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
| Arm | Description | Assigned 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. |
|
|
Placebo Comparator Placebo |
AKCEA-APOCIII-LRx-matching placebo will be administered once every 4 weeks by SC injection from Week 1 through Week 49. |
|
Recruiting Locations
Investigational Pharmacy Services
Nashville, Tennessee 37232
Nashville, Tennessee 37232
More Details
- Status
- Recruiting
- Sponsor
- Ionis Pharmaceuticals, Inc.
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.