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 | EUCTR2018-004740-36-DK (EUCTR) | 29/10/2019 | 25/06/2019 | A clinical study to evaluate how safe and effective the drug Deflazacort (EMFLAZA®) compared to placebo is in subjects with Limb-Girdle Muscular Dystrophy Type 2I | A MULTICENTER RANDOMIZED PLACEBO-CONTROLLED PHASE 3 STUDY ON THE SAFETY AND EFFICACY OF DEFLAZACORT (EMFLAZA®) IN SUBJECTS WITH LIMB-GIRDLE MUSCULAR DYSTROPHY 2I (LGMD2I | Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I) MedDRA version: 20.0;Level: PT;Classification code 10028356;Term: Muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) INN or Proposed INN: Deflazacort Other descriptive name: DEFLAZACORT Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) INN or Proposed INN: Deflazacort Other descriptive name: DEFLAZACORT Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) INN or Proposed INN: Deflazacort Other descriptive name: DEFLAZACORT | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | France;United States;Canada;Denmark;Russian Federation;Norway;Germany;Sweden | ||
| 2 | EUCTR2018-004740-36-DE (EUCTR) | 14/10/2019 | 29/04/2019 | A clinical study to evaluate how safe and effective the drug Deflazacort (EMFLAZA®) compared to placebo is in subjects with Limb-Girdle Muscular Dystrophy Type 2I | A MULTICENTER RANDOMIZED PLACEBO-CONTROLLED PHASE 3 STUDY ON THE SAFETY AND EFFICACY OF DEFLAZACORT (EMFLAZA®) IN SUBJECTS WITH LIMB-GIRDLE MUSCULAR DYSTROPHY 2I (LGMD2I | Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I) MedDRA version: 20.0;Level: PT;Classification code 10028356;Term: Muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) INN or Proposed INN: Deflazacort Other descriptive name: DEFLAZACORT Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) INN or Proposed INN: Deflazacort Other descriptive name: DEFLAZACORT Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) INN or Proposed INN: Deflazacort Other descriptive name: DEFLAZACORT | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | France;United States;Canada;Denmark;Russian Federation;Norway;Germany;Sweden | ||
| 3 | NCT03783923 (ClinicalTrials.gov) | May 15, 2019 | 19/12/2018 | A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) | A Multicenter Open Label Study on the Safety and Efficacy of Deflazacort (Emflaza®) in Subjects With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) | Limb-Girdle Muscular Dystrophy | Drug: Deflazacort | PTC Therapeutics | NULL | Active, not recruiting | 18 Years | N/A | All | 30 | Phase 3 | United States;Canada;Denmark;France;Germany;Norway;Russian Federation;Sweden |
| 4 | NCT02592941 (ClinicalTrials.gov) | March 1, 2019 | 29/10/2015 | Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy | An Open Label, Expanded Access Protocol Intended to Provide Treatment With MP-104 (Deflazacort) to U.S. Children, Adolescents, and/or Adults With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Deflazacort | PTC Therapeutics | Parexel;Dohmen Life Science Services | Approved for marketing | 5 Years | N/A | All | United States | ||
| 5 | NCT03642145 (ClinicalTrials.gov) | October 31, 2018 | 10/7/2018 | A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD) | A 52-Week Phase 3B Randomized Open-Label Study Evaluating the Safety and Pharmacokinetics of Emflaza® (Deflazacort) Compared to a Comparable Natural History Control Group in Males Aged =2 to <5 Years With Duchenne Muscular Dystrophy (DMD) Followed by a 52-Week Extension Period | Duchenne Muscular Dystrophy | Drug: Deflazacort | PTC Therapeutics | NULL | Withdrawn | 2 Years | 4 Years | Male | 0 | Phase 3 | United States |
| 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 | NCT02251600 (ClinicalTrials.gov) | December 2014 | 22/9/2014 | A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy | A Multi-center Study to Evaluate the Pharmacokinetics of 21-Desacetyldeflazacort and the Safety of Deflazacort After Oral Administration of Deflazacort Tablets to Children and Adolescent Subjects With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Deflazacort | PTC Therapeutics | NULL | Completed | 4 Years | 16 Years | Male | 24 | Phase 1 | United States |
| 7 | NCT02295748 (ClinicalTrials.gov) | December 2014 | 18/11/2014 | An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort | An Open-Label, Multi-Center, Long-Term Extension Study to Evaluate the Safety and Tolerability of Orally Administrated Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Deflazacort | PTC Therapeutics | NULL | Completed | 4 Years | N/A | Male | 24 | Phase 1 | United States |
| 8 | EUCTR2010-023744-33-IT (EUCTR) | 26/02/2013 | 28/12/2012 | Duchenne muscular dystrophy: a clinical trial to find the optimum steroid regimen. | Duchenne muscular dystrophy: double-blind randomized trial to find optimum steroid regimen. - FOR-DMD | Duchenne muscular dystrophy boys between 4 and 8 years of age, able to rise from the floor without support, not previously treated with steroids. MedDRA version: 14.1;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: DELTACORTENE*20CPR 5MG INN or Proposed INN: PREDNISONE Trade Name: DELTACORTENE*20CPR 5MG INN or Proposed INN: PREDNISONE Trade Name: DEFLAN*10CPR 6MG INN or Proposed INN: DEFLAZACORT | AZIENDA OSPEDALIERA DI PADOVA | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 300 | United States;Canada;United Kingdom;Italy | |||
| 9 | NCT01603407 (ClinicalTrials.gov) | January 2013 | 3/4/2012 | Finding the Optimum Regimen for Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid Regimen | Duchenne Muscular Dystrophy | Drug: Prednisone;Drug: Deflazacort | University of Rochester | Newcastle University;University Medical Center Freiburg;National Institute of Neurological Disorders and Stroke (NINDS) | Completed | 4 Years | 7 Years | Male | 196 | Phase 3 | United States;Canada;Germany;Italy;United Kingdom |
| 10 | EUCTR2010-023744-33-GB (EUCTR) | 12/11/2012 | 20/09/2012 | Trial to find best steroid treatment for Duchenne muscular dystrophy | Duchenne muscular dystrophy: double-blind randomized trial to find optimum steroid regimen - FOR-DMD | Duchenne muscular dystrophy (DMD) MedDRA version: 14.1;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: Deflazacort Product Name: Deflazacort 6 mg Tablets INN or Proposed INN: Deflazacort Trade Name: Prednisone Product Name: Prednisone 5 mg tablets INN or Proposed INN: Prednisone | University of Rochester | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 300 | United States;Canada;Germany;Italy;United Kingdom |