Long-term safety and benefit check-up for RP-L102 gene therapy
Part of Blood & lymphatic, Genetic & congenital, Hormones & metabolism clinical trials.
This is a follow-up study for patients who previously received RP-L102, an experimental gene therapy designed to treat Fanconi anemia by fixing a faulty gene. The study tracks how well the treatment continues to work and watches for any long-term side effects.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have been enrolled in one of the original RP-L102 parent studies (RP-L102-0418, RP-L102-0319, or RP-L102-0118)
- You must have received the RP-L102 infusion (the gene therapy treatment) in one of those parent studies
- You are able to attend scheduled study visits and follow the study plan
- You have signed a consent form (and assent form if applicable) agreeing to participate in this follow-up study
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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