Clinical trials
Pompe Disease (Infantile-Onset) clinical trials
Below are recruiting pompe disease (infantile-onset) clinical trials, each written for real people, not researchers. We’re tracking 4 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT04532047RecruitingPhase 1
Fetal enzyme therapy trial for certain inherited storage disorders
This Phase 1 trial tests an enzyme replacement treatment given to a developing baby before birth for specific inherited “lysosomal storage” diseases. The goal is to see if the treatment is safe and can improve outcomes for babies diagnosed in pregnancy.
San Francisco, CaliforniaAges 18–50 - NCT05619900Recruiting
Register people with lysosomal storage diseases
This study is a registry that collects information from people diagnosed with lysosomal storage diseases. It helps researchers better understand these conditions and support future studies.
San Francisco, CaliforniaAges Up to 64 years - NCT06833489Recruiting
Using genetic testing to find answers for rare muscle diseases
This study looks at leftover muscle tissue from people who have had genetic testing for rare muscle diseases but didn't get a clear diagnosis. The goal is to use newer genetic analysis to find the cause of their disease.
MarseilleAges Any age - NCT07072676Enrolling by invitation
Can a walker help prevent falls in neuromuscular disease?
This study tests if using assistive walking devices, after a short training period, can lower the risk of falls in people with neuromuscular diseases. It aims to help you stay steady and safe on your feet.
Bad FeilnbachAges 65–65
Hear when a new Pompe Disease (Infantile-Onset) trial opens
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Common questions
- Are there clinical trials for pompe disease (infantile-onset)?
- Yes. Clin2 currently lists 4 recruiting pompe disease (infantile-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 (infantile-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 (infantile-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.