A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)...
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain. This is...
A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to assess the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of satralizumab, a humanized anti-interleukin-6 receptor (aIL-6R) monoclonal antibody, in a...
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dy...
Once Weekly Infant Corticosteroid Trial for DMD
The hypothesis tested here is that a lower dose of intermittent oral corticosteroids (5mg/kg/week) will be equally effective to the 10mg/kg/week dose....
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
This is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to \<15 years with DMD due to mutations amenable to exon 44 skip...
NS-050/NCNP-03 in Boys With DMD (Meteor50)
This is a Phase 1/2 study of Multiple-Ascending Dose (MAD) levels for 12 weeks of treatment followed by 24 weeks of open-label treatment with a selected dose of NS-050/NCNP-03 administered once weekly...
A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Partici...
A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy
The goal of this clinical trial is to learn how safe and tolerable EPI-321 is and whether there may be early signs it is working in male or female adult (18 to 75 years) participants with facioscapulo...
Frequently Asked Questions
What clinical trials are available for Duchenne Muscular Dystrophy?
There are currently 9 actively recruiting clinical trials for duchenne muscular dystrophy. These studies are testing new treatments, therapies, and interventions at research sites across 41 cities.
How do I join a Duchenne Muscular Dystrophy clinical trial?
To join a duchenne muscular dystrophy clinical trial: 1) Browse the available studies above, 2) Click on a study that interests you, 3) Complete the eligibility form, and 4) A study coordinator will contact you.
Are clinical trials free for patients?
Yes, clinical trial participation is free. In most studies, the treatment, medical tests, and doctor visits related to the trial are provided at no cost. Some studies may also compensate for time and travel.
Can I leave a clinical trial if I change my mind?
Yes, participation in any clinical trial is completely voluntary. You can withdraw at any time, for any reason, without affecting your regular medical care.