A study of ENTR-601-45 for Duchenne MD (exon 45 skipping)
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This trial tests an experimental drug called ENTR-601-45 for people with Duchenne muscular dystrophy that can be treated with 'exon 45 skipping.' The goal is to see if it's safe and helps improve muscle function. It's for males between 4 and 20 who can still walk and have enough arm strength.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have Duchenne muscular dystrophy (DMD) caused by a genetic change that can be treated with 'exon 45 skipping' therapy, confirmed by a genetic counselor.
- You are male and have clear symptoms of DMD.
- You are between 4 and 20 years old (this applies to the first part of the study).
- You can still walk and have enough arm and hand movement to complete study tests.
- You haven’t taken any other exon-skipping treatment in the past 12 months, and never had gene therapy.
- You haven’t used blood thinners, certain immune-suppressing drugs, or an HDAC inhibitor (like givinostat) in the past 30 days.
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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