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Study on Itacitinib for Treating Non-Severe Hemophagocytic Lymphohistiocytosis in Adults

Verified siteInvestigationalNo placebo
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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 called Hemophagocytic Lymphohistiocytosis (HLH), specifically in adults with a non-severe form of the disease. HLH is a rare disorder where the body's immune system becomes overly activated, leading to symptoms like fever, enlarged spleen, and low blood cell counts. The trial will use a medication called Itacitinib, which is taken in tablet form. Itacitinib is being tested to see if it can help improve the symptoms of HLH by reducing the excessive immune response.

The purpose of this study is to evaluate how well Itacitinib works in treating non-severe HLH. Participants in the trial will take Itacitinib and be monitored for changes in their symptoms. The study will look at how many participants experience a complete or partial improvement in their condition after 15 days of treatment. This will help researchers understand the effectiveness of Itacitinib in managing HLH symptoms.

Throughout the study, participants will receive regular check-ups to assess their health and the impact of the treatment. The trial is designed to gather important information about the potential benefits of Itacitinib for people with non-severe HLH, contributing to the development of better treatment options for this condition.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Enrollment

    The patient provides written informed consent to participate in the study.

    Eligibility is confirmed based on criteria such as age, medical condition, and absence of severe symptoms.

  2. Step 2

    Initial assessment

    The patient undergoes a comprehensive evaluation to document baseline clinical and biological symptoms.

    This assessment includes checking for symptoms like fever, enlarged spleen, and blood cell counts.

  3. Step 3

    Treatment initiation

    The patient begins treatment with Itacitinib, administered orally in tablet form.

    The dosage and frequency are determined by the study protocol, focusing on the treatment of non-severe Hemophagocytosis Lymphohistiocytosis (HLH).

  4. Step 4

    Treatment period

    The patient continues taking Itacitinib as prescribed, with regular monitoring of symptoms and side effects.

    The treatment aims to achieve a complete or partial response by day 15, as measured by improvements in clinical and biological symptoms.

  5. Step 5

    Evaluation at day 15

    The patient's response to the treatment is evaluated on day 15.

    The primary goal is to determine if there is a complete or partial response to the treatment.

  6. Step 6

    Follow-up

    The patient continues to be monitored for any changes in symptoms and overall health.

    Further assessments may be conducted to ensure the safety and effectiveness of the treatment.

Who can join the trial?

21 criteria

  • Patients must be **18 years or older**.
  • Patients should have **non-severe HLH** (a condition affecting the immune system) that is either new or has come back.
  • Patients should not have other diseases that need specific treatment and should not have severe symptoms or organ failure.
  • Patients must show **major criteria** such as:
    • **Cytological features of hemophagocytosis** (a condition where certain cells eat other blood cells).
    • **Fever**.
    • **Splenomegaly** (enlarged spleen).
    • Patients must show **minor criteria** such as:
      • **Adenopathies** (swollen lymph nodes).
      • **Cytopenia** affecting more than two types of blood cells, such as:
        • **Hemoglobin** less than 9 g/dl (a measure of red blood cells).
        • **Platelets** less than 100,000/mm3 (cells that help with blood clotting).
        • **Neutrophils** less than 1,000/mm3 (a type of white blood cell).
        • **Hypertriglyceridemia** (high levels of fats in the blood) more than 3 mmol/l and/or **hypofibrinogenemia** (low levels of a protein that helps blood clot) less than 1.5 g/l.
        • **Ferritin** more than 500 μg/l (a protein that stores iron).
        • Patients must have an **HScore** of 169 or higher (a score used to assess the likelihood of HLH).
        • Patients must be willing to provide **written informed consent** and agree to follow the study rules.
        • Patients with **systemic juvenile idiopathic arthritis** (a type of arthritis in children) are included if they have:
          • **Ferritin** more than 684 ng/mL.
          • Any two of the following:
            • **Platelet count** less than 181,000/mm3.
            • **ASAT** more than 48 UI/ml (a liver enzyme).
            • **Triglyceride** more than 4 mmol/L.
            • **Fibrinogen** less than 3.6 g/L.
            • Women of childbearing potential must have a **negative pregnancy test** and use reliable contraception during the study.
            • Patients must be **affiliated with or a beneficiary of a social security category**.

Who cannot join the trial?

3 criteria

  • Patients with severe forms of HLH cannot participate. HLH stands for Hemophagocytic Lymphohistiocytosis, a condition where the body's immune system becomes overly active.
  • Patients who are not adults are excluded. This means only individuals who are 18 years or older can participate.
  • Patients who are part of a vulnerable population are not eligible. Vulnerable populations include groups like pregnant women, children, or those unable to give consent.
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Investigated drugs

Itacitinib is a medication being studied for its effectiveness in treating non-severe Hemophagocytosis Lymphohistiocytosis (HLH). The trial aims to determine how well patients respond to this treatment by evaluating improvements in clinical and biological symptoms. The goal is to see if patients achieve a complete or partial response after 15 days of using Itacitinib.

What is already known about the treatment

Itacitinib – Itacitinib is administered orally in tablet form. It is currently being studied in clinical trials for its effectiveness in treating non-severe Hemophagocytic Lymphohistiocytosis (HLH) in adults. The main therapeutic indication for Itacitinib is to manage symptoms of HLH, a condition characterized by excessive immune activation. At the molecular level, Itacitinib works by inhibiting the Janus kinase (JAK) pathway, which plays a crucial role in the signaling processes of immune cells. It is classified as a JAK inhibitor, a type of medication that helps regulate the immune system's activity.

Investigated diseases

Hemophagocytic Lymphohistiocytosis (HLH) – Hemophagocytic Lymphohistiocytosis is a severe systemic inflammatory syndrome characterized by excessive immune activation. It involves the overproduction of immune cells and cytokines, leading to symptoms such as prolonged fever, enlarged liver and spleen, and cytopenias. The disease can be triggered by infections, malignancies, or autoimmune conditions. In non-severe cases, symptoms may be less intense but still require careful monitoring. The progression involves persistent inflammation and immune system dysregulation, which can affect multiple organs. Early recognition and management are crucial to prevent complications.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-513173-44-00Estimated enrolment70 patientsSponsorAssistance Publique Hopitaux De Paris

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