A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)
Purpose
The purpose of the study is to evaluate the efficacy of Olezarsen 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. Patients without a documented history of pancreatitis are also eligible but their enrollment will be capped at 35% - 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 Olezarsen |
Olezarsen will be administered once every 4 weeks by subcutaneous (SC) injection from Week 1 through Week 49. |
|
|
Placebo Comparator Placebo |
Olezarsen-matching placebo will be administered once every 4 weeks by SC injection from Week 1 through Week 49. |
|
More Details
- Status
- Completed
- Sponsor
- Ionis Pharmaceuticals, Inc.
Study Contact
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 Olezarsen 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.