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Clinical trials for Ataluren (PTC124)

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    21 result(s) found for: Ataluren (PTC124). Displaying page 1 of 2.
    1  2  Next»
    EudraCT Number: 2016-001764-11 Sponsor Protocol Number: PTC124-GD-030-DMD Start Date*: 2020-01-21
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: A Phase 2 Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Ataluren (PTC124®) in Patients Aged ≥2 to <5 Years Old With Nonsense Mutation Dystrophinopathy
    Medical condition: nonsense mutation dystrophinopathy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    20.0 10010331 - Congenital, familial and genetic disorders 10059117 Becker's muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2009-016654-41 Sponsor Protocol Number: PTC124-GD-012-MMA Start Date*: 2010-03-02
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: A Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia
    Medical condition: Nonsense Mutation Methylmalonic Acidemia
    Disease: Version SOC Term Classification Code Term Level
    12.1 10064180 Blood methylmalonic acid increased LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Completed) GB (Prematurely Ended) IT (Prematurely Ended) BE (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2010-019692-30 Sponsor Protocol Number: PTC124-GD-009e-CF Start Date*: 2010-08-10
    Sponsor Name:PTC Therapeutics, Inc
    Full Title: A Phase 3 Extension Study of Ataluren (PTC124) in Subjects with Nonsense-Mutation-Mediated Cystic Fibrosis
    Medical condition: Nonsense-Mutation-Mediated Cystic Fibrosis
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10011762 Cystic fibrosis PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed) FR (Completed) NL (Completed) SE (Completed) IT (Completed) DE (Completed) ES (Completed) GB (Completed)
    Trial results: View results
    EudraCT Number: 2013-005489-20 Sponsor Protocol Number: PTC124-GD-020e-DMD Start Date*: 2014-08-22
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy
    Medical condition: Nonsense mutation dystrophinopathy
    Disease: Version SOC Term Classification Code Term Level
    18.1 10010331 - Congenital, familial and genetic disorders 10059117 Becker's muscular dystrophy PT
    18.1 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: SE (Completed) GB (Completed) BE (Completed) DE (Completed) IT (Completed) ES (Completed) CZ (Completed) FR (Completed) BG (Completed)
    Trial results: View results
    EudraCT Number: 2019-001767-67 Sponsor Protocol Number: PTC124-GD-045-DMD Start Date*: 2020-01-30
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: Phase 2 Clinical Pharmacology Study to Assess Dystrophin Levels in Subjects With nmDMD Before and After Treatment with Ataluren
    Medical condition: Non-Sense Mutation Duchenne Muscular Dystrophy (nmDMD)
    Disease:
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2019-001691-11 Sponsor Protocol Number: PTC124-GD-046-DMD Start Date*: 2020-03-06
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: Phase 2, Non-Interventional, Clinical Study to Assess Dystrophin Levels in Subjects With Nonsense Mutation Duchenne Muscular Dystrophy Who Have Been Treated With Ataluren for ≥9 Months
    Medical condition: Non-Sense Mutation Duchenne Muscular Dystrophy (nmDMD)
    Disease:
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2013-005449-35 Sponsor Protocol Number: PTC124-GD-023-CF Start Date*: 2014-08-26
    Sponsor Name: PTC Therapeutics, Inc.
    Full Title: An Open-Label Safety and Efficacy Study for Patients with Nonsense Mutation Cystic Fibrosis Previously Treated with Ataluren (PTC124®)
    Medical condition: Nonsense Mutation Cystic Fibrosis
    Disease: Version SOC Term Classification Code Term Level
    18.0 10010331 - Congenital, familial and genetic disorders 10011762 Cystic fibrosis PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: BE (Prematurely Ended) SE (Prematurely Ended) IT (Completed) DE (Completed) ES (Completed) FR (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2014-005355-83 Sponsor Protocol Number: PTC124-GD-021e-CF Start Date*: 2015-10-01
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: A Phase 3 extension Study of Ataluren (PTC124®) in Patients with Nonsense Mutation Cystic Fibrosis
    Medical condition: Nonsense Mutation Cystic Fibrosis
    Disease: Version SOC Term Classification Code Term Level
    18.0 10010331 - Congenital, familial and genetic disorders 10011762 Cystic fibrosis PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: BE (Prematurely Ended) IT (Prematurely Ended) ES (Completed) NL (Prematurely Ended) FR (Prematurely Ended) BG (Prematurely Ended) GR (Completed) GB (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2008-007648-32 Sponsor Protocol Number: PTC124 GD 007e DMD Start Date*: 2009-04-14
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy
    Medical condition: Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    9.1 10013801 Duchenne muscular dystrophy LLT
    9.1 10059117 Becker's muscular dystrophy LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: BE (Completed) FR (Completed) DE (Completed) SE (Prematurely Ended) GB (Prematurely Ended) ES (Completed) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2009-013169-24 Sponsor Protocol Number: PTC124-GD-008-DMD Start Date*: 2010-01-19
    Sponsor Name:PTC Therapeutics Inc
    Full Title: A Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients with Nonsense–Mutation-Mediated Duchenne/Becker Muscular Dystrophy
    Medical condition: Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    12.0 10013801 Duchenne muscular dystrophy LLT
    12.0 10059117 Becker's muscular dystrophy LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: GB (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2020-000980-21 Sponsor Protocol Number: PTC124-GD-048-DMD Start Date*: 2021-06-24
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: An Open-Label Study Evaluating the Safety and Pharmacokinetics of Ataluren in Children From ≥6 Months to <2 Years of Age with Nonsense Mutation Duchenne Muscular Dystrophy
    Medical condition: Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
    Disease:
    Population Age: Infants and toddlers, Under 18 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2015-003105-41 Sponsor Protocol Number: PTC124-GD-031-MPS Start Date*: 2015-10-13
    Sponsor Name:PTC Therapeutics Inc.
    Full Title: CNS Unmet Medical Need in Mucopolysaccharidosis: A Phase 2 Safety and Pharmacokinetics Study of Ataluren (COMPASS)
    Medical condition: Nonsense mutation Mucopolysaccharidosis Type I
    Disease: Version SOC Term Classification Code Term Level
    18.1 10010331 - Congenital, familial and genetic disorders 10056886 Mucopolysaccharidosis I PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2014-002596-28 Sponsor Protocol Number: PTC124-GD-024-MPS Start Date*: 2015-06-01
    Sponsor Name:PTC Therapeutics Inc.
    Full Title: CNS Unmet Medical Need in Mucopolysaccharidosis: A Phase 2 Safety and Pharmacokinetics Study of Ataluren (COMPASS)
    Medical condition: Nonsense mutation Mucopolysaccharidosis Type I
    Disease: Version SOC Term Classification Code Term Level
    18.0 10010331 - Congenital, familial and genetic disorders 10056886 Mucopolysaccharidosis I PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) GB (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2012-004527-20 Sponsor Protocol Number: PTC124-GD-020-DMD Start Date*: 2013-07-30
    Sponsor Name:PTC Therapeutics, Inc
    Full Title: A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy
    Medical condition: Nonsense mutation dystrophinopathy
    Disease: Version SOC Term Classification Code Term Level
    17.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    17.0 10010331 - Congenital, familial and genetic disorders 10059117 Becker's muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: BE (Completed) CZ (Completed) SE (Completed) GB (Completed) DE (Completed) IT (Completed) ES (Completed) PL (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2011-004853-18 Sponsor Protocol Number: PTC124-GD-019-DMD Start Date*: 2012-04-27
    Sponsor Name:PTC Therapeutics, Inc
    Full Title: An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy
    Medical condition: Nonsense mutation dystrophinopathy
    Disease: Version SOC Term Classification Code Term Level
    16.1 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    16.1 10010331 - Congenital, familial and genetic disorders 10059117 Becker's muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: BE (Completed) SE (Completed) DE (Completed) GB (Completed) ES (Completed) FR (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2013-004581-34 Sponsor Protocol Number: PTC124-GD-021-CF Start Date*: 2014-09-15
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: A Phase 3 Efficacy and Safety Study of Ataluren (PTC124®) in Patients with Nonsense Mutation Cystic Fibrosis
    Medical condition: Nonsense Mutation Cystic Fibrosis
    Disease: Version SOC Term Classification Code Term Level
    17.0 10010331 - Congenital, familial and genetic disorders 10011762 Cystic fibrosis PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Completed) BE (Completed) DE (Temporarily Halted) NL (Completed) ES (Completed) GB (Completed) BG (Completed) GR (Completed)
    Trial results: View results
    EudraCT Number: 2010-020224-22 Sponsor Protocol Number: PTC124-GD-011-HEM Start Date*: 2010-08-26
    Sponsor Name:PTC Therapeutics, Inc
    Full Title: A Phase 2a Study of Ataluren (PTC124) as an Oral Treatment for Nonsense-Mutation-Mediated Hemophilia A and B
    Medical condition: Nonsense-Mutation-Mediated Hemophilia A and B
    Disease: Version SOC Term Classification Code Term Level
    12.1 10060613 Hemophilia A (Factor VIII) LLT
    12.1 10060614 Hemophilia B (Factor IX) LLT
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2017-001223-49 Sponsor Protocol Number: PTC124-GD-041DMD Start Date*: 2018-01-16
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: A 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
    Medical condition: Nonsense Mutation Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: BG (Completed) PL (Completed)
    Trial results: (No results available)
    EudraCT Number: 2008-003924-52 Sponsor Protocol Number: PTC124-GD-009-CF Start Date*: 2009-04-08
    Sponsor Name:PTC Therapeutics, Inc
    Full Title: A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis
    Medical condition: Nonsense-Mutation-Mediated Cystic Fibrosis
    Disease: Version SOC Term Classification Code Term Level
    9.1 10011762 Cystic fibrosis LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Completed) NL (Completed) BE (Completed) ES (Completed) FR (Completed) GB (Completed) IT (Completed) DE (Completed)
    Trial results: View results
    EudraCT Number: 2022-001013-39 Sponsor Protocol Number: PTC124-GD-028-ANI Start Date*: 2022-04-14
    Sponsor Name:PTC Therapeutics, Inc
    Full Title: A Phase 2, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study of the Safety and Efficacy of Ataluren (PTC124) for the Treatment of Nonsense Mutation Aniridia
    Medical condition: Nonsense Mutation Aniridia
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
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