Gene therapy for inherited high cholesterol
Part of Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a one-time gene therapy (NGGT006) to treat a severe form of inherited high cholesterol that doesn't respond well to standard medications. It aims to lower cholesterol and reduce the risk of heart disease.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Be between 12 and 55 years old (first patient must be 18 or older)
- Have a genetic diagnosis of homozygous familial hypercholesterolemia with two LDL receptor gene mutations
- Have very high LDL cholesterol (≥10 mmol/L if untreated, or ≥7 mmol/L if on treatment, with xanthomas before age 18)
- Be on stable cholesterol-lowering medications for at least 30 days (or 60 days for certain injectables) and agree not to change them during the trial
- Agree to follow a low-fat diet and stick to a consistent exercise routine
- For women able to have children: must use highly effective birth control for at least one year after treatment and not be pregnant or breastfeeding
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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