Study of SRP-1001 for adults and teenagers with FSHD1
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This study tests a new medicine called SRP-1001 to see if it can help people with a type of muscular dystrophy called FSHD1. It aims to slow down muscle weakness and improve quality of life.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of FSHD1, which a doctor can verify with a genetic test.
- Your muscle weakness (on a scale from 0 to 10) should be between 3 and 8, meaning moderate weakness.
- You must be 18 to 70 years old (or 16 and older with special approval) and in good overall health.
- You must have a leg muscle that can be safely biopsied, as determined by an MRI.
- You must be willing to use effective birth control during the study and for 9 months after, if applicable.
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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