Clinical trials
Myopathy clinical trials
Below are recruiting myopathy clinical trials, each written for real people, not researchers. We’re tracking 123 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT06374719Recruiting
TNNT1 myopathy natural history study
This study follows people with a rare genetic muscle condition called TNNT1 myopathy over time. Researchers will track how the disease progresses to better understand it and help plan future treatments.
Gordonville, PennsylvaniaAges Any age - NCT04064307Recruiting
Register people with myotubular or centronuclear myopathy
This study sets up a patient registry for people diagnosed with myotubular myopathy or centronuclear myopathy. Your information can help researchers better understand these conditions and plan future studies or treatments.
Newcastle upon Tyne, Tyne and WearAges Any age - NCT06154252RecruitingPhase 2/Phase 3
Study of CABA-201 for active myositis in adults and children
This trial tests a new treatment called CABA-201, which uses your own immune cells modified to attack the overactive cells causing myositis. It is for people with active disease that hasn't improved enough with standard therapies.
Orange, CaliforniaAges 6–75 - NCT00272883Recruiting
Study genes in congenital muscle weakness
This research study looks at genes and molecules to better understand congenital myopathies (birth-related muscle weakness). It may help explain the cause of symptoms and improve future diagnoses for you and your family.
Boston, MassachusettsAges Any age - NCT07502989Recruiting
Study of Muscle Health Using Electrical Impedance Myography
This study uses a simple electrical test (EIM) and an MRI scan to check muscle health in people with muscle diseases. It may help researchers learn how to measure muscle changes without a biopsy.
Boston, MassachusettsAges 18–89 - NCT05979441Enrolling by invitationPhase 3
Long-term safety of efgartigimod injection for myositis
This study tests the long-term safety and effectiveness of a subcutaneous (under-the-skin) version of efgartigimod for adults with active idiopathic inflammatory myopathy. It is for people who have already completed an earlier study of this medicine.
Phoenix, ArizonaAges 18 years+ - NCT03749538Recruiting
Brain stimulation test for autoimmune muscle weakness
This trial tests whether a noninvasive brain stimulation technique (through the scalp) can improve muscle weakness in people with certain autoimmune muscle diseases. It may help by changing brain signals that affect how muscles work.
São PauloAges 18–80 - NCT04792931Recruiting
Study for adults with suspected autoimmune muscle disease
This study looks at adults who may have an autoimmune (self-immune) muscle disease. It aims to better understand and evaluate this condition, which may help guide future diagnosis and care.
BrestAges 18 years+ - NCT06157268Recruiting
Study of muscle tiredness in congenital myopathies
This study follows patients with congenital myopathies to understand how their muscles get tired over time. It may help develop better treatments.
Nijmegen, GelderlandAges 2 years+ - NCT01403402Recruiting
Study of congenital muscle disease patients and their families
This trial collects health information from patients with certain inherited (genetic) muscle diseases and from their families. It may help researchers better understand the disease and improve care by learning what symptoms and treatments patients experience over time.
Lakewood, CaliforniaAges Any age - NCT01353430Recruiting
Study of a rare muscle condition linked to Paget bone disease
This study looks at people and families with a rare muscle weakness condition that can be linked with Paget disease of the bone (and sometimes frontotemporal dementia). It aims to better describe how these conditions appear and relate, which can help future research and care.
Irvine, CaliforniaAges 18 years+ - NCT01793168Recruiting
Rare disease registry and history study
This study keeps a registry (a structured list) of people with rare diseases and may also track how the condition changes over time. It can help researchers better understand rare illnesses and find gaps in care, which may improve future treatments.
Sioux Falls, South DakotaAges Any age - NCT02398786Recruiting
Join a family registry for myotonic dystrophy (DM1 or DM2)
This study builds a registry of people and families affected by myotonic dystrophy types 1 and 2. It helps researchers learn about the condition over time and may support future studies that could lead to better care.
Oakland, CaliforniaAges Any age - NCT07478172Recruiting
Electrical muscle stimulation exercise for neuromuscular disease
This study tests whether whole-body electrical muscle stimulation (using small electrodes on the skin) can help adults with certain neuromuscular diseases exercise and improve muscle strength. It may be an option if you can stand for about 15 minutes at a time and have some muscle strength left.
Columbia, MissouriAges 18 years+ - NCT07488806Recruiting
Understanding Nemaline Myopathy Over Time (Spain)
This study follows people with nemaline myopathy over time to better understand how the disease progresses. It helps researchers learn more about the condition and plan future treatments.
BarcelonaAges Any age - NCT07052929RecruitingPhase 1/Phase 2
Gene therapy study for young boys with XLMTM on ventilators
This study tests a new gene therapy called ASP2957 in young boys with X-linked myotubular myopathy who are dependent on a ventilator. The goal is to see if it can help improve their breathing and muscle strength.
Chicago, IllinoisAges Up to 3 years - NCT07560020RecruitingPhase 2
Surlorian for RYR1 muscle weakness: strength and safety study
This trial tests whether a new drug called Surlorian can improve muscle strength in adults with a rare genetic muscle condition (RYR1-related myopathy). You'd receive either the drug or a placebo and be monitored for safety and changes in muscle function.
Marseille, Bouches-du-RhôneAges 18–65 - NCT07412821Enrolling by invitationPhase 1
Study of ASA-001 for ADSS1 deficiency muscle disease
This trial tests a drug called ASA-001 (adenylosuccinic acid) for people with a rare genetic muscle disease caused by ADSS1 deficiency. The goal is to see if it is safe and helps muscle function.
Los Angeles, CaliforniaAges 18 years+ - NCT05653544Recruiting
Study tracks symptoms in primary mitochondrial muscle conditions
This study observes people with primary mitochondrial muscle diseases over time to better understand how symptoms change. It may help improve future care by learning patterns of muscle weakness, fatigue, and related complications.
Madrid, MadridAges 16 years+ - NCT06574919Recruiting
Study of inherited muscle diseases in children
This study looks at children with inherited muscle diseases (not Duchenne muscular dystrophy) to understand their symptoms and muscle function better. It may help doctors identify what type of muscle disease a child has and how it affects them day-to-day.
SohagAges 2–18 - NCT06599697RecruitingPhase 2
IVIG for anti-HMGCR muscle disease
This trial tests if IVIG (intravenous immunoglobulin, a donated blood product that helps the immune system) can improve muscle strength and reduce damage in people with anti-HMGCR immune-mediated necrotizing myopathy (a rare muscle disease where the immune system attacks muscle cells). It may help you if your muscle weakness is not too severe and you haven't tried IVIG before.
Birmingham, AlabamaAges 16 years+ - NCT06132750Recruiting
Long-term study of LAMA2 and SELENON muscle conditions
This study follows people with LAMA2-related muscular dystrophy or SELENON-related myopathy over 5 years to understand how the condition changes over time. It may help you by tracking your disease progression and contributing to future research.
Nijmegen, GelderlandAges 1 day–100 years - NCT07715136RecruitingPhase 1
BCMA-CD19 CAR-T for Refractory Inflammatory Myopathy
This trial tests a new type of cell therapy (BCMA-CD19 cCAR-T) for people with inflammatory myopathy that hasn't improved with standard treatments. The goal is to see if this treatment can help control the disease.
Chengdu, SichuanAges 18–60 - NCT03092180Recruiting
Tests a treatment approach for certain muscle inflammation diseases
This trial looks at treatment strategies for people with idiopathic inflammatory myopathies, which are illnesses where the immune system attacks muscles. It may help improve how treatment is chosen and optimized for this group of conditions, but it specifically excludes several related muscle diseases.
São PauloAges 18 years+
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Common questions
- Are there clinical trials for myopathy?
- Yes. Clin2 currently lists 123 recruiting myopathy 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 myopathy 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 myopathy 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.