Olezarsen for Familial Chylomicronemia Syndrome in kids

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a Phase 3, multi-center, open-label trial to evaluate the safety, pharmacokinetics, efficacy, and pharmacodynamics of Oleazarsen in pediatric participants with Familial Chylomicronemia Syndrome. The study includes a screening period, a one-year treatment period, an optional one-year long-term extension, and a three-month post-treatment follow-up. Participants are divided into two age-based cohorts and must fast before certain visits, with confirmed genetic diagnosis and very high fasting triglycerides at screening. Lead sponsor is Ionis Pharmaceuticals, with three participating centers in the United States.

Why it matters

Who can join: the full rules

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.

Where it's running

• University of Alabama
  Birmingham, Alabama, United States Recruiting
• UCSF Benioff Children's Hospital
  San Francisco, California, United States Recruiting
• Children's Mercy Hospital
  Kansas City, Missouri, United States Recruiting
• University of Texas Southwestern Medical Center
  Dallas, Texas, United States Recruiting
• Cook Children's Medical Center
  Fort Worth, Texas, United States Recruiting
• Mary Bridge Children's
  Tacoma, Washington, United States Recruiting

Study contacts

• Study coordinator: Ionis Pharmaceuticals, Inc.
• Email: IonisOlezarsenPediatric@clinicaltrialmedia.com
• Phone: (844) 200-6410

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• CS-121 APOC3 base-edit gene therapy for familial chylomicronemia (FCS)

  This trial will try a single-course, dose-escalation APOC3 base-editing treatment (CS-121) delivered by lipid nanoparticles in adults 18–55 with genetically confirmed FCS and very high triglycerides to see if it lowers ApoC3 and triglycerides and is safe.

  Recruiting Early Phase 1 CorrectSequence Therapeutics Co., Ltd · Hefei, Anhui · NCT07176923

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.