Testing a New DMD Treatment Given Every 4 Weeks
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This study tests whether a new medicine called zeleciment rostudirsen can help ambulatory (walking) males with Duchenne muscular dystrophy (DMD). The medicine is given as an intravenous infusion (through a vein) every 4 weeks and works by helping the body produce a protective muscle protein.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are a male with DMD caused by a specific type of gene mutation (exon deletion amenable to exon 51 skipping)
- You can rise from the floor in less than 10 seconds—this will be tested twice during screening
- You have been taking steroids (glucocorticoids) at a stable dose for at least 24 weeks and plan to keep taking the same dose during the study
- You have not received other muscle-affecting medicines (besides steroids) in the past 12 weeks
- You have not had gene therapy at any time, and have not received other exon-skipping or DMD-modifying treatments in the past 24 weeks
- You have not had recent changes to heart failure treatment (if applicable) in the past 12 weeks
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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