New drug combo shows promise for kids with rare immune disease
NCT ID NCT02472054
First seen Apr 03, 2026 · Last updated Jun 23, 2026 · Updated 14 times
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.
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This is a summary of
the original study
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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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Contacts and locations
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Locations
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Hôpital Necker-Enfants Malades
Paris, 75015, France
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.
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.