113. 筋ジストロフィー Muscular dystrophy Clinical trials / Disease details
臨床試験数 : 646 / 薬物数 : 471 - (DrugBank : 105) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 170
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04336826 (ClinicalTrials.gov) | December 29, 2021 | 3/4/2020 | A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From =6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) | 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 | Nonsene Mutation Duchenne Muscular Dystrophy | Drug: Ataluren | PTC Therapeutics | NULL | Recruiting | 6 Months | 2 Years | Male | 10 | Phase 2 | United States |
| 2 | JPRN-jRCT2041200087 | 07/01/2021 | 25/12/2020 | An Open-label, Long-Term study of Ataluren in Nonsense Mutation Duchenne Muscular Dystrophy | An Open-Label, Long-Term Safety, Efficacy, and Tolerability Study for Ataluren (PTC124) in Patients with Nonsense Mutation Duchenne Muscular Dystrophy - PTC124-GD-049-DMD | Nonsense Mutation Duchenne Muscular Dystrophy | Subjects will receive ataluren tid as an oral suspension at respective morning, mid-day, and evening doses of 10 mg/kg, 10 mg/kg, and 20 mg/kg. | Penematsa Vinay | NULL | Recruiting | >= 5age old | Not applicable | Male | 25 | Phase 3 | China;Japan |
| 3 | NCT03796637 (ClinicalTrials.gov) | April 11, 2019 | 4/1/2019 | A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren | 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 | Duchenne Muscular Dystrophy | Other: Dystrophin levels;Drug: Ataluren | PTC Therapeutics | NULL | Completed | N/A | N/A | Male | 6 | Phase 2 | United States |
| 4 | NCT03648827 (ClinicalTrials.gov) | December 21, 2018 | 24/8/2018 | A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) | Phase 2 Clinical Pharmacology Study to Assess Dystrophin Levels in Subjects With nmDMD Before and After Treatment With Ataluren | Duchenne Muscular Dystrophy | Drug: Ataluren | PTC Therapeutics | NULL | Completed | 2 Years | 7 Years | Male | 20 | Phase 2 | United States |
| 5 | EUCTR2017-001223-49-BG (EUCTR) | 16/01/2018 | 11/10/2017 | A clinical trial to evaluate the safety and efficacy of Ataluren versus placebo in people who have specific genetic disease Duchenne Muscular Dystrophy | 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 | 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 340 | Phase 3 | United States;Taiwan;Hong Kong;Thailand;Turkey;Russian Federation;India;Mexico;Canada;Jordan;Argentina;Brazil;Malaysia;Poland;Australia;Bulgaria;China;Japan;Korea, Republic of | ||
| 6 | NCT03179631 (ClinicalTrials.gov) | July 6, 2017 | 1/6/2017 | Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy | 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 | Muscular Dystrophy, Duchenne;Muscular Dystrophies;Muscular Disorders, Atrophic;Muscular Diseases;Musculoskeletal Disease;Neuromuscular Diseases;Nervous System Diseases;Genetic Diseases, X-Linked;Genetic Diseases, Inborn | Drug: Ataluren;Drug: PLACEBO | PTC Therapeutics | NULL | Active, not recruiting | 5 Years | N/A | Male | 360 | Phase 3 | United States;Australia;Brazil;Bulgaria;Canada;China;Hong Kong;India;Japan;Korea, Republic of;Malaysia;Mexico;Poland;Puerto Rico;Russian Federation;Taiwan;Thailand;Turkey;Argentina;Chile;Jordan;Sri Lanka |
| 7 | EUCTR2013-005489-20-FR (EUCTR) | 18/10/2016 | 18/06/2015 | Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 18.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden | ||
| 8 | NCT02819557 (ClinicalTrials.gov) | June 9, 2016 | 16/6/2016 | Study of Ataluren in =2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy | 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 | Duchenne Muscular Dystrophy | Drug: Ataluren | PTC Therapeutics | NULL | Completed | 2 Years | 5 Years | Male | 14 | Phase 2 | United States |
| 9 | EUCTR2013-005489-20-BG (EUCTR) | 25/08/2015 | 26/06/2015 | Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 18.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden | ||
| 10 | EUCTR2013-005489-20-CZ (EUCTR) | 08/01/2015 | 24/10/2014 | Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 17.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden | ||
| 11 | EUCTR2013-005489-20-DE (EUCTR) | 29/09/2014 | 28/07/2014 | Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden | ||
| 12 | EUCTR2013-005489-20-IT (EUCTR) | 10/09/2014 | 30/07/2014 | Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden | ||
| 13 | EUCTR2013-005489-20-ES (EUCTR) | 01/09/2014 | 06/08/2014 | Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden | ||
| 14 | EUCTR2013-005489-20-BE (EUCTR) | 28/08/2014 | 11/07/2014 | Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;Bulgaria;Germany;Korea, Republic of;Sweden | ||
| 15 | EUCTR2013-005489-20-GB (EUCTR) | 22/08/2014 | 11/07/2014 | Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden | ||
| 16 | EUCTR2013-005489-20-SE (EUCTR) | 22/08/2014 | 11/07/2014 | Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Sweden;Korea, Republic of | ||
| 17 | NCT02090959 (ClinicalTrials.gov) | March 20, 2014 | 17/3/2014 | An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy | A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy | Muscular Dystrophy, Duchenne;Muscular Dystrophies;Muscular Disorders, Atrophic;Muscular Diseases;Musculoskeletal Diseases;Neuromuscular Diseases;Nervous System Diseases;Genetic Diseases, X-Linked;Genetic Diseases, Inborn | Drug: Ataluren | PTC Therapeutics | NULL | Terminated | 7 Years | 15 Years | Male | 219 | Phase 3 | United States;Australia;Belgium;Brazil;Bulgaria;Canada;Chile;Czechia;France;Germany;Israel;Italy;Korea, Republic of;Poland;Spain;Sweden;Switzerland;Turkey;United Kingdom;Czech Republic |
| 18 | EUCTR2012-004527-20-PL (EUCTR) | 12/02/2014 | 27/11/2013 | Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A | Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden | ||
| 19 | EUCTR2012-004527-20-SE (EUCTR) | 30/08/2013 | 24/06/2013 | Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A | Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Sweden;Korea, Republic of | ||
| 20 | EUCTR2012-004527-20-CZ (EUCTR) | 30/08/2013 | 24/06/2013 | Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A | Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden | ||
| 21 | EUCTR2012-004527-20-DE (EUCTR) | 20/08/2013 | 24/06/2013 | Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 17.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden | ||
| 22 | EUCTR2012-004527-20-GB (EUCTR) | 08/08/2013 | 24/06/2013 | Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A | Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 220 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;France;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden | ||
| 23 | EUCTR2012-004527-20-BE (EUCTR) | 30/07/2013 | 24/06/2013 | Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A | Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Poland;Belgium;Brazil;Australia;Germany;Korea, Republic of;Sweden | ||
| 24 | EUCTR2012-004527-20-ES (EUCTR) | 17/07/2013 | 12/07/2013 | Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A | Nonsense mutation dystrophinopathy MedDRA version: 16.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 16.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden | ||
| 25 | EUCTR2012-004527-20-IT (EUCTR) | 01/07/2013 | 27/06/2013 | Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A | Nonsense mutation dystrophinopathy MedDRA version: 16.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 16.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;United Kingdom;Italy;Switzerland;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden | ||
| 26 | NCT01826487 (ClinicalTrials.gov) | March 26, 2013 | 26/3/2013 | Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) | A Phase 3 Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Dystrophinopathy | Muscular Dystrophy, Duchenne;Muscular Dystrophies;Muscular Disorders, Atrophic;Muscular Diseases;Musculoskeletal Diseases;Neuromuscular Diseases;Nervous System Diseases;Genetic Diseases, X-Linked;Genetic Diseases, Inborn | Drug: Ataluren;Drug: Placebo | PTC Therapeutics | NULL | Completed | 7 Years | 16 Years | Male | 230 | Phase 3 | United States;Australia;Belgium;Brazil;Canada;Chile;Czechia;France;Germany;Israel;Italy;Korea, Republic of;Poland;Spain;Sweden;Switzerland;Turkey;United Kingdom;Bulgaria;Czech Republic |
| 27 | EUCTR2011-004853-18-IT (EUCTR) | 18/09/2012 | 27/09/2012 | An Open-Label Study for Previously Treated Ataluren (PTC124) Patients with Nonsense Mutation Dystrophinopathy | An Open-Label Study for Previously Treated Ataluren (PTC124) Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 15.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 15.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: na Product Name: ataluren Product Code: PTC124 INN or Proposed INN: na | PTC THERAPEUTICS, INC. | NULL | Not Recruiting | Female: no Male: yes | 96 | Phase 3 | Canada;Spain;Belgium;Australia;Israel;Germany;United Kingdom;Italy;Sweden | ||
| 28 | EUCTR2011-004853-18-GB (EUCTR) | 21/08/2012 | 29/03/2012 | Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy | An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 96 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden | ||
| 29 | EUCTR2011-004853-18-ES (EUCTR) | 13/06/2012 | 17/04/2012 | Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy | An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy - N/A | Nonsense mutation dystrophinopathy MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 96 | Phase 3 | Canada;Belgium;Spain;Australia;Israel;Germany;Italy;United Kingdom;Sweden | ||
| 30 | NCT01557400 (ClinicalTrials.gov) | May 20, 2012 | 15/3/2012 | Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada | An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients With Nonsense Mutation Dystrophinopathy | Duchenne Muscular Dystrophy;Becker Muscular Dystrophy;Dystrophinopathy | Drug: Ataluren | PTC Therapeutics | NULL | Completed | N/A | N/A | Male | 94 | Phase 3 | Australia;Belgium;Canada;France;Germany;Israel;Italy;Spain;Sweden;United Kingdom |
| 31 | EUCTR2011-004853-18-FR (EUCTR) | 02/05/2012 | 13/08/2012 | Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy | An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 14.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 96 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden | ||
| 32 | EUCTR2011-004853-18-DE (EUCTR) | 02/05/2012 | 12/03/2012 | Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy | An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 96 | Phase 3 | Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden | ||
| 33 | EUCTR2011-004853-18-BE (EUCTR) | 27/04/2012 | 03/01/2012 | Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy | An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 16.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 16.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 96 | Phase 3 | Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden | ||
| 34 | EUCTR2011-004853-18-SE (EUCTR) | 26/04/2012 | 27/02/2012 | Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy | An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy - N/A | Nonsense mutation dystrophinopathy MedDRA version: 17.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 96 | Phase 3 | Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden | ||
| 35 | NCT01247207 (ClinicalTrials.gov) | November 30, 2010 | 22/11/2010 | Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD) | An Open-Label, Safety Study for Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy | Duchenne Muscular Dystrophy | Drug: Ataluren | PTC Therapeutics | NULL | Enrolling by invitation | N/A | N/A | Male | 270 | Phase 3 | United States;Canada |
| 36 | EUCTR2009-013169-24-GB (EUCTR) | 19/01/2010 | 17/03/2010 | A Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients with Nonsense–Mutation-Mediated Duchenne/Becker Muscular Dystrophy - Study of Ataluren in Nonambulatory Patients with DMD/BMD | A Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients with Nonsense–Mutation-Mediated Duchenne/Becker Muscular Dystrophy - Study of Ataluren in Nonambulatory Patients with DMD/BMD | Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy MedDRA version: 12.0;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 12.0;Classification code 10059117;Term: Becker's muscular dystrophy | Product Name: Ataluren 125 mg Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: Ataluren 250 mg Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: Ataluren 1000 mg Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics Inc | NULL | Not Recruiting | Female: no Male: yes | 30 | Phase 2 | United Kingdom | ||
| 37 | NCT01009294 (ClinicalTrials.gov) | January 13, 2010 | 5/11/2009 | Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD) | A Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy | Duchenne Muscular Dystrophy;Becker Muscular Dystrophy | Drug: Ataluren;Drug: Chronic Corticosteroid Therapy | PTC Therapeutics | Genzyme, a Sanofi Company | Terminated | 7 Years | N/A | Male | 6 | Phase 2 | United States;United Kingdom |
| 38 | EUCTR2008-007648-32-ES (EUCTR) | 09/07/2009 | 04/05/2009 | Un estudio de extensión en Fase 2b de PTC124 en sujetos con distrofia muscular de Duchenne y Becker mediada por mutación terminadora. | Un estudio de extensión en Fase 2b de PTC124 en sujetos con distrofia muscular de Duchenne y Becker mediada por mutación terminadora. | Distrofia muscular de Duchenne, Distrofia muscular de Becker MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy | Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 174 | Phase 2 | France;Belgium;Spain;Germany;Italy;United Kingdom;Sweden | ||
| 39 | EUCTR2008-007648-32-GB (EUCTR) | 22/06/2009 | 29/04/2009 | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy | Product Name: Ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 174 | Phase 2 | Germany;United Kingdom;Belgium;France;Spain;Italy;Sweden | ||
| 40 | EUCTR2008-007648-32-DE (EUCTR) | 22/06/2009 | 08/04/2009 | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy | Product Name: Ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 174 | Phase 2 | France;Spain;Belgium;Germany;Italy;United Kingdom;Sweden | ||
| 41 | EUCTR2008-007648-32-SE (EUCTR) | 02/06/2009 | 17/04/2009 | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy | Product Name: Ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 174 | Phase 2 | United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden | ||
| 42 | EUCTR2008-007648-32-FR (EUCTR) | 28/05/2009 | 03/04/2009 | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy | Product Name: Ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 174 | Phase 2 | France;Spain;Belgium;Germany;Italy;United Kingdom;Sweden | ||
| 43 | EUCTR2008-007648-32-IT (EUCTR) | 27/05/2009 | 22/05/2009 | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy - ND | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy - ND | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801 MedDRA version: 9.1;Classification code 10059117 | Product Name: Ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Product Name: Ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Product Name: Ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN | PTC THERAPEUTICS, INC. | NULL | Not Recruiting | Female: no Male: yes | 174 | Phase 2 | United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden | ||
| 44 | EUCTR2007-005478-29-GB (EUCTR) | 27/04/2009 | 22/02/2008 | A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy | Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 174 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;Spain;Belgium;Germany;Italy;United Kingdom;Sweden | ||
| 45 | EUCTR2008-007648-32-BE (EUCTR) | 14/04/2009 | 25/02/2009 | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy | Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 174 | Phase 2 | United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden | ||
| 46 | NCT00847379 (ClinicalTrials.gov) | January 31, 2009 | 16/2/2009 | Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) | A Phase 2B Extension Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy;Becker Muscular Dystrophy | Drug: Ataluren | PTC Therapeutics | Genzyme, a Sanofi Company | Terminated | 5 Years | N/A | Male | 173 | Phase 2 | United States;Australia;Belgium;Canada;France;Germany;Israel;Italy;Spain;Sweden;United Kingdom |
| 47 | NCT00759876 (ClinicalTrials.gov) | August 13, 2008 | 23/9/2008 | Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD) | A Phase 2a Extension Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Ataluren | PTC Therapeutics | Genzyme, a Sanofi Company | Terminated | N/A | N/A | Male | 36 | Phase 2 | United States |
| 48 | EUCTR2007-005478-29-SE (EUCTR) | 16/04/2008 | 22/02/2008 | A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy | Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 174 | Phase 2 | United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden | ||
| 49 | EUCTR2007-005478-29-DE (EUCTR) | 14/04/2008 | 28/01/2008 | A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy | Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 174 | Phase 2 | United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden | ||
| 50 | EUCTR2007-005478-29-BE (EUCTR) | 11/03/2008 | 27/02/2008 | A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy | Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 174 | Phase 2 | United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden | ||
| 51 | NCT00592553 (ClinicalTrials.gov) | February 29, 2008 | 1/1/2008 | Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) | A Phase 2B Efficacy and Safety Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy;Becker Muscular Dystrophy | Drug: Ataluren;Drug: Placebo | PTC Therapeutics | NULL | Completed | 5 Years | N/A | Male | 174 | Phase 2 | United States;Australia;Belgium;Canada;France;Germany;Israel;Italy;Spain;Sweden;United Kingdom |
| 52 | EUCTR2020-000980-21-Outside-EU/EEA (EUCTR) | 24/06/2021 | A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Subjects From =6 Months to <2 Years of Age with Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) | 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 | Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD);Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: Translarna Product Name: Ataluren Product Code: PTC124 INN or Proposed INN: Ataluren | PTC Therapeutics, Inc. | NULL | NA | Female: no Male: yes | Phase 2 | United States | ||||
| 53 | EUCTR2012-004527-20-FR (EUCTR) | 25/09/2015 | Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A | Nonsense mutation dystrophinopathy MedDRA version: 18.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 220 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;France;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden | |||
| 54 | EUCTR2017-001223-49-PL (EUCTR) | 05/06/2018 | A clinical trial to evaluate the safety and efficacy of Ataluren versus placebo in people who have specific genetic disease Duchenne Muscular Dystrophy | 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 | 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. | NULL | NA | Female: no Male: yes | 340 | Phase 3 | United States;Taiwan;Hong Kong;Thailand;Turkey;Russian Federation;India;Mexico;Canada;Jordan;Argentina;Poland;Brazil;Malaysia;Australia;Bulgaria;China;Japan;Korea, Republic of |