Clinical trials
Myotonic Dystrophy clinical trials
Below are recruiting myotonic dystrophy clinical trials, each written for real people, not researchers. We’re tracking 42 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT07008469Enrolling by invitationPhase 3
Long-term safety study of del-desiran for DM1
This study tests whether the drug del-desiran continues to be safe and effective for people with DM1 (myotonic dystrophy type 1) after they complete an earlier study. You'll receive the drug and have regular check-ups to see how you're doing.
Los Angeles, CaliforniaAges 16 years+ - NCT07486934RecruitingPhase 3
Study of DYNE-101 for Myotonic Dystrophy Type 1
This trial tests an experimental drug called DYNE-101 to see if it can help people with myotonic dystrophy type 1 (DM1). The study aims to improve muscle function and daily life. It is for adults who can walk and stand up from a chair without help.
La Jolla, CaliforniaAges 16 years+ - NCT06138743RecruitingPhase 1/Phase 2
Investigational treatment for adult-onset myotonic dystrophy type 1
This trial is testing a new drug called SRP-1003 for people with myotonic dystrophy type 1 (DM1) who developed symptoms after age 12. The goal is to see if it can reduce muscle problems like myotonia (difficulty relaxing muscles) and improve daily function.
Liverpool, New South WalesAges 18–65 - NCT06747884Recruiting
Study for children with myotonic dystrophy
This study looks at how to best measure muscle health and function in children with myotonic dystrophy. It may help prepare for future treatment trials.
Little Rock, ArkansasAges 3–17 - NCT07700225Recruiting
Long-term study of myotonic dystrophy type 1
This study is a long-term follow-up for people with myotonic dystrophy type 1. It aims to find better ways to measure how the disease progresses, which could help develop future treatments.
Richmond, VirginiaAges 18–70 - NCT04003363Recruiting
Myotonic dystrophy registry to help track your condition
This is a national registry that collects information from people who have (or may soon have) myotonic dystrophy. By joining, you help researchers better understand the disease and may make future studies easier to access.
Newcastle upon TyneAges Any age - NCT05854433Enrolling by invitation
Study brain structure in adults with muscle weakness
This study uses scans and other tests to understand how brain structure relates to symptoms in people with myotonic dystrophy type 1 or type 2. It may help researchers connect measurable brain changes to movement and other clinical outcomes.
Winston-Salem, North CarolinaAges 30–65 - NCT05020002Recruiting
Checking RNA markers in blood and muscle in muscle disease
This study looks for tiny RNA “signals” in blood (and sometimes urine) and compares them with muscle samples in people with myotonic dystrophy (DM1 or DM2) and healthy controls. The goal is to find biomarkers that may help track the disease over time.
Boston, MassachusettsAges 5 years+ - NCT06075693Recruiting
Spinal fluid markers for myotonic dystrophy
This study looks for markers in spinal fluid that could help doctors better understand myotonic dystrophy type 1 (DM1). It involves a spinal tap and MRI scan. It may help people with DM1 and healthy volunteers learn more about the condition.
Boston, MassachusettsAges 18 years+ - NCT06316778Recruiting
Pelvic floor training for women with myotonic dystrophy
This study tests whether pelvic floor muscle training (exercises to strengthen the muscles that control urine flow) can help women with myotonic dystrophy type 1 who have bladder leakage. It's a gentle, non-surgical approach that may improve quality of life.
Jonquière, QuebecAges 18 years+ - NCT07362875Recruiting
Muscle imaging study for myotonic dystrophy
This study uses MRI scans to measure muscle changes in people with myotonic dystrophy (DM1 or DM2) and healthy volunteers. The goal is to find better ways to track the disease over time.
Winston-Salem, North CarolinaAges 18–65 - NCT07385443Recruiting
Spanish registry for myotonic dystrophy type 1
This registry collects information from people with myotonic dystrophy type 1 (DM1) to better understand the condition and improve care. Anyone with a confirmed genetic diagnosis can join.
Multiple Locations, AndalusiaAges Any age - 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 - 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 - NCT07505342Recruiting
Remote study for myotonic dystrophy type 1
This study tests whether remote assessments can track myotonic dystrophy type 1 (DM1) and looks at genetic factors. It aims to find better ways to monitor the disease from home.
Rochester, New YorkAges 18–88 - NCT07630389Recruiting
Remote monitoring for childhood myotonic dystrophy
This study uses video calls and online assessments to track how myotonic dystrophy (a muscle-weakening condition) affects children over time. Researchers will also look at genetic factors to better understand the disease and improve care.
Rochester, New YorkAges birth–17 years - NCT07072676Enrolling by invitation
Can a walker help prevent falls in neuromuscular disease?
This study tests if using assistive walking devices, after a short training period, can lower the risk of falls in people with neuromuscular diseases. It aims to help you stay steady and safe on your feet.
Bad FeilnbachAges 65–65 - NCT06926621Enrolling by invitationPhase 2
Long-term safety and effectiveness of VX-670 for myotonic dystrophy
This study is for people who already received VX-670 in a previous trial and want to continue treatment. It will check the drug's long-term safety and how well it works for myotonic dystrophy type 1.
St Louis, MissouriAges 18 years+ - NCT05004129RecruitingPhase 2/Phase 3
Tideglusib for genetic childhood myotonic dystrophy
This trial studies how safe and effective tideglusib is for children and young adults with congenital or childhood-onset myotonic dystrophy type 1 (DM1). It may help researchers learn whether the medicine improves symptoms while tracking side effects closely.
Little Rock, ArkansasAges 6–45 - NCT06667453RecruitingPhase 2
PGN-EDODM1 for myotonic dystrophy type 1
This study tests an experimental drug called PGN-EDODM1 for people with myotonic dystrophy type 1 (DM1). The goal is to see if it can help reduce symptoms like muscle stiffness (myotonia).
Calgary, AlbertaAges 16–65 - NCT05481879RecruitingPhase 1/Phase 2
Study medicine for DM1 to improve muscle function
This early-stage study tests DYNE-101 to see how safe it is and whether it can improve measurable muscle function in people with myotonic dystrophy type 1 (DM1). You may be asked to do several walking and strength tests and undergo heart and breathing screening measurements.
Stanford, CaliforniaAges 18–65 - NCT06844214RecruitingPhase 1/Phase 2
Gene therapy trial for myotonic dystrophy type 1
This trial tests a one-time gene therapy (SAR446268) for people with non-congenital myotonic dystrophy type 1. It aims to see if the treatment is safe and can improve muscle symptoms like stiffness and weakness.
Gainesville, FloridaAges 10–55 - NCT06979024Enrolling by invitation
Observational study of myotonic dystrophy type 1
This study follows people with myotonic dystrophy type 1 over time to learn more about the condition. It is for people of any age who have genetic confirmation of DM1, whether or not they have symptoms.
Fuzhou, FujianAges Any age - 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+
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Myotonic Dystrophy trials by city
Studies with a site in or near these metro areas.
Myotonic Dystrophy trials by state
Studies with a site anywhere in these states.
Common questions
- Are there clinical trials for myotonic dystrophy?
- Yes. Clin2 currently lists 42 recruiting myotonic 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 myotonic 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 myotonic 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.