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

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 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

I'm sorry, but the provided data does not include the name of any specific medication involved in the clinical trial. Please provide the name of the medication so I can create a detailed description for you.

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 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-517295-37-00Protocol codeUni-Koeln-4299Estimated enrolment280 patientsSponsorUniversity Of Cologne

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