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A Pivotal Phase 2 Trial of Antibody Naxitamab (hu3F8) and Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in High-Risk Neuroblastoma Patients with Primary Refractory Disease or Incomplete Response to Salvage Treatment in Bone and/or Bone Marrow.

Study ID: 37225
Short Title: Naxitamab and GM-CSF in High Risk Neuroblastoma Patients v6.0
Trust Name: UHS
Recruitment Site: Southampton General Hospital
Disease Area: CYP
Phase: III
Expected End Date: 30/06/2022
Postcode: SO16 6YD
Contact Name: Amanda Pattie
Contact Email: studysupport1and3.crnwessex@nihr.ac.uk
Active: Yes

1. Documented diagnosis of NB as defined per INRC as a. histopathology of tumor biopsy, or b. BM aspirate or biopsy indicative of NB by histology, plus high blood or urine catecholamine metabolite levels or Myelocytomatosis Viral-Related Oncogene, Neuroblastoma derived (MYCN) amplification, or c. MIBG-avid lesion(s) 2. High-risk NB patients with either primary refractory disease or incomplete response to salvage treatment (in both cases including SD, MR and PR) evaluable in bone and/or BM as defined in section 6.7 in the protocol. 3. Life expectancy > = 6 months 4. Age > = 12 months 5. Acceptable hematological status, (hematological support is allowed if administered > = 1 week before first infusion of naxitamab), defined as: a. Hemoglobin > = 8 g/dL (5.0 mmol/L) b. White blood cell count > = 1000/μL c. Absolute neutrophil count (ANC) > = 500/μL d. Platelet count > = 25,000/μL 6. Acceptable liver function defined as: a. Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) < = 5 times upper limit of normal (ULN) b. Bilirubin < = 1.5 x ULN 7. Acceptable kidney function defined as: a. Estimated Glomerular Filtration Rate (eGFR) > 60 mL/min/1.73 m2 calculated by the 2009 revised Bedside Schwartz Equation 8. Written informed consent from legal guardian(s) and/or patient in accordance with local regulations. Children must provide assent as required by local regulations.

1. Any systemic anti-cancer therapy, including chemotherapy or immunotherapy, within 3 weeks before 1st dose of GM-CSF 2. Evaluable NB outside bone and BM 3. Actively progressing disease at trial entry 4. Existing major organ dysfunction > Grade 2, with the exception of hearing loss, hematological status, kidney and liver function. 5. Active life-threatening infection 6. Prior treatment with naxitamab 7. Karnofsky/Lansky score < 50% 8. Pregnancy or a woman who is breast-feeding (women of child-bearing potential must have a negative pregnancy test at screening). A woman of child-bearing potential is excluded if she does not agree to use adequate contraception for a period of 40 days after the last naxitamab infusion: intrauterine devices or hormonal contraception (oral contraceptive pills, implants, transdermal patches, vaginal rings or long-acting injections). In certain cases, it is accepted to use double barrier methods (a condom combined with a diaphragm). A sterilized or infertile woman is exempt from the requirement to use contraception after naxitamab treatment: she must have undergone surgical sterilization (hysterectomy, or bilateral ovariectomy) 9. Inability to comply with protocol requirements, including PK studies, as determined by the investigator 10. History of allergy or known hypersensitivity to GM-CSF, yeast-derived products, or any component of the product.

Patients will be invited to take part in this study if they have neuroblastoma which has either not responded adequately to treatment or has relapsed in the bones and/or bone marrow. Neuroblastoma is one of the most common childhood cancers and accounts for a high proportion of childhood cancer deaths. The majority of patients have 'high risk' disease, with metastatic bone/bone marrow disease. Outcome in patients who don't respond well to frontline treatment, or who relapse after treatment, is extremely poor. The purpose of this research is to find out about the treatment effects of an antibody called humanized 3F8 (hu3F8/Naxitamab). In the last decade, antibody therapy has become part of the standard care for children with high risk neuroblastoma. Antibodies are proteins produced by the immune system to fight infections, but can also potentially be used to attack tumour cells. In order to enhance the positive effect of Naxitamab, the treatment will be combined with granulocyte-macrophage colony stimulating factor (GM-CSF). Granulocytes are white blood cells that are able to destroy cancer cells and GM-CSF increases the ability of granulocytes to more effectively kill cancer cells. The only treatment that is being tested in this trial is Naxitamab (hu3F8) in combination with GM-CSF, and all enrolled patients will receive both. Patients can receive treatment for up to 101 weeks following first administration via infusion of Naxitamab- hu3F8 and injections of GM-CSF. Depending on how the participant responds it is up to the treating investigator to decide how many cycles of treatment is required. After treatment has completed, patients will undergo long term follow up of 3 years during which patients will attend visits twice a year to monitor post treatment effects. This study will take place across 6 countries (UK, Spain, Denmark, United States Canada and Hong Kong.

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