Active, not recruiting
Phase 3
A Phase 3 Clinical Trial to Evaluate the Efficacy and Safety of VSA003 in Chinese HoFH Patients
Condition(s) studied
Homozygous Familial Hypercholesterolemia (HoFH)
Investigational drug(s) / intervention(s)
VSA003: subcutaneous injections
Placebo: subcutaneous injections
Study summary
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
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 |
|
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