Plain-English translation of NCT06164730 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Phase 1 — Testing in a small group (usually 20–80 people) to find a safe dose and watch for side effects.
This trial is testing , a brand-new medication that uses gene-editing technology to help lower cholesterol levels in the liver. The treatment is designed for people with inherited high cholesterol (familial hypercholesterolemia) or those who have had heart problems at a young age and still have high cholesterol despite taking other medications. Researchers want to understand whether this medication is safe and how well it works.
Some people have genetic conditions that make their cholesterol dangerously high, and current medications don't lower it enough. This medication uses a novel approach to address the root genetic cause, offering hope for patients who need stronger cholesterol control to prevent heart disease.
You likely qualify if…
You likely don't qualify if…
You will receive a single injection of the medication during a visit at the research clinic. The trial uses a 'dose-escalation' approach, meaning the first participants receive a lower dose to check safety, and later groups receive higher doses if the lower doses are well-tolerated. After receiving the treatment, you'll have follow-up visits and blood tests to monitor your cholesterol levels and check for any side effects.
AI-generated summary from trial data · Sep 15, 2026 · Not medical advice
United States