Duchenne Muscular Dystrophy



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
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


Virtual Reality in Individuals With Duchenne Muscular Dystrophy


Condition:   Duchenne Muscular Dystrophy
Interventions:   Behavioral: DMD gesture task;   Behavioral: DMD button-press task;   Behavioral: Control group gesture task;   Behavioral: Control group button-press task
Sponsor:   University of Sao Paulo
Completed


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


Analysis of a Virtual Reality Task in Patients With Duchenne Muscular Dystrophy


Condition:   Duchenne Muscular Dystrophy
Interventions:   Device: Acquisition on TouchScreen;   Device: Acquisition on Kinect;   Device: Acquisition on LeapMotion
Sponsor:   University of Sao Paulo
Completed


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
Active, not recruiting


A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy


Condition:   Duchenne Muscular Dystrophy
Interventions:   Drug: TAS-205;   Drug: Placebo
Sponsor:   Taiho Pharmaceutical Co., Ltd.
Active, not recruiting


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)
Recruiting


Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy


Condition:   Duchenne Muscular Dystrophy
Interventions:   Drug: Eplerenone;   Drug: Spironolactone
Sponsors:   Ohio State University;   University of California, Los Angeles;   University of Utah;   University of Colorado, Denver;   University of Kansas Medical Center;   Vanderbilt University Medical Center
Active, not recruiting


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


Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients


Condition:   Duchenne Muscular Dystrophy
Interventions:   Biological: Myoblast transplantation;   Procedure: Saline injection
Sponsor:   CHU de Quebec-Universite Laval
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


Duchenne Muscular Dystrophy < 18y in Norway: Genotype/Phenotype, Growth, Puberty, Bone Health and Quality of Life.


Condition:   Duchenne Muscular Dystrophy
Intervention:  
Sponsors:   Oslo University Hospital;   The Research Council of Norway;   University Hospital of North Norway
Completed


Whole Body Vibration Therapy in Boys With Duchenne Muscular Dystrophy


Condition:   Duchenne Muscular Dystrophy
Intervention:   Device: Whole Body Vibration Therapy
Sponsor:   Children's Hospital of Eastern Ontario
Active, not recruiting


Finding the Optimum Regimen for Duchenne Muscular Dystrophy


Condition:   Duchenne Muscular Dystrophy
Interventions:   Drug: Prednisone;   Drug: Deflazacort
Sponsors:   University of Rochester;   Newcastle University;   University Medical Center Freiburg;   National Institute of Neurological Disorders and Stroke (NINDS)
Active, not recruiting


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


The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor


Condition:   Duchenne Muscular Dystrophy
Intervention:  
Sponsor:   National Human Genome Research Institute (NHGRI)
Completed


This is a Study to Get More Information About Non Ambulatory Boys & Men With Duchenne Muscular Dystrophy


Condition:   Duchenne Muscular Dystrophy
Intervention:  
Sponsors:   Washington University School of Medicine;   Nationwide Children's Hospital;   Boston Children’s Hospital;   University of Minnesota - Clinical and Translational Science Institute;   University of California, Davis
Completed


Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)


Condition:   Duchenne Muscular Dystrophy
Intervention:   Drug: Ataluren
Sponsors:   PTC Therapeutics;   Genzyme, a Sanofi Company
Terminated

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