Long-term study of Pompe disease for monitoring
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study follows people with Pompe disease over time, even if they are already receiving treatment. It aims to learn more about the disease and how patients are doing over the long run.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of Pompe disease, which can be through low enzyme levels or genetic changes.
- You must not have had a severe allergic reaction to the treatment avalglucosidase alfa.
- You (or your parent or guardian) must agree to sign a consent form. If you are under 18, your parent or legal guardian must agree.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This study follows people with Pompe disease over a long period to learn how they do over time, especially those receiving enzyme replacement therapy. Your results could help doctors better understand expected outcomes and refine care.
This is a registry study that collects information over time from people with Pompe disease. It helps doctors and researchers better understand the condition and how it affects daily life, without testing any new treatments.
This study follows people with Pompe disease over time and checks their “CRIM status,” which is a lab result about a key protein. It may help researchers better understand the disease and how it progresses for different patient groups.
This trial tests an experimental medication called S-606001 for people with late-onset Pompe disease. The goal is to see if it can help improve breathing and walking ability. Participants must not have received enzyme replacement therapy for at least 3 months before starting.
This trial is a registry, meaning it collects health information from people with confirmed Pompe disease. It may help researchers better understand the disease and improve future treatments.
This study looks at how Pompe disease affects the brain and nervous system. You may be able to join if you have a confirmed diagnosis of infantile or late-onset Pompe disease and agree to participate.
Hear when a new Pompe Disease trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.