Clinical trials · California
Muscular Dystrophy clinical trials in California
We’re tracking 25 recruiting muscular dystrophy studies with a site in California — including Phase 1, Phase 1/Phase 2, Phase 2, Phase 2/Phase 3 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies in California
- NCT05230459RecruitingPhase 1/Phase 2
Safety study of a gene therapy for LGMD2I/R9
This trial tests the safety of AB-1003 (a gene therapy) in adults with a confirmed genetic form of LGMD2I/R9 caused by an FKRP gene mutation. It may help researchers learn whether the treatment is safe and how it affects the body in people with this condition.
Irvine, CaliforniaAges 18–65 - NCT07038200RecruitingPhase 3
Study of Del-brax (AOC 1020) for People with FSHD
This study tests an experimental medicine called Del-brax (AOC 1020) for people with facioscapulohumeral muscular dystrophy (FSHD). The goal is to see if it can help improve muscle strength and slow the disease. You may be able to join if you have a confirmed FSHD diagnosis and can walk at least 10 meters on your own.
Orange, CaliforniaAges 16–70 - 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 - NCT07674758Recruiting
Understanding heart risk in Duchenne and Becker muscular dystrophy
This study looks at heart health in people with Duchenne or Becker muscular dystrophy to find better ways to predict and track heart problems. The goal is to improve future treatment trials.
Sacramento, CaliforniaAges Any age - NCT06270719Enrolling by invitation
Comparing ELEVIDYS to standard care for Duchenne muscular dystrophy
This study compares the gene therapy ELEVIDYS (delandistrogene moxeparvovec) to usual treatments for people with Duchenne muscular dystrophy (DMD). It helps researchers understand how well ELEVIDYS works over time in real-world settings.
Los Angeles, CaliforniaAges 4 years+ - NCT06066580Enrolling by invitationPhase 2
Long-term sevasemten study for Becker muscular dystrophy
This trial offers continued treatment with sevasemten (EDG-5506) for people with Becker muscular dystrophy who already completed a previous study of this drug. It tests the long-term safety and effects of taking sevasemten over more time.
La Jolla, CaliforniaAges Any age - NCT06817382RecruitingPhase 1
Gene therapy for young boys with Duchenne muscular dystrophy
This study tests a one-time gene therapy given by injection into the spinal fluid (intrathecal) for boys with Duchenne muscular dystrophy. It aims to see if the treatment is safe and how it spreads in the body, and may offer a new way to slow the disease.
Davis, CaliforniaAges 2–4 - NCT07609394Recruiting
Duchenne and Becker Muscular Dystrophy Health Records Study
This study collects electronic health records (medical information already in your doctor's computer system) from people with Duchenne or Becker muscular dystrophy, or female carriers of these conditions. Researchers will use this data to better understand the diseases and improve care over time.
Sacramento, CaliforniaAges Any age - NCT06564974Recruiting
Long-term safety study of Agamree for Duchenne muscular dystrophy
This study watches boys with Duchenne muscular dystrophy who are already taking Agamree (vamorolone) to see how safe it is over a long period. It helps doctors understand the medicine's effects over time.
Loma Linda, CaliforniaAges 2 years+ - NCT06138639RecruitingPhase 1/Phase 2
Gene therapy SGT-003 for Duchenne muscular dystrophy
This trial tests a new gene therapy called SGT-003 for children with Duchenne muscular dystrophy. It aims to help the body produce a working version of dystrophin, the protein missing in Duchenne.
Los Angeles, CaliforniaAges birth–17 years - NCT07086521RecruitingPhase 1
Stem cell therapy for facioscapulohumeral muscular dystrophy
This trial tests a new stem cell treatment (ULSC) for people with FSHD, a type of muscular dystrophy. It aims to see if the treatment is safe and if it can help improve arm strength and daily movement.
Palo Alto, CaliforniaAges 15 years+ - NCT07287189RecruitingPhase 2
SAT-3247 for boys with Duchenne muscular dystrophy
This trial tests a new daily pill called SAT-3247 for boys with Duchenne muscular dystrophy (DMD) who are still able to walk. The goal is to see if it can help slow muscle decline.
Los Angeles, CaliforniaAges 7–9 - NCT06503367Recruiting
Observation study for children 0-5 with LAMA2 muscular dystrophy
This study follows young children with LAMA2-related congenital muscular dystrophy to learn more about the disease. It does not test a new treatment, so it may help families understand the condition better and prepare for future trials.
Los Angeles, CaliforniaAges Up to 5 years - NCT04626674RecruitingPhase 1
Gene therapy safety study for non-ambulatory Duchenne
This Phase 1 trial studies a one-time gene therapy treatment (SRP-9001) to see if it is safe and whether it can produce the needed gene activity in people with Duchenne muscular dystrophy (DMD) who are not walking. It may help researchers understand whether this approach could be useful for DMD patients, especially in later stages.
Sacramento, CaliforniaAges 2 years+ - NCT03373968RecruitingPhase 2/Phase 3
Long-term study of givinostat safety in Duchenne muscular dystrophy
This trial looks at how safely givinostat works over a longer time in people with Duchenne muscular dystrophy (DMD). It mainly checks long-term side effects and tolerability, especially in a specific subgroup of muscle fat levels.
Sacramento, CaliforniaAges 7 years+ - NCT06839469Recruiting
Walking study for rare muscle and nerve conditions
This study looks at how children and teens with Duchenne muscular dystrophy (DMD) or spinal muscular atrophy (SMA) walk, by having them do walking tests while wearing sensors. The goal is to find better ways to track how these diseases progress and how treatments work over time, without needing blood tests or scans.
Palo Alto, CaliforniaAges 5 years+ - NCT03698149Recruiting
Brain-signal training for hand and speech control after disability
This study tests whether recording brain signals can help people regain better control of movements in the arms and help with speech. It may be an option for people with significant disability from certain nerve and brain conditions who can safely take part in brain-monitoring procedures.
San Francisco, CaliforniaAges 21 years+ - NCT05724173Recruiting
Test of a brain-to-computer device for ALS hand paralysis
This study checks whether a brain-computer interface (a BrainGate2 system) is safe and workable for people with ALS. If you qualify, you may be asked to try the device and complete study tasks to help researchers learn how well it can support communication or control.
Stanford, CaliforniaAges 18–80 - NCT06094205Recruiting
BrainGate2 speech study for people with tetraplegia
This study tests a brain-computer interface to help people with severe paralysis communicate by decoding attempted speech or movements. It may offer a new way to interact with devices using only brain signals.
Sacramento, CaliforniaAges 18–80 - NCT06539169Recruiting
Following people with rare diseases over time
This study follows people with rare diseases over time to learn more about how these conditions progress and how they are treated. Joining may help researchers understand your disease better and find better ways to care for others.
Los Altos, CaliforniaAges Any age - NCT06847282Recruiting
Motor skills study for children with FSHD
This study looks at how children with FSHD move and function over time. It uses walking tests and optional MRI scans to understand muscle changes. Your child's participation could help researchers learn more about FSHD progression in kids.
Palo Alto, CaliforniaAges 5–17 - NCT00912041Recruiting
Test of a brain-to-computer device for quadriplegia
This study tests whether a tiny brain computer interface can help people with quadriplegia (loss of movement in all four limbs) communicate or control technology. It’s mainly a feasibility study, meaning the goal is to see if the device can work safely and reliably in real life.
Stanford, CaliforniaAges 18–80 - NCT06606340Enrolling by invitation
Long-term study of eteplirsen, golodirsen, or casimersen for DMD
This study follows people with Duchenne muscular dystrophy (DMD) who are taking eteplirsen, golodirsen, or casimersen. It aims to understand how these medicines work in everyday life over a long period.
Sacramento, CaliforniaAges Any age - NCT06491927Enrolling by invitation
Long-term follow-up for RGX-202 in Duchenne muscular dystrophy
This long-term follow-up study watches people who previously received the experimental gene therapy RGX-202 for Duchenne muscular dystrophy. It checks how patients are doing over time and does not involve any new treatment.
Palo Alto, CaliforniaAges Any age
Muscular Dystrophy trials by city in California
Where these studies are running in California
Institutions with a site for the recruiting muscular dystrophy studies listed above.
- Stanford University · 9 studies
- University of California, Davis Medical Center · 9 studies
- University of California, Los Angeles (UCLA) · 4 studies
- Children's Hospital Los Angeles · 3 studies
- Stanford Neuroscience Health Center · 2 studies
- University of California, Irvine Medical Center / UC Irvine Health · 2 studies
- Congenital Muscle Disease International Registry (www.cmdir.org)
- Loma Linda University Pediatric Clinics
What taking part in a muscular 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.
Muscular Dystrophy trials in other states
Common questions
- Are there muscular dystrophy clinical trials in California?
- Yes. We're currently tracking 25 recruiting muscular 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 muscular 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 muscular 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 muscular 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.