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โ† Trials
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A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric Participants

RecruitingPhase 3Ages 2 Years - 17 YearsAllIonis Pharmaceuticals, Inc.
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In plain words

What the trial is, and whether it could suit you

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

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
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 โ‰ฅ880 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) โ‰ฅ9.5% 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.
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What it tests

  • Olezarsen
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Where

5 sites
University of Alabama
Birmingham, United States
UCSF Benioff Children's Hospital
San Francisco, United States
University of Texas Southwestern Medical Center
Dallas, United States
Cook Children's Medical Center
Fort Worth, United States
Mary Bridge Children's
Tacoma, United States
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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