An Open-Label Extension Study of AKCEA-APOCIII-LRX 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: 10027433 - Metabolism and nutrition disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2021-003280-95-PT
• Lead Sponsor: Ionis Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2022-05-19
• Last Update Posted: 19 August 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

2. Satisfactory completion of the ISIS 678354 CS3 index study (last dose as scheduled at Week 49) with an acceptable safety profile, per Investigator judgement
3. Willing to follow a diet comprising = 20 g fat per day during the study

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

Have any new condition or worsening of existing condition which in the opinion of the Investigator would make the patient unsuitable for enrollment, or could interfere with the patient participating in or completing the study, including need for treatment with medications disallowed in the index study (ISIS 678354-CS3).

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified