New shot could slash Sky-High cholesterol in rare genetic disease

NCT ID NCT06712771

First seen May 13, 2026 · Last updated May 14, 2026 · Updated 2 times

Summary

This study tests a new medicine called VSA003 in 46 Chinese teenagers and adults with homozygous familial hypercholesterolemia (HoFH), a genetic condition causing extremely high cholesterol and early heart disease. VSA003 uses RNAi technology to lower a protein (ANGPTL3) that controls fat levels, aiming to reduce bad cholesterol and triglycerides. Participants must follow a low-fat diet and stable cholesterol meds; the main goal is to see how much LDL cholesterol drops after 6 months.

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Contacts and locations

Locations

  • Peking Union Medical College Hospital

    Beijing, China

Conditions

Explore the condition pages connected to this study.