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

NCT06712771 | Active Not Recruiting Phase 3

• 1 other identifier: VSA003-3001
• Study Type: interventional
• Target: 46
• Locations: 1 country
• Sites: 1

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.

Conditions

Homozygous Familial Hypercholesterolemia (HoFH)

Outcome Measures

Primary Outcomes (1)

• Percent change from baseline in serum LDL-C at month 6

  At month 6

Secondary Outcomes (8)

• 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

Study Arms (2)

VSA003

EXPERIMENTAL

subcutaneous injections

Drug: VSA003

Placebo

PLACEBO COMPARATOR

subcutaneous injections

Drug: Placebo

Interventions

VSA003 DRUG

subcutaneous injections

Also known as: AROANG3, Zodasiran

VSA003

Placebo DRUG

subcutaneous injections

Placebo

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Visirna Therapeutics HK Limited: lead

Study Sites (1)

Peking Union Medical College Hospital

Beijing, China

Location

MeSH Terms

Conditions

Homozygous Familial Hypercholesterolemia

Interventions

zodasiran

Condition Hierarchy (Ancestors)

Hyperlipoproteinemia Type II; Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hyperlipoproteinemias; Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 26, 2024
• First Posted: December 2, 2024
• Study Start: November 27, 2024
• Primary Completion: December 31, 2025
• Study Completion (Estimated): December 30, 2027
• Last Updated: May 5, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)