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Clinical trials for Nonsense Mutation

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    The EU Clinical Trials Register currently displays   44379   clinical trials with a EudraCT protocol, of which   7391   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    39 result(s) found for: Nonsense Mutation. Displaying page 1 of 2.
    1  2  Next»
    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: 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: 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: 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
    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: 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: 2007-000724-40 Sponsor Protocol Number: PTC124-GD-006-CF Start Date*: 2007-09-27
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: A Phase 2 Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis
    Medical condition: Cystic Fibrosis
    Disease: Version SOC Term Classification Code Term Level
    9.1 10011762 Cystic fibrosis LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: BE (Completed)
    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: 2018-000966-12 Sponsor Protocol Number: EL-004 Start Date*: 2019-11-18
    Sponsor Name:Eloxx Pharmaceuticals Inc
    Full Title: A Phase 2 Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Dose Levels of Subcutaneously Administered ELX-02 in Patients with Cystic Fibrosis...
    Medical condition: Cystic fibrosis (CF)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10011762 Cystic fibrosis PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DE (Completed)
    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: 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: 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: 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: 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: 2020-002337-15 Sponsor Protocol Number: 99625945 Start Date*: 2020-09-16
    Sponsor Name:Oslo University Hospital
    Full Title: TOPICAL GENTAMICIN TREATMENT OF PATIENTS WITH EPIDERMOLYSIS BULLOSA DUE TO NONSENSE MUTATIONS (THE GENTELBULL STUDY)
    Medical condition: Epidermolysis bullosa caused by nonsense mutations or splice site mutations
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: NO (Completed)
    Trial results: (No results available)
    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: 2007-005478-29 Sponsor Protocol Number: PTC124-GD-007-DMD Start Date*: 2008-03-17
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: A Phase 2b Efficacy and Safety 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 Gender: Male
    Trial protocol: FR (Completed) DE (Completed) SE (Completed) GB (Completed) BE (Completed) ES (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: 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
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