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

Purpose

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.

Condition

  • Familial Chylomicronemia Syndrome

Eligibility

Eligible Ages
Over 18 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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 - Stable doses of statins, omega-3 fatty acids, fibrates, or other lipid-lowering medications are allowed

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

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Double (Participant, Investigator)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
AKCEA-APOCIII-LRx
AKCEA-APOCIII-LRx will be administered once every 4 weeks by subcutaneous (SC) injection from Week 1 through Week 49.
  • Drug: AKCEA-APOCIII-LRx
    AKCEA-APOCIII-LRx will be administered by SC injection.
    Other names:
    • ISIS 678354
Placebo Comparator
Placebo
AKCEA-APOCIII-LRx-matching placebo will be administered once every 4 weeks by SC injection from Week 1 through Week 49.
  • Drug: Placebo
    AKCEA-APOCIII-LRx-matching placebo will be administered by SC injection.

Recruiting Locations

Investigational Pharmacy Services
Nashville, Tennessee 37232

More Details

Status
Recruiting
Sponsor
Ionis Pharmaceuticals, Inc.

Study Contact

Ionis Pharmaceuticals
(844) 414-1620
ionisNCT04568434study@clinicaltrialmedia.com

Detailed Description

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.