A Study of Olezarsen Administered Subcutaneously to Participants With Severe Hypertriglyceridemia

Ionis Pharmaceuticals

Status and phase: Active, not recruiting; Phase 3

Conditions: Severe Hypertriglyceridemia

Treatments: Drug: Placebo; Drug: Olezarsen

Study type: Interventional

Funder types: Industry

Identifiers

NCT05552326

ISIS 678354-CS6

2022-501420-20 (EudraCT Number)

Details and patient eligibility

About

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.

Full description

This is a Phase 3, multi-center, randomized, double-blind, placebo-controlled study in up to approximately 390 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.

The Screening Period was extended with no impact to overall study timelines. Endpoints added to provide assessment on the pancreatitis rate after olezarsen reaches steady state levels.

Enrollment: 446 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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446 participants in 2 patient groups, including a placebo group

Olezarsen

Experimental group

Description:

Olezarsen will be administered once every 4 weeks by subcutaneous (SC) injection from Week 1 through Week 49.

Treatment:

Drug: Olezarsen

Placebo

Placebo Comparator group

Description:

Olezarsen-matching placebo will be administered once every 4 weeks by subcutaneous (SC) injection from Week 1 through Week 49.

Treatment:

Drug: Placebo