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Phase 2 N=154 Randomized Quadruple-blind Treatment

A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Adults With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease (Established or at Increased Risk for), and/or With Severe Hypertriglyceridemia

Hypertriglyceridemia · Atherosclerotic Cardiovascular Disease · Severe Hypertriglyceridemia

Enrolled (actual)
154
Serious AEs
10.4%
Results posted
Apr 2026
Primary outcome: Primary: Percent Change From Baseline in Fasting TG at Month 6 — -7.77; -57.05; -60.92 Percent Change — p=<0.0001

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
Olezarsen (Drug); Placebo (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
Ionis Pharmaceuticals, Inc.
Primary completion
Mar 2023

Outcome Measures

OutcomeResultp-value
PRIMARY
Percent Change From Baseline in Fasting TG at Month 6
-7.77; -57.05; -60.92 <0.0001 sig
SECONDARY
Percent Change From Baseline in Fasting TG at Month 12
-5.59; -51.67; -60.39 <0.0001 sig
SECONDARY
Percentage of Participants Who Achieved Fasting TG <150 mg/dL (1.69 Millimoles Per Liter [mmol/L]) at Month 6 With Baseline TG <500 mg/dL (5.65 mmol/L)
11.4; 79.2; 84.0 <0.0001 sig
SECONDARY
Percentage of Participants Who Achieved Fasting TG <150 mg/dL (1.69 mmol/L) at Month 12 With Baseline TG <500 mg/dL (5.65 mmol/L)
5.7; 69.8; 82.0 <0.0001 sig
SECONDARY
Percent Change From Baseline in Fasting Apolipoprotein C-III (ApoC-III) at Month 6
-5.47; -69.63; -78.72 <0.0001 sig
SECONDARY
Percent Change From Baseline in Fasting ApoC-III at Month 12
-12.40; -69.09; -83.78 <0.0001 sig
SECONDARY
Percent Change From Baseline in Fasting Very Low-density Lipoprotein Cholesterol (VLDL-C) at Month 6
-9.74; -55.93; -59.46 <0.0001 sig
SECONDARY
Percent Change From Baseline in VLDL-C at Month 12
-14.90; -58.00; -66.67 <0.0001 sig
SECONDARY
Percent Change From Baseline in Fasting Non-high-density Lipoprotein Cholesterol (Non-HDL-C) at Month 6
3.28; -22.11; -19.79 <0.0001 sig
SECONDARY
Percent Change From Baseline in Fasting Non-HDL-C at Month 12
-2.55; -20.00; -26.57 =0.0006 sig
SECONDARY
Percent Change From Baseline in Fasting High-density Lipoprotein Cholesterol (HDL-C) at Month 6
8.38; 48.02; 48.01 <0.0001 sig
SECONDARY
Percent Change From Baseline in Fasting HDL-C at Month 12
10.53; 54.20; 57.43 <0.0001 sig
SECONDARY
Percent Change From Baseline Fasting Remnant Cholesterol (Remnant-C) at Month 6
-9.46; -55.98; -59.59 <0.0001 sig
SECONDARY
Percent Change From Baseline in Fasting Remnant-C at Month 12
-14.83; -58.17; -66.90 <0.0001 sig
SECONDARY
Percent Change From Baseline in Fasting Apolipoprotein B (ApoB) at Month 6
6.82; -11.42; -11.67 <0.0001 sig
SECONDARY
Percent Change From Baseline in Fasting ApoB at Month 12
0.68; -11.67; -18.53 =0.0038 sig
SECONDARY
Percent Change From Baseline in Fasting Apolipoprotein A-1 (ApoA-1) at Month 6
2.84; 17.37; 14.91 <0.0001 sig
SECONDARY
Percent Change From Baseline in Fasting ApoA-1 at Month 12
0.81; 16.37; 15.46 <0.0001 sig
SECONDARY
Percent Change From Baseline in Fasting Low-density Lipoprotein Cholesterol (LDL-C) at Month 6
40.46; 30.61; 32.74 =0.2413
SECONDARY
Percent Change From Baseline in Fasting LDL-C at Month 12
35.08; 32.99; 27.27 =0.7850
SECONDARY
Percent Change From Baseline in Fasting Total Cholesterol (Total-C) at Month 6
2.99; -6.78; -6.48 =0.0238 sig
SECONDARY
Percent Change From Baseline in Fasting Total-C at Month 12
-2.15; -6.08; -10.80 =0.3107
SECONDARY
Percentage of Participants Who Achieved Fasting TG <500 mg/dL (5.65 mmol/L) With Baseline TG ≥500 mg/dL at Month 6
75.0; 80.0; 100.0 =0.4212
SECONDARY
Percentage of Participants Who Achieved Fasting TG <500 mg/dL (5.65 mmol/L) With Baseline TG ≥500 mg/dL at Month 12
75.0; 100.0; 100.0 =0.4722
SECONDARY
Number of Participants With Adjudicated Acute Pancreatitis Events
0; 0; 0
SECONDARY
Number of Participants With Adjudicated Acute Pancreatitis Events (≥2 Events in 5 Years Prior to Enrollment)
0; 0; 0

Summary

The purpose of the study was to evaluate the effect of olezarsen on percent change in fasting triglyceride (TG) levels compared to placebo at Months 6 and 12 and the proportion of participants who achieve different thresholds in fasting TG. Other objectives were to evaluate the effect of olezarsen on percent change in fasting apolipoprotein C-III (apoC-III), very low-density lipoprotein cholesterol (VLDL-C), remnant cholesterol, non-high-density lipoprotein cholesterol (non-HDL-C), HDL-C, total cholesterol (TC), apolipoprotein B (apoB), low-density lipoprotein cholesterol (LDL-C), and apolipoprotein A-1 (apoA-1).

Eligibility Criteria

Inclusion Criteria

  • Aged ≥ 18 years at the time of informed consent
  • Fall into at least 1 of the following groups (a or b):
  • Hypertriglyceridemia with fasting TG ≥ 150 mg/dL (1.69 millimoles per liter [mmol/L]) and 20% within 3 months prior to Screening
  • For participants with type 1 diabetes: episode of diabetic ketoacidosis, or ≥ 3 episodes of severe hypoglycemia within 6 months prior to Screening
  • Acute coronary syndrome or stroke/transient ischemic attack (TIA) within 6 months prior to Screening
  • Major surgery, peripheral revascularization, or non-urgent percutaneous coronary intervention (PCI) within 3 months prior to Screening, or upcoming planned major surgery or major procedure (e.g., arterial revascularization) during the course of the study
  • Active pancreatitis within 4 weeks prior to Screening
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT05355402). 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.

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