Gene therapy for late-onset Pompe disease
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a new gene therapy for late-onset Pompe disease. The therapy aims to help your body produce the enzyme needed to break down glycogen, which may improve muscle and breathing function.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are 6 years old or older.
- You have been diagnosed with late-onset Pompe disease (LOPD).
- Your lung function test (FVC) is at least 30% of what is expected for your age and size.
- You can walk at least 40 meters in 6 minutes (with or without a walking aid).
- You have not taken part in a gene therapy trial before.
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 study tests a one-time gene therapy (AB-1009) for adults with late-onset Pompe disease. It aims to see if the treatment is safe and improves muscle and breathing function.
This trial tests if getting enzyme replacement therapy every 4 weeks instead of every 2 weeks is safe and effective for stable adults with late-onset Pompe disease. It may reduce the burden of frequent infusions.
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.
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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.
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