Skip to main content
OpenTrials
Active, Not Recruiting

NCT Number: NCT05130450

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

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Notify Me

Key information

Conditions

Familial Chylomicronemia Syndrome Familial hyperchylomicronemia syndrome

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Ecogene-21, Chicoutimi, Quebec, Canada

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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).

Treatment and study plan

Olezarsen

Drug

Olezarsen will be administered by SC injection.

Other names: ISIS 678354, AKCEA -APOCIII-LRx

Primary outcomes

  1. 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

  1. 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

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

    Time frame: At 6 months

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

    Time frame: At 12, 24, 36 months

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

    Time frame: Baseline and 6 months

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

    Time frame: At 12, 24, 36 months

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

    Time frame: At 6 months

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

    Time frame: At 12, 24, 36 months

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

    Time frame: Baseline and 6 months

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

    Time frame: At 12, 24, 36 months

  10. Percentage of Participants Who Achieve Fasting TG ≤ 880 milligrams per deciliter (mg/dL) at 6 Months

    Time frame: At 6 months

  11. Percentage of Participants Who Achieve Fasting TG ≤ 880 mg/dL at 12, 24, 36 months

    Time frame: At 12, 24, 36 months

  12. Adjudicated Acute Pancreatitis Event Rate During the Treatment Period in Participants With ≥ 2 Events of Adjudicated Acute Pancreatitis in 5 Years Prior to Treatment With Study Drug in the Index Study

    Time frame: Week 1 through Week 157

  13. Adjudicated Acute Pancreatitis Event Rate During the Treatment Period

    Time frame: Week 1 through Weeks 53, 105, or 157

  14. Adjudicated Acute Pancreatitis Event Rate During the Treatment Period in Patients with a Prior History of Pancreatitis within 10 Years Prior to Screening in the Index Study

    Time frame: Week 1 through Weeks 53, 105, or 157

  15. Percentage of Participants Who Achieve ≥ 70% Reduction in Fasting TG From Baseline at 6 Months

    Time frame: At 6 months

  16. Percentage of Participants Who Achieve ≥ 70% Reduction in Fasting TG From Baseline at 12, 24, 36 Months

    Time frame: At 12, 24, 36 months

  17. Percentage of Participants Who Achieve Fasting TG ≤ 500 mg/dL at 6 Months

    Time frame: At 6 months

  18. Percentage of Participants Who Achieve Fasting TG ≤ 500 mg/dL at 12, 24, 36 Months

    Time frame: At 12, 24, 36 months

Sponsors and collaborators

Lead sponsor

Ionis Pharmaceuticals, Inc.

Industry

Registry information

Official study title

An Open-Label Extension Study of AKCEA-APOCIII-LRx Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)

Important dates

Study start
2021
Primary completion
2028
Study completion
2028
First posted
Nov 23, 2021
Registry last updated
Dec 12, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.