Clinical trials
Pompe Disease (Late-onset) clinical trials
Below are recruiting pompe disease (late-onset) clinical trials, each written for real people, not researchers. We’re tracking 6 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT07282847RecruitingPhase 1/Phase 2
Gene therapy for adults with Pompe disease
This study tests a one-time gene therapy (AB-1009) for adults with late-onset Pompe disease. It aims to see if the treatment is safe and improves muscle and breathing function.
Phoenix, ArizonaAges 18–65 - NCT06391736RecruitingPhase 1/Phase 2
Gene therapy for late-onset Pompe disease
This trial tests a new gene therapy for late-onset Pompe disease. The therapy aims to help your body produce the enzyme needed to break down glycogen, which may improve muscle and breathing function.
BeijingAges 6 years+ - NCT07123155RecruitingPhase 2
Testing a new add-on treatment for late-onset Pompe disease
This trial tests a new medication, S-606001, given alongside your current enzyme replacement therapy (ERT) for late-onset Pompe disease. It may help improve your breathing and walking ability.
Irvine, CaliforniaAges 18 years+ - NCT00567073Recruiting
Register pregnant people with Pompe disease for pregnancy data
This study keeps a special record of pregnancies in people with Pompe disease to learn more about pregnancy outcomes and newborn information. It does not test a drug by itself—it collects health information to improve future care.
Phoenix, ArizonaAges Any age - NCT07478172Recruiting
Electrical muscle stimulation exercise for neuromuscular disease
This study tests whether whole-body electrical muscle stimulation (using small electrodes on the skin) can help adults with certain neuromuscular diseases exercise and improve muscle strength. It may be an option if you can stand for about 15 minutes at a time and have some muscle strength left.
Columbia, MissouriAges 18 years+ - NCT07354724RecruitingPhase 1
Study of DNL952 for adults with late-onset Pompe disease
This trial tests a new drug, DNL952, in adults with late-onset Pompe disease. It aims to see if the drug is safe and how it works in the body, with different groups for people currently on enzyme replacement therapy or those who haven't had it in the past year.
Irvine, CaliforniaAges 18–75
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Common questions
- Are there clinical trials for pompe disease (late-onset)?
- Yes. Clin2 currently lists 6 recruiting pompe disease (late-onset) 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 pompe disease (late-onset) 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 pompe disease (late-onset) 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.