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 | EUCTR2017-002704-27-NL (EUCTR) | 10/10/2019 | 25/02/2019 | 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] | 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;Netherlands;Germany;Sweden | |||
| 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 | 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 | ||
| 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-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 | ||
| 7 | 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 | ||
| 8 | 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 |
| 9 | EUCTR2017-003568-10-GB (EUCTR) | 05/01/2018 | 17/05/2018 | An Extension Study to Assess the Long-term Safety and Efficacy ofVamorolone in Boys With Duchenne Muscular Dystrophy(DMD) | A 24-month Phase II Open-label, Multicenter Long-term Extension Study toAssess the Long-term Safety and Efficacy of Vamorolone in Boys withDuchenne Muscular Dystrophy (DMD) - VBP15-LTE | 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 INN or Proposed INN: Vamorolone Other descriptive name: EV substance code: SUB188638 Product Name: Vamorolone INN or Proposed INN: Vamorolone Other descriptive name: EV substance code: SUB188638 Product Name: Vamorolone INN or Proposed INN: Vamorolone Other descriptive name: EV substance code: SUB188638 Product Name: Vamorolone INN or Proposed INN: Vamorolone Other descriptive name: EV substance code: SUB188638 | ReveraGen BioPharma Inc. | NULL | Not Recruiting | Female: no Male: yes | 48 | Phase 2 | United States;Canada;Australia;Israel;United Kingdom;Sweden | ||
| 10 | EUCTR2017-003568-10-SE (EUCTR) | 23/11/2017 | 05/10/2017 | An Extension Study to Assess the Long-term Safety and Efficacy ofVamorolone in Boys With Duchenne Muscular Dystrophy(DMD) | A 24-month Phase II Open-label, Multicenter Long-term Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (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 INN or Proposed INN: VAMOROLONE Product Name: Vamorolone INN or Proposed INN: VAMOROLONE Product Name: Vamorolone INN or Proposed INN: VAMOROLONE Product Name: Vamorolone INN or Proposed INN: VAMOROLONE | ReveraGen BioPharma Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 48 | Phase 2 | United States;Canada;Australia;Israel;United Kingdom;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 | NCT03863119 (ClinicalTrials.gov) | July 15, 2017 | 21/2/2019 | Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy | An Open-Label, Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy Who Have Completed the Long-Term Extension (VBP15-LTE) or VBP15-004 Studies | Duchenne Muscular Dystrophy | Drug: Vamorolone | ReveraGen BioPharma, Inc. | NULL | Available | N/A | N/A | Male | United States;Canada;Israel | ||
| 12 | EUCTR2016-004262-26-GB (EUCTR) | 16/02/2017 | 21/12/2020 | A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy(DMD) | VBP15-002 A Phase IIa Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability,Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys with Duchenne MuscularDystrophy (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: Vamarolone Product Code: VBP15 INN or Proposed INN: vamorolone Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: vamorolone Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: vamorolone Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: vamorolone | ReveraGen BioPharma, Inc. | NULL | Not Recruiting | Female: no Male: yes | 48 | Phase 2 | United States;Canada;Australia;Israel;United Kingdom;Sweden | ||
| 13 | EUCTR2016-004462-26-GB (EUCTR) | 16/02/2017 | 23/12/2016 | A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | VBP15-002 A Phase IIa Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) | Duchenne muscular dystrophy (DMD) MedDRA version: 19.0;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] | ReveraGen BioPharma, Inc. | NULL | Not Recruiting | Female: no Male: yes | 48 | Phase 2 | United States;Canada;Australia;Israel;United Kingdom | |||
| 14 | EUCTR2016-004263-38-SE (EUCTR) | 13/02/2017 | 16/12/2016 | An Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy(DMD) | A Phase II Open-label, Multicenter Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy (DMD) MedDRA version: 19.0;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: Vamarolone INN or Proposed INN: vamorolone Product Name: Vamorolone INN or Proposed INN: vamorolone Product Name: Vamorolone INN or Proposed INN: vamorolone Product Name: Vamorolone INN or Proposed INN: vamorolone | ReveraGen BioPharma Inc. | NULL | Not Recruiting | Female: no Male: yes | 48 | Phase 2 | United States;Canada;Australia;Israel;United Kingdom;Sweden | ||
| 15 | EUCTR2016-004262-26-SE (EUCTR) | 13/02/2017 | 16/12/2016 | A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | A Phase IIa, Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy (DMD) MedDRA version: 19.0;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: Vamarolone INN or Proposed INN: vamorolone Product Name: Vamorolone INN or Proposed INN: vamorolone Product Name: Vamorolone INN or Proposed INN: vamorolone Product Name: Vamorolone INN or Proposed INN: vamorolone | ReveraGen BioPharma Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 48 | Phase 2 | United States;Canada;Australia;Israel;United Kingdom;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 |
| 16 | EUCTR2016-004263-38-GB (EUCTR) | 10/02/2017 | 08/02/2019 | An Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | A Phase II Open-label, Multicenter Extension Study to Assess the Longterm Safety and Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (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] | ReveraGen BioPharma, Inc. | NULL | Not Recruiting | Female: no Male: yes | 48 | Phase 2 | United States;Canada;Australia;Israel;United Kingdom;Sweden | |||
| 17 | NCT03038399 (ClinicalTrials.gov) | February 2, 2017 | 30/1/2017 | Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | A 24-month Phase II Open-label, Multicenter Long-term Extension Study to Assess the Long-Term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | Drug: Vamorolone 0.25 mg/day/day;Drug: Vamorolone 0.75 mg/day/day;Drug: Vamorolone 2.0 mg/day/day;Drug: Vamorolone 6.0 mg/day/day | ReveraGen BioPharma, Inc. | University of Pittsburgh;Cooperative International Neuromuscular Research Group | Completed | 4 Years | 7 Years | Male | 46 | Phase 2 | United States;Australia;Canada;Israel;Sweden;United Kingdom |
| 18 | NCT02760277 (ClinicalTrials.gov) | July 28, 2016 | 28/4/2016 | An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | A Phase II Open-label, Multicenter Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | Drug: Vamorolone 0.25 mg/day/day;Drug: Vamorolone 0.75 mg/day/day;Drug: Vamorolone 2.0 mg/day/day;Drug: Vamorolone 6.0 mg/day/day | ReveraGen BioPharma, Inc. | University of Pittsburgh;National Institute of Neurological Disorders and Stroke (NINDS);Cooperative International Neuromuscular Research Group;National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) | Completed | 4 Years | 7 Years | Male | 48 | Phase 2 | United States;Australia;Canada;Israel;Sweden;United Kingdom |
| 19 | NCT02760264 (ClinicalTrials.gov) | June 2016 | 28/4/2016 | A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | A Phase IIa Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | Drug: Vamorolone 0.25 mg/kg/day;Drug: Vamorolone 0.75 mg/kg/day;Drug: Vamorolone 2.0 mg/kg/day;Drug: Vamorolone 6.0 mg/kg/day | ReveraGen BioPharma, Inc. | University of Pittsburgh;National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS);National Institute of Neurological Disorders and Stroke (NINDS);Cooperative International Neuromuscular Research Group | Completed | 4 Years | 6 Years | Male | 48 | Phase 2 | United States;Australia;Canada;Israel;Sweden;United Kingdom |