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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 (72) clinical trials

Patients are needed to participate in a clinical research study evaluating SRP-4045, SRP-4053, Placebo for the treatment of DUCHENNE MUSCULAR DYSTROPHY, Muscular Dystrophy

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 ...


A clinical trial to evaluate treatments for patients with DUCHENNE MUSCULAR DYSTROPHY

Each subject will receive pamrevlumab (35 mg/kg) every two weeks by intravenous infusion for up to 156 weeks. After at least 10 to 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 ...


A clinical trial sponsored by Pfizer for a research study for the treatment of 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 ...


Patients are needed to participate in a clinical research study to evaluate Facioscapulohumeral muscular dystrophy

Part 1 (dose escalation, open-label) Part 1 will consist of up to 6 cohorts (A to F) of patients and will evaluate multiple ascending dose levels of ACE-083 in either the tibialis anterior (TA) or biceps brachii (BB) muscle. Patients in each cohort will be enrolled in a 4-week screening ...


Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents

This single center open-label pilot study will enroll 15 non-ambulatory children with Duchenne muscular dystrophy at least 8 years of age and who demonstrate pre-clinical cardiomyopathy (defined as a cardiac ejection fraction >55% with abnormal LV strain by cardiac MRI). They will receive (+)-epicatechin at one of three doses during ...


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

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. ...


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 ...


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 ...


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 ...