Clinical trials
Muscular Dystrophy clinical trials
Below are recruiting muscular dystrophy clinical trials, each written for real people, not researchers. We’re tracking 133 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT07515235Recruiting
DMD Gene Variants and Heart Function in Young Males
This study examines how different genetic changes in the DMD gene affect heart function in boys with dystrophinopathy (a muscle-weakening condition). Understanding this connection may help doctors better monitor and treat heart problems in affected boys.
ThessalonikiAges 2–24 - NCT07118280Recruiting
Oral health and saliva study in OPMD patients and families
This study looks at the mouth health and saliva of people with OPMD and their household members. By understanding saliva changes, researchers hope to learn more about the disease and find ways to help manage symptoms.
Ramat GanAges 18 years+ - NCT06131983Enrolling by invitationPhase 1/Phase 2
Study of SRP-1001 for adults and teenagers with FSHD1
This study tests a new medicine called SRP-1001 to see if it can help people with a type of muscular dystrophy called FSHD1. It aims to slow down muscle weakness and improve quality of life.
Liverpool, New South WalesAges 16–70 - NCT00390104Recruiting
Study uses blood and skin samples to learn muscular disease causes
This study looks at samples from people with muscular dystrophy and from close relatives, to better understand neuromuscular diseases. You may be asked to provide a skin biopsy for research to help with genetic and molecular analysis.
Boston, MassachusettsAges 1 week–100 years - NCT07711730Recruiting
Telehealth support for kids with limb-girdle muscular dystrophy
This study tests an online program to help children and teens with limb-girdle muscular dystrophy improve their thinking, social skills, and emotional well-being. You join from home using a computer with a camera and internet.
Bilbao, BiscayAges 7–17 - NCT07332013Recruiting
Urine titin test for Duchenne muscular dystrophy
This study measures a protein called titin in urine to see if it can help track muscle damage in boys with Duchenne or Becker muscular dystrophy. It also includes healthy boys for comparison.
Philadelphia, PennsylvaniaAges 2–10 - NCT07188012RecruitingEarly Phase 1
Gene therapy trial for Duchenne muscular dystrophy in young boys
This trial tests a new gene therapy called SPOT-03 to see if it can help the body make dystrophin, a protein that is missing in Duchenne muscular dystrophy (DMD). It is for boys ages 2 to under 8 who can still walk at least 10 meters.
Shanghai, Shanghai MunicipalityAges 2–7 - 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 - NCT07086521RecruitingPhase 1
Stem cell therapy for facioscapulohumeral muscular dystrophy
This trial tests a new stem cell treatment (ULSC) for people with FSHD, a type of muscular dystrophy. It aims to see if the treatment is safe and if it can help improve arm strength and daily movement.
Palo Alto, CaliforniaAges 15 years+ - 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+ - NCT06185673RecruitingPhase 1/Phase 2
Gene therapy trial for OPMD with swallowing problems
This trial tests an experimental gene therapy (BB-301) for people with OPMD who have trouble swallowing. It involves a one-time injection into the throat muscles during a short surgery, and researchers will check if it is safe and improves swallowing.
New York, New YorkAges Up to 65 years - 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 - 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 - NCT04001582Recruiting
UK facial and shoulder muscle disease patient registry
This registry aims to collect health information from people in the UK who have facioscapulohumeral muscular dystrophy (FSHD). It helps researchers better understand the disease and plan future studies.
Newcastle upon TyneAges Any age - NCT07429240RecruitingPhase 1/Phase 2
Gene editing trial for Duchenne muscular dystrophy (ages 2-7)
This trial tests a new gene editing treatment called PBGENE-DMD for boys with Duchenne muscular dystrophy (DMD) whose genetic mutation falls within a specific range of the dystrophin gene (exons 45-55). The goal is to see if it is safe and can help improve muscle function.
Little Rock, ArkansasAges 2–7 - NCT07415837Recruiting
Studying a molecule in muscle diseases and healthy people
This study looks at a molecule called miR-1 in the blood to see if it can help understand muscle diseases. Researchers will compare levels in people with certain muscle conditions and in healthy volunteers.
Clermont-FerrandAges 2 years+ - NCT06868784Enrolling by invitation
Thinking and daily skills in children with Duchenne
This study looks at how thinking skills (like planning and memory) affect everyday activities such as dressing or playing in children with Duchenne muscular dystrophy. It aims to find ways to help kids do better in their daily lives.
Ankara, ÇankayaAges 6–12 - NCT06412328Enrolling by invitation
Psychoeducation for parents of children with DMD
This study offers a psychoeducation program for parents of children with Duchenne Muscular Dystrophy. It aims to provide support and education to help you care for your child.
Ankara, CankayaAges 18–65 - NCT05394506Recruiting
Study of factors that affect muscle laminopathy with LMNA changes
This study looks at what influences the course of a muscle disease caused by an LMNA genetic change (a type of laminopathy). It may help researchers better understand the disease and how it affects muscle and sometimes breathing or heart function.
Bron, Auvergne-Rhône-AlpesAges 2 years+ - NCT07745218Recruiting
Testing a new companion therapy for LAMA2 muscular dystrophy
This study looks at a new protein that might help people with LAMA2-related muscular dystrophy. It uses stored samples to see if the protein could be a useful addition to future treatments.
MilanAges Any age - NCT07146256Recruiting
OPMD Natural History Registry Study
This study is building a national registry (a database) for people with Oculo-Pharyngeal Muscular Dystrophy (OPMD) in Israel. Joining will help researchers understand how OPMD progresses over time and may lead to better care.
Ramat GanAges 18 years+ - NCT06503367Recruiting
Observation study for children 0-5 with LAMA2 muscular dystrophy
This study follows young children with LAMA2-related congenital muscular dystrophy to learn more about the disease. It does not test a new treatment, so it may help families understand the condition better and prepare for future trials.
Los Angeles, CaliforniaAges Up to 5 years - NCT04626674RecruitingPhase 1
Gene therapy for non-ambulatory Duchenne muscular dystrophy
This study tests a gene therapy that aims to help muscles produce a protein they are missing. It is for people with Duchenne muscular dystrophy who are no longer able to walk, and it may help slow the progression of the disease.
Little Rock, ArkansasAges 2 years+ - NCT07542314RecruitingPhase 4
Gene therapy for Duchenne muscular dystrophy
This study is checking how safe and effective ELEVIDYS, a gene therapy for Duchenne muscular dystrophy, is when used in everyday clinics. It will help doctors understand more about the treatment's real-world effects and provide care for those who receive it.
Houston, TexasAges 4 years+
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Common questions
- Are there clinical trials for muscular dystrophy?
- Yes. Clin2 currently lists 133 recruiting 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 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 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.