Can a new shot tame a rare fat disorder in kids?
NCT ID NCT07727538
First seen Jul 27, 2026 · Last updated Aug 27, 2026 · Updated 7 times
Summary
This trial tests whether olezarsen, an experimental drug given as a weekly injection, can lower dangerously high blood fat levels in children with familial chylomicronemia syndrome (FCS). FCS is a rare genetic condition that prevents the body from breaking down fats, leading to severe pancreatitis risk. The study enrolls children aged 2 to 17 and measures changes in triglyceride levels and safety over time.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- an experimental drug called olezarsen, given as a shot under the skin
- What this could lead to
- If it works, this could offer a treatment option for children with a rare, severe fat-processing disorder that currently has few therapies.
- What could go wrong
- This is a small, early-phase trial with only 12 participants, so results may not apply broadly. The drug may cause side effects or fail to lower triglycerides enough.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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About 12 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jul 2026
- Expected to finish
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Aug 2031
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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2 to 17 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
4 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Cook Children's Medical Center
RECRUITINGFort Worth, Texas, 76104, United States
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UCSF Benioff Children's Hospital
RECRUITINGSan Francisco, California, 94143, United States
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University of Alabama
RECRUITINGBirmingham, Alabama, 35294, United States
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University of Texas Southwestern Medical Center
RECRUITINGDallas, Texas, 75390, United States
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