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 | NCT04322357 (ClinicalTrials.gov) | October 2020 | 24/3/2020 | Weekend Steroids and Exercise as Therapy for DMD | Impact and Interplay of Corticosteroid Regimen and Exercise Training on DMD Muscle Function | Duchenne Muscular Dystrophy (DMD) | Drug: Prednisone;Behavioral: In-home Exercise Training | University of Florida | U.S. Army Medical Research and Development Command | Not yet recruiting | 5 Years | 8 Years | Male | 65 | Phase 2 | NULL |
| 2 | EUCTR2017-002704-27-ES (EUCTR) | 13/08/2019 | 11/06/2019 | A Study to Assess the efficacy and the safety of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) | A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - VISION DMD | Duchenne muscular dystrophy (DMD) 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] | Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE Product Name: Prednisone INN or Proposed INN: PREDNISONE Other descriptive name: PREDNISONE | ReveraGen BioPharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 120 | Phase 2 | United States;Greece;Spain;Israel;United Kingdom;Italy;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden | ||
| 3 | EUCTR2017-002704-27-GR (EUCTR) | 01/08/2019 | 10/06/2019 | A Study to Assess the efficacy and the safety of Vamorolone in Boys withDuchenne Muscular Dystrophy (DMD) | A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- andActive-controlled Study with Double-Blind Extension to Assess the Efficacyand Safety of Vamorolone in Ambulant Boys with Duchenne MuscularDystrophy (DMD) - VISION DMD | Duchenne muscular dystrophy (DMD) 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] | Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE Product Name: Prednisone INN or Proposed INN: PREDNISONE Other descriptive name: PREDNISONE | ReveraGen BioPharma, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 120 | Phase 2 | Czech Republic;Canada;Greece;Belgium;Spain;Australia;Israel;Netherlands;Germany;Italy;United Kingdom;Sweden | ||
| 4 | EUCTR2017-002704-27-BE (EUCTR) | 06/07/2019 | 21/02/2018 | A Study to Assess the efficacy and the safety of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) | A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - VISION DMD IIb | Duchenne muscular dystrophy (DMD) 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] | Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE Product Name: Prednisone INN or Proposed INN: PREDNISONE Other descriptive name: PREDNISONE | ReveraGen BioPharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 120 | Phase 2 | United States;Greece;Spain;Israel;United Kingdom;Italy;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden | ||
| 5 | NCT04054375 (ClinicalTrials.gov) | July 1, 2019 | 7/8/2019 | Weekly Steroids in Muscular Dystrophy | Open Label Safety and Efficacy of Once Weekly Steroid in Patients With LGMD and Becker Muscular Dystrophy | Limb-girdle Muscular Dystrophy;Becker Muscular Dystrophy | Drug: Prednisone | Northwestern University | NULL | Active, not recruiting | 18 Years | 65 Years | All | 20 | Phase 2 | 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 | EUCTR2017-002704-27-CZ (EUCTR) | 19/06/2019 | 22/03/2019 | A Study to Assess the efficacy and the safety of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) | A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - VISION DMD | Duchenne muscular dystrophy (DMD) 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] | Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE Product Name: Prednisone INN or Proposed INN: PREDNISONE Other descriptive name: PREDNISONE Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE | ReveraGen BioPharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 120 | Phase 2 | United States;Greece;Spain;Israel;United Kingdom;Italy;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden | ||
| 7 | EUCTR2017-002704-27-GB (EUCTR) | 21/09/2018 | 25/06/2019 | A Study to Assess the efficacy and the safety of Vamorolone in comparison to Prednisone in Boys with Duchenne Muscular Dystrophy (DMD) | A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - VISION DMD | Duchenne muscular dystrophy (DMD) 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] | Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE Product Name: Prednisone INN or Proposed INN: PREDNISONE Other descriptive name: PREDNISONE | ReveraGen BioPharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 120 | Phase 2 | United States;Greece;Spain;Israel;United Kingdom;Italy;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden | ||
| 8 | EUCTR2017-002704-27-SE (EUCTR) | 15/08/2018 | 12/03/2018 | A Study to Assess the efficacy and the safety of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) | A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - VISION DMD | Duchenne muscular dystrophy (DMD) 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] | Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE Product Name: Prednisone INN or Proposed INN: PREDNISONE Other descriptive name: PREDNISONE | ReveraGen BioPharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 120 | Phase 2 | United States;Greece;Spain;Israel;Italy;United Kingdom;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden | ||
| 9 | NCT03439670 (ClinicalTrials.gov) | June 19, 2018 | 9/1/2018 | A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study With Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | Drug: Vamorolone;Drug: Prednisone;Other: Placebo | ReveraGen BioPharma, Inc. | European Union;Cooperative International Neuromuscular Research Group;Newcastle University;University of Pittsburgh | Active, not recruiting | 4 Years | 7 Years | Male | 121 | Phase 2 | United States;Australia;Belgium;Canada;Czechia;Greece;Israel;Netherlands;Spain;Sweden;United Kingdom;Germany;Italy |
| 10 | NCT02036463 (ClinicalTrials.gov) | November 2014 | 6/1/2014 | A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular Dystrophy | CINRG0513: A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy (DMD) | Drug: Prednisone;Drug: Placebo | Ann & Robert H Lurie Children's Hospital of Chicago | Children's Research Institute | Withdrawn | 3 Years | 6 Years | Male | 0 | Phase 2 | 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 |
| 11 | 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 | |||
| 12 | 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 |
| 13 | 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 | |||
| 14 | NCT00308113 (ClinicalTrials.gov) | April 2007 | 27/3/2006 | CoQ10 and Prednisone in Non-Ambulatory DMD | PITT0503: Clinical Trial of Coenzyme Q10 and Prednisone in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Prednisone;Dietary Supplement: Coenzyme Q10 | Cooperative International Neuromuscular Research Group | United States Department of Defense | Terminated | 10 Years | 18 Years | Male | 3 | Phase 3 | United States;Australia;Puerto Rico |
| 15 | EUCTR2005-000663-26-AT (EUCTR) | 25/04/2005 | 21/03/2005 | Immunsuppressive Therapy in Duchenne Muscular Dystrophy - DMD-CsA | Immunsuppressive Therapy in Duchenne Muscular Dystrophy - DMD-CsA | Duchenne Muscular Dystrophy | Trade Name: Sandimmun Optoral Product Name: Sandimmun Optoral Product Code: 29180.00.00, 34681.00.00, 34681.01.00, 34681.02.00 Other descriptive name: Cyclosporin A Trade Name: Decortin Product Name: Decortin Product Code: not available Other descriptive name: Prednison | Universitätsklinik Freiburg | NULL | Not Recruiting | Female: no Male: yes | 150 | Phase 2;Phase 3 | Austria | ||
| 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 |
| 16 | NCT00110669 (ClinicalTrials.gov) | January 2004 | 12/5/2005 | High-dose Prednisone in Duchenne Muscular Dystrophy | A Randomized Study of Daily vs. High-dose Weekly Prednisone Therapy in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Prednisone | Cooperative International Neuromuscular Research Group | NULL | Completed | 4 Years | 10 Years | Male | 64 | Phase 3 | United States;India |
| 17 | NCT00004646 (ClinicalTrials.gov) | April 1995 | 24/2/2000 | Phase III Randomized, Double-Blind Study of Prednisone for Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: prednisone | National Center for Research Resources (NCRR) | National Institute of Neurological Disorders and Stroke (NINDS);University of Rochester | Completed | 5 Years | 15 Years | Male | 20 | Phase 3 | NULL |