New drug may help kids with rare immune disease reach life-saving transplant

NCT ID NCT05762640

First seen Apr 25, 2026 · Last updated Apr 28, 2026 · Updated 1 time

Summary

This study tests a drug called ruxolitinib as the first treatment for children with primary haemophagocytic lymphohistiocytosis (HLH), a rare and serious immune disorder. The goal is to see if ruxolitinib, given with steroids, can help children survive long enough to receive a stem cell transplant. About 20 children up to age 22 will take part.

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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.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Hopital Necker Enfants malades

    RECRUITING

    Paris, Île-de-France Region, 75015, France

    Contact Phone: •••-•••-•••• Email: •••••@•••••

Conditions

Explore the condition pages connected to this study.