N-acetyl-leucine trial for Niemann-Pick disease type C
Part of Blood & lymphatic, Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This Phase 3 trial tests whether N-acetyl-leucine can improve symptoms in people with Niemann-Pick disease type C (NPC). If you qualify, you will be asked to take study medicine and complete symptom and movement tests over time.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Be at least 4 years old and have a confirmed genetic diagnosis of Niemann-Pick disease type C (NPC)
- Be able to do study movement/ability tests (balance and hand-peg test results must fit the trial ranges)
- Weigh at least 15 kg at screening
- If you’re using medicines for NPC: stay on the same treatment and dose for at least 42 days before the first visit, and share your medication list with the study team
- You can’t take certain medicines around the start: you must avoid prohibited drugs for at least 42 days before the first visit and during the study
- If you can become pregnant or have a partner who can become pregnant: use the trial’s birth-control rules and pregnancy is not allowed during the 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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