Phase 3
N=37
A Phase 3 Study of ARO-APOC3 / VSA001 / SAR449124 (Plozasiran) in Chinese Adults With Familial Chylomicronemia Syndrome
Familial Chylomicronemia Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT05902598 ↗Enrolled (actual)
37
Serious AEs
13.5%
Results posted
Feb 2026
Primary outcome: Primary: Percent Change From Baseline in Fasting Serum Triglyceride (TG) at Month 10 — -85.7; -89.5; 38.8 Percent change — p=0.0022
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Plozasiran (Drug); Placebon (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Visirna Therapeutics HK Limited
- Primary completion
- Nov 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change From Baseline in Fasting Serum Triglyceride (TG) at Month 10 |
-85.7; -89.5; 38.8 | 0.0022 sig |
| SECONDARY Percent Change From Baseline in Fasting Serum TG at Months 10 and 12 (Averaged) |
-82.1; -86.6; 49.5 | 0.0055 sig |
| SECONDARY Percent Change From Baseline in Fasting Serum Apolipoprotein C3 (APOC3) at Month 10 |
-92.55; -91.83; 19.91 | 0.0008 sig |
| SECONDARY Percent Change From Baseline in Fasting Serum APOC3 at Month 12 |
-83.98; -88.16; 22.56 | 0.0016 sig |
| SECONDARY Percent Change From Baseline in Fasting Serum Non-high Density Lipoprotein Cholesterol (Non-HDL-C) and High Density Lipoprotein Cholesterol (HDL-C) at Month 10 |
-37.76; -17.83; 40.34; 71.62; 86.84; -3.51 | — |
| SECONDARY Percent Change From Baseline in Fasting Serum TG, Non-HDL-C, and HDL-C at Month 12 |
-71.56; -79.22; 79.41; -7.21; -29.22; 40.67 | — |
| SECONDARY Percentage of Participants Achieving Fasting Serum TG of <500 mg/dL at Month 10 |
90.0; 81.8; 9.1 | — |
| SECONDARY Percentage of Participants Achieving Fasting Serum TG of <500 mg/dL at Month 12 |
60.0; 72.7; 18.2 | — |
| SECONDARY Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 |
-12.716; -13.610; 0.568; -12.029; -10.892; -3.079 | — |
| SECONDARY Percent Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 |
-87.17; -91.71; 3.86; -87.31; -81.86; -30.22 | — |
| SECONDARY Number of Participants With Positively Adjudicated Events of Acute Pancreatitis |
0; 0; 1 | — |
Summary
This is a randomized, double-blinded, placebo controlled, two periods phase 3 clinical study. The primary objective of the study was to evaluate the efficacy and safety of Plozasiran injection in Chinese adults with familial chylomicronemia syndrome (FCS). A total of 37 participants were enrolled in the study. The duration of the study randomized period was approximately 112 weeks, including a screening period of up to 8 weeks and a treatment period of up to 104 weeks. Participants who completed the randomized period will continue in a 1-year open-label extension period where all participants will receive Plozasiran.
Eligibility Criteria
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:
- Newly diagnosed within 12 weeks of screening
- 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.
Data sourced from ClinicalTrials.gov (NCT05902598). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.