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Study of Naxitamab and Sargramostim for High-Risk Neuroblastoma in Patients with Refractory Bone or Bone Marrow Disease

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 type of cancer called high-risk neuroblastoma. Neuroblastoma is a cancer that most often affects children and develops from immature nerve cells found in several areas of the body. The study is specifically looking at patients who have a form of the disease that is either resistant to initial treatment or has not fully responded to additional treatments targeting the bone or bone marrow.

The trial will test a combination of two treatments: naxitamab and granulocyte-macrophage colony-stimulating factor (GM-CSF). Naxitamab is a type of medication known as a monoclonal antibody, which is designed to target and attach to specific cancer cells, helping the immune system to destroy them. GM-CSF is a protein that helps stimulate the growth of white blood cells, which are important for fighting infections and may help the body respond better to cancer treatment. Naxitamab is given as a solution for infusion, meaning it is administered directly into the bloodstream through a vein, while GM-CSF is given as a solution for injection under the skin.

The purpose of the study is to evaluate how well the combination of naxitamab and GM-CSF works in treating high-risk neuroblastoma. Participants in the study will receive these treatments over a period of time, and their response to the treatment will be closely monitored. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of the actual treatment. The study will also look at the safety of the treatment by monitoring any side effects that occur. The trial aims to provide valuable information on the effectiveness and safety of this treatment combination for patients with high-risk neuroblastoma.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the trial, an initial assessment is conducted to confirm eligibility. This includes verifying a documented diagnosis of high-risk neuroblastoma and ensuring the disease is present in bone or bone marrow.

    Eligibility criteria include a life expectancy of at least 6 months, age of at least 12 months, and acceptable blood, liver, and kidney function.

  2. Step 2

    Treatment initiation

    The treatment involves two main medications: naxitamab and sargramostim (also known as GM-CSF).

    Naxitamab is administered through an intravenous infusion, which means it is given directly into a vein.

    Sargramostim is given as a subcutaneous injection, which means it is injected under the skin.

  3. Step 3

    Treatment cycles

    The treatment is organized into cycles. Each cycle includes the administration of naxitamab and sargramostim according to the trial protocol.

    The frequency and duration of each cycle are determined by the trial's specific guidelines and the patient's response to treatment.

  4. Step 4

    Monitoring and evaluation

    Throughout the trial, regular monitoring is conducted to assess the response to treatment. This includes evaluating the objective response rate, which measures how well the cancer responds to the treatment.

    Safety is also monitored by tracking any adverse events or side effects experienced during the trial.

  5. Step 5

    Completion of trial

    The trial is expected to continue until April 2028, with ongoing assessments to determine the effectiveness and safety of the treatment.

    Upon completion, the results will contribute to understanding the treatment's impact on high-risk neuroblastoma.

Who can join the trial?

12 criteria

  • The patient must have a confirmed diagnosis of neuroblastoma, which is a type of cancer. This can be confirmed by a tumor biopsy, a bone marrow test showing neuroblastoma, or a special scan called MIBG that shows the cancer.
  • The patient should be considered high-risk with either a disease that hasn't responded to initial treatment or hasn't fully responded to additional treatment. The disease should be detectable in the bone or bone marrow. If it's only in the bone, it must be visible outside areas that have been treated with radiation. If it's only in the bone marrow, more than 5% of the marrow must be involved.
  • The patient should have a life expectancy of at least 6 months.
  • The patient must be at least 12 months old.
  • The patient should have an acceptable blood status, which means:
    • Hemoglobin (a protein in red blood cells) level of at least 8 g/dL.
    • White blood cell count of at least 1000 per microliter.
    • Absolute neutrophil count (a type of white blood cell) of at least 500 per microliter.
    • Platelet count of at least 25,000 per microliter.
    • The patient should have acceptable liver function, meaning:
      • Levels of liver enzymes (ALT and AST) should not be more than 5 times the normal limit.
      • Bilirubin (a substance made by the liver) should not be more than 1.5 times the normal limit.
      • The patient should have acceptable kidney function, which means an estimated filtration rate of more than 60 mL/min/1.73 m², calculated using a specific formula.
      • Written informed consent must be obtained from the patient's legal guardian(s) or the patient themselves, following local rules. If required, children must also agree to participate.

Who cannot join the trial?

8 criteria

  • Patients who have a different type of cancer than the one being studied cannot participate.
  • Patients who have not completed their previous cancer treatments cannot join the study.
  • Patients with severe or uncontrolled medical conditions that could interfere with the study cannot participate.
  • Patients who are pregnant or breastfeeding are not allowed to join the study.
  • Patients who have had another cancer within the last 5 years, except for certain skin cancers, cannot participate.
  • Patients who are unable to follow the study procedures or attend the required visits cannot join the study.
  • Patients who have allergies to the study drugs or similar medications cannot participate.
  • Patients who are currently participating in another clinical trial cannot join this study.
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Investigated drugs

  • Naxitamab (hu3F8)

    is an antibody used in this trial to help treat high-risk neuroblastoma, a type of cancer that occurs in certain types of nerve tissue. It works by targeting and binding to specific cancer cells, helping the immune system to recognize and destroy them.

  • Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF)

    is a therapy used to boost the immune system. It helps increase the number of white blood cells, which can improve the body's ability to fight cancer. In this trial, it is used in combination with naxitamab to enhance the treatment's effectiveness against neuroblastoma.

What is already known about the treatment

Naxitamab – Naxitamab is administered intravenously and is currently being studied in clinical trials for its effectiveness in treating high-risk neuroblastoma, particularly in patients who have not fully responded to initial treatments. It is an antibody that targets specific proteins on cancer cells, helping the immune system to identify and destroy these cells. Naxitamab is classified as a monoclonal antibody, and its mechanism involves binding to the GD2 antigen on neuroblastoma cells, facilitating immune-mediated cell destruction.

Investigated diseases

Neuroblastoma – Neuroblastoma is a type of cancer that most commonly affects children and develops from immature nerve cells found in several areas of the body. It often begins in the adrenal glands, but it can also develop in the neck, chest, abdomen, or spine. The disease can spread to other parts of the body, including the bone marrow, bones, lymph nodes, liver, and skin. Symptoms may vary depending on the location of the tumor and can include abdominal pain, a lump in the abdomen, or bone pain. As the disease progresses, it may cause fatigue, loss of appetite, and weight loss. Neuroblastoma is considered a high-risk condition when it does not respond well to initial treatments or when it recurs after treatment.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-508587-29-00Protocol code201Estimated enrolment122 patientsSponsorY-mAbs Therapeutics A/S

sourced from the EU Clinical Trials Register and site verification

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