Study of an experimental drug for late-onset Pompe disease
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study tests an experimental drug (S-606001) for people with late-onset Pompe disease. It aims to see if the drug can improve breathing and walking ability. You cannot have had enzyme replacement therapy for at least 3 months before joining.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You weigh at least 40 kilograms (about 88 pounds).
- You have a confirmed diagnosis of late-onset Pompe disease with low GAA enzyme activity and a genetic test showing the condition.
- Your breathing test (FVC) is between 30% and 80% of normal, or if it's higher, you have a significant drop when lying down.
- You can walk at least 75 meters in 6 minutes, and that distance is no more than 90% of what's expected for a healthy adult your age.
- You have not had enzyme replacement therapy in the last 3 months and agree not to have it during the study.
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 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.
This study is a follow-up for people with late-onset Pompe disease who have already completed a previous study of the investigational drug S-606001. It aims to continue monitoring safety and how well the treatment works over time.
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 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.
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.
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