A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)

Phase 3Active Not RecruitingNCT05130450

Ionis Pharmaceuticals, Inc.60 enrolled

Overview

The purpose of this study is to evaluate the effect of olezarsen (formerly known as AKCEA-APOCIII-LRx) on the percent change in fasting triglycerides (TG) from baseline.

This is a multi-center, open-label extension (OLE) study of up to 60 participants with FCS rolling-over from Study ISIS 678354-CS3 (NCT04568434). Participants will receive olezarsen during a 157-week treatment period, followed by a 13-week post-treatment follow-up period. The length of participation in this study is approximately 201 weeks, which includes an up to 31-day qualification period, a 157-week treatment period, and a 13-week post-treatment evaluation period. Treatment has been extended to obtain additional safety assessments and efficacy data and to provide patients with continued access to ISIS 678354 until the drug may be available commercially.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Familial Chylomicronemia Syndrome

Interventions

OlezarsenDRUG

Olezarsen will be administered by SC injection.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: • Satisfactory completion of treatment with olezarsen in the index study (ISIS 678354-CS3, last dose as scheduled at Week 49) with an acceptable safety profile, per Investigator judgement. Exclusion Criteria: • Have any new condition or worsening of existing condition which in the opinion of the Investigator would make the participant 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).

Locations (27)

Outcomes

Primary Outcomes

Percent Change From Baseline in Fasting TG at 6 Months (Average of Weeks 23, 25, and 27) Compared to Baseline

Time frame: Baseline and 6 months

Secondary Outcomes

Percent Change From Baseline in Fasting TG at 12 Months (Average of Weeks 51 and 53) at 24 Months (Average of Week 103 and Week 105) and 36 Months (Average of Week 155 and Week 157)

Time frame: Baseline and 36 months

Percentage of Participants Who Achieve ≥ 40% Reduction in Fasting TG From Baseline at 6 Months

Time frame: At 6 months

Percentage of Participants Who Achieve ≥ 40% Reduction in Fasting TG From Baseline at 12, 24, 36 Months

Time frame: At 12, 24, 36 months

Percent Change in Fasting apoC-III from Baseline at Month 6

Time frame: Baseline and 6 months

Percent Change in Fasting apoC-III from Month 12, 24, 36

Time frame: At 12, 24, 36 months

Percent Change From Baseline in Fasting Apolipoprotein 48 (apoB48) at 6 Months

Time frame: At 6 months

Percent Change From Baseline in Fasting apoB48 at 12, 24, 36 Months

Time frame: At 12, 24, 36 months

Percent Change in Fasting non-HDL-C from Baseline at Month 6

Time frame: Baseline and 6 months

Percent Change in Fasting non-HDL-C from Months 12, 24, 36

Data from ClinicalTrials.gov

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