Clinical trials
Myotonic Dystrophy 1 clinical trials
Below are recruiting myotonic dystrophy 1 clinical trials, each written for real people, not researchers. We’re tracking 35 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- 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 - 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 - 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 - 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 - NCT07136844Recruiting
Walking and arm movement study for nerve and muscle conditions
This study uses motion analysis to understand walking and arm function in adults with certain neurological or metabolic diseases. The goal is to find patterns that help doctors better manage these conditions.
LiègeAges 18 years+ - NCT06300307RecruitingPhase 1/Phase 2
Study of ATX-01 for Myotonic Dystrophy Type 1
This trial tests an experimental drug called ATX-01 for people with myotonic dystrophy type 1 (DM1), a condition that causes muscle weakness and stiffness. The study aims to see if ATX-01 can help reduce symptoms like muscle stiffness (myotonia) and improve daily function.
Los Angeles, CaliforniaAges 18–64 - NCT07587242RecruitingPhase 3
Testing a New Therapy for DMD with Exon 44 Mutations
This study tests whether a new medicine called AOC 1044 (delpacibart zotadirsen) can help boys with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation. The medicine works by helping the body skip a damaged part of the gene to make a functional protein.
Westmead, New South WalesAges 7–16 - 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 - NCT05224778Recruiting
Study for young children with neonatal myotonic dystrophy type 1
This study looks at measurements and child-focused outcomes in babies and toddlers with myotonic dystrophy type 1 (DM1) that started in the newborn period. It may help researchers better track how the condition affects children and evaluate care plans more accurately.
Los Angeles, CaliforniaAges Up to 4.9 years - NCT04369209Recruiting
Study of people with FSHD1 gene changes
This is a study that follows people who have a confirmed FSHD1 genetic change (and some healthy people for comparison). It may help researchers better understand the condition and how it affects people over time.
Fuzhou, FujianAges Any age - 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+
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Common questions
- Are there clinical trials for myotonic dystrophy 1?
- Yes. Clin2 currently lists 35 recruiting myotonic dystrophy 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 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 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.