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A study of ruxolitinib as first treatment for children with haemophagocytic lymphohistiocytosis (HLH)

Verified siteRegistered drugNo placebo
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What is this trial about?

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

This study focuses on haemophagocytic lymphohistiocytosis (HLH), a severe condition affecting the immune system in children where the body produces too many activated immune cells. The study will evaluate the effectiveness of ruxolitinib (also known as Jakavi) when used as a first treatment option along with corticosteroids in children who have primary HLH.

The medication being tested, Jakavi, comes in tablet form in different strengths (5 mg, 10 mg, 15 mg, and 20 mg) and is taken by mouth. The purpose of this research is to determine how well patients survive until they can receive a Haematopoietic Stem Cell Transplantation, which is a procedure where damaged bone marrow is replaced with healthy cells.

The treatment period will last up to 8 weeks, during which patients will receive daily doses of the medication. The maximum daily dose that may be given is 100 mg, and the total amount of medication received during the entire study will not exceed 5,750 mg. This is a Phase 2 study that will help researchers understand how effective this treatment approach is for children with this serious condition.

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

    Initial treatment phase

    You will receive Jakavi (ruxolitinib) tablets in combination with corticosteroids as the first treatment for your condition

    The medication will be taken by mouth (oral route)

    Jakavi comes in different strengths: 5 mg, 10 mg, 15 mg, and 20 mg tablets

  2. Step 2

    Treatment duration

    The treatment will continue until you are ready for Hematopoietic Stem Cell Transplantation (HSCT), which is a procedure to replace damaged bone marrow with healthy cells

    Your doctor will monitor your progress throughout the treatment period

  3. Step 3

    Safety measures

    If you are of childbearing age, you must use effective contraception during the study

    Male participants must continue contraception for 90 days after the end of study

    Female participants must continue contraception for 30 days after the end of study

  4. Step 4

    Study completion

    The study is expected to run until March 2028

    Your participation will end when you proceed to stem cell transplantation or if other factors require discontinuation of treatment

Who can join the trial?

15 criteria

  • Age between 0 to 22 years
  • Must have confirmed HLH syndrome through either:
    • Confirmed genetic diagnosis showing predisposition to primary HLH (including abnormal protein expression or family history), or
    • At least 5 of these conditions:
      • Fever
      • Enlarged spleen
      • Low blood cell counts in at least two types of blood cells
      • High levels of fats in blood or low blood clotting proteins
      • Presence of certain cells consuming other blood cells
      • Decreased immune system function
      • High levels of ferritin (an iron-storing protein) in blood
      • Presence of activated immune cells
      • Must not have received any previous treatment specifically for HLH syndrome
      • If able to have children, must agree to use effective birth control during the study and:
        • For males: continue for 90 days after study ends
        • For females: continue for 30 days after study ends
        • Must provide written informed consent (parent/guardian consent for minors)
        • Must have social security coverage

Who cannot join the trial?

12 criteria

  • Patients who have already received treatment for haemophagocytic lymphohistiocytosis (HLH) other than corticosteroids
  • Patients younger than 2 years or older than 18 years of age
  • Patients with severe liver dysfunction (poor liver function that significantly affects daily activities)
  • Patients with active infections that are not controlled by treatment
  • Patients who have received organ transplants
  • Patients with known allergies to Ruxolitinib or similar medications
  • Patients with severe heart conditions that could affect their ability to participate safely
  • Pregnant or breastfeeding females
  • Patients currently participating in other clinical trials
  • Patients with significant kidney problems (poor kidney function that requires special medical care)
  • Patients unable to follow study procedures or attend scheduled visits
  • Patients with other serious medical conditions that could interfere with the study treatment
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Investigated drugs

  • Ruxolitinib

    is a medication that helps control the immune system's response. It belongs to a group of drugs called JAK inhibitors. In this trial, it's being tested as a first-line treatment for children with a rare condition affecting the immune system. The medication works by reducing inflammation and controlling overactive immune responses in the body.

  • Corticosteroids

    are anti-inflammatory medications that help reduce inflammation and suppress immune system activity. They are commonly used to treat various inflammatory conditions and autoimmune disorders. In this trial, they are used in combination with Ruxolitinib to help control the immune system's response.

What is already known about the treatment

Ruxolitinib – An oral medication administered in tablet form that belongs to the class of JAK (Janus Kinase) inhibitors, currently used in clinical trials for treating haemophagocytic lymphohistiocytosis (HLH) in children. The drug works by blocking specific enzymes called JAK1 and JAK2, which are involved in inflammation and immune system responses, helping to control the overactive immune response characteristic of HLH. Ruxolitinib is being studied as a first-line treatment in combination with corticosteroids, aiming to improve survival rates in children with HLH until they can receive stem cell transplantation, representing a potentially promising therapeutic approach in managing this serious condition.

Investigated diseases

Haemophagocytic Lymphohistiocytosis (HLH) - A severe inflammatory condition where the immune system becomes overactive and damages the body's tissues and organs. The condition occurs when certain white blood cells (histiocytes and lymphocytes) build up in organs like the liver, spleen, and bone marrow. These cells begin to destroy other blood cells, leading to organ enlargement and potential dysfunction. In children, the disease can develop rapidly, causing fever, enlarged liver and spleen, and decreased blood cell counts. HLH can be inherited (primary) or triggered by infections, autoimmune conditions, or other diseases (secondary).
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-516105-23-01Estimated enrolment20 patientsSponsorAssistance Publique Hopitaux De Paris

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