New drug cocktail aims to tame deadly immune storm in kids

NCT ID NCT04551131

Ongoing Disease control Sponsor: St. Jude Children's Research Hospital Source: ClinicalTrials.gov ↗

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.

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Conditions

The condition(s) this trial relates to.

hemophagocytic syndrome Lymphohistiocytosis, Hemophagocytic Recurrence

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS

Contacts and locations

Locations

Children's Hospital of Orange County

Orange, California, 92868, United States
Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States
Children's Wisconsin/Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States
Cohen Children's Medical Center

New Hyde Park, New York, 11040, United States
Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States
John Hopkins University

Baltimore, Maryland, 21287, United States
Levine Children's Hospital

Charlotte, North Carolina, 28203, United States
Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States
St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States
Texas Children's Hospital

Houston, Texas, 77030, United States
University of California San Francisco

San Francisco, California, 94158, United States
University of Texas Southwestern Medical Center

Dallas, Texas, 75235, United States

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