Gene therapy trial for stubborn high cholesterol
Part of Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a new gene therapy (NGGT006) for people with a genetic form of high cholesterol that doesn't respond to standard treatments. It aims to lower cholesterol levels that remain high despite maximum medication.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 18 and 55 years old.
- You have a confirmed genetic diagnosis of familial hypercholesterolemia.
- Your cholesterol (LDL) is still high (≥70 or ≥100 mg/dL depending on your heart history) even after taking the highest tolerated doses of statins, ezetimibe, and a PCSK9 inhibitor.
- You have been on a stable healthy diet for at least 12 weeks.
- You are willing to use highly effective birth control for at least a year after the gene therapy dose.
- You must not have any active infections, liver problems, or a history of gene therapy.
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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This trial tests a new gene therapy called YOLT-101 for people with familial hypercholesterolemia, a genetic condition that causes very high cholesterol. The treatment aims to lower cholesterol levels and reduce heart risks.
This Phase 1 study tests a new treatment using tiny delivery particles (“exosomes”) to carry instructions (LDLR mRNA) that may help lower LDL (“bad”) cholesterol in people with homozygous familial hypercholesterolemia. The main goal is to see if it’s safe and learn how the treatment behaves in the body.
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