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 new gene-editing treatment designed to help lower harmful cholesterol levels. The medication works by targeting a specific gene in your liver that controls cholesterol production. Researchers want to understand whether this treatment is safe and how well it works for people with inherited high cholesterol or those who have had heart problems at an early age.
Many people with high cholesterol or early heart disease don't reach their cholesterol goals even with current medications. This medication uses new gene-editing technology to potentially offer a different approach to lowering cholesterol by addressing the problem at its source in the liver.
You likely qualify if…
You likely don't qualify if…
You will receive a single injection or infusion of the medication during the trial. The study is structured in groups, with each group receiving a different dose to test safety as the dose increases. You will have follow-up visits and blood tests to monitor how the medication affects your cholesterol levels and to check for any side effects.
AI-generated summary from trial data · Jul 1, 2026 · Not medical advice
United States