Overview
The primary purpose of the study is to evaluate the efficacy of olezarsen administered by subcutaneous injection to pediatric participants with FCS.
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.
Eligibility
Key Inclusion Criteria:
- 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.
- Must be able to comply with all study procedures.
- 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).
- Willing to fast for at least 10 hours before visits requiring fasted blood sampling.
- 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.
- 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:
- Diabetes mellitus with any of the following:
- Newly diagnosed within 12 weeks prior to screening or during the screening period.
- Hemoglobin A1c (HbA1c) ≥9.5% at screening.
- Change in basal insulin regimen \>20% within 3 months prior to screening or during the screening period.
- 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.
- History of bleeding, diathesis, or coagulopathy.
- Major surgery within 3 months of screening.
- Plasma apheresis within 4 weeks prior to screening or planned during the study.
- Treatment with another investigational drug, biological agent, or device within one month of screening, or 5 half-lives of investigational agent, whichever is longer.
- Active pancreatitis within 4 weeks prior to screening or during the screening period.
- Malignancy diagnosed or treated within 5 years prior to screening or during the screening period.
Note: Other protocol-specified inclusion/exclusion criteria may apply.
