Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at UCSF
Dates
study started
estimated completion

Description

Summary

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.

Official Title

A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of AKCEA-APOCIII-LRx Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)

Details

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.

Keywords

Familial Chylomicronemia Syndrome FCS Hyperlipoproteinemia Type I Syndrome Olezarsen

Eligibility

You can join if…

Open to people ages 18 years and up

  • 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

You CAN'T join if...

  • 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

Locations

  • University of California, San Francisco (UCSF) - Medical Center accepting new patients
    San Francisco California 94143 United States
  • Diabetes/Lipid Management & Research Center accepting new patients
    Huntington Beach California 92648 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Ionis Pharmaceuticals, Inc.
ID
NCT04568434
Phase
Phase 3 Familial Chylomicronemia Syndrome Research Study
Study Type
Interventional
Participants
Expecting 60 study participants
Last Updated