Print

Purpose

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

Condition

Eligibility

Eligible Ages
Between 2 Years and 17 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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.

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. For participants with type 1 diabetes: episode of diabetic ketoacidosis, or ≥3 episodes of severe hypoglycemia within 6 months prior to screening or during the screening period. 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

Phase
Phase 3
Study Type
Interventional
Allocation
N/A
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
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.
  • Drug: Olezarsen
    Olezarsen will be administered by subcutaneous injection.
    Other names:
    • ISIS 678354
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.
  • Drug: Olezarsen
    Olezarsen will be administered by subcutaneous injection.
    Other names:
    • ISIS 678354

Recruiting Locations

University of Texas Southwestern Medical Center
Dallas, Texas 75390

More Details

Status
Recruiting
Sponsor
Ionis Pharmaceuticals, Inc.

Study Contact

Ionis Pharmaceuticals, Inc.
(844) 200-6410
IonisOlezarsenPediatric@clinicaltrialmedia.com

Detailed Description

This is a Phase 3 multi-center open-label study to evaluate safety, pharmacokinetics (PK), efficacy, and pharmacodynamics (PD) of olezarsen in pediatric participants (aged 2 to less than (<)18 years) with FCS. This study consists of three to four periods with the following approximate timeframes: 1-month screening period, 1-year treatment period, an optional 1-year long-term extension treatment period, and a 3-month post-treatment follow-up period.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.