Early access program for arimoclomol in patients with Niemann-Pick disease type C
Part of Blood & lymphatic, Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This program provides early access to an experimental drug called arimoclomol for people with Niemann-Pick disease type C (NPC), a rare genetic condition. Arimoclomol may help with neurological symptoms, and this program allows patients to try it before it is fully approved.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of NPC (type C1 or C2) and at least one neurological symptom.
- You must be at least 2 years old.
- You must be a permanent resident of the United States.
- If you are taking miglustat (Zavesca), you must have been on the target dose for the last 6 weeks.
- If you are a woman who could become pregnant, you must agree to use highly effective birth control and have a negative pregnancy test.
- If you are a man with a partner who could become pregnant, you must agree to use a condom plus your partner's birth control.
- If you have seizures, they must be well-controlled and your seizure medication must be stable for at least 1 month before starting.
- You must not have severe liver or kidney problems, be allergic to arimoclomol, be pregnant or breastfeeding, or be in another clinical trial.
- You or your parent/guardian must sign a consent form to participate.
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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