NCT05130450

A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)

An Open-Label Extension Study of AKCEA-APOCIII-LRx Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 60 (actual)
• Sponsor: Ionis Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the effect of olezarsen (formerly known as AKCEA-APOCIII-LRx) on the percent change in fasting triglycerides (TG) from baseline.

Detailed description

This is a multi-center, open-label extension (OLE) study of up to 60 participants with FCS rolling-over from Study ISIS 678354-CS3 (NCT04568434). Participants will receive olezarsen during a 157-week treatment period, followed by a 13-week post-treatment follow-up period. The length of participation in this study is approximately 201 weeks, which includes an up to 31-day qualification period, a 157-week treatment period, and a 13-week post-treatment evaluation period. Treatment has been extended to obtain additional safety assessments and efficacy data and to provide patients with continued access to ISIS 678354 until the drug may be available commercially.

Conditions

• Familial Chylomicronemia Syndrome

Interventions

Type	Name	Description
DRUG	Olezarsen	Olezarsen will be administered by SC injection.

Timeline

• Start date: 2021-11-18
• Primary completion: 2028-02-01
• Completion: 2028-02-01
• First posted: 2021-11-23
• Last updated: 2025-12-12

Locations

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

Regulatory

• FDA-regulated drug study