Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

Not yet recruitingRare disease

Study on Etoposide for Patients with Severe Hemophagocytic Lymphohistiocytosis in Intensive Care

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 called Hemophagocytic Lymphohistiocytosis (HLH), which is a severe 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 Etoposide, which is commonly used in cancer treatment, to see how it can help manage this condition when it becomes severe and requires intensive care.

The purpose of the study is to compare two different strategies for starting treatment with Etoposide in patients with severe HLH. One strategy involves starting the treatment early, within 12 hours of the patient being included in the study. The other strategy involves delaying the start of the treatment until 48 hours later, but only if the patient's condition does not improve. This approach will help researchers understand which timing is more effective in managing the condition and preventing further organ damage.

Participants in the study will be monitored closely to see how their condition progresses with each treatment strategy. The study will look at various outcomes, such as the time it takes for symptoms to improve, the number of days patients spend on ventilators, and the overall length of their hospital stay. The study aims to provide valuable insights into the best way to use Etoposide for treating severe HLH in intensive care settings.

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

    Joining the study

    Upon joining the study, the patient is confirmed to have a diagnosis of hemophagocytic lymphohistiocytosis (HLH) and is admitted to the intensive care unit.

    The patient must meet specific criteria, including having one or more organ failures and a confirmed first episode of HLH.

  2. Step 2

    Randomization

    The patient is randomly assigned to one of two treatment strategies for the initiation of etoposide, a medication used in the study.

  3. Step 3

    Early etoposide initiation

    In the early strategy, etoposide is administered within 12 hours after inclusion in the study.

    The medication is given through an intravenous (IV) injection or infusion.

  4. Step 4

    Delayed etoposide initiation

    In the delayed strategy, etoposide is administered only if there is an unfavorable evolution after 48 hours.

    The medication is given through an intravenous (IV) injection or infusion.

  5. Step 5

    Monitoring and assessment

    The patient's condition is monitored using the modified Sequential Organ Failure Assessment (SOFA) score to evaluate organ failures.

    The occurrence of an event is defined as the onset or worsening of organ failures, with an increase of at least 1 point for at least two organ systems compared to Day 0.

  6. Step 6

    Follow-up and outcomes

    The study tracks various outcomes, including time to death, number of ventilator-free days, and length of stay in the intensive care unit.

    The cumulative dose of etoposide over the first 14 days is recorded, along with other health indicators such as normalization of fibrinogen, decrease in ferritin, and decrease in triglycerides.

Who can join the trial?

14 criteria

  • The patient must be an adult.
  • The patient must have a confirmed diagnosis of **Hemophagocytic Lymphohistiocytosis (HLH)**. This means they must meet 5 or more of the following criteria:
    • Fever
    • **Splenomegaly**: This is when the spleen is larger than normal.
    • **Cytopenias**: This means low blood cell counts affecting 2 or 3 types of blood cells.
    • **Hypertriglyceridemia** or **Hypofibrinogenemia**: High levels of fats in the blood or low levels of a protein that helps blood clot.
    • **Hemophagocytosis**: This is when certain cells eat other blood cells, and it can be found in the bone marrow, spleen, or lymph nodes.
    • **Hyperferritinemia**: High levels of a protein that stores iron in the blood.
    • The diagnosis of HLH must be confirmed by the medical team taking care of the patient.
    • This must be the patient's first episode of HLH.
    • The patient must be admitted to an **intensive care unit (ICU)**, which is a special hospital department for very sick patients.
    • The patient must have one or more **organ failures**. This means:
      • **Circulatory failure**: Blood pressure is too low (mBP < 65 mmHg) with high lactate levels (> 2 mmol/L), or the patient needs medication called catecholamines to support blood pressure.
      • **Respiratory failure**: The patient needs more than 6 liters of oxygen per minute, or needs special breathing support like non-invasive ventilation, high-flow nasal cannula, or a machine to help them breathe.
      • **Renal failure**: The kidneys are not working well, shown by certain levels of creatinine in the blood or low urine output.
      • **Neurological failure**: The patient has a **Glasgow Coma Scale (GCS)** score of 13 or less, which means they have reduced consciousness.

Who cannot join the trial?

3 criteria

  • Patients who do not have Hemophagocytic lymphohistiocytosis (HLH) cannot participate. HLH is a condition where the body's immune system becomes overly active and attacks its own tissues.
  • Patients who are not within the specified age range cannot participate. The age range for this study is not specified in the provided data.
  • Patients who are part of a vulnerable population cannot participate. A vulnerable population includes groups like children, pregnant women, or those unable to give consent.
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 yet 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

Etoposide is a medication used in this trial to treat severe hemophagocytic lymphohistiocytosis (HLH), a condition where the body's immune system becomes overly active and attacks its own tissues. The trial is comparing two strategies for starting etoposide treatment in patients with this condition who are in intensive care. One strategy involves starting etoposide early, within 12 hours of inclusion in the trial, while the other strategy involves delaying the start of etoposide until 48 hours later if the patient's condition does not improve.

What is already known about the treatment

Etoposide – Etoposide is administered intravenously, typically in a hospital setting, especially for severe conditions like hemophagocytic lymphohistiocytosis (HLH). It is currently being studied in clinical trials to determine the best timing for its initiation in intensive care settings. The main therapeutic indication for etoposide is the treatment of certain types of cancer and severe HLH. At the molecular level, etoposide works by inhibiting an enzyme called topoisomerase II, which is essential for DNA replication and cell division, leading to cell death. It is classified as a topoisomerase inhibitor and is part of the chemotherapy drug class.

Investigated diseases

Hemophagocytic lymphohistiocytosis (HLH) – This is a rare disease characterized by an overactive immune system, leading to excessive inflammation. It involves the body's immune cells attacking its own tissues, causing damage to organs such as the liver, spleen, and bone marrow. Symptoms often include prolonged fever, enlarged liver or spleen, and low blood cell counts. As the disease progresses, it can lead to organ dysfunction due to the excessive immune response. HLH can be triggered by infections, autoimmune diseases, or genetic mutations. The condition requires careful monitoring to manage the immune system's activity and prevent further organ damage.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-511807-41-00Protocol codeAPHP230874Estimated enrolment130 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.