Duchenne Muscular Dystrophy



A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)


Condition:   Duchenne Muscular Dystrophy
Interventions:   Drug: Vamorolone;   Drug: Prednisone;   Other: Placebo
Sponsors:   ReveraGen BioPharma, Inc.;   European Union;   Cooperative International Neuromuscular Research Group;   Newcastle University;   University of Pittsburgh
Not yet recruiting


Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)


Condition:   Duchenne Muscular Dystrophy
Intervention:   Drug: Idebenone
Sponsor:   Santhera Pharmaceuticals
Available


Systemic Gene Delivery Clinical Trial for Duchenne Muscular Dystrophy


Condition:   Duchenne Muscular Dystrophy
Intervention:   Drug: rAAVrh74.MHCK7.micro-dystrophin
Sponsors:   Nationwide Children's Hospital;   Washington University School of Medicine
Recruiting


Prognostic Factors Affecting Duchenne Muscular Dystrophy


Condition:   Duchenne Muscular Dystrophy
Intervention:  
Sponsor:   Assiut University
Not yet recruiting


Tamoxifen in Duchenne Muscular Dystrophy


Condition:   Duchenne Muscular Dystrophy
Interventions:   Drug: Tamoxifen;   Drug: Matching placebo
Sponsor:   University Hospital, Basel, Switzerland
Not yet recruiting


Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy


Condition:   Duchenne Muscular Dystrophy
Intervention:   Biological: rAAVrh74.MCK.GALGT2
Sponsor:   Kevin Flanigan
Enrolling by invitation


Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)


Condition:   Duchenne Muscular Dystrophy
Intervention:  
Sponsor:   Ann & Robert H Lurie Children's Hospital of Chicago
Enrolling by invitation


Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)


Condition:   Duchenne Muscular Dystrophy
Intervention:   Drug: NS-065/NCNP-01
Sponsors:   NS Pharma, Inc.;   Nippon Shinyaku Co., Ltd.;   Cooperative International Neuromuscular Research Group (CINRG);   Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS)
Enrolling by invitation


Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys With Duchenne Muscular Dystrophy


Condition:   Duchenne Muscular Dystrophy
Interventions:   Biological: BMS-986089;   Biological: Placebo for BMS-986089
Sponsor:   Hoffmann-La Roche
Active, not recruiting


Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)


Condition:   Duchenne Muscular Dystrophy
Interventions:   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
Sponsors:   ReveraGen BioPharma, Inc.;   University of Pittsburgh;   Cooperative International Neuromuscular Research Group
Enrolling by invitation


Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)


Condition:   Duchenne Muscular Dystrophy
Interventions:   Drug: BMN 044 IV 6 mg/kg;   Drug: BMN 044 IV 9 mg/kg;   Drug: BMN 044 SC 6 mg/kg
Sponsor:   BioMarin Pharmaceutical
Terminated


An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy


Condition:   Duchenne Muscular Dystrophy
Intervention:   Biological: PF-06252616
Sponsor:   Pfizer
Recruiting


An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)


Condition:   Duchenne Muscular Dystrophy
Interventions:   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
Sponsors:   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)
Active, not recruiting


A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)


Condition:   Duchenne Muscular Dystrophy
Interventions:   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
Sponsors:   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


Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2


Condition:   Duchenne Muscular Dystrophy
Interventions:   Biological: rAAVrh74.MCK.GALGT2;   Other: PLACEBO (Saline)
Sponsor:   Kevin Flanigan
Withdrawn


Study of DS-5141b in Patients With Duchenne Muscular Dystrophy


Condition:   Duchenne Muscular Dystrophy
Intervention:   Drug: DS-5141b
Sponsors:   Daiichi Sankyo Co., Ltd.;   Orphan Disease Treatment Institute Co., Ltd.
Active, not recruiting


Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients


Condition:   Duchenne Muscular Dystrophy
Interventions:   Drug: SRP-4045;   Drug: Placebo
Sponsor:   Sarepta Therapeutics
Active, not recruiting


Strength Training in Duchenne Muscular Dystrophy


Condition:   Duchenne Muscular Dystrophy
Interventions:   Procedure: Aim 2 Exercise group;   Procedure: Aim 1 Exercise Dosing;   Procedure: Aim 2 Control group
Sponsors:   University of Florida;   National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Active, not recruiting


Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)


Condition:   Duchenne Muscular Dystrophy
Interventions:   Drug: PRO044 SC 6 mg/kg;   Drug: PRO044 IV 6 mg/kg;   Drug: PRO044 IV 9 mg/kg
Sponsor:   BioMarin Pharmaceutical
Terminated


A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy


Condition:   Duchenne Muscular Dystrophy
Interventions:   Biological: PF-06252616;   Drug: Placebo
Sponsor:   Pfizer
Active, not recruiting


Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy


Condition:   Duchenne Muscular Dystrophy
Intervention:   Drug: Prednisolone
Sponsors:   Washington University School of Medicine;   Nationwide Children's Hospital;   Feinberg School of Medicine, Northwestern University;   University of Texas Southwestern Medical Center;   University of California, Davis;   Nemours Hospital, Orlando, FL
Completed


L-citrulline and Metformin in Duchenne's Muscular Dystrophy


Condition:   Duchenne's Muscular Dystrophy (DMD)
Interventions:   Drug: 750 mg metformin and 7.5 g L-citrulline daily p.o.;   Drug: Placebo
Sponsor:   University Hospital, Basel, Switzerland
Completed


A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)


Condition:   Duchenne Muscular Dystrophy
Interventions:   Drug: Regimen Selection Phase Group 2;   Drug: Regimen Selection Phase Group 3;   Drug: Treatment Phase Group 4;   Drug: Regimen Selection Phase Group 1 (COMPLETED);   Drug: Dosing Extension
Sponsor:   BioMarin Pharmaceutical
Terminated


Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy


Condition:   Duchenne Muscular Dystrophy
Interventions:   Drug: PRO045, 0.15 mg/kg/week;   Drug: PRO045, 1.0 mg/kg/week;   Drug: PRO045, 3.0 mg/kg/week;   Drug: PRO045, 6.0 mg/kg/week;   Drug: PRO045, 9.0 mg/kg/week;   Drug: PRO045, selected dose
Sponsor:   BioMarin Pharmaceutical
Terminated


Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy


Condition:   Duchenne Muscular Dystrophy
Interventions:   Drug: Epigallocatechin-Gallate;   Drug: Placebo
Sponsor:   Charite University, Berlin, Germany
Active, not recruiting

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