Timing is everything: new trial aims to save lives from immune storm
NCT ID NCT07497438
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study compares two strategies for starting the drug etoposide in adults with severe hemophagocytic lymphohistiocytosis (HLH) who are in the intensive care unit. HLH is a life-threatening overreaction of the immune system that can cause organ failure. The goal is to see if giving etoposide early, when organ failure first appears, reduces worsening organ damage compared to waiting to see if other treatments work first. About 176 participants will be randomly assigned to one of the two approaches.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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About 176 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Apr 2026
An estimate. Start dates often move.
- Expected to finish
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Feb 2030
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Adult patient * Confirmed diagnosis of HLH: * Hscore ≥ 169 * Diagnosis of HLH established by the multidisciplinary team caring for the patient(A 24/7 hotline will be available in case of diagnostic uncertainty: this will be the direct line to the on-call intensivist at Avicenne Hospital, who can contact the coordinating investigator as needed) * First episode of HLH * Admission to intensive care unit * Presence of one or more organ failures: * Circulatory: mBP \< 65 mmHg with lactate \> 2 mmol/L, or treatment with catecholamines * Respiratory: oxygen therapy \> 6 L/min or need for non-invasive ventilation, high-flow nasal cannula oxygen therapy, or invasive mechanical ventilation * Renal: stage 2 or higher according to KDIGO criteria, defined by a creatinine increase ≥ 2 times baseline, or urine output \< 0.5 mL/kg/h for ≥ 12 hours, or initiation of renal replacement therapy * Neurological: GCS ≤ 13 Exclusion Criteria: * Moribund patient with refractory distributive shock: multi-organ failure requiring noradrenaline \> 2.5 µg/kg/min and imminent risk of death. * Inability to administer etoposide within 12 hours. * Patient treated with etoposide prior to admission to the intensive care unit. * Hypersensitivity to etoposide or any of its excipients. * Hypersensitivity or contraindication to dexamethasone or any of its excipients, as described in the package insert for the dexamethasone specialty used in the trial and in the protocol. * Patient with a history of CAR-T cell therapy. * Patient developing HLH within 15 days following chemotherapy for cancer or hematologic malignancy. * Patient not covered by social security. * Patient under legal guardianship, tutelage, or curatorship. * Minor patient. * Pregnant women (positive βhCG), women for whom pregnancy has not been excluded (βhCG not performed in women of childbearing age), or lactating women. * Recent vaccination with a live attenuated vaccine. * Participation in another interventional research study.
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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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Intensive Care Unit - Avicenne Hospital, Assistance Publique des Hôpitaux de Paris
Bobigny, 93000, France
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