Exosome treatment to deliver LDL cholesterol gene instructions
Part of Genetic & congenital, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 18 and 45 years old.
- You have homozygous familial hypercholesterolemia confirmed by genetic testing.
- You can understand the study and sign informed consent.
- You must not have recent heart/stroke events or unstable heart/lung problems that needed hospitalization.
- Your recent blood, liver, kidney, and heart test results must be within study limits.
- You must not be pregnant or breastfeeding, and you must use reliable birth control during and for 3 months after the study.
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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