Clinical trial NCT06712771: A Phase 3 Clinical Trial to Evaluate the Efficacy and Safety of VSA003 in Chinese HoFH Patients
Official title: A Phase 3 Clinical Trial to Evaluate the Efficacy and Safety of VSA003 Injection in Chinese Adolescents and Adults with Homozygous Familial Hypercholesterolaemia (HoFH)
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE3
Conditions: Homozygous Familial Hypercholesterolemia (HoFH)
Lead sponsor: Visirna Therapeutics HK Limited
Sponsor class: INDUSTRY
First posted: 2024-12-02
Enrollment: 45
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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 criteria: 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 outcomes:
measure: Percent change from baseline in serum LDL-C at month 6
timeFrame: At month 6
Secondary outcomes:
measure: Percent change and absolute change in serum ANGPTL3 levels from baseline at month 6
timeFrame: At month 6
measure: Percent change and absolute change in serum LDL-C, ApoB, non-HDL-C, VLDL-C, TC and TG levels from baseline
timeFrame: Up to month 6, Up to month 18
measure: Percent change and absolute change in serum HDL-C, ApoC-III, ApoC-II, ApoA-I, ApoA-V and Lp[a] levels from baseline
timeFrame: Up to month 6, Up to month 18
measure: Percentage of patients achieving ≥30% reduction in serum LDL-C from baseline
timeFrame: At month 6, At month 18
measure: Percentage of patients achieving ≥50% reduction in serum LDL-C from baseline
timeFrame: At month 6, At month 18
measure: Number of Participants with Adverse Events
timeFrame: Up to month 6, Up to month 18
measure: Number of Participants with Anti-Drug Antibodies (ADAs) to VSA003 Over Time
timeFrame: Up to month 6, Up to month 18
measure: Correlation between VSA003 serum concentrations and QT intervals
timeFrame: Up to month 6, Up to month 18
Other outcomes:
Locations:
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