Gene therapy for inherited high cholesterol
Part of Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a one-time gene therapy to lower extremely high cholesterol in people with a rare inherited condition. It may help reduce heart attack risk in those who haven't responded to standard treatments.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 12 and 55 years old (first patient must be at least 18).
- You have a confirmed genetic diagnosis of homozygous familial hypercholesterolemia.
- Your LDL cholesterol is very high even with treatment, or you have cholesterol bumps on skin/tendons before age 18.
- You have not had gene therapy before.
- You are willing to follow a low-fat diet and maintain a stable exercise routine.
- You are not pregnant, breastfeeding, or planning pregnancy for at least a year after the treatment.
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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