Tested new drug for Gaucher disease affecting blood and bones
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This early-phase study tests PR001 in adults with Gaucher disease that affects organs and symptoms in the body. If you’re already on enzyme treatment, the trial checks whether this new treatment can safely help you, while doctors closely monitor side effects and organ changes.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You’re 18 years or older when you sign consent.
- Your genetic test shows Gaucher (GBA1) gene changes on both copies of the gene.
- You’ve been on enzyme-based therapy (ERT or SRT) for at least 2 years, and your dose hasn’t changed for at least 3 months.
- You can sign consent and agree to follow trial rules (including study-specific restrictions).
- If you can become pregnant, you must use highly effective birth control during the study and follow-up.
- You must agree not to donate blood, tissue, or organs during the study and follow-up.
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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