A Phase 3 Clinical Trial to Evaluate the Efficacy and Safety of VSA003 in Chinese HoFH Patients

Phase 3

Not yet recruiting

• Conditions: Homozygous Familial Hypercholesterolemia (HoFH)
• Interventions: Drug: VSA003; Drug: Placebo

• Registration Number: NCT06712771
• Lead Sponsor: Visirna Therapeutics HK Limited

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.

Detailed Description

Not available

Study Timeline

• Status Verified: 2024-11
• Study First Submitted: 2024-11-26
• Study First Submitted QC: 2024-11-26
• Last Update Submitted: 2024-11-26
• Study Start: 2024-12-02
• Study First Posted: 2024-12-02
• Last Update Posted: 2024-12-02
• Primary Completion: 2026-06-30
• Study Completion: 2027-12-30

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 45

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
VSA003	VSA003	subcutaneous injections
Placebo	Placebo	subcutaneous injections

Primary Outcome Measures

Name	Time	Method
Percent change from baseline in serum LDL-C at month 6	At month 6

Secondary Outcome Measures

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