Pompe Disease (Late-onset) research hub
4 indexed studies · 1 currently recruiting
- active not recruitingPHASE1, PHASE2
Gene Transfer Study in Patients With Late Onset Pompe Disease
This study is looking into a new gene therapy called zocaglusagene nuzaparvovec (AT845) for adults with Late Onset Pompe Disease. Researchers want to find out if it's safe and if it helps improve the condition. It's an early-stage study, giving different doses to see which works best.
United States · United Kingdom - recruiting
Pompe Pregnancy Sub-Registry
This study looks at how pregnancy affects women with Pompe disease. Researchers want to understand if different treatments, or no treatment, impact the health of both mum and baby. It's for women already in the Pompe Registry.
United States · Australia · Belgium - active not recruitingPHASE1, PHASE2
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
This study is testing a new medicine called SPK-3006 for adults with late-onset Pompe disease. It aims to see if it's safe and helps improve the condition. Participants will receive one dose of the new medicine and will be carefully checked.
United States · Canada · Denmark - active not recruitingNA
A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease
This study looks at blood markers and antibody levels in people with late-onset Pompe disease. It helps researchers understand the disease better and how bodies react to treatments, even without new medicines given during the study.
United States · Australia · Brazil