Familial Chylomicronemia SyndromePhase 3NCT05902598
A Phase 3 Study of ARO-APOC3 / VSA001 / SAR449124 (Plozasiran) in Chinese Adults With Familial Chylomicronemia Syndrome
A Phase 3 study testing Plozasiran in people with Familial Chylomicronemia Syndrome, run by Visirna Therapeutics HK Limited at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Familial Chylomicronemia Syndrome
- What is being tested?
- Plozasiran
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 37 participants
- Where
- 1 siteChina
- Study dates
- 10 Jul 2023to 9 Jan 2026
- Phase
- Phase 3
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
China1 site
Huashan Hospital Affiliated to Fudan University
Shanghai, Shanghai Municipality
About this study
Official title: A Phase 3 Study to Evaluate the Efficacy and Safety of ARO-APOC3 / VSA001 / SAR449124 Injection in Chinese Adults With Familial Chylomicronemia Syndrome
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.
- Conditions
- Familial Chylomicronemia Syndrome
- Being tested
- Plozasiran, Placebon
- Medical subject headings
- Familial hyperchylomicronemia syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlozasiranDrug
Also called ARO-APOC3, SAR449124, VSA001
Subcutaneous injection
PlacebonDrug
Also called Normal saline
Subcutaneous injection
| Study group | Role | What they receive |
|---|---|---|
| Plozasiran 25 mg | Experimental | Plozasiran 25 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months Receives: Drug: Plozasiran |
| Plozasiran 50 mg | Experimental | Plozasiran 50 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months Receives: Drug: Plozasiran |
| Placebo | Placebo comparator | Matching placebo administered every 3 months for 12 months Receives: Drug: Placebon |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Change From Baseline in Fasting Serum Triglyceride (TG) at Month 10
Blood samples for lipid parameters were collected at the specified time points. Fasting serum TG at Month 10 was defined as geometric mean of 2 measurements taken during Month 10, or the other non-missing measurement if any one of the two measurements was missing during Month 10. Analysis was performed on the basis of the handling strategy for intercurrent events, missing fasting serum was imputed mainly based on the Pattern Mixture Models (PMM). Descriptive statistics were calculated based on non-imputed data.
Measured: Baseline to Month 10
Secondary outcomes (10)
Percent Change From Baseline in Fasting Serum TG at Months 10 and 12 (Averaged)
Blood samples for lipid parameters were collected at the specified time points. Fasting serum TG at Month 10 and Month 12 was defined as the geometric mean of the measurements in Month 10 and Month 12 or the non-missing measurement in the other month if measurement in any one of the two months was missing. Analysis was performed on the basis of the handling strategy for intercurrent events, missing fasting serum TG was imputed mainly based on the PMM. Descriptive statistics were calculated based on non-imputed data.
Measured: Baseline to Months 10 and 12 (averaged)
Percent Change From Baseline in Fasting Serum Apolipoprotein C3 (APOC3) at Month 10
Blood samples for lipid parameters were collected at the specified time points. Baseline of fasting serum APOC 3 was defined as the last non-missing value prior to or on the first dosing date. Analysis was performed on the basis of the handling strategy for intercurrent events, missing values were imputed using the PMM. Descriptive statistics were calculated based on non-imputed data.
Measured: Baseline to Month 10
Percent Change From Baseline in Fasting Serum APOC3 at Month 12
Blood samples for lipid parameters were collected at the specified time points. Baseline of fasting serum APOC 3 was defined as the last non-missing value prior to or on the first dosing date. Analysis was performed on the basis of the handling strategy for intercurrent events, missing values were imputed using the PMM. Descriptive statistics were calculated based on non-imputed data.
Measured: Baseline to Month 12
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
Blood samples for lipid parameters were collected at the specified time points. Baseline was defined as the last non-missing value prior to or on the first dosing date.
Measured: Baseline to Month 10
Percent Change From Baseline in Fasting Serum TG, Non-HDL-C, and HDL-C at Month 12
Blood samples for lipid parameters were collected at the specified time points. Non-HDL-C and HDL-C baseline was defined as the last non-missing value prior to or on the first dosing date.
Measured: Baseline to Month 12
Percentage of Participants Achieving Fasting Serum TG of <500 mg/dL at Month 10
Blood samples for lipid parameters were collected at the specified time points.
Measured: 10 Month
Percentage of Participants Achieving Fasting Serum TG of <500 mg/dL at Month 12
Blood samples for lipid parameters were collected at the specified time points.
Measured: 12 Month
Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12
Blood samples for lipid parameters were collected at the specified time points.
Measured: From baseline up to Month 12, at Months 1,2,3,4,5,6,7,8,9,10,11 and 12
Percent Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12
Blood samples for lipid parameters were collected at the specified time points.
Measured: From baseline up to Month 12, at Months 1,2,3,4,5,6,7,8,9,10,11 and 12
Number of Participants With Positively Adjudicated Events of Acute Pancreatitis
Any AEs and SAEs reported by the investigator during the study that were consistent with acute pancreatitis events were adjudicated by the blinded Data Safety Committee based on the Atlanta Classification for Acute Pancreatitis 2013 for fulfillment of any 2 of the following 3 criteria:
- Abdominal pain consistent with symptoms of acute pancreatitis (acute episodes of persistent, severe upper abdominal pain often radiating to the back)
- Serum lipase activity (or amylase activity) ≥3 × upper limit of normal (ULN)
- Characteristic findings of acute pancreatitis on contrast-enhanced computed tomography (CECT) or magnetic resonance imaging (MRI) or transabdominal ultrasonography
Measured: From first administration of study treatment (Day 1) through Month 12.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jun 2023
- Study start10 Jul 2023
- Primary completion26 Nov 2024
- Study completion9 Jan 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Familial Chylomicronemia SyndromeStudies 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
- 37 participants (actual)
Sponsor and investigators
- Lead sponsor
- Visirna Therapeutics HK Limited
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Arrowhead Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Dong YOU, MD., PhD., Study chair, Visirna Therapeutics HK Limited
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05902598
- Sponsor's protocol ID
- VSA001-3001
- Other IDs
- U1111-1330-5258
- Status last verified
- Mar 2025
- First submitted
- 31 May 2023
- Last updated
- 27 Feb 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org