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Neuroblastoma – Trials in Disease

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Clinical Trials for Neuroblastoma: Current Research and Treatment Options

Neuroblastoma, a cancer that primarily affects children and develops from immature nerve cells, is the focus of 21 ongoing clinical trials across Europe and beyond. These studies are investigating various treatment approaches, including immunotherapies, chemotherapy combinations, stem cell transplantation, and innovative imaging techniques. Trials are currently recruiting patients in multiple countries, offering opportunities for children and young adults with newly diagnosed, high-risk, relapsed, or treatment-resistant neuroblastoma to access cutting-edge therapeutic options.

Clinical trial locations

Long-term safety study of GD2IL18CART treatment in patients with neuroblastoma, osteosarcoma, Ewing sarcoma, or advanced breast cancer

This study monitors the long-term safety of patients who have previously received GD2IL18CART cell therapy for several types of cancer, including neuroblastoma. The trial is specifically designed for follow-up monitoring rather than initial treatment.

Main inclusion criteria: Patients must have previously received GD2IL18CART treatment in the primary trial. They should be between 2 and 64 years of age and have one of the following conditions: neuroblastoma, osteosarcoma, Ewing sarcoma, or advanced breast cancer. Informed consent from the patient or caregiver is required.

Main exclusion criteria: The trial excludes pregnant or breastfeeding women, people with active autoimmune diseases, those with active or uncontrolled infections, and patients with severe heart, liver, or kidney problems. Individuals with active brain metastases that are not controlled, those who received other experimental treatments within 30 days, patients with inadequate bone marrow function, and those with known allergies to study treatment components are also excluded.

Study focus: The main goal is to understand the long-term safety of GD2IL18CART treatment by tracking any delayed side effects over time, including potential infections, cancer returning, or new health problems. The study involves regular check-ups to monitor health status, and for younger patients, growth and development are also assessed. Blood tests check for the presence of treatment cells, and the research team collects information about overall health and the treatment’s long-term effects.

Investigational treatment: GD2IL18CART is a type of cell therapy where a patient’s own immune cells (T cells) are modified in a laboratory to target a specific protein called GD2 found on cancer cells, enhanced with an IL18 component to boost immune response.

Study Comparing [18F]meta-fluorobenzylguanidine PET-CT and Iodine (123I) Iobenguane Scans for Detecting Neuroblastoma in Patients

This trial compares two different imaging methods for detecting neuroblastoma in the bones. The first method uses [18F]meta-fluorobenzylguanidine (also known as [18F]mFBG) in a PET-CT scan, while the second uses iodine (123I) iobenguane (also known as [123I]mIBG) in a SPECT-CT scan.

Main inclusion criteria: Patients must have a clinical suspicion of neuroblastoma or be referred for [123I]mIBG imaging. Written informed consent from the patient and/or parents or legal guardians is required, following local laws and regulations. The study is open to both male and female patients.

Main exclusion criteria: Patients without confirmed or suspected neuroblastoma cannot participate. Those outside the specified age range, not meeting gender requirements, or considered part of a vulnerable population may not be eligible.

Study focus: The trial aims to compare how well these two imaging methods detect cancer in the bones. Participants will undergo both types of scans, and results will be compared to determine which method is more effective at finding cancerous lesions. The study will also evaluate radiation absorption and the number of detected cancerous areas.

Investigational imaging agents: [18F]mFBG is used in PET-CT scans to help detect skeletal lesions in patients with neuroblastoma. [123I]mIBG is used in SPECT-CT scans as a conventional imaging method to visualize the spread of cancer in the body, especially in the bones.

Study of ABTL0812, Irinotecan, and Temozolomide for Children with Relapsed or Refractory Neuroblastoma and Other Solid Tumors

This trial explores treatment for children with relapsed or refractory neuroblastoma and other solid tumors using ABTL0812, a medication taken as a capsule, both alone and in combination with chemotherapy treatments including irinotecan (given by injection) and temozolomide (taken as a capsule).

Main inclusion criteria: Patients must be between 1 and 21 years old, able to swallow capsules, and have signed informed consent. They must have a solid tumor that has progressed, relapsed, or is refractory to at least one standard therapy, with at least one measurable site of disease. Adequate performance status and organ function are required, including specific blood count levels, normal kidney function (serum creatinine levels or creatinine clearance rate above 70 mL/min/1.73m2), and liver function within acceptable limits.

Main exclusion criteria: The trial excludes patients who have not experienced a return or worsening of their neuroblastoma or other solid tumors, those not within the specified age range, patients unable to take the study medication or combination, those unable to follow study procedures or attend required visits, and patients who are part of a vulnerable population not designed for the study.

Study focus: The purpose is to determine the best dose of ABTL0812 for children, both when used alone and when combined with other treatments. The study involves several phases, starting with a small group to find the safest and most effective dose, then expanding to include more participants. Throughout the trial, participants receive study medication and are monitored by healthcare professionals to assess effectiveness and identify any side effects.

Investigational drugs: ABTL0812 is an experimental medication being tested for its potential to inhibit cancer cell growth and improve treatment outcomes. Irinotecan is a chemotherapy drug that interferes with cancer cell DNA, preventing multiplication and spreading. Temozolomide is another chemotherapy drug that damages cancer cell DNA, leading to their death and helping reduce tumor size.

Study of Crizotinib and Temsirolimus for Children with ALK, ROS1, or MET Positive Cancers, Including Neuroblastoma and Rhabdomyosarcoma

This trial studies certain types of cancer in children that are positive for ALK, ROS1, or MET genes, including neuroblastoma, rhabdomyosarcoma, anaplastic large cell lymphoma, inflammatory myofibroblastic tumors, and other related malignancies. The treatment being tested is crizotinib, used alone or in combination with temsirolimus.

Main inclusion criteria: Patients must have a confirmed diagnosis of specific cancers such as neuroblastoma or rhabdomyosarcoma with ALK, ROS1, or MET positive genetic changes. Age must be between 1 and 21 years, with a performance status score above 60%. Patients must have measurable disease and specific genetic changes detected through special tests, a life expectancy of at least 12 weeks, and have completed previous cancer treatments at least 2 weeks before starting the study. They must not have had previous treatments directly targeting ALK, ROS1, or MET genes, or taken investigational drugs or had major surgery in the past 2 weeks.

Main exclusion criteria: The study excludes patients with different types of cancer not listed, those not within the specified age range, patients unable to follow study procedures, those with medical conditions that could interfere with treatment, pregnant or breastfeeding patients, participants in other clinical trials, those with recent surgery or medical procedures affecting the study, patients with allergies to study medications, those with certain heart conditions, uncontrolled infections, history of substance abuse, or inability to provide consent.

Study focus: The study aims to find the best dose of crizotinib when used with temsirolimus and assess the safety and initial effectiveness of crizotinib alone in treating gene-positive tumors. Participants receive either crizotinib alone or in combination with temsirolimus, depending on their cancer type and genetic markers. Throughout the study, regular monitoring assesses body response and any side effects.

Investigational drugs: Crizotinib is a medication that targets specific proteins in cancer cells to help stop their growth and spread. It is available in capsule form and oral solutions. Temsirolimus is combined with crizotinib to evaluate if the combination is more effective than crizotinib alone, working by blocking a protein that helps cancer cells grow.

Study on Dinutuximab Beta with Chemotherapy for Children and Teens with Newly Diagnosed High-Risk Neuroblastoma

This trial tests a treatment combining dinutuximab beta, a type of immunotherapy that helps the body’s immune system fight cancer, with various chemotherapy drugs for newly diagnosed high-risk neuroblastoma. The chemotherapy drugs include dacarbazine, etoposide, cisplatin, vincristine sulfate, ifosfamide, carboplatin, doxorubicin hydrochloride, and cyclophosphamide.

Main inclusion criteria: Patients must have confirmed diagnosis of neuroblastoma Stage M, be at least 18 months old and younger than 18 years, weigh more than 12 kilograms, and have liver function tests (ALT and AST) within specific limits.

Main exclusion criteria: The trial excludes patients not diagnosed with high-risk neuroblastoma, those younger than 18 months or older than 18 years, patients unable to safely receive study treatments due to other health conditions, pregnant or breastfeeding patients, recent participants in other clinical trials, those with allergies to study medications, patients with certain infections or diseases that could interfere, and those unable to follow study procedures.

Study focus: The purpose is to determine how safe and tolerable the combination of dinutuximab beta and chemotherapy is for patients, and to establish the best dose to use. Patients receive treatment in cycles with periods of medication followed by rest periods, closely monitored for side effects and cancer response.

Investigational drugs: Dinutuximab beta is used to treat high-risk neuroblastoma, being tested for safety and tolerability when combined with chemotherapy. Induction chemotherapy regimens (GPOH or rapid COJEC) are combinations of chemotherapy drugs designed to shrink tumors and kill cancer cells, tested with dinutuximab beta to see if they work better together.

Study on Stem Cell Transplantation and Immunotherapy with Rituximab, Dinutuximab Beta, and Drug Combination for Patients with High-Risk Relapsed Neuroblastoma

This trial is designed for patients with high-risk neuroblastoma whose disease has returned or is not responding to initial treatments. It evaluates the safety and effectiveness of haploidentical stem cell transplant with alfa-beta-CD19+ depletion, followed by immunotherapy including donor-derived NK cell infusion and anti-GD2 monoclonal antibody.

Main inclusion criteria: Patients must have high-risk neuroblastoma (stage 4 and older than 12 months at first diagnosis, or stage 2, 3, or 4s with MYCN amplification) that has come back or gotten worse after first treatment. Disease must be stable or better after receiving additional chemotherapy. A suitable family member who can donate haploidentical stem cells is required, able to donate from blood after receiving GCSF (and possibly Plerixafor). Parents or legal guardians must provide informed consent.

Main exclusion criteria: Patients not diagnosed with high-risk neuroblastoma that has returned or worsened, those not within the specified age range, patients not meeting specific health and medical conditions, and those unable to follow study procedures or give consent cannot participate.

Study focus: The study monitors various outcomes including overall survival, how well donor cells take hold in the patient’s body (engraftment), and any treatment-related side effects. The trial uses several medications administered as solutions for infusion, including Rituximab, Dinutuximab beta, Melphalan, Thiotepa, Fludarabine phosphate, and Anti-T lymphocyte immunoglobulin.

Investigational treatments: Haploidentical stem cell transplantation uses partially matched donor cells to help rebuild the immune system and fight cancer. Donor-NK cell infusion involves infusing natural killer cells from a donor to help attack cancer cells. Anti-GD2 monoclonal antibody is an immunotherapy using antibodies to target and bind to GD2 on neuroblastoma cells, helping the immune system recognize and attack cancer cells.

Study on the Safety and Dosage of GD2IL18CART for Patients with Relapsed or Refractory GD2 Positive Solid Cancers

This trial tests GD2IL18CART, a cell therapy where a patient’s own T cells are modified to target cancer cells, for patients with neuroblastoma, Ewing sarcoma, osteosarcoma, and advanced breast cancer that have returned or are not responding to standard treatments.

Main inclusion criteria: Patients must have relapsed or refractory neuroblastoma, Ewing sarcoma, osteosarcoma, or advanced breast cancer that cannot be cured with standard treatments. For advanced breast cancer, specific prior treatment requirements apply based on hormone receptor, HER2, or triple negative status. For neuroblastoma, Ewing sarcoma, and osteosarcoma, patients must have received specific prior therapies. GD2 expression must be present on tumor cells. Patients must be between 1 and 79 years old, have adequate CD3+ T cells, appropriate performance scores, and for females of childbearing potential, a negative pregnancy test. Written informed consent is required before any trial procedures.

Main exclusion criteria: The trial excludes patients with severe allergic reactions to similar treatments, those currently pregnant or breastfeeding, patients with uncontrolled infections or serious health conditions, those who received experimental treatment within 30 days, patients with certain heart problems or active autoimmune diseases, those unable to follow study procedures, patients with substance abuse history, and those who had major surgery within 4 weeks.

Study focus: The purpose is to determine the appropriate dose and assess how well GD2IL18CART works. Participants receive treatment through intravenous infusion and are monitored for side effects and cancer response using PET/CT scans. The study tracks the cancer’s response at week 12 and follows patients for a year to monitor treatment persistence and overall survival.

Investigational therapy: GD2IL18CART is a therapy using modified immune cells designed to target cancer cells with the GD2 marker, tested for safe and effective dosing in treating solid cancers that have not responded to other treatments.

Study on the Safety of 68Ga-SATO and Iodine (123I) Iobenguane in Children with Neuroblastoma

This trial explores the use of 68Ga-SATO, a solution for injection containing satoreotide trizoxetan gallium (Ga68), in children with neuroblastoma. The study assesses the short-term safety and tolerability of this new treatment.

Main inclusion criteria: Patients must be between 0 and 18 years old with written informed consent from a legal representative (and assent consent if applicable). They should have clinical suspicion of neuroblastoma and be referred for first-time conventional M123IBG imaging, or have known neuroblastoma and be referred for follow-up M123IBG imaging.

Main exclusion criteria: Patients without neuroblastoma, those not within the specified age range (children), those unable to follow study procedures or instructions, patients with other interfering medical conditions, those taking certain interfering medications, patients who had recent surgery or medical procedures that might interfere, pregnant or breastfeeding patients, and recent participants in other clinical trials cannot participate.

Study focus: The trial compares the effectiveness of 68Ga-SATO imaging with current standard imaging methods, such as M123IBG scintigraphy and whole-body MRI, to determine how well the new treatment detects cancer lesions. The study monitors safety, gathers information on radiation absorption, and evaluates the time required for preparing and conducting the imaging procedure.

Investigational imaging agent: 68Ga-SATO is a special imaging agent used to help doctors see certain types of cancer cells in children with neuroblastoma. It is not used to treat cancer but helps in diagnosing and understanding the disease extent, checking safety for children and evaluating image clarity.

Study on the Safety of Lutetium (177Lu) Oxodotreotide with Arginine and Lysine in Children with Refractory or Recurrent Neuroblastoma

This trial tests Peptide Receptor Radionuclide Therapy (PRRT) using 177Lu-DOTATATE medication for children whose neuroblastoma has either returned after treatment or has not responded to previous treatments. The medication is delivered through an infusion directly into the bloodstream.

Main inclusion criteria: Patients must have confirmed neuroblastoma diagnosis (with immunohistochemical staining for somatostatin receptors if available), provide written informed consent, and have relapsed or primary refractory high-risk neuroblastoma classified as INSS stage 4 or INRGSS stage M. They must be older than 18 months, have life expectancy over 3 months, performance status score greater than 50% (Karnofsky for over 12 years, Lansky for 12 years or younger), and have recovered from prior treatment and surgery. Specific diagnostic imaging tests must be completed within two months, and laboratory requirements must be met within 7 days before starting treatment, including adequate hemoglobin levels, neutrophil and platelet counts, liver and kidney function, and urinary catecholamine metabolites measured within 2 months. At least 2 x 106 CD34+ cells/kg of peripheral blood stem cells must be available.

Main exclusion criteria: Patients with different types of cancer than neuroblastoma, those who have not experienced return or worsening of high-risk neuroblastoma, patients not within the specified age range, those unable to follow study procedures, patients with other serious interfering health conditions, pregnant or breastfeeding patients, those who received certain recent treatments, and patients with allergies to study medication or ingredients cannot participate.

Study focus: The purpose is to find the highest dose of 177Lu-DOTATATE that can be safely given to children. During the study, children receive medication and are closely monitored for body reactions and cancer effects. LysaKare medication, containing arginine hydrochloride and lysine hydrochloride, may be used to help protect the kidneys during treatment.

Investigational therapy: 177Lu-DOTATATE is a targeted therapy involving a radioactive substance linked to a molecule designed to attach to specific receptors on cancer cells, aiming to deliver radiation directly to cancer cells to potentially reduce tumor size or slow growth.

Study of ABTL0812 with Irinotecan and Temozolomide for Children with Relapsed or Refractory Neuroblastoma and Other Solid Tumors

This trial focuses on studying relapsed or refractory neuroblastoma and other solid tumors in children using ABTL0812, a capsule containing sodium 2-hydroxylinoleate, both alone and in combination with chemotherapy or targeted therapies including irinotecan and temozolomide.

Main inclusion criteria: Patients must be between 1 and 21 years old, able to swallow capsules, with signed informed consent. They must have a solid tumor that has come back, not responded to treatment, or has no known cure (tissue sample confirmation recommended but not required), with at least one measurable disease area. Adequate performance status is required (at least 60% for under 16, more than 60% on Karnofsky scale for 16 and older). Adequate blood, liver, and kidney function must be shown by specific lab results within 7 days before starting the study drug, including hemoglobin at least 8 g/dL (transfusions allowed), appropriate white blood cell and platelet levels without recent growth factor or transfusion support, normal kidney function based on age and gender or specific creatinine clearance levels, and specific bilirubin and liver enzyme levels. Sexually active males and females must use appropriate birth control, and females of childbearing age must have negative pregnancy test. Patients must not have psychological, social, or geographical issues preventing them from following study rules and schedule.

Main exclusion criteria: Patients who have not been diagnosed with relapsed or refractory neuroblastoma or other solid tumors, those not within the specified age range, patients not eligible for specific treatments being tested (chemotherapy or targeted therapies), and those part of a vulnerable population needing special protection or care cannot participate.

Study focus: The study aims to determine the best dose of ABTL0812 for children when used alone or combined with other treatments. The trial involves treatment cycles with regular monitoring of effects and side effects, helping researchers understand the safest and most effective way to use ABTL0812. The study is expected to continue until 2028.

Investigational drugs: ABTL0812 is experimental, being tested for appropriate dosing when used alone or with other treatments. Irinotecan is a chemotherapy drug combined with other therapies to evaluate if it helps improve outcomes. Temozolomide is another chemotherapy medication tested in combination to evaluate effectiveness in children with relapsed or refractory neuroblastoma and other solid tumors.

Summary

The landscape of clinical trials for neuroblastoma demonstrates significant international collaboration, with trials spanning across multiple European countries. France, Germany, and Spain show particularly high concentrations of research activity, each hosting multiple studies. The Netherlands, Italy, and several Nordic countries are also actively engaged in neuroblastoma research.

A clear trend emerges in the therapeutic approaches being investigated. Immunotherapy, particularly using dinutuximab beta (an anti-GD2 monoclonal antibody), appears frequently across multiple trials, often in combination with traditional chemotherapy regimens. Several studies focus on CAR-T cell therapies targeting GD2, representing cutting-edge cellular immunotherapy approaches. Targeted radionuclide therapy using lutetium-based compounds is being explored in multiple countries, offering another innovative treatment avenue.

The trials address different disease stages and risk categories. Several studies specifically target newly diagnosed high-risk patients, while others focus on relapsed or refractory disease. Some trials explore treatments for low and intermediate risk patients, demonstrating a comprehensive approach to addressing the full spectrum of neuroblastoma presentations.

Combination therapies dominate the research landscape, with many trials investigating how traditional chemotherapy agents like temozolomide, irinotecan, topotecan, and cyclophosphamide work when combined with newer immunotherapies or targeted agents. Some studies are comparing different chemotherapy backbones or radiation doses, seeking to optimize existing treatment protocols.

Advanced imaging techniques are also under investigation, with trials comparing different scanning technologies for better disease detection and monitoring. These studies aim to improve diagnostic accuracy and treatment response assessment.

For patients and families seeking treatment options, the geographic distribution of trials offers opportunities across Europe, though some treatments are only available in specific countries. Eligibility criteria vary significantly between trials, with most accepting children and young adults, though specific age ranges differ. Many trials require previous treatment failure or disease progression, while others accept newly diagnosed high-risk patients.

Ongoing Clinical Trials on Neuroblastoma

  • Long-term safety study of GD2IL18CART treatment in patients with neuroblastoma, osteosarcoma, Ewing sarcoma, or advanced breast cancer

    Recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Germany

  • Study of Anti-GD2-800CW for Imaging in Children with Neuroblastoma

    Recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    The Netherlands

  • Study of GD2-CAR T Cells, Cyclophosphamide, and Fludarabine for Children with High-Risk or Relapsed Neuroblastoma and Other GD2+ Tumors

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Italy

  • Study on Stem Cell Transplantation and Immunotherapy with Rituximab, Dinutuximab Beta, and Drug Combination for Patients with High-Risk Relapsed Neuroblastoma

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Italy

  • Study Comparing [18F]meta-fluorobenzylguanidine PET-CT and Iodine (123I) Iobenguane Scans for Detecting Neuroblastoma in Patients

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    The Netherlands

  • Study of ABTL0812, Irinotecan, and Temozolomide for Children with Relapsed or Refractory Neuroblastoma and Other Solid Tumors

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Spain

  • Study of Crizotinib and Temsirolimus for Children with ALK, ROS1, or MET Positive Cancers, Including Neuroblastoma and Rhabdomyosarcoma

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Denmark Finland France Germany Italy The Netherlands +3

  • Study on the Safety and Dosage of GD2IL18CART for Patients with Relapsed or Refractory GD2 Positive Solid Cancers

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Germany

  • Study on Dinutuximab Beta with Chemotherapy for Children and Teens with Newly Diagnosed High-Risk Neuroblastoma

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Austria France Germany Italy The Netherlands Poland +1

  • Study of Lutetium (177Lu) Oxodotreotide for Children with Relapsed or Refractory High-Risk Neuroblastoma

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Denmark Lithuania The Netherlands Norway Spain Sweden

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