New drug aims to slash Sky-High cholesterol in rare genetic disease
NCT ID NCT06712771
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 3 trial tests VSA003, an RNAi drug that targets a protein called ANGPTL3, in 46 Chinese adolescents and adults with homozygous familial hypercholesterolemia (HoFH), a genetic condition causing extremely high cholesterol. Participants receive either VSA003 or a placebo injection, and researchers measure how much their LDL cholesterol drops after 6 months. The goal is to see if this drug can safely lower cholesterol and reduce heart disease risk in this hard-to-treat population.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- VSA003 (also called AROANG3 or Zodasiran), an RNAi drug targeting ANGPTL3
- What this could lead to
- If successful, this could provide a new treatment option to lower dangerously high cholesterol in people with HoFH, potentially reducing their risk of heart disease.
- What could go wrong
- This is a small, early-phase 3 trial with only 46 participants, so results may not apply to everyone. The drug is still experimental, and its long-term safety and effectiveness are not yet proven.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Peking Union Medical College Hospital
Beijing, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- New drug shows promise for rare high cholesterol condition
- CRISPR therapy takes aim at stubborn high cholesterol in first human trial
- New study screens newborns for inherited high cholesterol
- New hope for rare cholesterol disease: can evinacumab shrink dangerous plaque?
- Heart attack prevention hope for rare cholesterol disorder