Olezarsen: Olezarsen will be administered by subcutaneous injection.
Study summary
The primary purpose of the study is to evaluate the efficacy of olezarsen administered by subcutaneous injection to pediatric participants with FCS.
Eligibility
Sex
ALL
Min age
2 Years
Max age
17 Years
Healthy volunteers
No
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.
Primary outcome measure(s)
Percent Change from Baseline in Fasting Triglycerides (TG) — At 6 Months
Trial sites (4)
Facility
City
Region
Status
University of Alabama
Birmingham
Alabama
Recruiting
UCSF Benioff Children's Hospital
San Francisco
California
Recruiting
University of Texas Southwestern Medical Center
Dallas
Texas
Recruiting
Cook Children's Medical Center
Fort Worth
Texas
Recruiting
More Ionis Pharmaceuticals, Inc. trials in the USA
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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