Gene therapy study for young boys with XLMTM on ventilators
Treatments studied
Part of Bones, joints & muscles, Brain & nervous system clinical trials.
This study tests a new gene therapy called ASP2957 in young boys with X-linked myotubular myopathy who are dependent on a ventilator. The goal is to see if it can help improve their breathing and muscle strength.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must be 36 months old or younger at the time of treatment.
- Your child must need a ventilator (breathing machine) for at least 20 hours a day and have a tracheostomy tube.
- Your child must have a confirmed genetic diagnosis of X-linked myotubular myopathy (XLMTM).
- Your child must be up to date on recommended vaccinations (or have a medical reason for missing any).
- Your child must not have certain infections (like active hepatitis, HIV, or COVID-19) or serious liver problems.
- Your child must not have received other gene therapy or certain immune-suppressing medications 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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