Active clinical trials for "Muscular Dystrophy, Duchenne"

This study will help determine if CoQ10 and prednisone, alone and as a combination decrease the decline in cardiopulmonary and skeletal muscle function that occurs in the wheelchair confined phase of DMD. Participants who are enrolled in this study should not have taken any corticosteroids within the last six months. This is a 13-month, prospective, randomized study comparing a daily prednisone arm (0.75mg/kg/day), a CoQ10 arm (serum of greater than 2.5 ug/mL) and a combination arm (prednisone and CoQ10) with an enhanced standard of care arm in wheelchair confined males age 10 to 18 years with an established DMD diagnosis.

This study is a longitudinal, randomized control trial evaluating the use of two commercially available dynamic arm support devices (1) Armon Ayura-Kinova and 2) JAECO WREX) to promote participation in activities of daily living (ADLs) in non-ambulatory individuals with Duchenne muscular dystrophy (DMD) with upper extremity weakness.

The purpose of this study is to describe the progression of tissular and functional myocardial abnormalities in patients with Duchenne muscular dystrophy using cardiac magnetic resonance imaging and blood biomarkers assays.

To evaluate the positive effects of a home-based physical therapy intervention added to the usual physical therapy programs performed in children with neurological pathologies that induce sensorimotor impairments that affect their quality of life and the importance of the implications of their families in their treatment.

This is a phase 2, randomised, double-blind, placebo controlled study to evaluate the micro-macroscopic effects on muscles, the safety and tolerability, and the efficacy of givinostat in patients with Becker Muscular Dystrophy. Approximately 48 eligible patients will be randomized in a 2:1 ratio to be treated with givinostat or placebo for a period of 12 months.

This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.

This is a multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of two different weekly doses of RO7239361 in ambulatory boys with Duchenne Muscular Dystrophy (DMD).

Duchenne muscular dystrophy (DMD) is a progressive devastating disease that affects mainly boys, with an incidence of about 1:3,500 live births. The pathology of DMD is a result of non-repaired muscle damage that leads to muscle-tissue replacement by scar tissue, a process known as fibrosis. Currently, there is no effective treatment for the disease. The only therapy offered to these boys are steroids which slightly delayed the disease progression. The boys lose their ability to walk at around the age of 12, and die in the 4th decade of life from severe heart and lung problems. In this study investigators will test the efficacy of Tamoxifen treatment in ambulatory DMD boys. Tamoxifen is a drug used for palliative treatment of breast cancer patients and has an outstanding safety profile. In addition, Tamoxifen was tested in the past in boys, for other pediatric indications, and showed an excellent safety with no side effects. Tamoxifen is being tested in this study, as a therapy for DMD, for the following reasons: (i) it was shown to have anti-fibrotic effect in multiple in-vivo systems; (ii) it assists in the repair of damaged muscles. In other words, Tamoxifen is expected to have a synergistic effect on DMD patients, due to its dual mechanism of action. Indeed, Tamoxifen was shown to have significant beneficial effects in the mdx mouse model of DMD. Also, a small compassionate cohort of 3 boys, treated for 6 months with Tamoxifen, yielded very encouraging results.

This is an open label, long-term extension study in approximately 24 male DMD subjects consisting of children (ages 4-12, inclusive) and adolescents (ages 13-16, inclusive) who participated in the MP-104-CL-005 PK study.

The purpose of this study is to evaluate the safety and efficacy of exogenous gene transfer in DMD participants by measuring biological and clinical endpoints in three parts: two 48-week randomized, double-blinded, placebo-controlled periods (Part 1 and Part 2), and an open-label follow-up period (Part 3). Participants who are randomized to placebo in Part 1 will have the opportunity for treatment with delandistrogene moxeparvovec in Part 2. In order to provide a uniform approach to monitoring long-term safety and efficacy in participants who received SRP-9001 in a clinical trial, the Sponsor has amended Study Completion for this study to occur at Week 130. Therefore, participants have transitioned and will complete the remainder of the Part 3 follow up visits in a long-term extension study, SRP-9001-305 (NCT05967351).