New RNAi drug shows promise for rare, severe high cholesterol
NCT ID NCT06712771
First seen May 13, 2026 · Last updated May 24, 2026 · Updated 6 times
Summary
This study tests an experimental RNAi drug called VSA003 in 46 Chinese adolescents and adults with homozygous familial hypercholesterolemia (HoFH), a rare genetic condition causing extremely high cholesterol and early heart disease. The drug aims to lower harmful cholesterol by blocking a protein called ANGPTL3. Participants must already be on a stable cholesterol-lowering diet and medication. The main goal is to see how much LDL cholesterol drops after 6 months.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Locations
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Peking Union Medical College Hospital
Beijing, China
Conditions
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