Clinical trials
Congenital Myotonic Dystrophy clinical trials
Below are recruiting congenital myotonic dystrophy clinical trials, each written for real people, not researchers. We’re tracking 18 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- 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 - 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 - 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 - NCT05982119Recruiting
Assessing muscle disease patients with activity monitors
This study assesses physical activity in people with different muscle diseases (like Duchenne, FSHD, and others) and in healthy volunteers. It helps doctors understand how these conditions affect daily movement and function.
LiègeAges 1–80 - 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 - 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 - 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 - 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 - 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 - 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.
LeuvenAges 7–16 - 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 - 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 - 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 - 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+ - 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
Hear when a new Congenital Myotonic Dystrophy trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.
Congenital Myotonic Dystrophy trials by city
Studies with a site in or near these metro areas.
Congenital Myotonic Dystrophy trials by state
Studies with a site anywhere in these states.
Common questions
- Are there clinical trials for congenital myotonic dystrophy?
- Yes. Clin2 currently lists 18 recruiting congenital 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 congenital 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 congenital 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.