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A Phase 3 Trial of Antibody hu3F8 and Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in High-Risk Neuroblastoma Patients with Primary or Secondary Refractory Osteomedullary Disease
EU CTIS ID: 2023-508587-29-00
What this study is testing
To evaluate the centrally assessed objective response rate (ORR) to naxitamab + GM-CSF
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Documented diagnosis of neuroblastoma (NB) as defined per INRC as a. histopathology of tumor biopsy, or b. bone marrow (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)
- 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. If disease is only present in bone the patient must have evaluable disease outside the radiation areas for being eligible in the trial, please see section 7.2.1. If disease is only present in the BM the involvement must be >5%.
- Life expectancy ≥6 months
- Age ≥12 months
- Acceptable hematological status at screening, (hematological support is allowed if administered ≥1 week before first screening procedure), defined as: a. Hemoglobin ≥8 g/dL (5.0 mmol/L) b. White blood cell count ≥1000/μL (1.0 x109/L) c. Absolute neutrophil count (ANC) ≥500/μL (0.5 x109/L) d. Platelet count ≥25,000/μL (25 x109/L)
- 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
You likely can't join if
- Any systemic anti-cancer therapy, including chemotherapy or immunotherapy, within 3 weeks of 1st dose of GM-CSF
- History of allergy or known hypersensitivity to GM-CSF, yeast-derived products, or any component of GM-CSF or naxitamab
- History of anaphylactic reactions CTCAE grade 4 related to prior GD2 antibody therapy
- NB in central nervous system (CNS) within 6 months of 1st dose of GM-CSF
- Prior treatment with omburtamab (mu8H9) within 6 months of 1st dose of GM-CSF
- Patients who have had allogeneic hematopoietic stem cell transplantation (allo-SCT) or donor-lymphocyte-infusion (DLI). DLI or buffy coat infusion is defined as any kind of active allogenic lymphocyte suspension a. within 6 months of 1st dose of GM-CSF or b. with a lymphocyte count < 0.2 x109/L
See the full eligibility criteria
Who can join
- Documented diagnosis of neuroblastoma (NB) as defined per INRC as a. histopathology of tumor biopsy, or b. bone marrow (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)
- 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. If disease is only present in bone the patient must have evaluable disease outside the radiation areas for being eligible in the trial, please see section 7.2.1. If disease is only present in the BM the involvement must be >5%.
- Life expectancy ≥6 months
- Age ≥12 months
- Acceptable hematological status at screening, (hematological support is allowed if administered ≥1 week before first screening procedure), defined as: a. Hemoglobin ≥8 g/dL (5.0 mmol/L) b. White blood cell count ≥1000/μL (1.0 x109/L) c. Absolute neutrophil count (ANC) ≥500/μL (0.5 x109/L) d. Platelet count ≥25,000/μL (25 x109/L)
- 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
- 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
- Written informed consent from legal guardian(s) and/or patient in accordance with local regulations. Children must provide assent as required by local regulations.
What rules you out
- Any systemic anti-cancer therapy, including chemotherapy or immunotherapy, within 3 weeks of 1st dose of GM-CSF
- History of allergy or known hypersensitivity to GM-CSF, yeast-derived products, or any component of GM-CSF or naxitamab
- History of anaphylactic reactions CTCAE grade 4 related to prior GD2 antibody therapy
- NB in central nervous system (CNS) within 6 months of 1st dose of GM-CSF
- Prior treatment with omburtamab (mu8H9) within 6 months of 1st dose of GM-CSF
- Patients who have had allogeneic hematopoietic stem cell transplantation (allo-SCT) or donor-lymphocyte-infusion (DLI). DLI or buffy coat infusion is defined as any kind of active allogenic lymphocyte suspension a. within 6 months of 1st dose of GM-CSF or b. with a lymphocyte count < 0.2 x109/L
- Patients who received Hematopoietic Progenitor Cell (HPC) boost or “top-up” of allogenic stem cells (lymphocyte-depleted) within 2 months of 1st dose GM-CSF
- Any clinically meaningful abnormal finding in physical examination, vital signs, ECG, hematology, clinical chemistry, or urinalysis prior to inclusion into the trial, which in the opinion of the investigator, may put the subject at risk because of his/her participation in the study.
- Treatment with immunosuppressive agents (local steroids excluded) within a month prior to 1st dose of GM-CSF.
- Inadequate cardiac function defined as either left ventricular ejection fraction of < 50% by echocardiography or other clinically relevant cardiac disorders at the discretion of the investigator.
- Evaluable NB outside bone and BM defined as follows: • MIBG-avid tumor: Definite MIBG uptake in tumor tissues outside bone and BM • MIBG nonavid tumor: Definite uptake in tumor tissues outside bone and BM on FDG-PET
- Actively progressing disease at trial entry according to Park criteria (Park et al. 2017) (see section 6.7)
- Existing major organ dysfunction CTCAE >Grade 2, with the exception of hearing loss, hematological status, kidney and liver function.
- Active life-threatening infection
- Prior treatment with naxitamab
- Karnofsky/Lansky score <50%
- 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 highly effective contraception for a period of 42 days after the last naxitamab infusion according to section 9.2.5. 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).
- Inability to comply with protocol requirements, including PK studies, as determined by the investigator
The study team makes the final eligibility decision.
Where it's taking place
- United States
- United Kingdom
- Hong Kong
- Canada
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; United Kingdom; Hong Kong; Canada. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.