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Study on Melphalan and Drug Combination for Low and Intermediate Risk Neuroblastoma Patients

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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 neuroblastoma, a type of cancer that most commonly affects children. The trial aims to improve the survival of patients with low and intermediate risk neuroblastoma by using a variety of treatments. The treatments being studied include several medications: Melphalan, Dinutuximab Beta, Filgrastim, Vindesine Sulfate, Vincristine Sulfate, Busulfan, Cyclophosphamide, Etoposide, Carboplatin, Dacarbazine, Ifosfamide, Cisplatin, Clonazepam, Lenograstim, Mesna, Doxorubicin Hydrochloride, and etoposide phosphate. These medications are used in different forms such as infusions or injections, and some are specifically designed to protect the bladder or support the immune system.

The purpose of the study is to determine the best treatment approach for patients by assessing their risk through gene expression analysis. This means that the study will look at how genes are expressed in the cancer cells to decide on the most suitable treatment plan. The trial will involve different groups of patients who will receive either intensified or reduced treatment based on their risk level. The study will monitor the patients over time to see how well they respond to the treatment and to check for any side effects.

Throughout the study, researchers will track the time from diagnosis to any significant events, such as the cancer returning or worsening. They will also look at overall survival rates and other factors that might affect the outcomes, like the age at diagnosis and specific genetic changes in the cancer cells. The trial will compare the results with historical data to understand the effectiveness of the treatments and the impact of any side effects. This research is expected to continue until 2035, with patient recruitment starting in 2024.

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 trial

    Upon joining the clinical trial, you will be informed about the study's purpose, procedures, and potential risks and benefits. You will be asked to provide informed consent, which means you agree to participate after understanding all the information provided.

  2. Step 2

    Initial assessment

    An initial assessment will be conducted to confirm your eligibility for the trial. This may include a review of your medical history, physical examination, and laboratory tests to ensure you meet the criteria for participation.

  3. Step 3

    Treatment allocation

    Based on the results of your initial assessment, you will be assigned to a treatment group. The treatment is determined by a process called risk stratification, which uses gene expression analysis to decide the most appropriate treatment plan for you.

  4. Step 4

    Treatment phase

    During the treatment phase, you will receive medications as part of your treatment plan. These may include drugs such as melphalan, dinutuximab beta, filgrastim, and others. The medications will be administered through different methods, such as intravenous infusion or subcutaneous injection. The dosage, frequency, and duration of each medication will be explained to you by the medical team.

  5. Step 5

    Monitoring and follow-up

    Throughout the trial, your health and response to treatment will be closely monitored. This includes regular check-ups, blood tests, and imaging studies to assess the effectiveness of the treatment and to identify any side effects. Adjustments to your treatment may be made based on these assessments.

  6. Step 6

    End of trial

    At the end of the trial, a final evaluation will be conducted to determine the overall outcome of your treatment. You will be informed about the results and any further steps that may be necessary for your care.

Who can join the trial?

10 criteria

  • Diagnosis of neuroblastoma, a type of cancer, confirmed by either:
    • Examination of tumor tissue under a microscope
    • Finding specific neuroblastoma cells in the bone marrow
    • High levels of certain substances (catecholamine metabolites like HVA and VMA) in blood or urine
    • MYCN not amplified: This means that the MYCN gene, which can affect cancer growth, is not present in high numbers.
    • Specific stages and ages at diagnosis:
      • Localized neuroblastoma (stages L1/L2 or stages 1-3) and age between 18 months and less than 21 years
      • Stage 4S or stage MS
      • Stage 4 or stage M and age less than 18 months
      • Stage L1/L2 and age less than 18 months with a return or worsening of neuroblastoma
      • Enough tumor tissue must be available from the initial diagnosis for further testing.
      • Consent must be given by guardians or the patient, depending on the patient's age and mental development.

Who cannot join the trial?

5 criteria

  • Patients with medical conditions other than low and intermediate risk neuroblastoma cannot participate.
  • Patients who are not within the specified age range for the study cannot participate.
  • Patients who are not part of the specified clinical trial groups cannot participate.
  • Patients who are not male or female cannot participate.
  • Patients who are not considered part of a vulnerable population cannot participate.
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Investigated drugs

  • Cyclophosphamide

    is a medication used in this trial to help treat neuroblastoma, a type of cancer. It works by slowing or stopping the growth of cancer cells in the body. Cyclophosphamide is often used in combination with other cancer treatments to improve their effectiveness.

  • Doxorubicin

    is another medication involved in the trial. It is used to treat various types of cancer, including neuroblastoma. Doxorubicin works by interfering with the DNA inside cancer cells, which prevents them from growing and dividing. This helps to reduce the size of the tumor and slow the progression of the disease.

  • Vincristine

    is a medication that is part of the treatment plan in this trial. It is used to treat neuroblastoma by stopping the cancer cells from dividing and spreading. Vincristine is often used in combination with other medications to enhance its cancer-fighting effects.

  • Carboplatin

    is included in the trial as a treatment option for neuroblastoma. It works by damaging the DNA of cancer cells, which prevents them from multiplying. This helps to control the growth of the tumor and can be an important part of the overall treatment strategy.

  • Etoposide

    is a medication used in this trial to treat neuroblastoma. It works by blocking the action of an enzyme that cancer cells need to divide and grow. By doing so, etoposide helps to slow down or stop the progression of the cancer.

What is already known about the treatment

  • Cyclophosphamide

    Cyclophosphamide is a well-established chemotherapy medicine that in this trial is given as an infusion into a vein and also as tablets taken by mouth. It has been used for decades to treat many cancers, including childhood tumours such as neuroblastoma, as well as lymphomas and leukaemias. It belongs to the group of alkylating agents, which means that inside the body it is converted into an active form that attaches to the DNA of cancer cells, damaging it so the cells can no longer divide and eventually die. Because fast-growing cells are most sensitive to this damage, the drug mainly affects tumour tissue.

  • Cisplatin

    Cisplatin is a platinum-based chemotherapy medicine given as an infusion into a vein. It is one of the most widely used anticancer drugs worldwide and is a standard treatment for many solid tumours, including childhood cancers such as neuroblastoma. The platinum atom in the drug binds to the DNA of cancer cells and creates cross-links that block the copying of genetic material, which stops cell division and triggers cell death. It is classified as an alkylating-like (platinum compound) chemotherapy agent.

  • Etoposide

    Etoposide is a chemotherapy medicine given as an infusion into a vein, sometimes in the form of a water-soluble version called etoposide phosphate that works in the same way. It is firmly established in cancer medicine and is used for neuroblastoma, lung cancer, leukaemias and several other tumours. It blocks an enzyme called topoisomerase II, which cancer cells need to untangle their DNA during division; when the enzyme is blocked, the DNA breaks and the cell dies. It belongs to the group of medicines known as topoisomerase inhibitors derived from the plant substance podophyllotoxin.

  • Ifosfamide

    Ifosfamide is a chemotherapy medicine administered as an intravenous infusion. It is a close relative of cyclophosphamide and is commonly used for childhood solid tumours, sarcomas and testicular cancer. As an alkylating agent, it is activated in the liver and then damages the DNA of rapidly dividing cells, preventing them from multiplying. Because it can irritate the bladder, it is usually given together with a protective medicine called mesna.

  • Dacarbazine

    Dacarbazine is a chemotherapy medicine given as an infusion into a vein. It has long been used to treat melanoma, Hodgkin lymphoma and some soft-tissue tumours, and it appears in combination regimens for childhood cancers. In the body it is converted into an active compound that attaches small chemical groups to the DNA of cancer cells, which stops them from dividing correctly and leads to their death. It is classified as an alkylating agent.

  • Vincristine

    Vincristine is a chemotherapy medicine, given as an injection or short infusion into a vein, that is derived from the periwinkle plant. It is a standard part of treatment for many childhood cancers, including neuroblastoma, leukaemias and lymphomas. It works by binding to a cell protein called tubulin, which prevents the formation of the tiny internal scaffolding (the mitotic spindle) that cells need in order to divide, so the cancer cells are stopped in the middle of division and die. It belongs to the group of vinca alkaloids.

Investigated diseases

Neuroblastoma – Neuroblastoma is a type of cancer that forms in certain types of nerve tissue, most often in the adrenal glands, but it can also develop in the neck, chest, abdomen, or spine. It primarily affects children, especially those under the age of five. The disease begins when neuroblasts, which are immature nerve cells, grow uncontrollably. As it progresses, neuroblastoma can spread to other parts of the body, including lymph nodes, bones, bone marrow, liver, and skin. The progression can vary significantly, with some cases showing spontaneous regression, while others may become more aggressive. The risk and progression are often assessed based on factors like age at diagnosis, stage of the disease, and genetic characteristics.
Trial detailsLast updated 22 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-517295-37-00Protocol codeUni-Koeln-4299Estimated enrolment231 patientsSponsorUniversity Of Cologne

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