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.
| 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 | 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 2a | United Kingdom | ||
| 2 | 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 | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 174 | United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden | |||
| 3 | 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 2b | Germany;United Kingdom;Belgium;France;Spain;Italy;Sweden | ||
| 4 | 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 2b | France;Spain;Belgium;Germany;Italy;United Kingdom;Sweden | ||
| 5 | 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 2b | United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden | ||
| 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 |
| 6 | 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 | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 174 | Phase 2b | United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden | ||
| 7 | 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 2b | United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden | ||
| 8 | EUCTR2007-005478-29-ES (EUCTR) | 20/05/2008 | 10/03/2008 | Estudio de fase 2b sobre la eficacia y la seguridad de PTC124 en pacientes con distrofia muscular de Duchenne y Becker mediada por mutación terminadora | Estudio de fase 2b sobre la eficacia y la seguridad de PTC124 en pacientes 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: PTC124 Product Code: PTC124 INN or Proposed INN: PTC124 Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 165 | United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden | |||
| 9 | 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 2b | United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden | ||
| 10 | 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 2b | United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden | ||
| 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 |
| 11 | EUCTR2007-005478-29-FR (EUCTR) | 17/03/2008 | 24/01/2008 | 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 Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 165 | Phase 2b | United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden | ||
| 12 | 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 2b | United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden |