Comparing ELEVIDYS to standard care for Duchenne muscular dystrophy
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This study compares the gene therapy ELEVIDYS (delandistrogene moxeparvovec) to usual treatments for people with Duchenne muscular dystrophy (DMD). It helps researchers understand how well ELEVIDYS works over time in real-world settings.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed diagnosis of Duchenne muscular dystrophy (DMD) through genetic testing.
- You are currently taking or have been prescribed to start taking chronic steroids (like prednisone) for DMD.
- If you receive ELEVIDYS, you must be at least 4 years old at the time of the infusion.
- If you are part of the standard care group, you must be at least 4 years old and have never had gene therapy for DMD.
- You do not have a deletion of exon 8 and/or exon 9 in the DMD gene.
- You are not currently taking part in any other research study for DMD treatments.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This Phase 1 trial studies a one-time gene therapy treatment (SRP-9001) to see if it is safe and whether it can produce the needed gene activity in people with Duchenne muscular dystrophy (DMD) who are not walking. It may help researchers understand whether this approach could be useful for DMD patients, especially in later stages.
This study checks how people with Duchenne muscular dystrophy are doing long-term after receiving delandistrogene moxeparvovec (SRP-9001) in an earlier trial. It helps researchers understand the long-term safety and effects of that treatment.
This study follows people with Duchenne muscular dystrophy (DMD) who are taking eteplirsen, golodirsen, or casimersen. It aims to understand how these medicines work in everyday life over a long period.
This study tests a one-time gene therapy given by injection into the spinal fluid (intrathecal) for boys with Duchenne muscular dystrophy. It aims to see if the treatment is safe and how it spreads in the body, and may offer a new way to slow the disease.
This long-term follow-up study watches people who previously received the experimental gene therapy RGX-202 for Duchenne muscular dystrophy. It checks how patients are doing over time and does not involve any new treatment.
This trial tests a new gene therapy called SGT-003 in boys and men with Duchenne muscular dystrophy who are still able to walk. The treatment aims to deliver a working version of the dystrophin gene to help muscle cells produce the protein they are missing.
Hear when a new Duchenne Muscular Dystrophy trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.