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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.

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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:
Female Icon + Male Icon

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

Full Details:

https://clinicaltrials.gov/ct2/show/NCT04568434

39 Study Locations

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