Safety study of a gene therapy for LGMD2I/R9
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This trial tests the safety of AB-1003 (a gene therapy) in adults with a confirmed genetic form of LGMD2I/R9 caused by an FKRP gene mutation. It may help researchers learn whether the treatment is safe and how it affects the body in people with this condition.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You’re 18 to 65 years old and have LGMD2I/R9 confirmed by an FKRP gene mutation test
- You can walk or run 10 meters in under 30 seconds
- You can understand the study and follow all study visits and procedures
- If you can become pregnant: you must not be pregnant, and you must use reliable birth control (and follow partner contraception rules) for 6 months after dosing
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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