Clinical Trials in the USA / NCT07727538
Recruiting
Phase 3
A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric Participants
NCT07727538 · tracked via the Priya Life Science USA tracker
Condition(s) studied
Familial Chylomicronemia Syndrome
Investigational drug(s) / intervention(s)
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
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 (5)
| 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 |
| Mary Bridge Children's | Tacoma | Washington | Recruiting |
More Ionis Pharmaceuticals, Inc. trials in the USA
CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)
Active, not recruiting
A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
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Other trials for the same condition
Official registry record
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.
View NCT07727538 on ClinicalTrials.gov ↗ ← All trials in the USA