Can a new shot tame a rare fat disorder in kids?
NCT ID NCT07727538
First seen Jul 27, 2026 · Last updated Jul 30, 2026 · Updated 3 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.
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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.
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 Texas Southwestern Medical Center
RECRUITINGDallas, Texas, 75390, United States
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