Gene therapy trial for Gaucher disease type 1
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a gene therapy called FLT201 for adults with Gaucher disease type 1. It aims to provide a long-term treatment option for people who have been stable on enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least two years.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be 18 years or older.
- You must have been diagnosed with Gaucher disease type 1 (not type 2 or 3).
- Your hemoglobin and platelet counts must be stable at the start of the trial.
- You must have been receiving ERT or SRT regularly for at least 2 years without stopping.
- You must not have had a splenectomy (spleen removal), a bone marrow or organ transplant, or any prior gene therapy.
- You must not have antibodies against the virus used in the gene therapy (AAVS3).
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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