Long-term safety study of S-606001 for late-onset pompe disease
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have already completed a study of the drug S-606001 for Pompe disease.
- You must weigh at least 40 kilograms (about 88 pounds).
- You cannot be taking part in any other research study right now.
- You cannot be pregnant or breastfeeding, and you cannot plan to have a child 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 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 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 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 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.
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.