Clinical trials
Myopathy clinical trials
Below are recruiting myopathy clinical trials, each written for real people, not researchers. We’re tracking 121 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT06754098RecruitingPhase 2
Testing two drugs for TK2 deficiency
This trial tests two experimental drugs, Doxecitin and Doxribthymine, for adults with a rare genetic condition called TK2 deficiency that causes muscle weakness and breathing problems. The goal is to see if these drugs can slow or stop the disease.
Madrid, MadridAges 18 years+ - NCT06733935RecruitingPhase 1/Phase 2
Study of NKX019 for immune diseases
This trial tests an investigational cell therapy called NKX019 for people with certain autoimmune diseases like scleroderma, myositis, vasculitis, or rheumatoid arthritis. It aims to see if NKX019 can help control the disease when standard treatments have not worked well enough.
Orange, CaliforniaAges 18–75 - NCT06723106Enrolling by invitationPhase 1
Long-term safety study of RAY121 for immune conditions
This study is for people who already took RAY121 in a previous trial and did well. It tests whether continuing the treatment is safe and effective over a longer period. The goal is to manage chronic immune conditions.
Camperdown, New South WalesAges 18–85 - NCT06698796RecruitingPhase 3
Study of dazukibart for ongoing idiopathic inflammatory myopathies
This trial studies the long-term effects of dazukibart in people who completed an earlier study for idiopathic inflammatory myopathies (a group of rare conditions causing muscle inflammation and weakness). It helps researchers understand how well the medicine works over a longer period.
Glendale, ArizonaAges 18 years+ - NCT06666816Recruiting
Watching changes in walking for muscle and nerve diseases
This study watches how people with certain muscle or nerve diseases walk over time. It may help doctors understand changes in walking patterns and is open to adults who can walk and have a genetic diagnosis of a neuromuscular condition.
Bosisio Parini, LeccoAges 18–75 - NCT06614270Recruiting
CAR-NK cell therapy for severe autoimmune diseases
This trial tests a new type of immune cell therapy using modified natural killer cells (CAR-NK) that target a protein called CD19 and produce helpful signals (IL-10 and IL-15). It is for people with certain autoimmune diseases that have not improved with standard treatments or have come back after getting better.
Hangzhou, ZhejiangAges 18–65 - NCT06581146Recruiting
Liver health study for boys with XLMTM
This study looks at how the liver works in boys with a serious muscle condition called XLMTM. It checks whether their liver is healthy, which may help doctors better understand the condition and guide future treatments.
Santa Monica, CaliforniaAges Up to 17 years - NCT06548607RecruitingEarly Phase 1
CAR-T therapy for autoimmune diseases
This early-phase trial tests a new CAR-T cell therapy that targets CD19 or both CD19 and BCMA to treat several autoimmune diseases like lupus, scleroderma, and vasculitis. It aims to calm the overactive immune system with a single infusion of your own engineered cells.
Bengbu, AnhuiAges 18–70 - NCT06502015Recruiting
Biomarkers in nervous system autoimmune diseases
This study looks for biomarkers (biological clues) in people with autoimmune diseases of the nervous system. It aims to discover better ways to diagnose and treat these conditions.
WuhanAges 18 years+ - NCT06479863RecruitingEarly Phase 1
Testing a new drug combo for inclusion body myositis
This trial tests a combination of two investigational drugs (pozelimab and cemdisiran) to see if they can help people with sporadic inclusion body myositis, a muscle disease that causes progressive weakness. If the drugs work, they may slow down the disease and help you stay stronger longer.
Austin, TexasAges 45–75 - NCT06455449RecruitingPhase 3
Injectable anifrolumab for polymyositis or dermatomyositis
This study tests a new injectable medicine (anifrolumab) added to your usual treatments for polymyositis or dermatomyositis. It may help reduce disease activity if you have moderate to severe symptoms and are already on stable therapy.
Phoenix, ArizonaAges 18–75 - NCT06375993Enrolling by invitationPhase 1
Prula-cel for autoimmune diseases: a phase 1 study
This trial tests a new treatment called prulacabtagene leucel (prula-cel), which is a type of immune cell therapy (CAR T-cells) for people with certain autoimmune diseases. It aims to see if it can reset the immune system and reduce disease activity in conditions like lupus, scleroderma, vasculitis, or myositis.
Redwood City, CaliforniaAges 18–80 - NCT06371417RecruitingPhase 1
Testing RAY121 for several immune diseases (RAINBOW trial)
This trial tests a new medicine called RAY121 (an antibody) for people with certain autoimmune diseases. It aims to see if RAY121 is safe and how the body handles it. If you have one of these conditions and meet the basic health requirements, you might be able to join.
Orange, CaliforniaAges 18–85 - NCT06287762Recruiting
Natural history study of RYR1-related disorders
This study follows people with RYR1-related disorders over time to learn how the condition progresses. It may help researchers plan future treatments.
Bethesda, MarylandAges 7–100 - NCT06285539RecruitingPhase 2
Testing a drug for rare inflammatory diseases
This trial is testing a new drug (a JAK inhibitor) for people with rare inflammatory diseases like Behçet's, myositis, or IgG4-related disease that hasn't improved with standard treatments. The goal is to see if it can help control symptoms and reduce inflammation.
AmsterdamAges 18–65 - NCT00082108Recruiting
Muscle Disease and Family Health Registry
This is a registry study that collects health information from people with myotonic dystrophy (a genetic muscle disease that causes weakness and stiffness) or facioscapulohumeral muscular dystrophy (a genetic condition affecting shoulder and face muscles), as well as their unaffected family members. The information helps researchers better understand these conditions and track how they progress over time.
Rochester, New YorkAges Any age - NCT06092346Recruiting
Understanding rare purine and pyrimidine metabolism disorders
This study aims to learn more about rare disorders of purine and pyrimidine metabolism (DPPM), which can affect the brain, immune system, kidneys, and muscles. Researchers will study people with these disorders, their family members, and healthy volunteers to better understand the condition over time.
Bethesda, MarylandAges 1 month–100 years - NCT05569122RecruitingPhase 1
Studying an MRI contrast method in mitochondrial muscle disease
This Phase 1 study tests a special MRI approach (using a contrast agent) to better measure changes in muscles in people with mitochondrial diseases. It also includes some healthy volunteers and other critically ill patients to compare results and check safety.
Philadelphia, PennsylvaniaAges 10–60 - NCT05895786RecruitingPhase 3
Study drug for active dermatomyositis or polymyositis
This Phase 3 trial tests how an investigational medicine (PF-06823859) works in adults with active dermatomyositis or polymyositis. It aims to improve disease control by targeting the immune system, but you must meet several safety and diagnosis requirements.
Glendale, ArizonaAges 18 years+ - NCT05859997Enrolling by invitation
CAR-T cell therapy for active autoimmune disease flare-ups
This study tests an immune-cell treatment (CAR-T, called BRL-301) in people with certain autoimmune diseases that are active and hard to control. It may help your immune system calm down if standard treatments have not worked or your disease has returned.
Shanghai, Shanghai MunicipalityAges 18–65 - NCT05554835Recruiting
Mitochondrial disease registry and history study
This study collects information about people who have a suspected or confirmed mitochondrial disease. It aims to better understand how the disease looks over time, which can help future treatments.
InnsbruckAges Any age - NCT05250375Recruiting
Study of mitochondrial muscle diseases over time
This natural history study follows children and adults with suspected or confirmed mitochondrial myopathy to better understand symptoms and how the condition changes. It may also include healthy participants to compare test results and improve future treatments.
Philadelphia, PennsylvaniaAges birth–100 years - NCT05211661Recruiting
Diaphragm health during breathing machine support
This study looks at how the diaphragm (the main breathing muscle) changes when a person is on a breathing machine in the intensive care unit. It may help doctors understand how to better support breathing and recovery.
AachenAges 18–75 - NCT04824040Enrolling by invitation
Study of immune, muscle, and genetic features in LGMD 2B
This study looks at immune function, muscle appearance, and genetic information in people with limb-girdle muscular dystrophy type 2B. It may help researchers understand the condition better, which can support future treatments.
MoscowAges 18–85
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Common questions
- Are there clinical trials for myopathy?
- Yes. Clin2 currently lists 121 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.