Olezarsen for Familial Chylomicronemia Syndrome in Kids
A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric Participants
Recruiting
Phase 3Interventional Study
Familial Chylomicronemia Syndrome
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
Ready to participate?
Review the details below, then apply to join this clinical trial.
At a Glance
Age
2 – 17
Sex
Any
Trial phase
Phase 3
Study type
Interventional
Purpose
Treatment
Participants needed
12 (estimated)
Sponsor
Ionis Pharmaceuticals, Inc. · Industry
Who this trial is looking for
This trial is looking for children aged 2 to under 18 who have Familial Chylomicronemia Syndrome. Participants will receive olezarsen through an injection and must be willing to fast before blood tests.
Are You a Good Fit for This Trial?
Rules you outDiabetesActive pancreatitisMalignancy
You may be able to join if
I have been diagnosed with Familial Chylomicronemia Syndrome.
I am between 2 and 18 years old.
I am willing to fast for at least 10 hours before blood tests.
My fasting triglycerides are 880 mg/dL or higher.
You may not be able to join if
I have been diagnosed with diabetes in the last 12 weeks.
My hemoglobin A1c is 9.5% or higher.
I have had major surgery in the last 3 months.
I have active pancreatitis.
I have had cancer diagnosed or treated in the last 5 years.
Summarized in plain language from this trial's official eligibility criteria.
The full criteria are further down this page — only the research team can
confirm whether you qualify.
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
The primary purpose of the study is to evaluate the efficacy of olezarsen administered by subcutaneous injection to pediatric participants with FCS.
Trial Locations
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Eligibility Criteria
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 …
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. 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.
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