Testing a new add-on treatment for late-onset Pompe disease
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are 18 years or older and weigh at least 88 pounds (40 kg)
- You have been on enzyme replacement therapy (ERT) for at least 2 years, with no changes in the last 6 months
- Your breathing function (FVC) is between 30% and 80% of normal when sitting up, or you have a significant drop in breathing when lying down
- You can walk at least 75 meters (about the length of a city block) in 6 minutes
- You have not had gene therapy or other experimental Pompe treatments recently
- You are not pregnant, breastfeeding, or planning to conceive 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.
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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 study is a long-term follow-up for people who previously finished a trial of the experimental medication S-606001 for late-onset Pompe disease. It tests how safe and effective the drug is over a longer period.
This trial provides access to an investigational combination therapy for Pompe disease when standard treatments are not suitable. It aims to help people with Pompe disease who cannot join other clinical studies or are no longer benefiting from current enzyme replacement therapy.
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 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.
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.
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