A Study of Olezarsen Administered Subcutaneously to Participants With Severe Hypertriglyceridemia

Purpose

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.

Condition

  • Severe Hypertriglyceridemia

Eligibility

Eligible Ages
Over 18 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Triple (Participant, Care Provider, Investigator)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Olezarsen
Olezarsen will be administered once every 4 weeks by subcutaneous (SC) injection from Week 1 through Week 49.
  • Drug: Olezarsen
    Olezarsen will be administered by SC injection.
    Other names:
    • ISIS 678354
    • AKCEA-APOCIII-LRx
Placebo Comparator
Placebo
Olezarsen-matching placebo will be administered once every 4 weeks by subcutaneous (SC) injection from Week 1 through Week 49.
  • Drug: Placebo
    Olezarsen-matching placebo will be administered by SC injection.

More Details

Status
Completed
Sponsor
Ionis Pharmaceuticals, Inc.

Study Contact

Detailed Description

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.