There are currently 25 active clinical trials seeking participants for Duchenne Muscular Dystrophy research studies. The states with the highest number of trials for Duchenne Muscular Dystrophy participants are California, Ohio, Pennsylvania and Florida.
Assessment of Neurodevelopmental Needs in Duchenne Muscular Dystrophy
NCT05280730
Recruiting
Duchenne Muscular Dystrophy is a genetic disease that causes progressive muscle weakness. There is now substantial evidence that boys with this disease do not demonstrate age-related gains in their cognitive skills. The goals of this study are (i) to use a technology-enabled neurobehavioral assessment called National Institutes of Health Toolbox Cognition Battery (NIHTB-CB) to assess brain development over time; (ii) engage with key-stakeholders to understand how neurodevelopmental problems lik... Read More
Gender:
ALL
Ages:
3 years and above
Trial Updated:
03/25/2025
Locations: Children's Hospital of Richmond, Richmond, Virginia
Conditions: Duchenne Muscular Dystrophy
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A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
NCT06138639
Recruiting
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 dystrophy. There will be 2 cohorts in this study. Cohort 1 will include participants 4 to \<7 years of age. Cohort 2 will include participants 7 to \<12 years of age. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.
Gender:
MALE
Ages:
Between 4 years and 11 years
Trial Updated:
03/25/2025
Locations: Arkansas Children's Hospital, Little Rock, Arkansas +5 locations
Conditions: Duchenne Muscular Dystrophy
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Trial of Cell Based Therapy for DMD
NCT06692426
Recruiting
This is a single-center, single-arm, interventional phase 1 trial to evaluate the safety and tolerability of local injection of induced pluripotent stem cell (iPSC)- derived CD54+ allogeneic muscle progenitor cells in individuals with Duchenne muscular dystrophy (DMD)
Gender:
ALL
Ages:
18 years and above
Trial Updated:
03/21/2025
Locations: Masonic Cancer Center, Minneapolis, Minnesota
Conditions: Duchenne Muscular Dystrophy
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AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants with Duchenne Muscular Dystrophy (DMD)
NCT05693142
Recruiting
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 multicenter, open-label dose evaluation clinical study to assess the safety, tolerability, and clinical efficacy of a one-time intravenous (IV) dose of RGX-202 in participants with Duchenne.
Gender:
MALE
Ages:
1 year and above
Trial Updated:
03/10/2025
Locations: Arkansas Children's Hospital, Little Rock, Arkansas +5 locations
Conditions: Duchenne Muscular Dystrophy
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Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients with Duchenne Muscular Dystrophy.
NCT06564974
Recruiting
The goal of this observational study is to follow patients being treated with the FDA approved drug AGAMREE® in male patients 2 years of age or older with Duchenne's Muscular Dystrophy for long term safety and quality of life.
Gender:
MALE
Ages:
2 years and above
Trial Updated:
03/07/2025
Locations: Arkansas Childrens Hospital, Little Rock, Arkansas +8 locations
Conditions: Duchenne Muscular Dystrophy
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A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)
NCT06450639
Recruiting
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 ambulatory and non-ambulatory participants with DMD age ≥ 8 to \< 18 years old receiving corticosteroid therapy.
Gender:
MALE
Ages:
Between 8 years and 17 years
Trial Updated:
03/05/2025
Locations: Corewell Health, Grand Rapids, Michigan +11 locations
Conditions: Duchenne Muscular Dystrophy
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A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74
NCT06597656
Recruiting
This is a gene transfer therapy study evaluating the safety of and delandistrogene moxeparvovec dystrophin protein expression from delandistrogene moxeparvovec following therapeutic plasma exchange (plasmapheresis) in ambulatory male participants with DMD and pre-existing antibodies to AAVrh74 over a period of 58 weeks.
Gender:
MALE
Ages:
Between 4 years and 8 years
Trial Updated:
02/28/2025
Locations: Washington University School of Medicine in St. Louis, Saint Louis, Missouri +1 locations
Conditions: Duchenne Muscular Dystrophy
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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)
NCT05881408
Recruiting
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 moxeparvovec in either Part 1 or Part 2.
Gender:
MALE
Ages:
All
Trial Updated:
02/14/2025
Locations: Arkansas Children's Hospital, Little Rock, Arkansas +41 locations
Conditions: Duchenne Muscular Dystrophy
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Once Weekly Infant Corticosteroid Trial for DMD
NCT05412394
Recruiting
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.
Gender:
MALE
Ages:
Between 1 month and 30 months
Trial Updated:
02/11/2025
Locations: Lurie Children's Hospital of Chicago, Chicago, Illinois +3 locations
Conditions: Duchenne Muscular Dystrophy
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The Duchenne Registry
NCT02069756
Recruiting
The Duchenne Registry is an online, patient-report registry for individuals with Duchenne and Becker muscular dystrophy and carrier females. The purpose of the Registry is to connect Duchenne and Becker patients with actively recruiting clinical trials and research studies, and to educate patients and families about Duchenne and Becker care and research. At the same time, The Duchenne Registry is a valuable resource for clinicians and researchers in academia and industry, allowing access to de-i... Read More
Gender:
ALL
Ages:
All
Trial Updated:
02/06/2025
Locations: The Duchenne Registry / PPMD, Washington, District of Columbia
Conditions: Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
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NS-050/NCNP-03 in Boys With DMD (Meteor50)
NCT06053814
Recruiting
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 to ambulant boys with DMD, who have a DMD exon deletion amenable to exon 50 skipping.
Gender:
MALE
Ages:
Between 4 years and 14 years
Trial Updated:
12/12/2024
Locations: Children's Hospital Los Angeles, Los Angeles, California +10 locations
Conditions: Duchenne Muscular Dystrophy
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An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy
NCT06270719
Recruiting
This is a multicenter, prospective, observational Phase 4 study in the United States. The study is designed to collect both medical history and prospective data on Duchenne muscular dystrophy (DMD) treatment outcomes in participants receiving delandistrogene moxeparvovec as part of clinical care, compared to participants with DMD receiving or prescribed to start chronic glucocorticoid treatment at baseline in routine clinical practice.
Gender:
MALE
Ages:
4 years and above
Trial Updated:
11/27/2024
Locations: Arkansas Children's Hospital, Little Rock, Arkansas +12 locations
Conditions: Duchenne Muscular Dystrophy
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