ClinicalTrials.gov record NCT06951971

Dose-modified Emapalumab and Ruxolitinib (E-Ru) Regimens for Hemophagocytic Lymphohistiocytosis

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

A Multiple Center, Prospective, Single Arm, Phase III Clinical Study to Evaluate the Efficacy and Safety of Dose-modified Emapalumab and Ruxolitinib in the Treatment of Hemophagocytic Lymphohistiocytosis

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 2/3A study that tests whether a treatment works while continuing to check safety, in a larger group than a phase 2 study alone. It combines the aims of phase 2 and phase 3 in one protocol.
Age1 year old and older
SexAll
LocationChina
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This multicenter, single‑arm Phase II/III trial tests a combination regimen of lower‑dose emapalumab and higher‑dose ruxolitinib in people with active HLH. Treatment is given for eight weeks and predefined rescue treatments (including HLH‑94/DEP regimens or lymphoma chemotherapy) or allo‑HSCT are allowed when specified. The main outcome is overall survival at 60 days, with response, safety, and biomarker measurements at days 7, 14, 28, and 56, and survival followed up to six months.

What it is studying. The study is evaluating whether a dose‑modified combination of emapalumab (an interferon‑gamma blocking antibody given at a lower dose than usual) and ruxolitinib (given at a higher dose than usual) is effective and safe for treating active HLH. It collects short‑term survival and response data, measures adverse events, and tracks changes in related biomarkers.

What the researchers hope to learn. To evaluate the efficacy and safety of dose‑modified emapalumab plus ruxolitinib in active HLH. Primary scientific goal: measure 60‑day overall survival. Secondary goals: assess response rates at days 7, 14, 28, and 56; record adverse events using CTCAE v5.0; measure event‑free survival and overall survival up to six months; and describe trends in related biomarkers from baseline through treatment.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

This study is trying to evaluate the efficacy and safety of dose-modified Emapalumab and Ruxolitinib (E-Ru) regimens for the treatment of active hemophagocytic lymphohistiocytosis.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • dose-modified E-Ru
Other
  • Salvage treatment and follow-up

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • Fulfillment of at least five of the eight HLH-2004 criteria for HLH;
  • Age 1-70 years old;
  • No prior chemotherapy for HLH;
  • Confirmed not pregnant and willing to use effective contraceptive measures during the study period, with the last dose of medication administered at least 12 months prior;
  • Signed informed consent prior to study participation.

Exclusion criteria

  • According to the New York Heart Association (NYHA) score, patients with heart disease of grade II or above (including grade II);
  • HIV-infected patients;
  • Patients with severe renal dysfunction (glomerular filtration rate <15 mL/min);
  • Patients with severe liver cirrhosis (MELD score>20);
  • Uncontrollable infections (including lung infections, intestinal infections, etc.);
  • Have serious mental illness;
  • Have a history of active tumor;
  • Participate in other clinical investigators at the same time;
  • People with central nervous system involvement;
  • Exclusion criteria related to concomitant medication.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

This clinical trial is designed to evaluate a new treatment strategy for patients with active Hemophagocytic Lymphohistiocytosis (HLH)-a rare but severe immune disorder characterized by excessive inflammation and immune system activation. HLH can be life-threatening if not treated effectively. The study is prospective and multicenter, meaning it will be conducted at multiple hospitals and medical institutions, and patients will be followed over time to assess treatment outcomes. We aim to test the combination of two medications:

  • Emapalumab, a monoclonal antibody that blocks interferon-gamma, a key driver of the overactive immune response seen in HLH. In this trial, it will be used at a lower-than-standard dose to reduce potential side effects.
  • Ruxolitinib, a JAK1/2 inhibitor that can reduce inflammation by interfering with immune signaling pathways. It will be administered at a higher-than-standard dose to enhance its therapeutic effects.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants receive an 8‑week regimen of dose‑modified Emapalumab (given IV once weekly) plus Ruxolitinib (oral, twice daily). They will be monitored frequently for response and safety (assessments noted at day 7, 14, 28, and 56 and outcomes tracked through 6 months). If there is no response after 7 days or if relapse occurs, a predefined salvage regimen (for example HLH‑94 or DEP) may be given; chemotherapy will be started if lymphoma is diagnosed and allo‑HSCT may be performed if criteria are met. Adverse events and clinical response are recorded; informed consent is required before any study procedures.

How the study is structured. Interventional, prospective, multicenter, single‑group (all participants receive the same treatment), open‑label Phase II/III study. Participants receive emapalumab intravenously once weekly and oral ruxolitinib twice daily for eight weeks; predefined rescue treatments or follow‑up (including allo‑HSCT when criteria are met) are part of the plan.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The registry says recruitment has not started. When is it expected to open, and can I register interest now?
  • The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on hemophagocytic lymphohistiocytosis

Clinical trials34 trials listed for hemophagocytic lymphohistiocytosisEvery trial the registry lists for hemophagocytic lymphohistiocytosis, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in hemophagocytic lymphohistiocytosis researchLatest monthly update: September 2026. Includes: Multiple test results. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Jun 1, 2025 (estimated)
Primary completion
Jun 1, 2027 (estimated)
Study completion
Jun 1, 2028 (estimated)
Target enrollment
30 participants (estimated)
Last updated on registry
Apr 30, 2025
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
The First Affiliated Hospital of Soochow University
Principal investigator
Xuefeng He, The First Affiliated Hospital of Soochow University
The First Affiliated Hospital of Soochow University

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

Terms used on this page · explanations by MedicalUpdates.ai

informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
glomerular filtration rate
A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
Exclusion criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
concomitant medication
Other medicines a participant takes during the study. Some must be reported; some are restricted.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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