A Study of AKCEA-APOCIII-LRx Administered to Patients With Familial Chylomicronemia Syndrome (FCS)
The purpose of the study is to evaluate the efficacy of AKCEA-APOCIII-LRx as compared to placebo on the percent change in fasting triglycerides (TG) from baseline.
- Familial Chylomicronemia Syndrome
- Eligible Ages
- Over 18 Years
- Eligible Genders
- Accepts Healthy Volunteers
- 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 - Stable doses of statins, omega-3 fatty acids, fibrates, or other lipid-lowering medications are allowed
- 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
- Phase 3
- Study Type
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Double (Participant, Investigator)
|AKCEA-APOCIII-LRx will be administered once every 4 weeks by subcutaneous (SC) injection from Week 1 through Week 49.||
|AKCEA-APOCIII-LRx-matching placebo will be administered once every 4 weeks by SC injection from Week 1 through Week 49.||
- Ionis Pharmaceuticals, Inc.
Study ContactIonis Pharmaceuticals
This is a multi-center, double-blind, Phase 3 study in up to 60 patients with FCS. Participants will be randomized in a 2:1 ratio to receive AKCEA-APOCIII-LRx or matching placebo in a 53-week treatment period. The length of participation in the study is approximately 74 weeks, which includes 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 may have the option of enrolling in an open label extension study.