New drug could tame deadly immune storm in kids before transplant
NCT ID NCT05762640
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a drug called ruxolitinib as the first treatment for children with a rare, life-threatening immune condition called primary HLH. The goal is to calm the overactive immune system and help more children survive long enough to receive a stem cell transplant. The trial will enroll 20 children aged 0 to 22 years and give them ruxolitinib along with steroids for up to 9 weeks or until transplant.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Ruxolitinib (a targeted drug that calms an overactive immune system)
- What this could lead to
- If successful, this could offer a safer, more effective way to control HLH before a stem cell transplant, potentially improving survival and reducing the need for harsh chemotherapy.
- What could go wrong
- This is a small, early-phase trial with only 20 children, so results may not apply to all patients. Ruxolitinib can cause side effects like low blood counts and infections, and it is not yet proven to be better than current treatments.
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
-
Hopital Necker Enfants malades
RECRUITINGParis, Île-de-France Region, 75015, France