Muscular Dystrophy



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


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


A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Participants With Duchenne Muscular Dystrophy (DMD)


Condition:   Muscular Dystrophy, Duchenne
Intervention:   Drug: SRP-5051
Sponsor:   Sarepta Therapeutics
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


(-)- Epicatechin Becker Muscular Dystrophy


Condition:   Becker Muscular Dystrophy
Intervention:   Drug: (-)-Epicatechin
Sponsors:   University of California, Davis;   Cardero Therapeutics, Inc.
Recruiting


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


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
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


The Study of Skeletal Muscle Blood Flow in Becker Muscular Dystrophy


Condition:   Muscular Dystrophy
Interventions:   Drug: Tadalafil 20 MG;   Other: beetroot juice extract
Sponsor:   Cedars-Sinai Medical Center
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


Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy


Condition:   Muscular Dystrophy, Duchenne
Interventions:   Drug: Edasalonexent;   Drug: Placebo
Sponsor:   Catabasis Pharmaceuticals
Active, not recruiting


Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin to Patients With Duchenne Muscular Dystrophy


Condition:   Duchenne Muscular Dystrophy
Intervention:   Biological: rAAVrh74.MCK.micro-Dystrophin
Sponsors:   Jerry R. Mendell;   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Completed


Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy


Condition:   Duchenne Muscular Dystrophy
Intervention:   Biological: rAAV1.CMV.huFollistin344
Sponsors:   Jerry R. Mendell;   Duchenne Alliance;   Milo Therapeutics
Completed


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


Limb Girdle Muscular Dystrophy (LGMD) Natural History


Condition:   Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED)
Intervention:  
Sponsors:   Carolinas Healthcare System;   Muscular Dystrophy Association
Completed


Heart Imaging in Children With Muscular Dystrophy


Condition:   Muscular Dystrophy
Intervention:  
Sponsor:   National Heart, Lung, and Blood Institute (NHLBI)
Terminated

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