Clinical trials
Duchenne Muscular Dystrophy clinical trials
Below are recruiting duchenne muscular dystrophy clinical trials, each written for real people, not researchers. We’re tracking 82 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT07587242RecruitingPhase 3
Testing a New Therapy for DMD with Exon 44 Mutations
This study tests whether a new medicine called AOC 1044 (delpacibart zotadirsen) can help boys with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation. The medicine works by helping the body skip a damaged part of the gene to make a functional protein.
LeuvenAges 7–16 - NCT07092540Recruiting
Baby Duchenne Study: Early Years in Boys With DMD
This study tracks development and health in young boys with Duchenne muscular dystrophy during the first three years of life. It aims to better understand early signs and how the condition progresses, which may help improve care and future treatments.
Rochester, New YorkAges birth–3 years - NCT07766980Recruiting
Nutritional supplement for quality of life in Duchenne muscular dystrophy
This study tests whether a daily nutritional supplement called VM100 can improve quality of life for people with Duchenne muscular dystrophy. If you are 8 or older and your medications have been stable for at least 3 months, you may be able to participate.
Gainesville, FloridaAges 6 years+ - NCT07160634RecruitingPhase 3
Gene therapy for ambulant males with Duchenne muscular dystrophy
This trial tests a new gene therapy called SGT-003 in boys and men with Duchenne muscular dystrophy who are still able to walk. The treatment aims to deliver a working version of the dystrophin gene to help muscle cells produce the protein they are missing.
Little Rock, ArkansasAges 7–11 - NCT05016908RecruitingTeam says not enrolling
Testing blood and other fluid markers in Duchenne or Becker
This study looks for signals in blood and other body fluids that may help track Duchenne or Becker muscular dystrophy. It may be helpful for patients by improving how future monitoring tests are developed.
Boston, MassachusettsAges 5 years+ - NCT07573631Enrolling by invitationPhase 2
Long-term Safety Study of BMN 351 for Duchenne Muscular Dystrophy
This study continues treatment with an investigational medicine called BMN 351 for people with Duchenne muscular dystrophy (a genetic muscle-weakening condition). It checks whether the medicine remains safe and effective when used over a longer period, building on results from an earlier trial.
Milan, ItalyAges 4 years+ - NCT07172971RecruitingPhase 1
SGLT2i drug for heart health in Duchenne muscular dystrophy
This trial tests if an SGLT2i medication can help protect heart function in people with Duchenne muscular dystrophy (DMD). The drug is already approved for other conditions, and researchers want to see if it can slow or prevent heart muscle damage.
Nashville, TennesseeAges 8–18 - NCT06564974Recruiting
Long-term safety study of Agamree for Duchenne muscular dystrophy
This study watches boys with Duchenne muscular dystrophy who are already taking Agamree (vamorolone) to see how safe it is over a long period. It helps doctors understand the medicine's effects over time.
Phoenix, ArizonaAges 2 years+ - NCT05933057RecruitingPhase 3
Givinostat for wheelchair-bound boys with Duchenne muscular dystrophy
This Phase 3 trial tests givinostat to see if it can improve safety and functional outcomes in boys with Duchenne muscular dystrophy who cannot walk. You might be a candidate if you meet age, genetic diagnosis, wheelchair-level mobility, and specific arm-strength/respiratory/heart criteria.
LeuvenAges 9–17 - NCT03373968RecruitingPhase 2/Phase 3
Long-term study of givinostat safety in Duchenne muscular dystrophy
This trial looks at how safely givinostat works over a longer time in people with Duchenne muscular dystrophy (DMD). It mainly checks long-term side effects and tolerability, especially in a specific subgroup of muscle fat levels.
Sacramento, CaliforniaAges 7 years+ - NCT03882827Recruiting
Natural history study of Duchenne muscular dystrophy in young boys
This study follows boys with Duchenne muscular dystrophy over time to better understand how the condition changes and how standard treatments affect health and function. It does not appear to test a new medicine, but it may help doctors plan better future care and studies.
BrusselsAges 4–9 - NCT04012671Recruiting
Study of Duchenne muscular dystrophy in children and carriers
This is a research study that follows people with Duchenne muscular dystrophy and female genetic carriers to better understand the condition. It may help researchers measure health patterns over time, which can support future treatments.
FuzhouAges 2 years+ - NCT04626674RecruitingPhase 1
Gene therapy safety study for non-ambulatory Duchenne
This Phase 1 trial studies a one-time gene therapy treatment (SRP-9001) to see if it is safe and whether it can produce the needed gene activity in people with Duchenne muscular dystrophy (DMD) who are not walking. It may help researchers understand whether this approach could be useful for DMD patients, especially in later stages.
Little Rock, ArkansasAges 2 years+ - NCT04906460RecruitingPhase 1/Phase 2
Testing WVE-N531 for Duchenne muscular dystrophy exon 53
This early-phase study tests WVE-N531 in people with Duchenne muscular dystrophy (DMD) whose genetics specifically match an “exon 53” change. It may help by targeting the muscle disease process, and it includes procedures like muscle biopsies and regular heart/lung checks.
Little Rock, ArkansasAges 4–18 - NCT05019625Recruiting
Study samples for biomarkers in myotonic dystrophy
This study collects blood and, for some people, muscle tissue to look for signs (biomarkers) that can help track and better understand myotonic dystrophy. You may help by providing samples and basic muscle function testing, depending on your age and condition type.
Boston, MassachusettsAges 5 years+ - NCT05982119Recruiting
Assessing muscle disease patients with activity monitors
This study assesses physical activity in people with different muscle diseases (like Duchenne, FSHD, and others) and in healthy volunteers. It helps doctors understand how these conditions affect daily movement and function.
LiègeAges 1–80 - NCT05996003RecruitingPhase 2
Exon 44 skipping drug for Duchenne muscular dystrophy
This trial tests a new drug (NS-089/NCNP-02) that aims to help boys with Duchenne muscular dystrophy produce a shorter but still working version of the dystrophin protein. It's for boys who are still walking and have a specific genetic type that can be fixed by skipping a part of the gene called exon 44.
Aurora, ColoradoAges 4–14 - NCT06138639RecruitingPhase 1/Phase 2
Gene therapy SGT-003 for Duchenne muscular dystrophy
This trial tests a new gene therapy called SGT-003 for children with Duchenne muscular dystrophy. It aims to help the body produce a working version of dystrophin, the protein missing in Duchenne.
Little Rock, ArkansasAges birth–17 years - NCT06147414Recruiting
Blood test for genetic disorders in pregnancy
This study tests a safer blood test to check for certain genetic disorders during pregnancy, instead of an invasive procedure like amniocentesis. It's for pregnant women who are at risk of passing on a known genetic condition.
ParisAges 18 years+ - NCT06290713RecruitingPhase 2
Blood flow and exercise study for Duchenne muscular dystrophy
This trial tests if a medicine that improves blood flow, combined with exercise, can help muscles in boys with Duchenne muscular dystrophy. It is for boys who can still walk and are on a stable steroid medication.
Gainesville, FloridaAges 6 years+ - NCT06363357Recruiting
Shoulder brace study for arm weakness from nerve or muscle diseases
This study tests a fabric shoulder brace that moves like a muscle. It may help people with nerve or muscle diseases improve arm movements like reaching and lifting.
Seoul, Jongno-guAges 10 years+ - NCT06366815Recruiting
A study of Duchenne muscular dystrophy in boys who no longer walk
This study is for boys with Duchenne muscular dystrophy who can no longer walk. It aims to describe how the condition changes over time once walking is lost, without testing any new treatments.
Bosisio Parini, LcAges 8–35 - NCT06402942Recruiting
Game-based occupational therapy for teens with Duchenne
This trial tests a fun, game-based occupational therapy program for teens with Duchenne muscular dystrophy. It aims to help you build skills and stay engaged through activities you can do at home on a computer or tablet.
AnkaraAges 13–18 - NCT06641895RecruitingEarly Phase 1
Testing BBM-D101 gene therapy for Duchenne muscular dystrophy
This early-phase study tests a new gene therapy called BBM-D101 for boys with Duchenne muscular dystrophy (DMD). The goal is to see if it is safe and helps improve muscle function in young boys.
Shanghai, Shanghai MunicipalityAges 4–8
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Duchenne Muscular Dystrophy trials by city
Studies with a site in or near these metro areas.
Duchenne Muscular Dystrophy trials by state
Studies with a site anywhere in these states.
Common questions
- Are there clinical trials for duchenne muscular dystrophy?
- Yes. Clin2 currently lists 82 recruiting duchenne muscular dystrophy studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a duchenne muscular dystrophy trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a duchenne muscular dystrophy trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.