New drug cocktail aims to tame deadly immune storm in kids
NCT ID NCT04551131
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a combination of three drugs—ruxolitinib, dexamethasone, and etoposide—for children and young adults with hemophagocytic lymphohistiocytosis (HLH), a rare condition where the immune system attacks the body. The trial includes two groups: newly diagnosed patients and those whose disease has come back or not responded to prior treatment. The goal is to see if this tailored regimen can safely control the disease and improve survival.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Ruxolitinib (Jakafi) combined with dexamethasone and etoposide
What this could lead to
If successful, this could provide a more effective and tolerable frontline treatment for HLH, a rare and life-threatening immune disorder, and offer a new option for patients who relapse.
What could go wrong
This is an early-phase trial with only 10 participants, so results may not apply to all patients. The combination includes strong chemotherapy-like drugs, which carry risks of severe side effects like infections and organ damage.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
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Locations
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Children's Hospital of Orange County
Orange, California, 92868, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
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Children's Wisconsin/Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
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Cohen Children's Medical Center
New Hyde Park, New York, 11040, United States
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Dana-Farber Cancer Institute
Boston, Massachusetts, 02215, United States
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John Hopkins University
Baltimore, Maryland, 21287, United States
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Levine Children's Hospital
Charlotte, North Carolina, 28203, United States
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Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
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St. Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
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Texas Children's Hospital
Houston, Texas, 77030, United States
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University of California San Francisco
San Francisco, California, 94158, United States
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University of Texas Southwestern Medical Center
Dallas, Texas, 75235, United States