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New drug combo shows promise for kids with rare immune disease

NCT ID NCT02472054

Completed Disease control Sponsor: Assistance Publique - Hôpitaux de Paris Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a drug called alemtuzumab (Campath) as a first treatment for children with hemophagocytic lymphohistiocytosis (HLH), a rare and life-threatening immune disorder. The goal was to see if it could help children survive until they could receive a stem cell transplant. The trial included 29 children under 18 with confirmed HLH. Researchers measured how many survived to transplant and how many achieved complete remission.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
Alemtuzumab (also called Campath), given with methylprednisolone and cyclosporin A
What this could lead to
If successful, this could offer a better first-line treatment for children with HLH, helping them survive long enough to receive a stem cell transplant.
What could go wrong
This is a small, early-phase trial (Phase 1/2) with only 29 participants, so results may not apply to all patients. Alemtuzumab can cause serious side effects, including infections and immune reactions.

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.

Chediak-Higashi syndrome Griscelli syndrome type 2 hemophagocytic syndrome hereditary hemophagocytic lymphohistiocytosis Lymphohistiocytosis, Hemophagocytic X-linked lymphoproliferative disease due to XIAP deficiency X-linked lymphoproliferative syndrome

As listed by the trial registrant

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

HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS (HLH)

Contacts and locations

Locations

  • Hôpital Necker-Enfants Malades

    Paris, 75015, France

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