Familial Chylomicronemia Syndrome| AKCEA-APOCIII-LRx
A Study of AKCEA-APOCIII-LRx Administered to Patients With Familial Chylomicronemia Syndrome (FCS)
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.
AKCEA-APOCIII-LRx
Purpose of Study
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.
Eligibility
Age:
18 +Sex:
+ 
Key Inclusion / Exclusion Criteria
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
Conditions:
Familial Chylomicronemia Syndrome
Status:
Recruiting
Phase:
Phase 3
Clinical Trials ID
NCT04568434
Read Detailed Summary:
Start Date / End Date:
11 2020 - 06 2023
Intervention:
AKCEA-APOCIII-LRx
Enrollment:
60