Translarna 1000 mg granules for oral suspension    (DrugBank: -)

1 disease
告示番号疾患名(ページ内リンク)臨床試験数
113筋ジストロフィー2

113. 筋ジストロフィー [臨床試験数:567,薬物数:442(DrugBank:93),標的遺伝子数:55,標的パスウェイ数:151
Searched query = "Muscular dystrophy", "Dystrophinopathies", "Myotilinopathy", "Laminopathy", "Caveolinopathy", "LGMD1C", "Desminopathy", "Sarcoglycanopathy", "α-dystroglycanopathy", "FCMD", "Walker-Warburg syndrome", "Muscle-eye-brain disease", "Myotonic dystrophy", "Integrin α7 deficient CMD", "Rigid spine syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 567 trials found
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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Inclusion_
agemin
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PhaseCountries
1EUCTR2017-001223-49-BG
(EUCTR)
16/01/201811/10/2017A clinical trial to evaluate the safety and efficacy of Ataluren versus placebo in people who have specific genetic disease Duchenne Muscular DystrophyA Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension Nonsense Mutation Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Translarna 125 mg granules for oral suspension
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
Trade Name: Translarna 250 mg granules for oral suspension
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
Trade Name: Translarna 1000 mg granules for oral suspension
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
PTC Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
340Phase 3United States;Taiwan;Hong Kong;Thailand;Turkey;Russian Federation;India;Mexico;Canada;Jordan;Argentina;Brazil;Malaysia;Poland;Australia;Bulgaria;China;Japan;Korea, Republic of
2EUCTR2017-001223-49-PL
(EUCTR)
05/06/2018A clinical trial to evaluate the safety and efficacy of Ataluren versus placebo in people who have specific genetic disease Duchenne Muscular DystrophyA Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension Nonsense Mutation Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Translarna 125 mg granules for oral suspension
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
Trade Name: Translarna 250 mg granules for oral suspension
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
Trade Name: Translarna 1000 mg granules for oral suspension
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
PTC Therapeutics, Inc.NULLNAFemale: no
Male: yes
340Phase 3United States;Taiwan;Hong Kong;Thailand;Turkey;Russian Federation;India;Mexico;Canada;Jordan;Argentina;Poland;Brazil;Malaysia;Australia;Bulgaria;China;Japan;Korea, Republic of