Clinical trials
Myotonic Dystrophy Type 1 clinical trials
Below are recruiting myotonic dystrophy type 1 clinical trials, each written for real people, not researchers. We’re tracking 28 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 - 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+ - 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 - 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 - NCT03981575Recruiting
Biomarker and health marker study in myotonic dystrophy type 1
This study measures physical and lab “health markers” in people with myotonic dystrophy type 1 (DM1) to better understand how the disease changes over time. Some participants may also have a muscle biopsy to study tissue differences.
La Jolla, CaliforniaAges 18–70 - 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 - 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 - NCT05532813RecruitingPhase 3
Metformin for adult myotonic dystrophy type 1 muscle weakness
This Phase 3 trial tests whether metformin can improve symptoms and safety in adults with genetically confirmed myotonic dystrophy type 1 (DM1). It may help reduce muscle problems, but you must meet specific walking, breathing, heart, and kidney requirements.
GarchesAges 18–70 - 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+ - NCT06523400RecruitingPhase 3
Mexiletine for myotonic dystrophy types 1 and 2
This trial tests a once-daily drug called mexiletine PR to see if it can help reduce muscle stiffness (myotonia) in people with myotonic dystrophy type 1 or 2. It may be a good option if you have a confirmed genetic diagnosis and are at least 16 years old.
LeuvenAges 16 years+ - NCT06708468Recruiting
Personalized training for rare neuromuscular disorders
This study tests a personalized exercise program for people with rare neuromuscular diseases like FSHD, DM1, or CMT to see if it improves their physical function and quality of life.
BergenAges 18–70 - NCT06716931Recruiting
Exercise study for myotonic dystrophy type 2
This study tests if a structured exercise program is safe and helpful for people with myotonic dystrophy type 2 (DM2). It aims to improve strength and daily function.
Boston, MassachusettsAges 18–70 - 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 - NCT07220603RecruitingPhase 2
Long-term safety study for people with myotonic dystrophy type 1
This is a follow-up study for people with myotonic dystrophy type 1 who already received the study drug PGN-EDODM1 in an earlier trial. It lets you continue treatment and helps researchers learn more about long-term safety and effects.
Calgary, AlbertaAges Any age - 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 - 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 - NCT07608432RecruitingPhase 3
Testing a New DMD Treatment Given Every 4 Weeks
This study tests whether a new medicine called zeleciment rostudirsen can help ambulatory (walking) males with Duchenne muscular dystrophy (DMD). The medicine is given as an intravenous infusion (through a vein) every 4 weeks and works by helping the body produce a protective muscle protein.
Hillsborough, North CarolinaAges 4–18 - 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 - 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 - NCT06549400Enrolling by invitationPhase 3
Study on long-term safety of mexiletine for myotonic dystrophy
This study tests if a daily capsule of mexiletine is safe and helpful over 26 weeks for people with myotonic dystrophy type 1 or 2. It's for people who finished a previous related study and want to continue treatment.
LeuvenAges 16 years+
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Myotonic Dystrophy Type 1 trials by city
Studies with a site in or near these metro areas.
Myotonic Dystrophy Type 1 trials by state
Studies with a site anywhere in these states.
Common questions
- Are there clinical trials for myotonic dystrophy type 1?
- Yes. Clin2 currently lists 28 recruiting myotonic dystrophy type 1 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 type 1 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 type 1 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.