Aprepitant for CLIFAHDD syndrome
Part of Brain & nervous system, Mental health clinical trials.
This trial tests a drug called aprepitant to see if it can help people with CLIFAHDD, a rare genetic condition that affects movement and development. The study will check if the drug is safe and if it improves symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of CLIFAHDD with a specific genetic change (NALCN variant) that increases gene activity.
- You must be older than 6 months and weigh more than 6 kilograms (about 13 pounds).
- You must be able to travel to San Antonio, Texas for study visits and follow the treatment plan.
- Your organ function (like blood counts, kidney, and liver) must be within certain safe limits, as shown by lab tests.
- If you are a woman of childbearing age, you must use highly effective birth control or not be sexually active.
- You must not be pregnant or have certain medical conditions or take medicines that could interact with the study drug.
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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