A Study of AKCEA-APOCIIILRx Administered to Patients With Familial Chylomicronemia Syndrome (FCS)

Phase 1

Conditions: Familial Chylomicronemia Syndrome (FCS)
MedDRA version: 20.0Level: PTClassification code 10059183Term: Familial hypertriglyceridaemiaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.1Level: LLTClassification code 10020607Term: HyperchylomicronemiaSystem Organ Class: 100000004861
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Registration Number: EUCTR2020-002536-67-PT

Lead Sponsor: Ionis Pharmaceuticals, Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 60

Inclusion Criteria

•A diagnosis of genetically confirmed Familial Chylomicronemia Syndrome (type 1 Hyperlipoproteinemia)
•Fasting TG = 880 mg/dL (10 mmol/L) at Screening
•History of pancreatitis. Patients without a documented history of pancreatitis are also eligible but their enrollment will be capped at 20%

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 55
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5

Exclusion Criteria

•Acute coronary syndrome within 6 months of Screening
•Major surgery within 3 months of Screening
•Active pancreatitis within 4 weeks of Screening
•Platelet count < 100K/mm3 at Screening or Qualification.
•Have any other conditions, which, in the opinion of the Investigator would make the patient unsuitable for inclusion, or could interfere with the patient participating in or completing the study

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the efficacy of ISIS 678354 as compared to placebo on the percent change in fasting triglycerides (TG) from Baseline;Secondary Objective: •Proportion of patients who achieve = 40% reduction in fasting TG from Baseline<br>•Percent change in fasting apoB-48 from Baseline<br>•Proportion of patients who achieve fasting TG = 750 mg/dL (8.4 mmol/L)<br>•Adjudicated acute pancreatitis event rate in patients with = 2 events of adjudicated acute pancreatitis in 5 years prior to enrollment <br>•Adjudicated acute pancreatitis event rate <br>•Proportion of patients who achieve = 70% reduction in fasting TG from Baseline<br>•Proportion of patients who achieve fasting TG = 500 mg/dL (5.7 mmol/L)<br><br>;Primary end point(s): The primary endpoint is the percent change in fasting TG from Baseline at 6 months (average of Weeks 23, 25 and 27) compared to placebo;Timepoint(s) of evaluation of this end point: 6 months

Secondary Outcome Measures