A Study of Olezarsen Administered Subcutaneously to Participants With Severe Hypertriglyceridemia

Ionis Pharmaceuticals, Inc.446 enrolled

Overview

The purpose of this study is to evaluate the efficacy of olezarsen as compared to placebo on the percent change in fasting triglycerides (TG) from baseline.

This is a Phase 3, multi-center, randomized, double-blind, placebo-controlled study in 446 participants. Participants will be randomized to receive olezarsen or placebo in a 53-week treatment period. The length of participation in the study will be approximately 78 weeks, which includes an up to 12-week screening period, a 53-week treatment period, and a 13-week post-treatment evaluation period or transition to open-label extension (OLE) study with up to 1-year treatment.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

446

Conditions

Severe Hypertriglyceridemia

Interventions

OlezarsenDRUG

Olezarsen will be administered by SC injection.

PlaceboDRUG

Olezarsen-matching placebo will be administered by SC injection.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Fasting TG ≥ 500 mg/dL (5.65 mmol/L) at Screening and Qualification 2. Participants must be on lipid-lowering therapy that should adhere to standard of care (SOC) per local guidelines. Lipid-lowering medications should be optimized and stabilized for at least 4 weeks prior to screening to minimize changes in these medications during the study. 3. Participants must be willing to comply with diet and lifestyle recommendations as able. Exclusion Criteria: 1. Hemoglobin A1c (HbA1c) ≥ 9.5% at Screening 2. Alanine aminotransferase or aspartate aminotransferase \> 3.0 × upper limit of normal 3. Total bilirubin \> 1.5 ULN unless due to Gilbert's syndrome 4. Estimated GFR \< 30 mL/min/1.73 m\^2

Locations (205)

Outcomes

Primary Outcomes

Percent Change from Baseline in Fasting TG Compared to Placebo

Time frame: Baseline and Month 6

Secondary Outcomes

Percent Change from Baseline in Fasting TG Compared to Placebo

Time frame: Baseline and Month 12

Percent Change from Baseline in Fasting Apolipoprotein C-III (ApoC-III), Remnant Cholesterol and Fasting Non-High-Density Lipoprotein-Cholesterol (Non-HDL-C) Compared to Placebo

Time frame: Baseline, Month 6 and 12

Proportion of Patients Who Achieve Fasting TG <500 mg/dL (5.65 mmol/L) Compared to Placebo

Time frame: Month 12

Proportion of Participants Who Achieve Fasting TG < 880 mg/dL (10 mmol/L) Compared to Placebo in the Subgroup of Participants with Baseline TG ≥ 880 mg/dL

Time frame: Month 12

Adjudicated Acute Pancreatitis Event Rate During the Treatment Period Compared to Placebo

Time frame: Week 1 through Week 53

Absolute Change in Hepatic Fat Fraction (HFF) Between Olezarsen Treatment Group and Pooled Placebo

Time frame: Baseline through Month 12

Data from ClinicalTrials.gov

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