Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

Not recruiting

Study on Ruxolitinib and Drug Combination for Critically Ill Adults with Acquired Hemophagocytic Syndrome

Verified siteRegistered drugNo placebo
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition known as acquired hemophagocytic syndrome (HS), which is a serious disorder where the body's immune system becomes overly active and starts attacking its own tissues. The study is investigating the use of a medication called ruxolitinib, which is a type of drug known as a JAK inhibitor. This medication is being tested to see if it can help improve the condition of patients with HS who are critically ill and admitted to the intensive care unit (ICU). The goal is to determine if ruxolitinib, when used alongside standard treatments, can better reverse organ failure compared to standard treatments alone.

In this study, patients will receive either ruxolitinib or a placebo, in addition to the usual care they would receive for HS. The treatment period for ruxolitinib is up to 28 days. Other medications involved in the study include lidocaine hydrochloride monohydrate, methylprednisolone acetate, etoposide, and betamethasone sodium phosphate, which are used for various supportive treatments. The study will monitor the patients' health, focusing on improvements in organ function and overall survival, as well as any changes in symptoms related to HS.

The trial will assess the safety of ruxolitinib in these patients and track various health indicators, such as temperature, blood cell counts, and levels of certain proteins in the blood that indicate inflammation. The primary measure of success will be a significant improvement in the patients' organ function scores after seven days of treatment. The study aims to provide valuable insights into the potential benefits of using ruxolitinib for treating critically ill patients with acquired hemophagocytic syndrome.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon meeting the eligibility criteria, which include being an adult over 18 years old with acquired hemophagocytic syndrome (HS) and admitted to the intensive care unit (ICU), participation in the study begins.

    Informed consent is required, either from the patient or a family member if the patient is unable to provide it.

  2. Step 2

    Treatment initiation

    The treatment involves the administration of ruxolitinib in combination with standard care. Ruxolitinib is provided in the form of Jakavi 5 mg tablets and is taken orally.

    The objective is to improve organ function as measured by the SOFA score, which assesses the extent of a patient's organ function or rate of failure.

  3. Step 3

    Monitoring and assessment

    The primary endpoint is survival with a decrease in the SOFA score by at least 3 points by day 7.

    Secondary endpoints include overall survival, changes in temperature, SOFA score, and various biological markers related to HS, such as ferritin levels and white blood cell count.

    Safety of ruxolitinib is also monitored throughout the study.

  4. Step 4

    Completion of the study

    The study is estimated to conclude by October 16, 2025.

    Upon completion, data regarding the effectiveness and safety of the treatment will be analyzed.

Who can join the trial?

6 criteria

  • Must be an adult older than 18 years.
  • Must have acquired hemophagocytic syndrome (HS), which is a condition diagnosed by specific criteria.
  • Must be admitted to the Intensive Care Unit (ICU).
  • Must need treatment for HS related to organ failure, as shown by a specific score called the SOFA score of 4 or higher. The SOFA score is a way to measure how well the organs in the body are working.
  • Must have informed consent signed by the patient or a family member/trustworthy person if the patient cannot sign due to their condition. In emergencies, consent can be obtained later when the patient is able.
  • If the patient is a woman who can have children, she must use a highly effective contraceptive method during the study and for one day after treatment ends.
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Not recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

Ruxolitinib is a medication being studied for its potential to help patients with acquired hemophagocytic syndrome, especially those who are critically ill. It is being tested to see if it can improve organ function when used alongside the usual treatments. The goal is to see if it can help patients recover better than with standard treatments alone.

What is already known about the treatment

Ruxolitinib – Ruxolitinib is administered orally in the form of tablets. It is currently being studied in clinical trials for its potential to improve outcomes in critically ill patients with acquired hemophagocytic syndrome (HS) when used alongside standard care. The main therapeutic indication for ruxolitinib in this context is to help reverse organ failure in these patients. At the molecular level, ruxolitinib works by inhibiting certain enzymes known as Janus kinases (JAKs), which play a role in the signaling pathways that can lead to inflammation and immune system activation. It is classified as a JAK inhibitor in pharmacology.

Investigated diseases

Hemophagocytic Syndrome – Hemophagocytic Syndrome (HS) is a severe systemic inflammatory condition characterized by excessive immune activation. It involves the overproduction of immune cells and cytokines, leading to widespread inflammation and tissue damage. The disease can cause symptoms such as prolonged fever, enlarged liver or spleen, and cytopenias, which are low blood cell counts. As the condition progresses, it can lead to organ dysfunction, including liver, kidney, and lung involvement. The progression of HS is often rapid, requiring close monitoring and management.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-504513-77-00Protocol codeAPHP220919Estimated enrolment42 patientsSponsorAssistance Publique Hopitaux De Paris

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.