Recruiting
Phase 3

Olezarsen

Sponsor:

Ionis Pharmaceuticals, Inc.

Code:

NCT07727538

Conditions

Familial Chylomicronemia Syndrome

Eligibility Criteria

Sex: All

Age: 2 - 17

Healthy Volunteers: Not accepted

Interventions

Olezarsen

Study Details

Brief summary:

The primary purpose of the study is to evaluate the efficacy of olezarsen administered by subcutaneous injection to pediatric participants with FCS.

Conditions

Familial Chylomicronemia Syndrome

Study ID

NCT07727538

Start date

Jul 31, 2026

Status verified date

Aug, 2026

Completion date

Aug, 2031

Anticipated

Primary completion date

Aug, 2030

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 2 - 17

Healthy Volunteers: Not accepted

Key Inclusion Criteria:

1. Parental or legally authorized representative consent must be obtained, and the participants must provide age-appropriate or cognition-appropriate assent, as determined by the Investigator. The parent or legal guardian must be able to understand and comply with the study visit schedule and all other study procedures.
2. Must be able to comply with all study procedures.
3. Age 12 to less than 18 years at the time of informed consent/assent (Cohort 1); age 2 to less than 12 years at time of informed consent/assent (Cohort 2).
4. Willing to fast for at least 10 hours before visits requiring fasted blood sampling.
5. A diagnosis of Familial Chylomicronemia Syndrome (type 1 Hyperlipoproteinemia) by documentation of confirmed homozygote, compound heterozygote or double heterozygote for loss-of-function mutations in type 1-causing genes.
6. Fasting TGs ≥880 mg/dL at screening. If fasting TG is < 880 mg/dL, up to two additional tests may be performed during the screening period with any single test used to qualify.

Key Exclusion Criteria:

1. Diabetes mellitus with any of the following:

1. Newly diagnosed within 12 weeks prior to screening or during the screening period.
2. Hemoglobin A1c (HbA1c) ≥9.5% at screening.
3. Change in basal insulin regimen >20% within 3 months prior to screening or during the screening period.
4. Type 1 diabetes.
2. History of bleeding, diathesis, or coagulopathy.
3. Major surgery within 3 months of screening.
4. Plasma apheresis within 4 weeks prior to screening or planned during the study.
5. Treatment with another investigational drug, biological agent, or device within one month of screening, or 5 half-lives of investigational agent, whichever is longer.
6. Active pancreatitis within 4 weeks prior to screening or during the screening period.
7. Malignancy diagnosed or treated within 5 years prior to screening or during the screening period.

Note: Other protocol-specified inclusion/exclusion criteria may apply.

Study Design

Enrollment

12 participants

Anticipated

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Cohort 1

Participants aged 12 to <18 years will receive multiple doses of olezarsen, at a dose level that depends on body weight, once every month by subcutaneous injection for up to 1 year. All participants may continue to the optional 1-year long-term extension period with the last dose received being after 2 years of treatment.

experimental: Cohort 2

Participants aged 2 to <12 years will receive multiple doses of olezarsen, at a dose level based on information obtained from Cohort 1, once every month by subcutaneous injection for up to 1 year. All participants may continue to the optional 1-year long-term extension period with the last dose received being after 2 years of treatment.

Interventions

Olezarsen

Olezarsen will be administered by subcutaneous injection.

Primary outcome measure

  • Percent Change from Baseline in Fasting Triglycerides (TG) [ Time Frame: At 6 Months ]

Central Contacts and Locations

Central contacts

Locations

University of Alabama

Recruiting

Birmingham, Alabama, United States, 35294

UCSF Benioff Children's Hospital

Recruiting

San Francisco, California, United States, 94143

University of Texas Southwestern Medical Center

Recruiting

Dallas, Texas, United States, 75390

Cook Children's Medical Center

Recruiting

Fort Worth, Texas, United States, 76104

More Information

Sponsor

Ionis Pharmaceuticals, Inc.

Last update posted

Aug 26, 2026

Last verified

Aug, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Ionis Pharmaceuticals, Inc. on 2026-08-26.