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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.

RESULTS

Found (27) clinical trials

Study of SRP-4045 and SRP-4053 in DMD Patients

This is a double-blind, placebo-controlled, multi-center study to evaluate the efficacy and safety of SRP-4045 and SRP-4053. Eligible patients with out-of-frame deletion mutations amenable to exon 45 or 53 skipping will be randomized to receive once weekly intravenous (IV) infusions of 30 mg/kg SRP-4045 or 30 mg/kg SRP-4053 respectively (combined-active ...

Phase

Peak Cough Flow and Cough Clearance in Patients With Muscular Dystrophy

This study is to determine whether physiologic measures (peak cough flow, measures of respiratory muscle strength including MIP, MEP ,SNIP, and spirometry) can predict spontaneous cough clearance (as measured by a nuclear medicine study) in children with neuromuscular disease. It will also determine whether airway clearance is augmented by high ...

Phase N/A

Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy

This is a multicenter, observational study of patients receiving Translarna based on inclusion of their data in a registry. This study is intended to enroll 200 patients across ~50 care centers in Europe and other regions over a period of ~ 2 years. The study population will include patients who ...

Phase N/A

Trial of Pamrevlumab (FG-3019) in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)

Each subject will receive pamrevlumab (35 mg/kg) every two weeks by intravenous infusion for up to 104 weeks. After 12 subjects complete one year of treatment interim analysis may increase total subjects enrolled to approximately 32. All subjects will be closely monitored for safety. Efficacy assessments will be performed routinely ...

Phase

Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

This is a Phase II, multiple center, 2-period, randomized, placebo-controlled, dose finding study of NS-065/NCNP-01 administered by infusion once weekly for 24 weeks to ambulant boys ages 4-<10 years with DMD. Two dose level cohorts will be enrolled. Period 1 of this study will be conducted in a double-blind fashion. ...

Phase

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

This study will evaluate the safety and tolerability of a new steroid-like medication called vamorolone in boys with DMD ages ≥ 4 years and < 7 years. Enrolled participants will take the study medication for 14 days followed by a 14 day follow-up period. The potential effectiveness of vamorolone in ...

Phase

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

This study will evaluate if it is safe to use a new medication called vamorolone for more than two weeks in children with DMD, if boys with DMD who take the study medication have improved muscle function compared to boys with DMD in other studies who did not take any ...

Phase

A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids

The SIDEROS trial is a randomized, placebo controlled, parallel group study of the efficacy of idebenone in delaying the loss of respiratory function, whilst also monitoring safety and tolerability of idebenone in at least 266 DMD patients taking stable dose of concomitant glucocorticoid steroids. The study treatment period will be ...

Phase

Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy

Givinostat or placebo oral suspension (10 mg/mL) will be administered orally as 2 oral doses daily while the subject is in fed state, according to the child's weight. Study drug should be permanently stopped if any of the following occur: - severe drug-related diarrhoea; - any drug-related Serious Adverse Event; ...

Phase

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

This study is an open-label extension to protocol B5161002 and will provide an assessment of the long term safety, efficacy, pharmacodynamics and pharmacokinetics of intravenous dosing of PF 06252616 in boys with Duchenne muscular dystrophy. Approximately 105 eligible subjects will be assigned to receive a monthly individualized maximum tolerated dose ...

Phase