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New injection shows promise for rare High-Fat disease

NCT ID NCT05902598

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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 study tested a drug called Plozasiran in 37 Chinese adults with Familial Chylomicronemia Syndrome (FCS), a rare condition causing extremely high blood fats. The trial compared the drug to a placebo over about two years, measuring how much it lowered triglyceride levels. All participants who finished could then receive the drug in an open-label extension.

What this could mean

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

Active substance
Plozasiran (a drug injection that targets a protein to lower blood fats)
What this could lead to
If it works, this could provide a new treatment option to control severe high triglycerides and reduce risk of pancreatitis in people with Familial Chylomicronemia Syndrome.
What could go wrong
This is a small, early-phase trial with only 37 participants, so results may not apply to everyone. The drug may cause side effects or fail to show lasting benefit.

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

37 people

The number who actually took part.

Started

Jul 2023

Finished

Jan 2026

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

18 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: * Males or nonpregnant (who do not plan to become pregnant), nonlactating females ≥18 years of age * Fasting triglycerides (TG) ≥10 mmol/L (\~880 mg/dL) at screening, that is refractory to standard lipid lowering therapy (sample drawn after at least the minimum time on stable lipid-lowering regimen described in protocol). Two repeat tests are allowed to qualify. * A diagnosis of FCS * Willing to follow dietary counseling as per PI judgment based on local standard of care, consistent with an intake of ≤20 g of fat per day during the study * If on medications for management of type 2 diabetes, or other medications specified in protocol, the dosing regimen must be stable before collection of qualified lipid parameter at screening. * Participants with a medical history of clinical atherosclerotic cardiovascular disease (ASCVD) or those with elevated 10-year ASCVD risk (eg, ≥7.5% per American Heart Association / American College of Cardiology risk calculator) must be on appropriate lipid-lowering therapy as per local standard of care (ie, including moderate to high intensity statin, as indicated) prior to collection of qualifying TG levels. * Participants of childbearing potential must agree to use a highly effective form of contraception in addition to a male condom, during the study and for at least 24 weeks after the last dose of investigational product (IP). Women of childbearing potential on a hormonal contraceptive must be stable on the medication for ≥1 menstrual cycles prior to Day 1. Men must not donate sperm during the study and for at least 24 weeks after the last dose of IP. Exclusion Criteria: * Current use or use within the last 365 days from Day 1 of any hepatocyte-targeted siRNA or antisense oligonucleotide molecule * Diabetes mellitus with any of the following: 1. Newly diagnosed within 12 weeks of screening 2. HbA1c ≥9.0% at screening * Active pancreatitis within 12 weeks before Day 1 * History of acute coronary syndrome event within 24 weeks of Day 1 The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

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Conditions

The condition(s) this trial relates to.

familial chylomicronemia syndrome Familial hyperchylomicronemia syndrome

As listed by the trial registrant

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

Contacts and locations

Locations

  • Huashan Hospital Affiliated to Fudan University

    Shanghai, Shanghai Municipality, China

More trials for these conditions

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