Homozygous Familial Hypercholesterolemia (HoFH)Phase 3NCT06712771
A Phase 3 Clinical Trial to Evaluate the Efficacy and Safety of VSA003 in Chinese HoFH Patients
A Phase 3 study testing VSA003 in people with Homozygous Familial Hypercholesterolemia (HoFH), run by Visirna Therapeutics HK Limited at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Homozygous Familial Hypercholesterolemia (HoFH)
- What is being tested?
- VSA003
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 46 participants
- Where
- 1 siteChina
- Study dates
- 27 Nov 2024to 30 Dec 2027
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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;
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- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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)
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.
- Being tested
- VSA003, Placebo
- Medical subject headings
- Homozygous Familial Hypercholesterolemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VSA003Drug
Also called AROANG3, Zodasiran
subcutaneous injections
PlaceboDrug
subcutaneous injections
| Study group | Role | What they receive |
|---|---|---|
| VSA003 | Experimental | subcutaneous injections Receives: Drug: VSA003 |
| Placebo | Placebo comparator | subcutaneous injections Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent change from baseline in serum LDL-C at month 6
Measured: At month 6
Secondary outcomes (8)
Percent change and absolute change in serum ANGPTL3 levels from baseline at month 6
Measured: At month 6
Percent change and absolute change in serum LDL-C, ApoB, non-HDL-C, VLDL-C, TC and TG levels from baseline
Measured: 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
Measured: Up to month 6, Up to month 18
Percentage of patients achieving ≥30% reduction in serum LDL-C from baseline
Measured: At month 6, At month 18
Percentage of patients achieving ≥50% reduction in serum LDL-C from baseline
Measured: At month 6, At month 18
Number of Participants with Adverse Events
Measured: Up to month 6, Up to month 18
Number of Participants with Anti-Drug Antibodies (ADAs) to VSA003 Over Time
Measured: Up to month 6, Up to month 18
Correlation between VSA003 serum concentrations and QT intervals
Measured: Up to month 6, Up to month 18
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Dec 2024
- Study start27 Nov 2024
- Primary completion31 Dec 2025
- Study completion30 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Homozygous Familial Hypercholesterolemia (HoFH)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 46 participants (actual)
Sponsor and investigators
- Lead sponsor
- Visirna Therapeutics HK Limited
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06712771
- Sponsor's protocol ID
- VSA003-3001
- Status last verified
- Apr 2026
- First submitted
- 26 Nov 2024
- Last updated
- 5 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT06712771
- Acronym / sponsor ID
- VSA003-3001
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 46 participants (actual)
- Lead sponsor
- Visirna Therapeutics HK LimitedIndustry (pharma, biotech, devices)
- Study start
- 27 Nov 2024 (actual)
- Primary completion
- 31 Dec 2025 (actual)
- Study completion
- 30 Dec 2027 (estimated)
- First posted
- 2 Dec 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| VSA003 | Experimental | Drug: VSA003subcutaneous injections |
| Placebo | Placebo comparator | Drug: Placebosubcutaneous injections |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (11 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VSA003Also: AROANG3, Zodasiransubcutaneous injections | Drug | VSA003 |
| Placebosubcutaneous injections | Drug | Placebo |
Primary outcome
Percent change from baseline in serum LDL-C at month 6
Time frame: At month 6
8 secondary outcomes
Percent change and absolute change in serum ANGPTL3 levels from baseline at month 6
Time frame: At month 6
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
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
Percentage of patients achieving ≥30% reduction in serum LDL-C from baseline
Time frame: At month 6, At month 18
Percentage of patients achieving ≥50% reduction in serum LDL-C from baseline
Time frame: At month 6, At month 18
Number of Participants with Adverse Events
Time frame: Up to month 6, Up to month 18
Number of Participants with Anti-Drug Antibodies (ADAs) to VSA003 Over Time
Time frame: Up to month 6, Up to month 18
Correlation between VSA003 serum concentrations and QT intervals
Time frame: Up to month 6, Up to month 18
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 May 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing VSA003 in people with Homozygous Familial Hypercholesterolemia (HoFH), run by Visirna Therapeutics HK Limited at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 5 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent change from baseline in serum LDL-C at month 6 (time frame: At month 6). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 May 2026.