New injection shows promise for rare High-Fat disease
NCT ID NCT05902598
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
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37 people
The number who actually took part.
- Started
-
Jul 2023
- Finished
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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
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Anyone
- Healthy volunteers
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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 Hide 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.
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.