Clinical trials · California
Congenital Myotonic Dystrophy clinical trials in California
We’re tracking 6 recruiting congenital myotonic dystrophy studies with a site in California — including Phase 1/Phase 2, Phase 2/Phase 3 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies in California
- 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+ - 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.
Los Angeles, CaliforniaAges 6–45 - 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 - 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 - 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
Where these studies are running in California
Institutions with a site for the recruiting congenital myotonic dystrophy studies listed above.
- University of California, Los Angeles (UCLA) · 3 studies
- Altman Clinical and Translational Research Institute
- Congenital Muscle Disease International Registry (www.cmdir.org)
- Myotonic Dystrophy Foundation
- Stanford University
- UCLA Medical Center
What taking part in a congenital myotonic dystrophy study involves
A screening visit first
Before anything else, the study team checks whether you fit — usually a visit with some tests. You can stop at any point, and screening is typically free.
Care at a nearby site
Study visits happen at a clinic or hospital taking part. Many studies cover the cost of the study treatment and related visits, and some reimburse travel.
You stay in control
Taking part is voluntary and you can leave a study at any time, for any reason, without affecting your regular care.
The team decides eligibility
Our fit check is a helpful first read, not a decision. The study team makes the final call after reviewing your health history.
Congenital Myotonic Dystrophy trials in other states
Common questions
- Are there congenital myotonic dystrophy clinical trials in California?
- Yes. We're currently tracking 6 recruiting congenital myotonic dystrophy studies with a site in California, each rewritten in plain language so you can see what it's testing and who it's for.
- How do I find out if I qualify for a congenital myotonic dystrophy study in California?
- Each study lists its eligibility rules — age, diagnosis, prior treatments. On every trial page we explain these in plain language and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Do I have to live in California to take part?
- Not necessarily. These studies have a site in California, but eligibility is about your health, not your address — some people travel to take part, and a few studies reimburse travel. The study team can tell you what's required.
- Does it cost anything to join a congenital myotonic dystrophy trial?
- Using Clin2 is always free. Many trials cover the study treatment and related visits; some reimburse travel. The study team explains exactly what's covered before you decide.
Not the right time?
New congenital myotonic dystrophy studies open in California regularly. Set up a health profile and we’ll quietly watch for studies that fit you and email you when one opens.
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.