Safety and effectiveness of ELEVIDYS for Duchenne muscular dystrophy
Treatments studied
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This study evaluates how safe and effective ELEVIDYS (a gene therapy) is when used in real-world clinical practice for boys with Duchenne muscular dystrophy. The trial includes two groups: boys starting ELEVIDYS for the first time, and boys who have already received it commercially.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Be male at birth
- For Cohort 1 (new patients): be ambulatory (able to walk) and at least 4 years old; for Cohort 2: have already received ELEVIDYS in a commercial setting
- Not have HIV, hepatitis B, or hepatitis C infection
- Not have a recent infection (within 4 weeks) such as a respiratory infection or pneumonia
- For Cohort 1: agree to use condom protection and have female partners use birth control; be able to take sirolimus (a medication) without allergies or contraindications
- Have a parent or legal guardian (if under 18) able to give informed consent and follow study visits, or be 18+ and able to consent independently
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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