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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
19 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2017-002704-27-NL
(EUCTR)
10/10/201925/02/2019A 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.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
120Phase 2United States;Greece;Spain;Israel;United Kingdom;Italy;Czech Republic;Canada;Belgium;Australia;Netherlands;Germany;Sweden
2EUCTR2017-002704-27-ES
(EUCTR)
13/08/201911/06/2019A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
120Phase 2United States;Greece;Spain;Israel;United Kingdom;Italy;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
3EUCTR2017-002704-27-GR
(EUCTR)
01/08/201910/06/2019A 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, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
120Phase 2Czech Republic;Canada;Greece;Belgium;Spain;Australia;Israel;Netherlands;Germany;Italy;United Kingdom;Sweden
4EUCTR2017-002704-27-BE
(EUCTR)
06/07/201921/02/2018A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
120Phase 2United States;Greece;Spain;Israel;United Kingdom;Italy;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
5EUCTR2017-002704-27-CZ
(EUCTR)
19/06/201922/03/2019A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
120Phase 2United States;Greece;Spain;Israel;United Kingdom;Italy;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2017-002704-27-GB
(EUCTR)
21/09/201825/06/2019A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
120Phase 2United States;Greece;Spain;Israel;United Kingdom;Italy;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
7EUCTR2017-002704-27-SE
(EUCTR)
15/08/201812/03/2018A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
120Phase 2United States;Greece;Spain;Israel;Italy;United Kingdom;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
8NCT03439670
(ClinicalTrials.gov)
June 19, 20189/1/2018A 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 DystrophyDrug: Vamorolone;Drug: Prednisone;Other: PlaceboReveraGen BioPharma, Inc.European Union;Cooperative International Neuromuscular Research Group;Newcastle University;University of PittsburghActive, not recruiting4 Years7 YearsMale121Phase 2United States;Australia;Belgium;Canada;Czechia;Greece;Israel;Netherlands;Spain;Sweden;United Kingdom;Germany;Italy
9EUCTR2017-003568-10-GB
(EUCTR)
05/01/201817/05/2018An 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.NULLNot RecruitingFemale: no
Male: yes
48Phase 2United States;Canada;Australia;Israel;United Kingdom;Sweden
10EUCTR2017-003568-10-SE
(EUCTR)
23/11/201705/10/2017An 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
48Phase 2United States;Canada;Australia;Israel;United Kingdom;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT03863119
(ClinicalTrials.gov)
July 15, 201721/2/2019Expanded Access Protocol for Boys With Duchenne Muscular DystrophyAn Open-Label, Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy Who Have Completed the Long-Term Extension (VBP15-LTE) or VBP15-004 StudiesDuchenne Muscular DystrophyDrug: VamoroloneReveraGen BioPharma, Inc.NULLAvailableN/AN/AMaleUnited States;Canada;Israel
12EUCTR2016-004262-26-GB
(EUCTR)
16/02/201721/12/2020A 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.NULLNot RecruitingFemale: no
Male: yes
48Phase 2United States;Canada;Australia;Israel;United Kingdom;Sweden
13EUCTR2016-004462-26-GB
(EUCTR)
16/02/201723/12/2016 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.NULLNot Recruiting Female: no
Male: yes
48Phase 2United States;Canada;Australia;Israel;United Kingdom
14EUCTR2016-004262-26-SE
(EUCTR)
13/02/201716/12/2016A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
48Phase 2United States;Canada;Australia;Israel;United Kingdom;Sweden
15EUCTR2016-004263-38-SE
(EUCTR)
13/02/201716/12/2016An 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.NULLNot RecruitingFemale: no
Male: yes
48Phase 2United States;Canada;Australia;Israel;United Kingdom;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16EUCTR2016-004263-38-GB
(EUCTR)
10/02/201708/02/2019 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.NULLNot Recruiting Female: no
Male: yes
48Phase 2United States;Canada;Australia;Israel;United Kingdom;Sweden
17NCT03038399
(ClinicalTrials.gov)
February 2, 201730/1/2017Long-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 DystrophyDrug: 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/dayReveraGen BioPharma, Inc.University of Pittsburgh;Cooperative International Neuromuscular Research GroupCompleted4 Years7 YearsMale46Phase 2United States;Australia;Canada;Israel;Sweden;United Kingdom
18NCT02760277
(ClinicalTrials.gov)
July 28, 201628/4/2016An 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 DystrophyDrug: 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/dayReveraGen 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)Completed4 Years7 YearsMale48Phase 2United States;Australia;Canada;Israel;Sweden;United Kingdom
19NCT02760264
(ClinicalTrials.gov)
June 201628/4/2016A 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 DystrophyDrug: 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/dayReveraGen 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 GroupCompleted4 Years6 YearsMale48Phase 2United States;Australia;Canada;Israel;Sweden;United Kingdom