Phase 3
N=66
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)
Familial Chylomicronemia Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT04568434 ↗Enrolled (actual)
66
Serious AEs
24.2%
Results posted
Mar 2025
Primary outcome: Primary: Percent Change From Baseline in Fasting TG at Month 6 — 11.52; -10.85; -31.99 percent change — p==0.0009
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Olezarsen (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Ionis Pharmaceuticals, Inc.
- Primary completion
- Jul 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change From Baseline in Fasting TG at Month 6 |
11.52; -10.85; -31.99 | =0.0009 sig |
| SECONDARY Percent Change From Baseline in Fasting TG at Month 12 |
20.89; -22.92; -38.50 | =0.0044 sig |
| SECONDARY Percent Change From Baseline in Fasting Apolipoprotein C-III (apoC-III) at Months 6 and 12 |
7.57; -57.91; -66.13; 17.08; -59.98; -64.20 | <0.0001 sig |
| SECONDARY Percentage of Participants With ≥ 40% Reduction in Fasting TG at Month 6 |
4.3; 33.3; 40.9 | — |
| SECONDARY Percent Change From Baseline in Fasting Apolipoprotein B-48 (apoB-48) at Months 6 and 12 |
24.50; -8.78; -59.46; -3.52; -36.41; -79.15 | =0.1860 |
| SECONDARY Percent Change From Baseline in Fasting Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) at Months 6 and 12 |
5.33; -12.35; -18.86; 12.01; -17.84; -27.69 | =0.0401 sig |
| SECONDARY Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years During the Treatment Period (Week 1 Through Week 53) in Participants With Prior History of Pancreatitis |
66.22; 6.73 | =0.0052 sig |
| SECONDARY Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years During the Treatment Period (Week 1 Through Week 53) |
36.31; 4.37 | =0.0144 sig |
| SECONDARY Adjudicated Acute Pancreatitis Mean Event Per 100 Participant-Years Rate During Week 13 Through Week 53 in Participants With Prior History of Pancreatitis |
57.89; 8.17 | =0.0174 sig |
| SECONDARY Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years During Week 13 to Week 53 |
33.43; 5.42 | =0.0314 sig |
| SECONDARY Percentage of Participants With ≥ 70% Reduction in Fasting TG at Month 6 |
0; 4.8; 9.1 | — |
| SECONDARY Percentage of Participants With Fasting TG ≤ 880 mg/dL at Month 6 |
0; 10.0; 14.3 | — |
| SECONDARY Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years During Treatment Period in Participants With ≥ 2 Events in 5 Years Prior to Enrollment |
118.59; 16.59 | =0.0137 sig |
| SECONDARY Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years From Week 13 to Week 53 in Participants With ≥ 2 Events in 5 Years Prior to Enrollment |
106.91; 21.36 | =0.0480 sig |
| SECONDARY Percentage of Participants With Fasting TG ≤ 500 mg/dL at Month 6 |
0; 0; 13.6 | — |
Summary
The purpose of the study was to evaluate the efficacy of olezarsen as compared to placebo on the percent change in fasting triglycerides (TG) from baseline.
Eligibility Criteria
Key Inclusion Criteria
- A diagnosis of genetically confirmed Familial Chylomicronemia Syndrome (type 1 Hyperlipoproteinemia)
- Fasting TG ≥ 880 mg/dL (10 millimoles per liter (mmol/L) at Screening
- History of pancreatitis. Patients without a documented history of pancreatitis are also eligible but their enrollment will be capped at 35%
- Stable doses of statins, omega-3 fatty acids, fibrates, or other lipid-lowering medications are allowed
Key Exclusion Criteria
- Acute coronary syndrome within 6 months of Screening
- Major surgery within 3 months of Screening
- Have any other conditions, which, in the opinion of the Investigator would make the participant unsuitable for inclusion, or could interfere with participating in or completing the study
Data sourced from ClinicalTrials.gov (NCT04568434). 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.