Study of ENTR-601-44 for Duchenne muscular dystrophy (exon 44 skipping)
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This trial tests a new medicine called ENTR-601-44 for people with Duchenne muscular dystrophy whose genetic mutation can be treated by skipping a part of the gene called exon 44. The goal is to see if it is safe and helps with muscle function.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed Duchenne muscular dystrophy diagnosis and a specific genetic change that allows for exon 44 skipping.
- You must be male and have symptoms of Duchenne muscular dystrophy.
- For Part A, you must be between 4 and 20 years old.
- You must be able to walk and have enough arm and hand function as checked by the study team.
- You must have enough muscle for a biopsy sample.
- You cannot have used certain medicines like other exon skipping drugs, gene therapy, blood thinners, immunosuppressants (except steroids), or histone deacetylase inhibitors like givinostat.
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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