (RFM), together with the outcomes of the clinical trials conducted to date with this new antiepileptic drug."( [Rufinamide. A review of its pharmacokinetic and pharmacodynamic properties].
Herranz, JL,
)
1.26
"The objective of this study was to determine the pharmacokinetic properties and short-term adverse effect profile of single-dose oral rufinamide in healthy dogs."( Pharmacokinetics of oral rufinamide in dogs.
Chen, AV; Davies, NM; Martinez, SE; Wright, HM, 2012
)
0.89
"To evaluate the relationship between the pharmacokinetic (PK) parameters and therapeutic and adverse effects of rufinamide (RUF) in children with epileptic encephalopathies (EE) aged <4 years."( A pharmacokinetic study and correlation with clinical response of rufinamide in infants with epileptic encephalopathies.
Della Bona, ML; Falchi, M; Guerrini, R; la Marca, G; Malvagia, S; Pellacani, S; Rosati, A, 2013
)
0.84
"53 mg/l) and half-life (13."( A pharmacokinetic study and correlation with clinical response of rufinamide in infants with epileptic encephalopathies.
Della Bona, ML; Falchi, M; Guerrini, R; la Marca, G; Malvagia, S; Pellacani, S; Rosati, A, 2013
)
0.63
" This work addresses that area by describing in a rapid pharmacokinetic study the main pharmacokinetic properties of rufinamide at three different doses of 1 mg/kg body weight (bw), 5 mg/kg bw, and 20 mg/kg bw."( Dose-dependent pharmacokinetics and brain penetration of rufinamide following intravenous and oral administration to rats.
Gáll, O; Gáll, Z; Kolcsár, M; Szilágyi, T; Vancea, S, 2015
)
0.87
"The purpose of this study was to determine the pharmacokinetic and safety profile of single and multiple doses of rufinamide in healthy Chinese subjects."( Pharmacokinetics and Tolerability of Rufinamide Following Single and Multiple Oral Doses and Effect of Food on Pharmacokinetics in Healthy Chinese Subjects.
Chen, H; He, X; Li, H; Li, W; Ni, Y; Xu, M; Zhou, Y, 2016
)
0.92
" Pharmacokinetic parameters, including the maximum plasma concentration (C max), the time to peak concentration (t max), the area under the plasma concentration versus time curve from time 0 to the last measurable concentration (AUC0-t ) and from time 0 to infinity (AUC0-∞), terminal elimination half-life (t 1/2), apparent volume of distribution (V d), apparent clearance (CL), average residence time (MRT), area under the plasma concentration versus time curve from time 0 to the last measurable concentration at steady state (AUCss), peak concentration (C max,ss) and trough level concentration (C min,ss) at steady state were calculated using non-compartmental models."( Pharmacokinetics and Tolerability of Rufinamide Following Single and Multiple Oral Doses and Effect of Food on Pharmacokinetics in Healthy Chinese Subjects.
Chen, H; He, X; Li, H; Li, W; Ni, Y; Xu, M; Zhou, Y, 2016
)
0.71

Compound-Compound Interactions

ExcerptReferenceRelevance
" Hence, their metabolic stability and potential involvement in relevant drug-drug interactions (DDI) are of great clinical interest, being HepaRG cells herein used as an in vitro human model."( Study of the metabolic stability profiles of perampanel, rufinamide and stiripentol and prediction of drug interactions using HepaRG cells as an in vitro human model.
Alves, G; Falcão, A; Fortuna, A; Meirinho, S; Rodrigues, M, 2022
)
0.97

Bioavailability

Rufinamide is well absorbed when taken with food, with an absolute bioavailability between 70% and 85%. This single-dose study found no statistically significant differences in relative bioavailability among each of the 3 test suspensions and the currently markete.

ExcerptReferenceRelevance
" Bioavailability of tablets relative to suspension and intra- and inter-subject variability were assessed by statistical analysis."( Intra- and inter-subject variabilities of CGP 33101 after replicate single oral doses of two 200-mg tablets and 400-mg suspension.
Chan, K; Cheung, WK; Cook, T; John, V; Kianifard, F; Mathieu, J; Redalieu, E; Wong, A, 1995
)
0.29
" When taken with food, rufinamide is relatively well absorbed in the lower dose range, with approximately dose-proportional plasma concentrations up to 1,600 mg/day, but less than dose-proportional plasma concentrations at higher doses due to reduced oral bioavailability."( Rufinamide: clinical pharmacokinetics and concentration-response relationships in patients with epilepsy.
Cloyd, J; Critchley, D; Fuseau, E; Perucca, E, 2008
)
2.1
" Rufinamide is well absorbed when taken with food, with an absolute bioavailability between 70% and 85%."( Rufinamide for pediatric patients with Lennox-Gastaut syndrome: a comprehensive overview.
Cerna, A; So, TY; Wier, HA, 2011
)
2.72
"The primary purpose of this study was to compare the relative bioavailability and other pharmacokinetics of rufinamide administered as a 400-mg tablet formulation (reference) with 10 mL of a newly developed 40-mg/mL suspension (test) manufactured using 3 different homogenization speeds in healthy subjects under fed conditions."( Bioavailability of three rufinamide oral suspensions compared with the marketed 400-mg tablet formulation: results from a randomized-sequence, open-label, four-period, four-sequence crossover study in healthy subjects.
Aluri, J; Bibbiani, F; Boyd, P; Critchley, DJ; Delargy, H; Narurkar, M; Whayman, M, 2011
)
0.89
"This single-dose study in a small population of fed, healthy subjects found no statistically significant differences in relative bioavailability among each of the 3 test suspensions and the currently marketed 400-mg tablet formulation of rufinamide, meeting FDA and EMA regulatory requirements for assuming bioequivalence."( Bioavailability of three rufinamide oral suspensions compared with the marketed 400-mg tablet formulation: results from a randomized-sequence, open-label, four-period, four-sequence crossover study in healthy subjects.
Aluri, J; Bibbiani, F; Boyd, P; Critchley, DJ; Delargy, H; Narurkar, M; Whayman, M, 2011
)
0.86
" The bioavailability of rufinamide is high, but decreases with the dose and increases with food intake."( [Therapeutic drug monitoring of rufinamide].
Bentué-Ferrer, D; Tribut, O; Verdier, MC,
)
0.72
"The ATP-binding cassette transporter P-glycoprotein (P-gp) is known to limit both brain penetration and oral bioavailability of many chemotherapy drugs."( A High-Throughput Screen of a Library of Therapeutics Identifies Cytotoxic Substrates of P-glycoprotein.
Ambudkar, SV; Brimacombe, KR; Chen, L; Gottesman, MM; Guha, R; Hall, MD; Klumpp-Thomas, C; Lee, OW; Lee, TD; Lusvarghi, S; Robey, RW; Shen, M; Tebase, BG, 2019
)
0.51

Dosage Studied

This work is concerned with development of two methods for determination of rufinamide (RUF) in presence of 1-[(2,6-difluorophenyl)methyl]-1H-1,2,3-triazole-4 carboxylic acid. We undertook a retrospective analysis of 77 patients with refractory epilepsy and receiving rufi.

ExcerptRelevanceReference
" Initial dosage and titration schedule of rufinamide were at the discretion of the treating physician according to medical need."( Effectiveness and tolerability of rufinamide in children and adults with refractory epilepsy: first European experience.
Bast, T; Boor, R; Ernst, JP; Haberlandt, E; Kluger, G; Kurlemann, G; Makowski, C; Runge, U; Schneider, F, 2009
)
0.9
" To explore rufinamide exposure under different dosing regimens in LGS patients, clinical trial simulations were performed."( Supporting the recommended paediatric dosing regimen for rufinamide in Lennox-Gastaut syndrome using clinical trial simulation.
Critchley, DJ; Fuseau, E; Marchand, M, 2010
)
0.98
" A liquid dosage form does not exist at the present time."( Stability of extemporaneously prepared rufinamide oral suspensions.
Best, R; Hutchinson, DJ; Liou, Y; Zhao, F, 2010
)
0.63
" We undertook a retrospective analysis of 77 patients with refractory epilepsy and receiving rufinamide to evaluate the drug's efficacy, tolerability, safety, and dosing schedules."( Experience with rufinamide in a pediatric population: a single center's experience.
Bergin, AM; Bourgeois, BF; Duffy, FH; Eksioglu, YZ; Gooty, VD; Kothare, SV; Libenson, M; Loddenkemper, T; Poduri, A; Rotenberg, A; Takeoka, M; Vendrame, M, 2010
)
0.93
" Serial blood samples were collected for 72 hours after dosing for the measurement of rufinamide in plasma."( Bioavailability of three rufinamide oral suspensions compared with the marketed 400-mg tablet formulation: results from a randomized-sequence, open-label, four-period, four-sequence crossover study in healthy subjects.
Aluri, J; Bibbiani, F; Boyd, P; Critchley, DJ; Delargy, H; Narurkar, M; Whayman, M, 2011
)
0.9
" These data complement the clinical trial results, by providing information on the efficacy and tolerability of rufinamide when used on an individualised basis in real-world practice, under less tightly restricted conditions in terms of patient population and dosing strategies."( Rufinamide from clinical trials to clinical practice in the United States and Europe.
Arzimanoglou, A; Brown, LW; Flamini, R; Harrison, M; Kerr, M; Kluger, G; Kothare, S; Narurkar, M; Philip, S; Resnick, T, 2011
)
2.02
" RUF was recommenced in two patients using a lower and slower dosing strategy; one patient showed improvement in seizure control and no weight loss but RUF was re-stopped in the second patient because of continued weight loss."( Dramatic weight loss with rufinamide.
Crespel, A; Gelisse, P; Mourand, I, 2013
)
0.69
" Final rufinamide mean dosage was 31."( Efficacy and safety of rufinamide in children under four years of age with drug-resistant epilepsies.
Accorsi, P; Balestri, P; Coppola, G; Dontin, SD; Gobbi, G; Grosso, S; Parisi, P; Pruna, D; Verrotti, A, 2014
)
1.17
" While in fed condition at the dosage of 200 mg, mean C max (SD) and mean AUC0-t (SD) were 2363 (582) μg/L and 40,593 (10,516) μg·h/L, respectively."( Pharmacokinetics and Tolerability of Rufinamide Following Single and Multiple Oral Doses and Effect of Food on Pharmacokinetics in Healthy Chinese Subjects.
Chen, H; He, X; Li, H; Li, W; Ni, Y; Xu, M; Zhou, Y, 2016
)
0.71
" No adjustments to body weight-based rufinamide dosing in subjects ≥ 1 to < 4 years are necessary."( Safety and pharmacokinetic profile of rufinamide in pediatric patients aged less than 4 years with Lennox-Gastaut syndrome: An interim analysis from a multicenter, randomized, active-controlled, open-label study.
Arzimanoglou, A; Bibbiani, F; Critchley, D; Dhadda, S; Ferreira, JA; Hussein, Z; Kumar, D; Mendes, S; Satlin, A; Schuck, E; Williams, B, 2016
)
0.98
" In clinical practice, rufinamide dosing and titration may differ from the trial setting."( Dosing considerations for rufinamide in patients with Lennox-Gastaut syndrome: Phase III trial results and real-world clinical data.
Bibbiani, F; Kluger, G; Kothare, S; Olhaye, O; Perdomo, C; Sachdeo, R; Williams, B, 2017
)
1.07
" Mean rufinamide dosage was 33."( Rufinamide efficacy and association with phenotype and genotype in children with intractable epilepsy: A retrospective single center study.
Bozarth, XL; Oesch, G, 2020
)
2.48
" The final mean dosage of rufinamide was 31."( Rufinamide as add-on therapy in children with epileptic encephalopathies other than Lennox-Gastaut syndrome: A study of 34 patients.
Caraballo, RH; Espeche, A; Fasulo, L; Galichio, S; Pociecha, J; Reyes, G; Semprino, M, 2020
)
2.3
" Median treatment duration was 15 months, and median rufinamide dosage at the last follow-up was 42 mg/kg/d (interquartile range 34-56)."( Efficacy and Tolerability of Rufinamide in Epileptic Children Younger Than 4 Years.
Loddenkemper, T; Tanritanir, A; Wang, X, 2021
)
1.16
"This work is concerned with development of two methods for determination of rufinamide (RUF) in presence of 1-[(2,6-difluorophenyl)methyl]-1H-1,2,3-triazole-4 carboxylic acid as its alkaline degradation product in dosage form."( Determination of Rufinamide in the Presence of 1-[(2,6-Difluorophenyl)Methyl]-1H-1,2,3-Triazole-4 Carboxylic Acid Using RP-HPLC and Derivative Ratio Methods as Stability Indicating Assays to Be Applied on Dosage Form.
Hashem, HMA; Hassib, ST; Mahrouse, MA; Mostafa, EA, 2020
)
1.13
"The first method was capable of determing RUF in the presence of its alkaline degradation product and in dosage form."( Determination of Rufinamide in the Presence of 1-[(2,6-Difluorophenyl)Methyl]-1H-1,2,3-Triazole-4 Carboxylic Acid Using RP-HPLC and Derivative Ratio Methods as Stability Indicating Assays to Be Applied on Dosage Form.
Hashem, HMA; Hassib, ST; Mahrouse, MA; Mostafa, EA, 2020
)
0.9
[information is derived through text-mining from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Drug Classes (2)

ClassDescription
heteroareneA heterocyclic compound formally derived from an arene by replacement of one or more methine (-C=) and/or vinylene (-CH=CH-) groups by trivalent or divalent heteroatoms, respectively, in such a way as to maintain the continuous pi-electron system characteristic of aromatic systems and a number of out-of-plane pi-electrons corresponding to the Hueckel rule (4n+2).
aromatic amideAn amide in which the amide linkage is bonded directly to an aromatic system.
[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 (11)

Potency Measurements

ProteinTaxonomyMeasurementAverage (µ)Min (ref.)Avg (ref.)Max (ref.)Bioassay(s)
Chain A, MAJOR APURINIC/APYRIMIDINIC ENDONUCLEASEHomo sapiens (human)Potency4.46680.003245.467312,589.2998AID2517
thioredoxin reductaseRattus norvegicus (Norway rat)Potency0.86750.100020.879379.4328AID488773; AID588453
Smad3Homo sapiens (human)Potency1.00000.00527.809829.0929AID588855
ParkinHomo sapiens (human)Potency14.58100.819914.830644.6684AID720573
arylsulfatase AHomo sapiens (human)Potency0.30131.069113.955137.9330AID720538
chromobox protein homolog 1Homo sapiens (human)Potency0.00600.006026.168889.1251AID488953
TAR DNA-binding protein 43Homo sapiens (human)Potency35.48131.778316.208135.4813AID652104
ATP-dependent phosphofructokinaseTrypanosoma brucei brucei TREU927Potency0.75690.060110.745337.9330AID485368
[prepared from compound, protein, and bioassay information from National Library of Medicine (NLM), extracted Dec-2023]

Inhibition Measurements

ProteinTaxonomyMeasurementAverageMin (ref.)Avg (ref.)Max (ref.)Bioassay(s)
Carbonic anhydrase 1Homo sapiens (human)Ki10.00000.00001.372610.0000AID1564385
Carbonic anhydrase 2Homo sapiens (human)Ki10.00000.00000.72369.9200AID1564386
Carbonic anhydrase 5A, mitochondrialHomo sapiens (human)Ki0.34380.00001.27259.9000AID1564387
[prepared from compound, protein, and bioassay information from National Library of Medicine (NLM), extracted Dec-2023]

Biological Processes (30)

Processvia Protein(s)Taxonomy
one-carbon metabolic processCarbonic anhydrase 1Homo sapiens (human)
morphogenesis of an epitheliumCarbonic anhydrase 2Homo sapiens (human)
positive regulation of synaptic transmission, GABAergicCarbonic anhydrase 2Homo sapiens (human)
positive regulation of cellular pH reductionCarbonic anhydrase 2Homo sapiens (human)
angiotensin-activated signaling pathwayCarbonic anhydrase 2Homo sapiens (human)
regulation of monoatomic anion transportCarbonic anhydrase 2Homo sapiens (human)
secretionCarbonic anhydrase 2Homo sapiens (human)
regulation of intracellular pHCarbonic anhydrase 2Homo sapiens (human)
neuron cellular homeostasisCarbonic anhydrase 2Homo sapiens (human)
positive regulation of dipeptide transmembrane transportCarbonic anhydrase 2Homo sapiens (human)
regulation of chloride transportCarbonic anhydrase 2Homo sapiens (human)
carbon dioxide transportCarbonic anhydrase 2Homo sapiens (human)
one-carbon metabolic processCarbonic anhydrase 2Homo sapiens (human)
one-carbon metabolic processCarbonic anhydrase 5A, mitochondrialHomo sapiens (human)
negative regulation of protein phosphorylationTAR DNA-binding protein 43Homo sapiens (human)
mRNA processingTAR DNA-binding protein 43Homo sapiens (human)
RNA splicingTAR DNA-binding protein 43Homo sapiens (human)
negative regulation of gene expressionTAR DNA-binding protein 43Homo sapiens (human)
regulation of protein stabilityTAR DNA-binding protein 43Homo sapiens (human)
positive regulation of insulin secretionTAR DNA-binding protein 43Homo sapiens (human)
response to endoplasmic reticulum stressTAR DNA-binding protein 43Homo sapiens (human)
positive regulation of protein import into nucleusTAR DNA-binding protein 43Homo sapiens (human)
regulation of circadian rhythmTAR DNA-binding protein 43Homo sapiens (human)
regulation of apoptotic processTAR DNA-binding protein 43Homo sapiens (human)
negative regulation by host of viral transcriptionTAR DNA-binding protein 43Homo sapiens (human)
rhythmic processTAR DNA-binding protein 43Homo sapiens (human)
regulation of cell cycleTAR DNA-binding protein 43Homo sapiens (human)
3'-UTR-mediated mRNA destabilizationTAR DNA-binding protein 43Homo sapiens (human)
3'-UTR-mediated mRNA stabilizationTAR DNA-binding protein 43Homo sapiens (human)
nuclear inner membrane organizationTAR DNA-binding protein 43Homo sapiens (human)
amyloid fibril formationTAR DNA-binding protein 43Homo sapiens (human)
regulation of gene expressionTAR DNA-binding protein 43Homo sapiens (human)
[Information is prepared from geneontology information from the June-17-2024 release]

Molecular Functions (15)

Processvia Protein(s)Taxonomy
arylesterase activityCarbonic anhydrase 1Homo sapiens (human)
carbonate dehydratase activityCarbonic anhydrase 1Homo sapiens (human)
protein bindingCarbonic anhydrase 1Homo sapiens (human)
zinc ion bindingCarbonic anhydrase 1Homo sapiens (human)
hydro-lyase activityCarbonic anhydrase 1Homo sapiens (human)
cyanamide hydratase activityCarbonic anhydrase 1Homo sapiens (human)
arylesterase activityCarbonic anhydrase 2Homo sapiens (human)
carbonate dehydratase activityCarbonic anhydrase 2Homo sapiens (human)
protein bindingCarbonic anhydrase 2Homo sapiens (human)
zinc ion bindingCarbonic anhydrase 2Homo sapiens (human)
cyanamide hydratase activityCarbonic anhydrase 2Homo sapiens (human)
carbonate dehydratase activityCarbonic anhydrase 5A, mitochondrialHomo sapiens (human)
zinc ion bindingCarbonic anhydrase 5A, mitochondrialHomo sapiens (human)
RNA polymerase II cis-regulatory region sequence-specific DNA bindingTAR DNA-binding protein 43Homo sapiens (human)
DNA bindingTAR DNA-binding protein 43Homo sapiens (human)
double-stranded DNA bindingTAR DNA-binding protein 43Homo sapiens (human)
RNA bindingTAR DNA-binding protein 43Homo sapiens (human)
mRNA 3'-UTR bindingTAR DNA-binding protein 43Homo sapiens (human)
protein bindingTAR DNA-binding protein 43Homo sapiens (human)
lipid bindingTAR DNA-binding protein 43Homo sapiens (human)
identical protein bindingTAR DNA-binding protein 43Homo sapiens (human)
pre-mRNA intronic bindingTAR DNA-binding protein 43Homo sapiens (human)
molecular condensate scaffold activityTAR DNA-binding protein 43Homo sapiens (human)
[Information is prepared from geneontology information from the June-17-2024 release]

Ceullar Components (16)

Processvia Protein(s)Taxonomy
cytosolCarbonic anhydrase 1Homo sapiens (human)
extracellular exosomeCarbonic anhydrase 1Homo sapiens (human)
cytoplasmCarbonic anhydrase 2Homo sapiens (human)
cytosolCarbonic anhydrase 2Homo sapiens (human)
plasma membraneCarbonic anhydrase 2Homo sapiens (human)
myelin sheathCarbonic anhydrase 2Homo sapiens (human)
apical part of cellCarbonic anhydrase 2Homo sapiens (human)
extracellular exosomeCarbonic anhydrase 2Homo sapiens (human)
cytoplasmCarbonic anhydrase 2Homo sapiens (human)
plasma membraneCarbonic anhydrase 2Homo sapiens (human)
apical part of cellCarbonic anhydrase 2Homo sapiens (human)
mitochondrial matrixCarbonic anhydrase 5A, mitochondrialHomo sapiens (human)
mitochondrionCarbonic anhydrase 5A, mitochondrialHomo sapiens (human)
cytoplasmCarbonic anhydrase 5A, mitochondrialHomo sapiens (human)
mitochondrionCarbonic anhydrase 5A, mitochondrialHomo sapiens (human)
intracellular non-membrane-bounded organelleTAR DNA-binding protein 43Homo sapiens (human)
nucleusTAR DNA-binding protein 43Homo sapiens (human)
nucleoplasmTAR DNA-binding protein 43Homo sapiens (human)
perichromatin fibrilsTAR DNA-binding protein 43Homo sapiens (human)
mitochondrionTAR DNA-binding protein 43Homo sapiens (human)
cytoplasmic stress granuleTAR DNA-binding protein 43Homo sapiens (human)
nuclear speckTAR DNA-binding protein 43Homo sapiens (human)
interchromatin granuleTAR DNA-binding protein 43Homo sapiens (human)
nucleoplasmTAR DNA-binding protein 43Homo sapiens (human)
chromatinTAR DNA-binding protein 43Homo sapiens (human)
[Information is prepared from geneontology information from the June-17-2024 release]

Bioassays (64)

Assay IDTitleYearJournalArticle
AID1347105qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for MG 63 (6-TG R) cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347107qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for Rh30 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347104qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for RD cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1745845Primary qHTS for Inhibitors of ATXN expression
AID1347093qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for SK-N-MC cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347099qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for NB1643 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347103qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for OHS-50 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347091qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for SJ-GBM2 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347407qHTS to identify inhibitors of the type 1 interferon - major histocompatibility complex class I in skeletal muscle: primary screen against the NCATS Pharmaceutical Collection2020ACS chemical biology, 07-17, Volume: 15, Issue:7
High-Throughput Screening to Identify Inhibitors of the Type I Interferon-Major Histocompatibility Complex Class I Pathway in Skeletal Muscle.
AID1347089qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for TC32 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1296008Cytotoxic Profiling of Annotated Libraries Using Quantitative High-Throughput Screening2020SLAS discovery : advancing life sciences R & D, 01, Volume: 25, Issue:1
Cytotoxic Profiling of Annotated and Diverse Chemical Libraries Using Quantitative High-Throughput Screening.
AID1347106qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for control Hh wild type fibroblast cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347092qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for A673 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347101qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for BT-12 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347108qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for Rh41 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1508630Primary qHTS for small molecule stabilizers of the endoplasmic reticulum resident proteome: Secreted ER Calcium Modulated Protein (SERCaMP) assay2021Cell reports, 04-27, Volume: 35, Issue:4
A target-agnostic screen identifies approved drugs to stabilize the endoplasmic reticulum-resident proteome.
AID1347082qHTS for Inhibitors of the Functional Ribonucleoprotein Complex (vRNP) of Lassa (LASV) Arenavirus: LASV Primary Screen - GLuc reporter signal2020Antiviral research, 01, Volume: 173A cell-based, infectious-free, platform to identify inhibitors of lassa virus ribonucleoprotein (vRNP) activity.
AID1347097qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for Saos-2 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347154Primary screen GU AMC qHTS for Zika virus inhibitors2020Proceedings of the National Academy of Sciences of the United States of America, 12-08, Volume: 117, Issue:49
Therapeutic candidates for the Zika virus identified by a high-throughput screen for Zika protease inhibitors.
AID1347090qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for DAOY cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID651635Viability Counterscreen for Primary qHTS for Inhibitors of ATXN expression
AID1347083qHTS for Inhibitors of the Functional Ribonucleoprotein Complex (vRNP) of Lassa (LASV) Arenavirus: Viability assay - alamar blue signal for LASV Primary Screen2020Antiviral research, 01, Volume: 173A cell-based, infectious-free, platform to identify inhibitors of lassa virus ribonucleoprotein (vRNP) activity.
AID1347098qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for SK-N-SH cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347100qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for LAN-5 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347096qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for U-2 OS cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347094qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for BT-37 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347095qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for NB-EBc1 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347102qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for Rh18 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347425Rhodamine-PBP qHTS Assay for Modulators of WT P53-Induced Phosphatase 1 (WIP1)2019The Journal of biological chemistry, 11-15, Volume: 294, Issue:46
Physiologically relevant orthogonal assays for the discovery of small-molecule modulators of WIP1 phosphatase in high-throughput screens.
AID1347086qHTS for Inhibitors of the Functional Ribonucleoprotein Complex (vRNP) of Lymphocytic Choriomeningitis Arenaviruses (LCMV): LCMV Primary Screen - GLuc reporter signal2020Antiviral research, 01, Volume: 173A cell-based, infectious-free, platform to identify inhibitors of lassa virus ribonucleoprotein (vRNP) activity.
AID1347424RapidFire Mass Spectrometry qHTS Assay for Modulators of WT P53-Induced Phosphatase 1 (WIP1)2019The Journal of biological chemistry, 11-15, Volume: 294, Issue:46
Physiologically relevant orthogonal assays for the discovery of small-molecule modulators of WIP1 phosphatase in high-throughput screens.
AID1346987P-glycoprotein substrates identified in KB-8-5-11 adenocarcinoma cell line, qHTS therapeutic library screen2019Molecular pharmacology, 11, Volume: 96, Issue:5
A High-Throughput Screen of a Library of Therapeutics Identifies Cytotoxic Substrates of P-glycoprotein.
AID1346986P-glycoprotein substrates identified in KB-3-1 adenocarcinoma cell line, qHTS therapeutic library screen2019Molecular pharmacology, 11, Volume: 96, Issue:5
A High-Throughput Screen of a Library of Therapeutics Identifies Cytotoxic Substrates of P-glycoprotein.
AID1474166Liver toxicity in human assessed as induction of drug-induced liver injury by measuring severity class index2016Drug discovery today, Apr, Volume: 21, Issue:4
DILIrank: the largest reference drug list ranked by the risk for developing drug-induced liver injury in humans.
AID1564386Inhibition of recombinant human carbonic anhydrase 2 preincubated with enzyme for 15 mins by phenol red dye based stopped flow CO2 hydration assay2019European journal of medicinal chemistry, Nov-01, Volume: 181A computer-assisted discovery of novel potential anti-obesity compounds as selective carbonic anhydrase VA inhibitors.
AID1564385Inhibition of recombinant human carbonic anhydrase 1 preincubated with enzyme for 15 mins by phenol red dye based stopped flow CO2 hydration assay2019European journal of medicinal chemistry, Nov-01, Volume: 181A computer-assisted discovery of novel potential anti-obesity compounds as selective carbonic anhydrase VA inhibitors.
AID1564387Inhibition of recombinant human carbonic anhydrase VA preincubated with enzyme for 15 mins by phenol red dye based stopped flow CO2 hydration assay2019European journal of medicinal chemistry, Nov-01, Volume: 181A computer-assisted discovery of novel potential anti-obesity compounds as selective carbonic anhydrase VA inhibitors.
AID1474167Liver toxicity in human assessed as induction of drug-induced liver injury by measuring verified drug-induced liver injury concern status2016Drug discovery today, Apr, Volume: 21, Issue:4
DILIrank: the largest reference drug list ranked by the risk for developing drug-induced liver injury in humans.
AID588501High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Lethal Factor Protease, MLPCN compound set2010Current protocols in cytometry, Oct, Volume: Chapter 13Microsphere-based flow cytometry protease assays for use in protease activity detection and high-throughput screening.
AID588501High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Lethal Factor Protease, MLPCN compound set2006Cytometry. Part A : the journal of the International Society for Analytical Cytology, May, Volume: 69, Issue:5
Microsphere-based protease assays and screening application for lethal factor and factor Xa.
AID588501High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Lethal Factor Protease, MLPCN compound set2010Assay and drug development technologies, Feb, Volume: 8, Issue:1
High-throughput multiplex flow cytometry screening for botulinum neurotoxin type a light chain protease inhibitors.
AID588499High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Botulinum neurotoxin light chain A protease, MLPCN compound set2010Current protocols in cytometry, Oct, Volume: Chapter 13Microsphere-based flow cytometry protease assays for use in protease activity detection and high-throughput screening.
AID588499High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Botulinum neurotoxin light chain A protease, MLPCN compound set2006Cytometry. Part A : the journal of the International Society for Analytical Cytology, May, Volume: 69, Issue:5
Microsphere-based protease assays and screening application for lethal factor and factor Xa.
AID588499High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Botulinum neurotoxin light chain A protease, MLPCN compound set2010Assay and drug development technologies, Feb, Volume: 8, Issue:1
High-throughput multiplex flow cytometry screening for botulinum neurotoxin type a light chain protease inhibitors.
AID588497High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Botulinum neurotoxin light chain F protease, MLPCN compound set2010Current protocols in cytometry, Oct, Volume: Chapter 13Microsphere-based flow cytometry protease assays for use in protease activity detection and high-throughput screening.
AID588497High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Botulinum neurotoxin light chain F protease, MLPCN compound set2006Cytometry. Part A : the journal of the International Society for Analytical Cytology, May, Volume: 69, Issue:5
Microsphere-based protease assays and screening application for lethal factor and factor Xa.
AID588497High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Botulinum neurotoxin light chain F protease, MLPCN compound set2010Assay and drug development technologies, Feb, Volume: 8, Issue:1
High-throughput multiplex flow cytometry screening for botulinum neurotoxin type a light chain protease inhibitors.
AID504812Inverse Agonists of the Thyroid Stimulating Hormone Receptor: HTS campaign2010Endocrinology, Jul, Volume: 151, Issue:7
A small molecule inverse agonist for the human thyroid-stimulating hormone receptor.
AID504810Antagonists of the Thyroid Stimulating Hormone Receptor: HTS campaign2010Endocrinology, Jul, Volume: 151, Issue:7
A small molecule inverse agonist for the human thyroid-stimulating hormone receptor.
AID504749qHTS profiling for inhibitors of Plasmodium falciparum proliferation2011Science (New York, N.Y.), Aug-05, Volume: 333, Issue:6043
Chemical genomic profiling for antimalarial therapies, response signatures, and molecular targets.
AID1347057CD47-SIRPalpha protein protein interaction - LANCE assay qHTS validation2019PloS one, , Volume: 14, Issue:7
Quantitative high-throughput screening assays for the discovery and development of SIRPα-CD47 interaction inhibitors.
AID1347405qHTS to identify inhibitors of the type 1 interferon - major histocompatibility complex class I in skeletal muscle: primary screen against the NCATS LOPAC collection2020ACS chemical biology, 07-17, Volume: 15, Issue:7
High-Throughput Screening to Identify Inhibitors of the Type I Interferon-Major Histocompatibility Complex Class I Pathway in Skeletal Muscle.
AID1347045Natriuretic polypeptide receptor (hNpr1) antagonism - Pilot counterscreen GloSensor control cell line2019Science translational medicine, 07-10, Volume: 11, Issue:500
Inhibition of natriuretic peptide receptor 1 reduces itch in mice.
AID588349qHTS for Inhibitors of ATXN expression: Validation of Cytotoxic Assay
AID504836Inducers of the Endoplasmic Reticulum Stress Response (ERSR) in human glioma: Validation2002The Journal of biological chemistry, Apr-19, Volume: 277, Issue:16
Sustained ER Ca2+ depletion suppresses protein synthesis and induces activation-enhanced cell death in mast cells.
AID1347410qHTS for inhibitors of adenylyl cyclases using a fission yeast platform: a pilot screen against the NCATS LOPAC library2019Cellular signalling, 08, Volume: 60A fission yeast platform for heterologous expression of mammalian adenylyl cyclases and high throughput screening.
AID1347058CD47-SIRPalpha protein protein interaction - HTRF assay qHTS validation2019PloS one, , Volume: 14, Issue:7
Quantitative high-throughput screening assays for the discovery and development of SIRPα-CD47 interaction inhibitors.
AID1347059CD47-SIRPalpha protein protein interaction - Alpha assay qHTS validation2019PloS one, , Volume: 14, Issue:7
Quantitative high-throughput screening assays for the discovery and development of SIRPα-CD47 interaction inhibitors.
AID588378qHTS for Inhibitors of ATXN expression: Validation
AID1347050Natriuretic polypeptide receptor (hNpr2) antagonism - Pilot subtype selectivity assay2019Science translational medicine, 07-10, Volume: 11, Issue:500
Inhibition of natriuretic peptide receptor 1 reduces itch in mice.
AID1347049Natriuretic polypeptide receptor (hNpr1) antagonism - Pilot screen2019Science translational medicine, 07-10, Volume: 11, Issue:500
Inhibition of natriuretic peptide receptor 1 reduces itch in mice.
AID1347151Optimization of GU AMC qHTS for Zika virus inhibitors: Unlinked NS2B-NS3 protease assay2020Proceedings of the National Academy of Sciences of the United States of America, 12-08, Volume: 117, Issue:49
Therapeutic candidates for the Zika virus identified by a high-throughput screen for Zika protease inhibitors.
AID1794808Fluorescence-based screening to identify small molecule inhibitors of Plasmodium falciparum apicoplast DNA polymerase (Pf-apPOL).2014Journal of biomolecular screening, Jul, Volume: 19, Issue:6
A High-Throughput Assay to Identify Inhibitors of the Apicoplast DNA Polymerase from Plasmodium falciparum.
AID1794808Fluorescence-based screening to identify small molecule inhibitors of Plasmodium falciparum apicoplast DNA polymerase (Pf-apPOL).
[information is prepared from bioassay data collected from National Library of Medicine (NLM), extracted Dec-2023]

Research

Studies (168)

TimeframeStudies, This Drug (%)All Drugs %
pre-19900 (0.00)18.7374
1990's4 (2.38)18.2507
2000's25 (14.88)29.6817
2010's108 (64.29)24.3611
2020's31 (18.45)2.80
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Market Indicators

Research Demand Index: 62.75

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 very strong demand-to-supply ratio for research on this compound.

MetricThis Compound (vs All)
Research Demand Index62.75 (24.57)
Research Supply Index5.29 (2.92)
Research Growth Index5.50 (4.65)
Search Engine Demand Index103.38 (26.88)
Search Engine Supply Index2.00 (0.95)

This Compound (62.75)

All Compounds (24.57)

Study Types

Publication TypeThis drug (%)All Drugs (%)
Trials22 (12.57%)5.53%
Reviews41 (23.43%)6.00%
Case Studies15 (8.57%)4.05%
Observational4 (2.29%)0.25%
Other93 (53.14%)84.16%
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Clinical Trials (13)

Trial Overview

TrialPhaseEnrollmentStudy TypeStart DateStatus
Pharmacokinetics and Tolerability of Rufinamide Following Single and Multiple Oral Doses and Effect of Food on Pharmacokinetics in Healthy Chinese Subjects [NCT02332174]Phase 140 participants (Actual)Interventional2014-03-31Completed
Effect of Rufinamide on Chronic Postthoracotomy Pain Syndrome. A Prospective, Randomized, Double-blind, Placebo Controlled, Monocenter Study. [NCT02095899]Phase 20 participants (Actual)Interventional2014-03-31Withdrawn(stopped due to Poor study patient recruitment)
A Randomised Controlled Trial of the Ketogenic Diet in the Treatment of Epilepsy in Children Under the Age of Two Years [NCT02205931]Phase 4160 participants (Anticipated)Interventional2015-01-31Recruiting
An Open-Label Extension Study of Rufinamide Given as Adjunctive Therapy in Patients With Refractory Partial Seizures [NCT00448539]Phase 3286 participants (Actual)Interventional2007-03-15Terminated(stopped due to The study was terminated early by the sponsor due to the discontinuation of clinical development for rufinamide.)
A Double-Blind, Placebo-Controlled, Parallel-Group Study of Rufinamide Given as Adjunctive Therapy in Patients With Refractory Partial Seizures [NCT04558580]Phase 30 participants (Actual)Interventional2006-04-30Withdrawn
An Extended Access Program (EAP) for Rufinamide in Pediatric Participants With Inadequately Controlled Lennox-Gastaut Syndrome [NCT03778424]0 participants Expanded AccessAvailable
A Double-Blind, Placebo-Controlled, Parallel-Group Study of Rufinamide Given as Adjunctive Therapy in Patients With Refractory Partial Seizures [NCT00334958]Phase 3356 participants (Actual)Interventional2006-02-13Completed
A Placebo-Controlled, Double-Blind Comparative Study of E2080 in Lennox-Gastaut Syndrome Patients [NCT01146951]Phase 366 participants (Actual)Interventional2010-06-30Completed
A Long Term Extension Study of E2080 in Lennox-Gastaut Patients [NCT01151540]Phase 354 participants (Actual)Interventional2010-11-30Completed
[NCT02175173]707 participants (Actual)Observational2013-06-13Completed
A Multi-Center,Randomized, Double-Blind, Placebo-Controlled, Phase 2, Exploratory Study to Evaluate the Effect of Rufinamide on Anxiety in Patients With Moderate to Severe Generalized Anxiety Disorder [NCT00595231]Phase 2239 participants (Actual)Interventional2008-03-31Completed
Population Pharmacokinetics of Antiepileptic in Pediatrics [NCT03196466]1,000 participants (Anticipated)Observational2017-06-19Recruiting
A Multicenter, Randomized, Controlled, Open-label Study to Evaluate the Cognitive Development Effects and Safety, and Pharmacokinetics of Adjunctive Rufinamide Treatment in Pediatric Subjects 1 to Less Than 4 Years of Age With Inadequately Controlled Lenn [NCT01405053]Phase 337 participants (Actual)Interventional2011-06-16Completed
[information is prepared from clinicaltrials.gov, extracted Sep-2024]

Trial Outcomes

TrialOutcome
NCT00334958 (4) [back to overview]Log10 Transformed Total Partial Seizure Frequency Per 28 Days During the Baseline Phase and Maintenance Phase
NCT00334958 (4) [back to overview]Percentage Change in Total Partial Seizure Frequency Per 28 Days During Maintenance Phase Relative to the Baseline Phase
NCT00334958 (4) [back to overview]Percentage of Participants With 50% or Greater Reduction in Total Partial Seizure Frequency Per 28 Days During the Maintenance Phase Relative to the Baseline Phase
NCT00334958 (4) [back to overview]Reduction From Baseline in Total Partial Seizure Frequency Rate (RRATIO) During Maintenance Phase
NCT00448539 (1) [back to overview]Percentage Change in Total Partial Seizure Frequency Per 28 Days Relative to the Baseline Phase
NCT00595231 (6) [back to overview]Change From Baseline in Clinical Global Impression Scale for Severity of Illness: (CGI-S)
NCT00595231 (6) [back to overview]Change From Baseline in Montgomery-Asberg Depression Scale (MADRS) Summary Statistics.
NCT00595231 (6) [back to overview]Change From Baseline Raskin Depression Scale.
NCT00595231 (6) [back to overview]Change From Baseline in Covi Anxiety Scale (CAS) - Index Total Score: Summary Statistics Observed
NCT00595231 (6) [back to overview]Change From Baseline in Hamilton Anxiety Scale (HAMA) - Total Score: Summary Statistics - Observed
NCT00595231 (6) [back to overview]Change From Baseline in Hospital Anxiety and Depression Scale (HADS). Summary Statistics
NCT01146951 (5) [back to overview]Percent Change in Tonic-Atonic Seizure Frequency From Baseline (Per 28 Days)
NCT01146951 (5) [back to overview]Percent Change in Total Seizure Frequency (Per 28 Days)
NCT01146951 (5) [back to overview]Clinical Global Impression of Change (CGIC)
NCT01146951 (5) [back to overview]Number of Participants Achieving a 50% Reduction in Tonic-atonic Seizure Frequency
NCT01146951 (5) [back to overview]Percentage Change in the Frequency of Seizures Other Than Tonic-atonic Seizures (Per 28 Days)
NCT01151540 (6) [back to overview]Number of Participants With Adverse Events as a Measure of Safety and Tolerability of Rufinamide
NCT01151540 (6) [back to overview]Percent Change in the Frequency of Seizures Other Than Tonic-Atonic Seizures
NCT01151540 (6) [back to overview]Percent Change in the Total Seizure Frequency From Baseline (Per 28 Days)
NCT01151540 (6) [back to overview]Percent Change in Tonic-Atonic Seizure Frequency From Baseline (Per 28 Days)
NCT01151540 (6) [back to overview]Percentage of Participants Who Achieved 100%, 75%, 50% or 25% Reduction in Tonic-Atonic Seizure Frequency (Responders)
NCT01151540 (6) [back to overview]Percentage of Participants With An Increase In Tonic-Atonic Seizure Frequency
NCT01405053 (10) [back to overview]Child Behavior Checklist (CBCL) Total Problem T-scores at the End of 2-year Treatment Period
NCT01405053 (10) [back to overview]Percent Change in Total Seizure Frequency Per 28 Days
NCT01405053 (10) [back to overview]Time to Withdrawal From Treatment Due to an Adverse Event or Lack of Efficacy
NCT01405053 (10) [back to overview]Change From Baseline in CBCL Sub Scores at Week 106
NCT01405053 (10) [back to overview]Change From Baseline in CBCL Total Problem T-Scores at End of 2-year Treatment Period
NCT01405053 (10) [back to overview]Change From Baseline in Language Development Survey (LDS) Scores During Maintenance Period
NCT01405053 (10) [back to overview]Change From Baseline in Sub-scores in QoLCE
NCT01405053 (10) [back to overview]Change From Baseline in Total Score of Quality of Life in Childhood Epilepsy (QoLCE) Scale
NCT01405053 (10) [back to overview]Incidence of Worsening of Seizures
NCT01405053 (10) [back to overview]Percent Change in Seizure Frequency by Individual Seizure Type Per 28 Days

Log10 Transformed Total Partial Seizure Frequency Per 28 Days During the Baseline Phase and Maintenance Phase

Total partial seizure frequencies per 28 days during the double-blind Maintenance and Baseline Phases were transformed using logarithms to the base 10 (log10), because it was expected from previous studies that the results would not be normally distributed. (NCT00334958)
Timeframe: Days 13 to 96

InterventionSeizures per 28-days (log-transformed) (Mean)
Rufinamide0.98
Placebo1.13

[back to top]

Percentage Change in Total Partial Seizure Frequency Per 28 Days During Maintenance Phase Relative to the Baseline Phase

Seizure data was collected via patient diary, which was used to record daily seizure count and type. Intent-to-treat (ITT) population: All randomized participants who had baseline Patient Seizure Diary data and had at least completed the titration period. (NCT00334958)
Timeframe: Baseline, Days 13 to 96

InterventionPercentage change (Median)
Rufinamide-23.25
Placebo-9.80

[back to top]

Percentage of Participants With 50% or Greater Reduction in Total Partial Seizure Frequency Per 28 Days During the Maintenance Phase Relative to the Baseline Phase

Seizure data was collected via patient diary, which was used to record daily seizure count and type. (NCT00334958)
Timeframe: Baseline, Days 13 to 96

InterventionPercentage of Participants (Number)
Rufinamide32.5
Placebo14.3

[back to top]

Reduction From Baseline in Total Partial Seizure Frequency Rate (RRATIO) During Maintenance Phase

RRATIO= 100*(T-B)/(T+B) where T= total seizure frequency per 28 days during the Maintenance Phase, and B=total seizure frequency per 28 days during the Baseline Phase. (NCT00334958)
Timeframe: Baseline, Days 13 to 96

InterventionRRATIO (Mean)
Rufinamide-18.76
Placebo-6.90

[back to top]

Percentage Change in Total Partial Seizure Frequency Per 28 Days Relative to the Baseline Phase

"Seizure data was collected via patient diaries. OL refers to open-label." (NCT00448539)
Timeframe: Baseline, Titration Phase (Days 1 to 18), Maintenance Phase

,
InterventionPercentage change (Median)
OL Titration PhaseOL Maintenance Phase
Rufinamide (Placebo During Core Study)-45.10-31.10
Rufinamide (Rufinamide During Core Study)-35.65-30.95

[back to top]

Change From Baseline in Clinical Global Impression Scale for Severity of Illness: (CGI-S)

Severity of illness is the first scale in the CGI. A rating is filled in by the investigator at the start of treatment based on a 0-7 point weighted scale. It goes from not assessed (0), to among the most extremely ill patients (7). (NCT00595231)
Timeframe: 8 weeks

Interventionscore on a scale (Mean)
Rufinamide4.5
Placebo4.4

[back to top]

Change From Baseline in Montgomery-Asberg Depression Scale (MADRS) Summary Statistics.

The MADRS (Montgomery and Asberg 1979) is a clinician-rated instrument that measures the presence and severity of depression. This instrument consists of 10 items. Each item is rated on a defined step scale of 0 to 6 with anchors at 2-point intervals. The MADRS total score is the sum of the 10 items and ranges from 0 to 60. A high numeric rating shows a greater degree of symptom severity. (NCT00595231)
Timeframe: 8 Weeks

Interventionscore on a scale (Mean)
Rufinamide15.7
Placebo16.4

[back to top]

Change From Baseline Raskin Depression Scale.

It explores the extent to which an individual demonstrates depression on three sub-scales (rated 1-5): verbal self-report, behavior and secondary symptoms of depression. Scores range from 3-15, with higher scores indicating greater severity. (NCT00595231)
Timeframe: 8 weeks

Interventionscore on a scale (Mean)
Rufinamide5.2
Placebo5.3

[back to top]

Change From Baseline in Covi Anxiety Scale (CAS) - Index Total Score: Summary Statistics Observed

"The Covi Anxiety Scale is a simple 3 item scale for the assessment of severity of anxiety symptoms. The scale measures 3 dimensions: verbal report, behavior and somatic symptoms of anxiety. Each item scored on a scale of 1 - 5. (1)not at all, (2)somewhat, (3)moderately, (4) considerably, and (5)very much), hence the scale is a 5- to 15 point range. The three items are the patient's verbal report (feeling shaky, jittery, jumpy), observed behavior consistent with anxiety during the interview (e.g. appearing frightened, shaky, restless) and somatic complains (e.g., sweating, trembling, heart pounding).~COVI Rating Scale total score is the sum of items 1 - 3." (NCT00595231)
Timeframe: 8 weeks

,
Interventionscore on a scale (Mean)
ScreeningBaselineWeek 3Week 6Week 8Last Observation
Placebo10.510.68.17.67.47.7
Rufinamide10.710.78.37.57.17.7

[back to top]

Change From Baseline in Hamilton Anxiety Scale (HAMA) - Total Score: Summary Statistics - Observed

The Hamilton Anxiety Scale is a 14-item test measuring the severity of anxiety symptoms. It provides measures of overall anxiety, psychic anxiety (mental agitation and psychological distress), and somatic anxiety (physical complaints related to anxiety). The interviewer then rated the individuals on a 5-point scale for each of the 14 items. Seven of the items specifically address psychic anxiety and the remaining 7 items address somatic anxiety. The total anxiety score ranges from 0 to 56. The 7 psychic anxiety items elicit a psychic anxiety score that ranges from 0 to 28. The remaining 7 items yield a somatic anxiety score that also ranges from 0 to 28. A score of 17 or less indicates mild anxiety severity. A score from 18 to 24 indicates mild to moderate anxiety severity, a score of 25 to 30 indicates a moderate to severe anxiety and lastly a score of 31-56 is very severe. HAM-A total score is the sum of items 1 - 14. (NCT00595231)
Timeframe: 8 Weeks

,
Interventionscore on a scale (Mean)
ScreeningBaselineWeek 1Week 2Week 3Week 6Week 8Last Observation
Placebo25.625.821.419.617.816.715.116.1
Rufinamide25.525.521.920.018.015.715.116.7

[back to top]

Change From Baseline in Hospital Anxiety and Depression Scale (HADS). Summary Statistics

The Hospital Anxiety and Depression Scale is a self screening questionnaire for depression and anxiety. It consists of 14 questions, seven for anxiety and seven for depression. The 14 statements are relevant to either generalized anxiety (7 statements) or 'depression' (again 7). Each question has 4 possible responses. Responses are scored on a scale from 3 to 0. The maximum score is therefore 21 for depression and 21 for anxiety. A score of 11 or higher indicates the probable presence of the mood disorder with a score of 8 to 10 being just suggestive of the presence of the respective state. The 2 subscales, anxiety and depression, have been found to be independent measures. In its current form the HADS is now divided into 4 ranges: normal (0-7), mild (8-10), moderate (11-15) and severe (16-21). Anxiety score = sum of items 1, 3, 5, 7, 9, 11, and 13. Depression score = sum of items 2, 4, 6, 8, 10, 12, and 14. (NCT00595231)
Timeframe: 8 Weeks

,
Interventionscore on a scale (Mean)
Week 1Week 2Week 3Week 6Week 8Last Observation
Placebo-0.1-0.4-0.5-1.2-1.6-1.4
Rufinamide-0.2-0.4-1.0-1.4-2.0-1.2

[back to top]

Percent Change in Tonic-Atonic Seizure Frequency From Baseline (Per 28 Days)

"The sum of the frequencies of tonic seizures and atonic seizures was defined as the tonic-atonic seizure frequency and the percent change in tonic-atonic seizure frequency per 28 days was assessed. The percent change in tonic-atonic seizure frequency was calculated using the tonic-atonic seizure frequency per 28 days of the Observation Period as the baseline and the tonic-atonic seizure frequency per 28 days of the Treatment Period as the post-treatment value. Percentage change in tonic - atonic seizure frequency was calculated as follows: [100 x (post-treatment value - baseline)/ baseline].~The frequency of epileptic seizures was recorded in the seizure diary by the recorder. Seizure frequency was counted based on the classification established by the International League Against Epilepsy (ILAE). The diary recorder monitored the participant and recorded the seizure diary in a consistent manner, and continued these practices throughout the study period." (NCT01146951)
Timeframe: Baseline (28 day observational period) and End of Treatment (28 day treatment period)

InterventionPercent Change (Median)
Rufinamide (E2080)-24.20
Placebo-3.25

[back to top]

Percent Change in Total Seizure Frequency (Per 28 Days)

Percent change in the total seizure frequency (per 28 days) was calculated using the total seizure frequency per 28 days of the Observation Period as the baseline and the total seizure frequency per 28 days of the Treatment Period as the post-treatment value. Percentage change in total seizure frequency was calculated as follows: [100 x (post-treatment value - baseline)/ baseline]. (NCT01146951)
Timeframe: Baseline (28 day observational period) and End of Treatment (28 day treatment period)

InterventionPercent Change (Median)
Rufinamide (E2080)-32.90
Placebo-3.05

[back to top]

Clinical Global Impression of Change (CGIC)

"CGIC in participants with Lennox-Gastaut Syndrome (LGS) relative to placebo was presented as number of participants in each category at the final assessment (last observation carried forward [LOCF]) & at Week 12 of the Treatment Period. The investigator assessed the CGIC by comparing the participants' condition during the 4 weeks immediately before the completion (or discontinuation [d/c]) of the Treatment Period to his/her condition during the 4-week Observation Period (for participants who d/c'd the study during the Treatment Period, the CGIC was assessed by comparing the participant's condition from the start to discontinuation of the study treatment to his/her condition during the 4-week Observation Period).~The CGIC was assessed according to the following 7-grade scale based on the frequency & severity of seizures, AEs, and overall conditions of daily life.~Markedly improved, Improved, Slightly improved, Unchanged, Slightly worsened, Worsened, Markedly worsened." (NCT01146951)
Timeframe: Up to Week 12 of the treatment period

,
Interventionparticipants (Number)
Week 12: Markedly improved (n=25, 29)Week 12: Improved (n=25, 29)Week 12: Slightly Improved (n=25, 29)Week 12: Unchanged (n=25, 29)Week 12: Slightly Worsened (n=25, 29)Week 12: Worsened (n=25, 29)Week 12: Markedly Worsened (n=25, 29)LOCF: Markedly Improved (n=28, 30)LOCF: Improved (n=28, 30)LOCF: Slightly Improved (n=28, 30)LOCF: Unchanged (n=28, 30)LOCF: Slightly Worsened (n=28, 30)LOCF: Worsened (n=28, 30)LOCF: Markedly Worsened (n=28, 30)
Placebo0091811000919110
Rufinamide (E2080)0941011039410110

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Number of Participants Achieving a 50% Reduction in Tonic-atonic Seizure Frequency

50% Responder Rate in Tonic-Atonic Seizure Frequency was presented as the number of participants who achieved a 50% reduction in tonic-atonic seizure frequency. (NCT01146951)
Timeframe: 12 weeks

,
InterventionParticipants (Number)
Yes (50% Reduction Achieved)No
Placebo228
Rufinamide (E2080)721

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Percentage Change in the Frequency of Seizures Other Than Tonic-atonic Seizures (Per 28 Days)

"Percent change in the frequency of seizures other than tonic-atonic seizures (per 28 days) was calculated using the total seizure frequency per 28 days of the Observation Period as the baseline and the total seizure frequency per 28 days of the Treatment Period as the post-treatment value. Percentage change in total seizure frequency was calculated as follows: [100 x (post-treatment value - baseline)/ baseline].~Seizures analyzed other than tonic-atonic seizures included:~Partial seizure freq. (frequency), Absence seizure, Atyp. (atypical) absence seizure, Myoclonic seizure, Clonic seizure, Tonic seizure, Tonic-clonic seizure, Atonic seizure, & Uncla. (unclassified) epileptic seizure.~The frequency of epileptic seizures was recorded in the diary by the recorder. Seizure frequency was counted based on the classification established by the International League Against Epilepsy (ILAE). The diary recorder monitored the participant and recorded the seizure diary in a consistent manner." (NCT01146951)
Timeframe: Baseline (28 day observational period) and End of Treatment (28 day treatment period)

,
InterventionPercent change (Median)
Partial Seizure Freq. % Change (n=4,6)Absence Seizure Freq. % Change (n=1,0)Atyp. Absence Seizure Freq. % Change (n=12,19)Myoclonic Seizure Freq. % Change (n=10,10)Clonic Seizure Freq. % Change (n=1,0)Tonic Seizure Freq. % Change (n=28,28)Tonic-clonic Seizure Freq. % Change (n=2,10)Atonic Seizure Freq. % Change (n=10,12)Uncla. Epileptic Seizure Freq. % Change (n=1,0)
Placebo4.5NA-21.106.60NA-3.602.35-6.10NA
Rufinamide (E2080)-52.203.40-59.00-52.35-81.20-24.20-57.35-63.10-88.70

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Number of Participants With Adverse Events as a Measure of Safety and Tolerability of Rufinamide

Safety was assessed by monitoring and recording all adverse events (AEs), serious adverse events (SAEs), clinical laboratory tests, blood pressure, pulse rate, physical examination, and 12-lead electrocardiogram (ECG). Treatment-emergent adverse events (TEAEs) were defined as AEs that started on or after the date and time of administration of first dose of test drug, but not later than 30 days after discontinuation from the study, or if the AE was present prior to the administration of the first dose of test drug and increased in National Cancer Institute Common Toxicity Criteria (NCI CTC version 3.0) grade during the study or 30 days after discontinuation from the study. AEs were considered serious if it resulted in; death, was life-threatening, hospitalization/prolonged hospitalization, persistent or significant disability/incapacity, or a congenital anomaly/birth defect. (NCT01151540)
Timeframe: From date of first dose up to 30 days after the last dose of study treatment, up to approximately 2 years 10 months

InterventionParticipants (Number)
TEAEsTreatment-related TEAEsSAEsTreatment-related SAEsAEs leading to study drug withdrawalAEs leading to study drug dose reduction
Rufinamide543892312

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Percent Change in the Frequency of Seizures Other Than Tonic-Atonic Seizures

Percent change in the frequency of seizures other than tonic-atonic seizures (per 28 days) was calculated using the total seizure frequency per 28 days of the Observation Period as the baseline and the total seizure frequency per 28 days of the Weeks, 12, 24, 32, 40, 52 and 52 LOCF as the post-treatment value. Percentage change in total seizure frequency was calculated as follows: [100 x (post-treatment value - baseline)/ baseline]. Seizures analyzed other than tonic-atonic seizures included: Partial seizure frequency, Absence seizure, Atypical absence seizure, Myoclonic seizure, Tonic seizure, Tonic-clonic seizure, Atonic seizure, and Unclassified epileptic seizure. This data was based on the diary data collected for 7 days after each visit. Seizure frequency was counted based on the classification established by the ILAE. The diary recorder monitored the participant and recorded the seizure diary in a consistent manner. (NCT01151540)
Timeframe: Baseline (Observation period in Study 304), Week 12, Week 24, Week 32, Week 40, Week 52 and Week 52 LOCF

InterventionPercent Change in Seizure Frequency (Median)
Partial Seizure Week 12Partial Seizure Week 24Partial Seizure Week 32Partial Seizure Week 40Partial Seizure Week 52Partial Seizure Week 52 LOCFAbsence Seizure Week 12Absence Seizure Week 24Absence Seizure Week 32Absence Seizure Week 40Absence Seizure Week 52Absence Seizure Week 52 LOCFAtypical Absence Seizure Week 12Atypical Absence Seizure Week 24Atypical Absence Seizure Week 32Atypical Absence Seizure Week 40Atypical Absence Seizure Week 52Atypical Absence Seizure Week 52 LOCFMyoclonic Seizure Week 12Myoclonic Seizure Week 24Myoclonic Seizure Week 32Myoclonic Seizure Week 40Myoclonic Seizure Week 52Myoclonic Seizure Week 52 LOCFTonic Seizure Week 12Tonic Seizure Week 24Tonic Seizure Week 32Tonic Seizure Week 40Tonic Seizure Week 52Tonic Seizure Week 52 LOCFTonic-clonic Seizure Week 12Tonic-clonic Seizure Week 24Tonic-clonic Seizure Week 32Tonic-clonic Seizure Week 40Tonic-clonic Seizure Week 52Tonic-clonic Seizure Week 52 LOCFAtonic Seizure Week 12Atonic Seizure Week 24Atonic Seizure Week 32Atonic Seizure Week 40Atonic Seizure Week 52Atonic Seizure Week 52 LOCFUnclassified Seizure Week 12Unclassified Seizure Week 24Unclassified Seizure Week 32Unclassified Seizure Week 40Unclassified Seizure Week 52Unclassified Seizure Week 52 LOCF
Rufinamide-95-80.9-70.5-85.7-77.3-77.3-87.7-100-100-100-100-100-86.7-92.85-92.3-100-100-100-100-100-100-100-100-100-35.4-37.85-49.4-47.05-36.4-46.2-61.55-44.1-22.6-46.7-35.5-38.1-60.35-84.3-100-67.2-67.55-67.55-1006932.3-100-100-100-100

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Percent Change in the Total Seizure Frequency From Baseline (Per 28 Days)

Percent change in the total seizure frequency (per 28 days) was calculated using the total seizure frequency per 28 days of the Observation Period of Study 304 as the baseline and the total seizure frequency per 28 days at Weeks 12, 24, 32, 40, 52 and Week 52 LOCF as the post-treatment value. Percentage change in total seizure frequency was calculated as follows: [100 x (post-treatment value - baseline)/ baseline]. (NCT01151540)
Timeframe: Baseline (Observation period in Study 304), Week 12, Week 24, Week 32, Week 40, Week 52 and Week 52 LOCF

InterventionPercent Change in Seizure Frequency (Median)
Percent Change in Week 12Percent Change in Week 24Percent Change in Week 32Percent Change in Week 40Percent Change in Week 52Percent Change in Week 52 LOCF
Rufinamide-47.7-48.9-50.6-52-47.35-46.3

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Percent Change in Tonic-Atonic Seizure Frequency From Baseline (Per 28 Days)

"The sum of the frequencies of tonic seizures and atonic seizures was defined as the tonic-atonic seizure frequency. The percent change in tonic-atonic seizure frequency was calculated using the tonic-atonic seizure frequency per 28 days of the Observation Period in Study 304 as the baseline and the tonic-atonic seizure frequency at Weeks 12, 24, 32, 40, 52 and Week 52 Last Observation Carried Forward (LOCF) as the post-treatment value. Percentage change in tonic - atonic seizure frequency was calculated as follows: [100 x (post-treatment value - baseline)/ baseline]. The frequency of epileptic seizures was recorded in the seizure diary by the recorder. Seizure frequency was counted based on the classification established by the International League Against Epilepsy (ILAE). The diary recorder monitored the participant and recorded the seizure diary in a consistent manner, and continued these practices throughout the study period." (NCT01151540)
Timeframe: Baseline (Observation period in Study 304), Week 12, Week 24, Week 32, Week 40, Week 52 and Week 52 LOCF

InterventionPercent Change in Seizure Frequency (Median)
Percent Change in Week 12Percent Change in Week 24Percent Change in Week 32Percent Change in Week 40Percent Change in Week 52Percent Change in Week 52 LOCF
Rufinamide-39.3-40.6-46.8-47.6-36.05-39.25

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Percentage of Participants Who Achieved 100%, 75%, 50% or 25% Reduction in Tonic-Atonic Seizure Frequency (Responders)

Categorized percent change in Tonic-atonic seizure frequency per 28 Days by visit relative to the baseline (Observation Phase in Study 304) was determined based on the diary data collected for 7 days after each visit. The Efficacy Analysis Set was used. (NCT01151540)
Timeframe: Week 12, Week 24, Week 32, Week 40, Week 52 and Week 52 LOCF

InterventionPercentage of participants (Number)
Week 12 100% Reduction - YesWeek 12 100% Reduction - NoWeek 12 75% Reduction - YesWeek 12 75% Reduction - NoWeek 12 50% Reduction - YesWeek 12 50% Reduction - NoWeek 12 25% Reduction - YesWeek 12 25% Reduction - NoWeek 24 100% Reduction - YesWeek 24 100% Reduction - NoWeek 24 75% Reduction - YesWeek 24 75% Reduction - NoWeek 24 50% Reduction - YesWeek 24 50% Reduction -NoWeek 24 25% Reduction - YesWeek 24 25% Reduction - NoWeek 32 100% Reduction - YesWeek 32 100% Reduction - NoWeek 32 75% Reduction - YesWeek 32 75% Reduction - NoWeek 32 50% Reduction - YesWeek 32 50% Reduction - NoWeek 32 25% Reduction - YesWeek 32 25% Reduction - NoWeek 40 100% Reduction - YesWeek 40 100% Reduction - NoWeek 40 75% Reduction - YesWeek 40 75% Reduction - NoWeek 40 50% Reduction - YesWeek 40 50% Reduction - NoWeek 40 25% Reduction - YesWeek 40 25% Reduction - NoWeek 52 100% Reduction - YesWeek 52 100% Reduction - NoWeek 52 75% Reduction - YesWeek 52 75% Reduction -NoWeek 52 50% Reduction - YesWeek 52 50% Reduction - NoWeek 52 25% Reduction - YesWeek 52 25% Reduction - NoWeek 52 (LOCF) 100% Reduction - YesWeek 52 (LOCF) 100% Reduction - NoWeek 52 (LOCF) 75% Reduction - YesWeek 52 (LOCF) 75% Reduction -NoWeek 52 (LOCF) 50% Reduction - YesWeek 52 (LOCF) 50% Reduction - NoWeek 52 (LOCF) 25% Reduction - YesWeek 52 (LOCF) 25% Reduction - No
Rufinamide6.593.517.482.643.556.571.728.32.397.711.688.439.560.565.134.92.497.6198147.652.466.733.34.995.117.182.948.851.26139595208037.562.560408.791.321.778.339.160.96337

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Percentage of Participants With An Increase In Tonic-Atonic Seizure Frequency

Number of participants with an increase in Tonic-atonic seizure frequency per 28 Days by visit relative to the baseline (Observation Phase in Study 304) was determined based on the diary data collected for 7 days after each visit. The Efficacy Analysis Set was used. (NCT01151540)
Timeframe: Week 12, Week 24, Week 32, Week 40, Week 52 and Week 52 LOCF

InterventionPercentage of participants (Number)
Week 12 Increase - YesWeek 12 Increase - NoWeek 24 Increase - YesWeek 24 Increase - NoWeek 32 Increase - YesWeek 32 Increase - NoWeek 40 Increase - YesWeek 40 Increase - NoWeek 52 Increase - YesWeek 52 Increase -NoWeek 52 LOCF Increase - YesWeek 52 LOCF Increase - No
Rufinamide21.778.323.376.716.783.39.890.222.577.519.680.4

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Child Behavior Checklist (CBCL) Total Problem T-scores at the End of 2-year Treatment Period

CBCL: 99-item questionnaire measures specific behavioral problems or developmental delays, answered by a parent/legal guardian or suitable caregiver. Each item were rated using 3-point scale (0=Not True, 1=Somewhat/Sometimes True, 2=Very True/ Often True) to indicate how often or typical the behavior was. The 99 items were combined to yield scores for 8 problem area scales (emotionally reactive, anxious/depressed, somatic complaints, withdrawn, sleep problems, attention problems, aggressive behavior, and other problems) and 3 summary scores (internalizing, externalizing, and total problems). Total Problem score was sum of all the problem areas plus 1 additional item, ranging from 0 to 198. Total raw scores are converted to t-scores with mean of 50 and standard deviation (SD) of 10. T-scores were standardized test scores that indicate same degree of elevation in problems relative to the normative sample of peers. Higher scores were indicative of more problems. (NCT01405053)
Timeframe: End of Treatment Period (up to approximately Week 106)

Interventionscore on a scale (Mean)
Rufinamide55.7
Any Other Approved Antiepileptic Drug54.8

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Percent Change in Total Seizure Frequency Per 28 Days

The frequency per 28 days was defined as (S/D)*28 where, S was equal to the sum of the seizures reported in the participant seizure diary during the specified time interval and D was equal to the number of days with non-missing data in the participant seizure diary for the specified study phase. The number of seizures was assessed and recorded by the participant's parent(s)/caregiver(s) in the participant seizure diary. (NCT01405053)
Timeframe: Baseline up to End of the Treatment Period (up to approximately Week 106)

Interventionpercent change in seizure frequency (Median)
Rufinamide-7.05
Any Other Approved Antiepileptic Drug-20.15

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Time to Withdrawal From Treatment Due to an Adverse Event or Lack of Efficacy

Withdrawal from either rufinamide or other AED was due to the occurrence of an adverse event or for lack of efficacy. Data was obtained till Week 106 and was extrapolated using Kaplan-Meier method to determine the overall survival time (in weeks) to withdrawal from treatment (excluding taper) due to an adverse event or lack efficacy. (NCT01405053)
Timeframe: Baseline up to the End of the Treatment Period (up to approximately Week 106)

Interventionweeks (Median)
Rufinamide142.0
Any Other Approved Antiepileptic Drug28.0

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Change From Baseline in CBCL Sub Scores at Week 106

CBCL: 99-item questionnaire, measures behavioral problems/developmental delays, answered by parent/guardian/caregiver. Each item rated on 3-point scale (0=Not True,1=Somewhat/Sometimes True, 2=Very/Often True). 99 items were combined to give scores for 8 problem area scales, where 1 for each 8 syndrome (emotionally reactive, anxious/depressed, somatic, withdrawn, sleep, attention, aggressive behavior, and other problems) were calculated, range: 0 (normal) to 16 (clinical behavior) and 3 summary scores (internalizing, externalizing, and total problems). All 3 summary scores reported scaled to T-scores. Total Problem score was sum of all the problem areas plus 1 additional item, ranging from 0 to 198. Total raw score were converted to t-scores with mean of 50 and SD of 10. T-scores were standardized test scores that indicate same degree of elevation in problems relative to normative sample of peers. Higher scores were indicative of more problems. (NCT01405053)
Timeframe: Baseline and Week 106

,
Interventionscore on a scale (Mean)
Baseline: Total emotional reactive scoresChange at Week 106:Total emotional reactive scoresBaseline: Total anxious/depression scoresChange at Week 106:Total anxious/depression scoresBaseline: Total Somatic Complaints ScoresChange at Week 106:Total Somatic Complaints ScoresBaseline: Total withdrawn scoresChange at Week 106:Total withdrawn scoresBaseline: Total Sleep Problems ScoresChange at Week 106:Total sleep problem scoresBaseline: Total attention problems scoresChange at Week 106:Total attention problems scoresBaseline: Total Aggressive Behavior ScoresChange at Week106:Total aggressive behavior scoresBaseline: Total internalizing scoresChange at Week 106:Total internalizing scoresBaseline: Total externalizing scoresChange at Week 106:Total externalizing scores
Any Other Approved Antiepileptic Drug60.9-1.354.60.754.9-1.772.1-7.062.4-5.765.9-7.758.6-0.360.6-2.758.1-3.7
Rufinamide59.0-1.156.40.559.40.171.5-2.257.8-1.959.3-1.152.53.261.6-1.547.54.7

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Change From Baseline in CBCL Total Problem T-Scores at End of 2-year Treatment Period

CBCL: 99-item questionnaire measures specific behavioral problems or developmental delays, answered by a parent/legal guardian or suitable caregiver. Each item were rated using 3-point scale (0=Not True, 1=Somewhat/Sometimes True, 2=Very True/ Often True) to indicate how often or typical the behavior was. The 99 items were combined to yield scores for 8 problem area scales (emotionally reactive, anxious/depressed, somatic complaints, withdrawn, sleep problems, attention problems, aggressive behavior, and other problems) and 3 summary scores (internalizing, externalizing, and total problems). Total Problem score was sum of all the problem areas plus 1 additional item, ranging from 0 to 198. Total raw scores are converted to t-scores with mean of 50 and standard deviation (SD) of 10. T-scores were standardized test scores that indicate same degree of elevation in problems relative to the normative sample of peers. Higher scores were indicative of more problems. (NCT01405053)
Timeframe: Baseline and End of Treatment Period (up to approximately Week 106)

,
Interventionscore on a scale (Geometric Mean)
BaselineChange at Week 106
Any Other Approved Antiepileptic Drug62.8-6.7
Rufinamide56.6-0.3

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Change From Baseline in Language Development Survey (LDS) Scores During Maintenance Period

LDS, a caregiver-administered survey consisted of 8-item questionnaire and vocabulary list of 310 words organized within 14 semantic categories. List contained high frequency words (e.g. more), less common words (e.g. hamburger), and lexical chunks (e.g. Sesame Street). Average LDS score, calculated by dividing total number of words across all valid phrases by number of phrases with greater than (>) 0words; for participants with no words, average was 0. This value was compared to standardized chart to obtain percentile rating. LDS provided 2 scores: average phrase length (number of words/phrase) and number of endorsed vocabulary words. LDS phrase length was categorized into delay (less than or equal to [<=] 20th percentile) and no delay (>20th percentile). LDS vocabulary was categorized into delay(<=15th percentile)and no delay(>15th percentile). Both raw scores were used to provide 2 normative scores based on child's age in months. Higher scores indicated better language development. (NCT01405053)
Timeframe: Baseline, Weeks 24, 56, 88, and 106

,
Interventionwords (Mean)
Baseline: LDS average phrase lengthChange at Week 24: LDS average phrase lengthChange at Week 56: LDS average phrase lengthChange at Week 88: LDS average phrase lengthChange at Week 106: LDS average phrase lengthBaseline:LDS Vocabulary ScoreChange at Week 24:LDS Vocabulary ScoreChange at Week 56:LDS Vocabulary ScoreChange at Week 88:LDS Vocabulary ScoreChange at Week 106:LDS Vocabulary Score
Any Other Approved Antiepileptic Drug00.70.00.00.00.64.8-0.400.01.0
Rufinamide0.30.20.10.10.410.47.117.925.439.6

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Change From Baseline in Sub-scores in QoLCE

The QoLCE was a 76-item questionnaire designed specifically to measure quality of life in children with epilepsy. QOLCE consists of 16 quality of life subscales (14 multi-item and 2 single item). Each subscales had number of items or questions with responses as excellent, very good, good, fair, and poor. They were changed to 1, 2, 3, 4, and 5 as per instructions. Then changed on a scale of 100, where 1=0, 2=25, 3=50, 4=75, and 5=100. Items corresponding to each subscale were marked and there mean score was score of that subscale. The form was completed by a parent or caregiver who interacted with the child on a consistent, daily basis and took about 20 to 30 minutes to complete. The higher the score, the better the child's quality of life. (NCT01405053)
Timeframe: Baseline and Week 106

,
Interventionscore on a scale (Mean)
Baseline: Physical restrictionChange at Week 106: Physical restrictionBaseline: Energy/FatigueChange at Week 106: Energy/FatigueBaseline: Attention/ConcentrationChange at Week 106:Attention/ConcentrationBaseline: MemoryChange at Week 106: MemoryBaseline: LanguageChange at Week 106: LanguageBaseline: Other cognitiveChange at Week 106: Other cognitiveBaseline: DepressionChange at Week 106: DepressionBaseline: AnxietyChange at Week 106: AnxietyBaseline: Control/HelplessnessChange at Week 106: Control/HelplessnessBaseline: Self-esteemChange at Week 106: Self-esteemBaseline: Social interactionsChange at Week 106: Social interactionsBaseline: Social activitiesChange at Week 106: Social activitiesBaseline: StigmaChange at Week 106: StigmaBaseline: BehaviorChange at Week 106: BehaviorBaseline: General-healthChange at Week 106: General healthBaseline: Quality-of-lifeChange at Week 106: Quality-of-life
Any Other Approved Antiepileptic Drug49.9-6.844.32.851.12.552.60.553.1-0.553.1-1.045.32.348.51.347.83.050.5-6.050.54.046.63.550.74.545.87.349.0-2.050.73.3
Rufinamide50.1-1.051.6-3.149.9-2.150.2-0.549.8-0.548.60.351.2-2.650.1-0.150.70.250.1-1.349.9-1.550.50.948.9-0.151.40.250.5-1.349.51.1

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Change From Baseline in Total Score of Quality of Life in Childhood Epilepsy (QoLCE) Scale

The QoLCE was a 76-item questionnaire designed specifically to measure quality of life in children with epilepsy. QOLCE consists of 16 quality of life subscales (14 multi-item and 2 single item). Each subscales had number of items or questions with responses as excellent, very good, good, fair, and poor. They were changed to 1, 2, 3, 4, and 5 as per instructions. Then changed on a scale of 100, where 1 is equal to (=) 0, 2=25, 3=50, 4=75, and 5=100. Items corresponding to each subscale were marked and there mean score was score of that subscale. The form was completed by a parent or caregiver who interacted with the child on a consistent, daily basis and took about 20 to 30 minutes to complete. The higher the score, the better the child's quality of life. (NCT01405053)
Timeframe: Baseline and Week 106

,
Interventionscore on a scale (Mean)
BaselineWeek 106
Any Other Approved Antiepileptic Drug49.61.5
Rufinamide50.4-1.3

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Incidence of Worsening of Seizures

Worsening of seizures was summarized by the incidence of participants with doubling in total seizure frequency, doubling in frequency of major seizures (generalized tonic-clonic, drop attacks), or occurrence of new seizure type during each successive 3 to 4 month visit interval of the Maintenance Period relative to baseline. (NCT01405053)
Timeframe: Baseline up to End of Treatment Period (up to approximately Week 106)

,
InterventionParticipants (Count of Participants)
Doubling in total seizure frequencyDoubling in frequency of major seizuresOccurrence of a new seizure type
Any Other Approved Antiepileptic Drug110
Rufinamide450

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Percent Change in Seizure Frequency by Individual Seizure Type Per 28 Days

The frequency per 28 days was defined as (S/D)*28 where, S was equal to the sum of the seizures reported in the participant seizure diary during the specified time interval and D was equal to the number of days with non-missing data in the participant seizure diary for the specified study phase. The number of seizures was assessed and recorded by the participant's parent(s)/caregiver(s) in the participant seizure diary. (NCT01405053)
Timeframe: Baseline up to End of Treatment Period (up to approximately Week 106)

,
Interventionpercent change in seizure frequency (Median)
Partial seizuresAbsence seizuresAtypical absence seizuresMyoclonic seizuresClonic seizuresTonic-atonic seizuresPrimary generalized tonic-clonic seizuresOther seizures
Any Other Approved Antiepileptic Drug-57.65-49.704.90-27.90-48.35-31.80-96.60-100.00
Rufinamide-39.8-23.6-70.95-24.60-60.85-35.20-97.80-90.65

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