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Study on Langerhans Cell Histiocytosis Treatment in Children and Adolescents Using Cladribine, Prednisolone, and Cytarabine

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 clinical trial focuses on studying Langerhans Cell Histiocytosis (LCH), a rare disease that can affect various parts of the body, including bones, skin, and organs. The study aims to find better ways to treat children and adolescents with this condition. The trial will use several medications, including Cladribine, Prednisolone, Fludarabine Phosphate, Cytarabine, Vinblastine Sulfate, Human Normal Immunoglobulin (IV), Mercaptopurine Monohydrate, Melphalan, Alemtuzumab, Indometacin, and Methotrexate. Some participants may receive a placebo as part of the study.

The purpose of the study is to reduce the risk of death in patients with multi-system LCH by quickly switching to alternative treatments if the first treatment does not work. The study will also explore whether extending or intensifying ongoing treatment can help reduce the chances of the disease coming back and prevent long-term problems. For patients with single-system LCH, the study will investigate if longer treatment can help prevent the disease from returning and reduce long-term issues.

Participants will follow a treatment plan that may include different combinations of the medications mentioned above. The study will last for a total of 24 months, with some treatments lasting for shorter periods. The goal is to achieve disease resolution, prevent reactivations, and minimize long-term consequences. The trial will also look at how well the treatments work and their safety. Participants will be monitored throughout the study to assess their response to the treatments and any side effects they may experience.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying the diagnosis of Langerhans Cell Histiocytosis (LCH) and ensuring that the participant is under 18 years of age.

    No prior systemic therapy should have been administered, and informed consent must be signed.

  2. Step 2

    First-line therapy

    Participants begin with a first-line therapy aimed at reducing mortality in multi-system LCH. The specific medications and dosages are determined based on individual needs and responses.

  3. Step 3

    Monitoring response

    The response to the first-line therapy is closely monitored. If there is no adequate response, an early switch to a salvage treatment may be considered.

  4. Step 4

    Continuation therapy

    For those with single-system LCH or specific lesions, continuation therapy is investigated. This involves either prolongation or intensification of treatment to reduce reactivations and late effects.

    The duration of this therapy can vary, with options for 6 or 12 months based on the specific condition.

  5. Step 5

    Second-line treatment

    For patients with non-risk organ involvement, a second-line treatment is provided. This includes a combination of Prednisolone, Cytarabine, and Vinblastine for 24 weeks.

    Following this, a continuation therapy is administered for a total of 24 months, which may involve Indomethacin or a combination of Mercaptopurine and Methotrexate.

  6. Step 6

    Regular follow-ups

    Regular follow-ups are scheduled to monitor the participant's health, response to treatment, and any side effects. These follow-ups are crucial for adjusting treatment plans as necessary.

  7. Step 7

    End of trial

    The trial is estimated to conclude by December 30, 2025. At the end of the trial, a final assessment is conducted to evaluate the overall health and outcomes for the participant.

Who can join the trial?

5 criteria

  • Diagnosis of Langerhans Cell Histiocytosis (LCH) confirmed by a tissue sample test.
  • Must be younger than 18 years old.
  • No previous treatment with medicines that affect the whole body.
  • Signed agreement to participate in the study, known as informed consent.
  • Must meet specific criteria for the group they are being considered for in the study.

Who cannot join the trial?

10 criteria

  • Patients who have not responded to the first-line therapy for **Langerhans Cell Histiocytosis** with involvement of risk organs cannot participate.
  • Patients with a medical condition that might interfere with the study treatment or procedures are excluded.
  • Patients who are unable to comply with the study protocol or follow-up procedures are not eligible.
  • Pregnant or breastfeeding women are not allowed to participate in the study.
  • Patients with a history of another significant medical condition that could affect the study results are excluded.
  • Patients who are currently participating in another clinical trial are not eligible.
  • Patients with known allergies to any of the study medications are excluded.
  • Patients with severe liver or kidney disease are not allowed to participate.
  • Patients with a history of drug or alcohol abuse that could interfere with the study are excluded.
  • Patients who have received certain treatments or medications that could affect the study results are not eligible.
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Investigated drugs

  • Prednisone

    is a medication that helps reduce inflammation in the body. It is often used to treat conditions where the immune system is overactive, such as in Langerhans Cell Histiocytosis (LCH). In this trial, it is part of the treatment plan to help manage symptoms and control the disease.

  • Cytarabine

    , also known as Ara-C, is a type of chemotherapy drug. It works by slowing or stopping the growth of cancer cells. In this trial, it is used as part of a combination therapy to treat patients with LCH, especially those who do not respond to the first line of treatment.

  • Vincristine

    is another chemotherapy medication used in this trial. It helps stop the growth of cancer cells by interfering with their ability to divide. It is used in combination with other drugs to treat LCH, particularly in patients who need a more aggressive treatment approach.

  • Indomethacin

    is a medication that reduces inflammation and pain. It is often used to treat conditions like arthritis. In this trial, it is used as part of a continuation therapy to help prevent the disease from coming back and to manage any long-term effects of LCH.

  • 6-Mercaptopurine

    , also known as 6-MP, is a medication that suppresses the immune system. It is used to treat certain types of cancer and autoimmune diseases. In this trial, it is part of a continuation therapy to help maintain disease control and prevent reactivation of LCH.

  • Methotrexate

    is a medication that can slow down the growth of cancer cells and suppress the immune system. It is used in various treatments for cancer and autoimmune diseases. In this trial, it is combined with other medications as part of a long-term treatment strategy to manage LCH and prevent the disease from returning.

What is already known about the treatment

  • Prednisone

    Prednisone is taken orally in the form of tablets and is used to treat Langerhans Cell Histiocytosis (LCH), a rare disease that can affect various organs in the body. It is a well-established medication in medical literature and is commonly used to reduce inflammation and suppress the immune system. At the molecular level, prednisone works by mimicking the effects of cortisol, a natural hormone, to decrease inflammation and immune responses. It is classified as a corticosteroid, which is a type of steroid hormone.

  • Cytarabine

    Cytarabine is administered through injection and is used in the treatment of Langerhans Cell Histiocytosis (LCH), particularly in cases where the disease affects multiple systems. It is a recognized medication in medical literature and is used to interfere with the growth of cancer cells. At the molecular level, cytarabine works by incorporating itself into the DNA of rapidly dividing cells, preventing them from multiplying. It is classified as an antimetabolite, which is a type of chemotherapy drug.

  • Vincristine

    Vincristine is given by injection and is used to treat Langerhans Cell Histiocytosis (LCH), especially when the disease involves multiple systems. It is a well-documented medication in medical literature and is used to stop the growth of cancer cells. At the molecular level, vincristine works by binding to proteins in the cell, preventing them from dividing and spreading. It is classified as a vinca alkaloid, which is a type of chemotherapy drug.

  • Indomethacin

    Indomethacin is taken orally in the form of capsules and is used in the treatment of Langerhans Cell Histiocytosis (LCH) to help reduce inflammation and pain. It is a known medication in medical literature and is used to relieve symptoms associated with inflammation. At the molecular level, indomethacin works by inhibiting the production of substances in the body that cause inflammation and pain. It is classified as a nonsteroidal anti-inflammatory drug (NSAID).

  • Mercaptopurine

    Mercaptopurine is taken orally in the form of tablets and is used in the treatment of Langerhans Cell Histiocytosis (LCH) to help prevent the disease from returning. It is a recognized medication in medical literature and is used to interfere with the growth of cancer cells. At the molecular level, mercaptopurine works by incorporating itself into the DNA of rapidly dividing cells, preventing them from multiplying. It is classified as an antimetabolite, which is a type of chemotherapy drug.

  • Methotrexate

    Methotrexate is taken orally or by injection and is used in the treatment of Langerhans Cell Histiocytosis (LCH) to help control the disease. It is a well-established medication in medical literature and is used to slow down the growth of cancer cells. At the molecular level, methotrexate works by inhibiting an enzyme needed for cell division, thereby preventing the cells from multiplying. It is classified as an antimetabolite, which is a type of chemotherapy drug.

Investigated diseases

Langerhans Cell Histiocytosis – Langerhans Cell Histiocytosis is a rare disease characterized by an abnormal proliferation of Langerhans cells, which are a type of dendritic cell that normally helps regulate the immune system. The disease can affect various organs, including bones, skin, lungs, and the central nervous system. In its progression, Langerhans Cell Histiocytosis can present as either a single-system disease, affecting one organ or system, or as a multi-system disease, involving multiple organs. The condition may lead to the formation of lesions or tumors in the affected areas, which can cause pain, swelling, or dysfunction of the involved organs. Over time, the disease may lead to chronic inflammation and tissue damage, potentially resulting in long-term complications. The course of the disease can vary significantly, with some cases resolving spontaneously while others may persist or recur.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IVTrial ID2024-512676-36-00Protocol code042011Estimated enrolment2 030 patientsSponsorSt. Anna Childrens Cancer Research Institute GmbH

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