A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS) (BALANCE)

Ionis Pharmaceuticals

Status and phase

Completed

Phase 3

Conditions

Familial Chylomicronemia Syndrome

Treatments

Drug: Olezarsen

Drug: Placebo

Study type

Interventional

Funder types

Industry

Identifiers

NCT04568434

ISIS 678354-CS3

2020-002536-67 (EudraCT Number)

Details and patient eligibility

About

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.

Full description

This was a multi-center, double-blind, Phase 3 study in up to 60 patients with FCS. Participants were randomized in a 2:1 ratio to receive Olezarsen or matching placebo in a 53-week treatment period. The length of participation in the study was approximately 74 weeks, which included an up to 8-week screening period, a 53-week treatment period, and a 13-week post-treatment evaluation period. Following the treatment period, eligible patients had the option to enroll in the Open-label Extension (OLE) Study ISIS 678354-CS13 (NCT05130450).

Enrollment

66 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Parallel Assignment

Masking

Double Blind

66 participants in 3 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

Participants received olezarsen-matching placebo, once every 4 weeks by subcutaneous (SC) injection, during Weeks 1 to 49 of the 53-week treatment period.

Treatment:

Drug: Placebo

Olezarsen 50 mg

Experimental group

Description:

Participants received olezarsen, 50 milligrams (mg), once every 4 weeks by SC injection, during Weeks 1 to 49 of the 53-week treatment period.

Treatment:

Drug: Olezarsen

Olezarsen 80 mg

Experimental group

Description:

Participants received olezarsen 80 mg, once every 4 weeks by SC injection, during Weeks 1 to 49 of the 53-week treatment period.