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New drug aims to slash Sky-High cholesterol in rare genetic disease

NCT ID NCT06712771

Disease control Sponsor: Visirna Therapeutics HK Limited Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This phase 3 trial tests VSA003, an RNAi drug that targets a protein called ANGPTL3, in 46 Chinese adolescents and adults with homozygous familial hypercholesterolemia (HoFH), a genetic condition causing extremely high cholesterol. Participants receive either VSA003 or a placebo injection, and researchers measure how much their LDL cholesterol drops after 6 months. The goal is to see if this drug can safely lower cholesterol and reduce heart disease risk in this hard-to-treat population.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
VSA003 (also called AROANG3 or Zodasiran), an RNAi drug targeting ANGPTL3
What this could lead to
If successful, this could provide a new treatment option to lower dangerously high cholesterol in people with HoFH, potentially reducing their risk of heart disease.
What could go wrong
This is a small, early-phase 3 trial with only 46 participants, so results may not apply to everyone. The drug is still experimental, and its long-term safety and effectiveness are not yet proven.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

46 people

The number who actually took part.

Started

Nov 2024

Expected to finish

Dec 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

homozygous familial hypercholesterolemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Homozygous familial hypercholesterolemia (HOFH)

Contacts and locations

Locations

  • Peking Union Medical College Hospital

    Beijing, China

More trials for these conditions

Other studies related to the condition(s) this trial covers.