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Study of Temozolomide, Topotecan, Bevacizumab, Irinotecan and Dinutuximab Beta in Patients with High-Risk Relapsed Neuroblastoma

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 treating high-risk relapsed neuroblastoma, a type of cancer that has returned after initial treatment or has progressed after being classified as high-risk at any point following diagnosis. The study aims to test new treatments against currently available options for patients whose cancer has returned. The trial will evaluate several medications including temozolomide, topotecan, bevacizumab, dinutuximab beta, and irinotecan.

The study is designed as a platform trial with multiple treatment arms, meaning different treatment combinations will be tested simultaneously. Some medications will be given by mouth, while others will be administered through an intravenous line (directly into the vein). The treatments will be given in cycles, and patients will be monitored for up to 12 treatment cycles.

During the study, doctors will track how well the treatments work by measuring if the cancer stops growing or shrinks, how long patients live without their disease getting worse, and how long they survive overall. They will also monitor side effects and assess quality of life using questionnaires. Patients will have regular medical examinations, including scans and blood tests, to evaluate their response to treatment.

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 medical assessment

    Your doctor will perform medical tests to confirm neuroblastoma diagnosis and evaluate your eligibility for the study

    Blood tests will check your liver function, blood count, and kidney function

    Blood pressure measurement must be performed

    Your overall physical condition will be assessed using a performance status scale

  2. Step 2

    Treatment assignment

    Based on your medical history, you will be assigned to one of two treatment tiers

    If this is your first relapse, you may be eligible for Tier 1

    If you have had more than one relapse, you will be assigned to Tier 2

  3. Step 3

    Treatment administration

    You will receive a combination of medications that may include:

    Temozolomide - taken by mouth

    Topotecan - given through an intravenous (IV) line

    Dinutuximab beta - given through an IV line

    Bevacizumab - given through an IV line

    Irinotecan - given through an IV line

    The treatment will continue for up to 12 cycles

  4. Step 4

    Regular monitoring

    Your disease will be monitored regularly using imaging scans

    Blood tests will be performed to check your health status

    Side effects will be monitored and recorded

    Quality of life assessments will be conducted using questionnaires

  5. Step 5

    Treatment completion

    The study will evaluate how long you remain free from disease progression

    Your overall survival will be monitored

    The effectiveness of the treatment will be assessed based on your response to therapy

    Follow-up assessments will continue after treatment completion

Who can join the trial?

12 criteria

  • Age must be 1 year or older
  • Must have confirmed high-risk neuroblastoma that has returned or gotten worse after previous treatment
  • Disease must be measurable through imaging scans or special tests (called MIBG scan). Disease detected only in bone marrow is not eligible
  • Must have adequate physical function with a performance score of at least 50% (ability to perform daily activities)
  • Expected survival time of at least 12 weeks

  • Must have adequate blood test results:

    • Blood platelets at least 50,000 per microliter without transfusions
    • White blood cells (neutrophils) at least 500 per microliter without medication support
    • Hemoglobin above 8 g/dL (blood transfusions allowed)
  • Liver function must be within acceptable limits (blood tests required within 72 hours before starting)
  • Kidney function must be within acceptable limits (tests required within 72 hours before starting)
  • Blood pressure must be below the 95th percentile for age and sex. For adults (18+ years), blood pressure must be below 150/90 mmHg
  • Must not have uncontrolled blood clotting problems (patients on blood thinners are allowed if stable)
  • Must have experienced more than one relapse or be ineligible for Tier 1 treatment
  • Written informed consent must be provided by the patient, parent, or guardian

Who cannot join the trial?

12 criteria

  • Previous participation in any other clinical trial within 30 days before starting this study
  • Having any serious medical conditions that could interfere with study participation, including:
    • Severe heart problems
    • Serious liver dysfunction
    • Severe kidney problems
    • Known allergic reactions to similar medications
    • Current pregnancy or breastfeeding
    • Inability to follow study procedures
    • Having received other cancer treatments within the last 4 weeks
    • Active, uncontrolled infections requiring treatment
    • Major surgery within 4 weeks before starting the study
    • Presence of any condition that the study doctor believes would make participation unsafe
    • Mental conditions that could affect the ability to provide informed consent or follow study instructions
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Investigated drugs

  • Temozolomide

    is a chemotherapy medicine taken by mouth. It works by stopping cancer cells from growing and dividing. In this trial it is given to try to keep the neuroblastoma from getting worse again after it has come back.

  • Topotecan

    is a chemotherapy drug given through an IV (a needle placed in a vein). It interferes with the DNA of cancer cells, which can stop them from multiplying. The study uses it to see if it can improve the time before the disease progresses.

  • Irinotecan

    is another IV chemotherapy medication. It blocks an enzyme that cancer cells need to copy their DNA, leading to cell death. Researchers are testing it as part of new treatment combinations for relapsed neuroblastoma.

  • Kimozo

    is an oral suspension (a liquid you can swallow) that contains temozolomide. It is used for patients who have trouble swallowing pills. Like regular temozolomide, it aims to stop cancer cells from growing when taken by mouth.

  • Bevacizumab

    is a targeted therapy given by IV infusion. It works by blocking a protein that helps tumors grow new blood vessels, which can starve the cancer of nutrients. The trial includes it to see if cutting off the tumor’s blood supply helps control the disease.

  • Qarziba

    is an IV infusion of dinutuximab beta, a type of immunotherapy. It helps the body’s immune system recognize and attack neuroblastoma cells. In the study it is tested to determine if it can improve outcomes for children whose cancer has returned.

What is already known about the treatment

  • Temozolomide

    This drug is taken by mouth, often as a capsule or oral suspension, and works by adding a small chemical group to DNA which stops cancer cells from copying themselves. It is an established chemotherapy medicine used mainly for brain tumors and some skin cancers, and it is being tested for relapsed neuroblastoma. Temozolomide belongs to the alkylating agent class of drugs, which damage DNA to kill rapidly growing cells. Its safety and effectiveness are well documented in medical literature, and it is approved in many countries for cancer treatment.

  • Topotecan

    Topotecan is given through an IV line and works by blocking an enzyme called topoisomerase I, which normally helps untangle DNA during cell division; this blockage causes cancer cells to break and die. It is approved for ovarian and small‑cell lung cancers and is now being studied for use in high‑risk relapsed neuroblastoma. The medication is classified as a topoisomerase I inhibitor chemotherapy. Clinical experience and research show it is an effective, though sometimes toxic, option for several adult cancers.

  • Irinotecan

    Irinotecan is administered intravenously and also blocks topoisomerase I, leading to DNA damage and death of cancer cells. It is approved for colorectal cancer and is being explored as part of combination regimens for relapsed neuroblastoma. This drug belongs to the topoisomerase I inhibitor class of chemotherapy agents. Its use is supported by extensive trial data and it is widely available in oncology practice.

  • Bevacizumab

    Bevacizumab is given as an IV infusion and works by attaching to a protein called VEGF, which tumors need to grow new blood vessels; by blocking VEGF, the drug starves the tumor of blood supply. It is an approved treatment for several adult cancers such as colorectal, lung, and kidney cancers and is being evaluated for neuroblastoma. The medication is classified as an anti‑angiogenic monoclonal antibody. Safety and efficacy have been well described in many clinical studies, leading to its worldwide approval.

  • Dinutuximab beta

    Dinutuximab beta is delivered by IV infusion and targets a molecule called GD2 that is found on most neuroblastoma cells, marking them for attack by the immune system. It is an approved immunotherapy for high‑risk neuroblastoma after standard chemotherapy and is included in the trial to improve outcomes in relapsed disease. This drug belongs to the anti‑GD2 monoclonal antibody class. Its clinical benefit and safety profile are documented in pediatric oncology guidelines.

Investigated diseases

Neuroblastoma - A rare type of cancer that develops from immature nerve cells, typically starting in the adrenal glands but can develop in other areas of the body. The disease most commonly occurs in children under the age of 5 years. Neuroblastoma can start in different places along the sympathetic nervous system, which controls body functions like heart rate, blood pressure, and digestion. The cancer cells can form solid tumors that grow and spread to other parts of the body, including bone marrow, bones, lymph nodes, liver, and skin. High-risk neuroblastoma refers to cases where the cancer has unfavorable biological features or has spread widely in the body.
Trial detailsLast updated 21 Oct 2026
Age18+ yearsPhasePhase ITrial ID2024-516115-24-00Protocol codeRG_22-136Estimated enrolment154 patientsSponsorThe University Of Birmingham

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