NCT04568434

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

A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of AKCEA-APOCIII-LRx Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 66 (actual)
• Sponsor: Ionis Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed 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).

Conditions

• Familial Chylomicronemia Syndrome

Interventions

Type	Name	Description
DRUG	Olezarsen	Olezarsen was administered by SC injection.
DRUG	Placebo	Olezarsen-matching placebo was administered by SC injection.

Timeline

• Start date: 2020-11-18
• Primary completion: 2023-07-14
• Completion: 2023-10-17
• First posted: 2020-09-29
• Last updated: 2025-03-06
• Results posted: 2025-03-06

Locations

47 sites across 11 countries: United States, Canada, France, Italy, Netherlands, Norway, Portugal, Slovakia, Spain, Sweden, United Kingdom

Regulatory

• FDA-regulated drug study