Significant reduction in ANGPLT3 levels is expected to reduce plasma LDL-C and TG levels and may lead to the reduction of risk of coronary heart disease and cardiovascular events associated with hypertriglyceridemia and LDL-C in subjects with persistent dyslipidemia. In this clinical study, the efficacy and safety of VSA003, an RNAi-based therapeutic targeting ANGPTL3, will be evaluated in subjects with HoFH.
Eligibility
Sex
ALL
Min age
12 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
* Male or female aged ≥ 12 years and weighing ≥ 40 kg at the time of ICF signature;
* Patients with HoFH who meet genetic or clinical diagnosis;
* Willing to follow a daily low-fat diet for the duration of the study;
* Receiving stable and tolerable lipid-lowering therapy prior to LDL-C testing during the screening period;
* Fasting LDL-C ≥ 2.6 mmol/L.
Exclusion Criteria:
* Have received or are receiving targeted ANGPTL3 treatment within 5 months or 5 half-lives, whichever is longer, prior to screening;
* Fasting TG ≥ 4.5 mmol/L at screening;
* Presence of uncontrolled endocrine disease affecting lipids or lipoproteins;
* Weight change of more than 10% in the 4 weeks prior to randomisation;
* Initiation of a new dietary plan or significant differences from previous dietary structure and habits within 4 weeks prior to screening;
* Women who are pregnant (including planned pregnancies) or breastfeeding;
* Refusal to limit alcohol consumption to moderate limits and below during the study period, specifically no more than 14 units per week;
* Uncontrolled hypertension (blood pressure \> 160/100 mmHg at screening);
* New York Heart Association (NYHA) class IV heart failure or left ventricular ejection fraction \<30% within 12 months prior to screening;
* History of major surgery within 12 weeks prior to baseline, or planning to undergo major surgery during the study;
* Known allergy to any component of the study drug.
Primary outcome measure(s)
Percent change from baseline in serum LDL-C at month 6 — At month 6
Trial sites (1)
Facility
City
Region
Status
Peking Union Medical College Hospital
Beijing
China
More Visirna Therapeutics HK Limited trials in China
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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