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Active, not recruitingNCT06712771Updated May 5, 2026

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

A Phase 3 interventional study of VSA003 and Placebo in Homozygous Familial Hypercholesterolemia (HoFH), sponsored by Visirna Therapeutics HK Limited. Active, not recruiting at 1 site in China. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2026-05-05.

Sponsored by Visirna Therapeutics HK Limited · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
46
Allocation
Randomized
Ages
12 Years and older
Sex
All
01

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.

02

Conditions studied

  • Homozygous Familial Hypercholesterolemia (HoFH)
03

In context

Homozygous Familial Hypercholesterolemia

70 studies on the registry are indexed under Homozygous Familial Hypercholesterolemia; 12 are open to participants now.

This study's enrollment of 46 is above the median of 20 across 51 interventional studies indexed under Homozygous Familial Hypercholesterolemia.

Browse Homozygous Familial Hypercholesterolemia studies →

Lead sponsor

Visirna Therapeutics HK Limited is the lead sponsor of 5 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
12 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

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

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.
05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
46 participants (actual)

Study arms

  • Experimental
    VSA003

    subcutaneous injections

    Drug: VSA003

  • Placebo comparator
    Placebo

    subcutaneous injections

    Drug: Placebo

Interventions

  • DrugVSA003

    subcutaneous injections

    Also known as: AROANG3, Zodasiran

  • DrugPlacebo

    subcutaneous injections

06

What researchers measure

Primary outcomes

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

    Time frame: At month 6

Secondary outcomes

  1. Percent change and absolute change in serum ANGPTL3 levels from baseline at month 6

    Time frame: At month 6

  2. Percent change and absolute change in serum LDL-C, ApoB, non-HDL-C, VLDL-C, TC and TG levels from baseline

    Time frame: Up to month 6, Up to month 18

  3. Percent change and absolute change in serum HDL-C, ApoC-III, ApoC-II, ApoA-I, ApoA-V and Lp[a] levels from baseline

    Time frame: Up to month 6, Up to month 18

  4. Percentage of patients achieving ≥30% reduction in serum LDL-C from baseline

    Time frame: At month 6, At month 18

  5. Percentage of patients achieving ≥50% reduction in serum LDL-C from baseline

    Time frame: At month 6, At month 18

  6. Number of Participants with Adverse Events

    Time frame: Up to month 6, Up to month 18

  7. Number of Participants with Anti-Drug Antibodies (ADAs) to VSA003 Over Time

    Time frame: Up to month 6, Up to month 18

  8. Correlation between VSA003 serum concentrations and QT intervals

    Time frame: Up to month 6, Up to month 18

07

Study locations

1 site
  • Peking Union Medical College Hospital
    Beijing, China
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on May 5, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT06712771
Lead sponsor
Visirna Therapeutics HK Limited
Responsible party
Sponsor
First posted
Dec 2, 2024
Start date
Nov 27, 2024
Primary completion
Dec 31, 2025
Completion
Dec 30, 2027 (estimated)
Last update
May 5, 2026

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is active, not recruiting, as verified in Apr 2026. You cannot join it, but the record below documents what was studied.

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Discussion

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