Sirolimus for Leigh Syndrome
Treatments studied
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests if sirolimus can help people with Leigh syndrome. It aims to see if the drug can improve neurological symptoms and is looking for participants aged 6 months to 55 years.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a genetically confirmed diagnosis of Leigh syndrome with developmental delays or abnormal neurological exam findings.
- You must be between 6 months and 55 years old and weigh at least 5 kg.
- You need to have adequate liver, kidney, and blood function, as determined by lab tests.
- You must not have serious heart problems, unstable medical conditions, or a history of allergic reaction to sirolimus.
- You must agree to use effective contraception if sexually active, and females must have negative pregnancy tests.
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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