Early access treatment for Pompe disease
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have been diagnosed with Pompe disease, based on a lab test showing low GAA enzyme activity or a genetic test finding.
- You are not eligible for any other Amicus-sponsored clinical trial, or you are declining currently approved enzyme replacement therapy (like Myozyme).
- You are willing to receive the study treatment and share your health data with the study team and sponsor.
- You are not planning to have a child during the treatment program (for men or women).
- You have not had gene therapy at any time.
- You are not taking certain diabetes or other medicines like miglitol, acarbose, or voglibose.
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 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 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 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, 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 study collects safety information about avalglucosidase alfa when it’s taken during pregnancy and/or while breastfeeding. If you (or your baby) were exposed to this medicine and consent to be in a pregnancy safety registry, your information may help researchers better understand risks.
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.
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