Gene therapy trial for Duchenne muscular dystrophy in young boys
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This trial tests a gene therapy called GNR-097 for boys with Duchenne muscular dystrophy (DMD). The therapy aims to help improve muscle function, and the study will check how safe and effective it is.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Boys aged 4 to 9 who can walk and have a confirmed DMD diagnosis
- A specific type of DNA change (frameshift or nonsense mutation) in the DMD gene
- Must be taking a stable dose of oral steroids for at least 12 weeks
- Meningitis vaccine received at least 4 weeks before the study treatment
- Able to complete standard physical tests like standing up quickly and walking a certain distance
- No prior gene therapy or certain other medications
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 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.
This trial tests a new gene therapy called SGT-003 for children with Duchenne muscular dystrophy. It aims to help the body produce a working version of dystrophin, the protein missing in Duchenne.
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 study follows boys with Duchenne muscular dystrophy over time to better understand how the condition changes and how standard treatments affect health and function. It does not appear to test a new medicine, but it may help doctors plan better future care and studies.
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 trial tests a new drug (NS-089/NCNP-02) that aims to help boys with Duchenne muscular dystrophy produce a shorter but still working version of the dystrophin protein. It's for boys who are still walking and have a specific genetic type that can be fixed by skipping a part of the gene called exon 44.
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.