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

A Phase 3 Clinical Trial to Evaluate the Efficacy and Safety of VSA003 Injection in Chinese Adolescents and Adults With Homozygous Familial Hypercholesterolaemia (HoFH)

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Homozygous Familial Hypercholesterolemia (HoFH)
• Intervention / Treatment: Drug: Placebo; Drug: VSA003

• Study Type: Interventional
• Enrollment (Actual): 46
• Phase: Phase 3

Contacts and Locations

Study Locations

• China
  • Beijing, China
    • Peking Union Medical College Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: VSA003; subcutaneous injections	Drug: VSA003; subcutaneous injections; Other Names: AROANG3; Zodasiran
Placebo Comparator: Placebo; subcutaneous injections	Drug: Placebo; subcutaneous injections

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Percent change from baseline in serum LDL-C at month 6	At month 6

Secondary Outcome Measures

Outcome Measure	Time Frame
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

Collaborators and Investigators

• Sponsor: Visirna Therapeutics HK Limited

Study record dates

Study Major Dates

• Study Start (Actual): November 27, 2024
• Primary Completion (Actual): December 31, 2025
• Study Completion (Estimated): December 30, 2027

Study Registration Dates

• First Submitted: November 26, 2024
• First Submitted That Met QC Criteria: November 26, 2024
• First Posted (Actual): December 2, 2024

Study Record Updates

• Last Update Posted (Actual): May 5, 2026
• Last Update Submitted That Met QC Criteria: April 28, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Lipid Metabolism, Inborn Errors; Hyperlipoproteinemias; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases; Hyperlipoproteinemia Type II; Homozygous Familial Hypercholesterolemia; zodasiran
• Other Study ID Numbers: VSA003-3001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No