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Muscular Dystrophy Clinical Trials

A listing of Muscular Dystrophy medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

Found 136 clinical trials
E Eytan Ben-Ami, Dr

Subcutaneous Administration of MD-18 in Healthy Subjects

A Single Center, Single Dose, Double-blind, Randomized, Placebo-controlled Dose-Escalating Study to Evaluate Safety, Tolerability and Pharmacokinetics of Subcutaneously Administered MD-18 in healthy subjects.

18 - 70 years of age Both Phase 1

Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)

A study of sevasemten (EDG-5506) in Becker muscular dystrophy (known as CANYON) and pivotal cohort (known as GRAND CANYON). The EDG-5506-201 CANYON study was expanded to include an additional 120 adult participants in a cohort called GRAND CANYON, that is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety …

12 - 50 years of age Male Phase 2
H Hugh McMillan

Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy

This is a randomised, double-blind, placebo-controlled, multicentre study to evaluate the efficacy, safety, and tolerability of givinostat in non-ambulant male paediatric (aged 9 to <18 years) patients with DMD. 138 patients will be randomised 2:1 to givinostat or placebo and will be treated for 18 months. Planned screening duration: approximately …

9 - 17 years of age Male Phase 3
M Mariacristina Scoto

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The study consists of 3 periods: a multiple-ascending dose (MAD) / placebo-controlled …

4 - 16 years of age Male Phase 1/2
C Carmen Silva

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy

The purpose of this study is to test the safety and tolerability of BMN 351 in participants with Duchenne Muscular Dystrophy (DMD) with a genetic mutation amenable to exon 51 skipping.

4 - 10 years of age Male Phase 1/2
E Eugenio Mercuri

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene …

- 100 years of age Male Phase 3
J John Brandsema, MD

Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I)

This study will evaluate the safety and efficacy of long-term administration of BBP-418 in patients with LGMD2I/R9. The study will include patients ages 12 to 60, consistent with the existing preclinical toxicology profile. This will encompass the significant majority of existing diagnosed patients based upon the established epidemiology of the …

12 - 60 years of age Both Phase 3
K Kina Bennett

Hydrotherapy in Duchenne Muscular Dystrophy (DMD)

The goal of this clinical trial aims to establish if there are meaningful benefits to providing a hydrotherapy service for young people with Duchenne muscular dystrophy (DMD). The main aims are to: 1. to allocate a clinical physiotherapist to a project implementing hydrotherapy in young patients with DMD to establish …

6 - 25 years of age Male Phase N/A
R Roxanna M Bendixen, Ph.D.

Sleep Intervention in Young Boys With Duchenne Muscular Dystrophy

This project will systematically plan and evaluate the implementation of the Transdiagnostic Sleep and Circadian Intervention for youth (TranS-CY). As an early stage study, investigators will focus on recruitment strategies to reach the target population and collection of preliminary data on primary and secondary effects of the TranS-CY. Weekly remote …

6 - 18 years of age Both Phase N/A

Gamified Occupational Therapy for Adolescents With Duchenne Muscular Dystrophy

This research aims to improve the quality of life, occupational performance, occupational satisfaction and emotional health of young people with Duchenne muscular dystrophy compared to the classical occupational therapy program. The findings are planned to shed light on the development of new and effective strategies in the rehabilitation of adolescents …

13 - 18 years of age Male Phase N/A

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