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| Diagnosis | Neuroblastoma | Study Status | Open |
| Phase | II |
| Age | Child, Adult, Older Adult - (1 Year and older) | Randomisation | NO |
| Line of treatment | Disease relapse or progression |
| Routes of Treatment Administration | naxitamab (intravenous); GM-CSF (sub-cutaneous) |
| Last Posted Update | 2024-01-19 |
| ClinicalTrials.gov # | NCT03363373 |
International Sponsor
Y-mAbs TherapeuticsPrincipal Investigators for Canadian Sites
The Hospital for Sick Children – Dr. Daniel MorgensternCentres
Study Description
Brief Summary:
Children and adults diagnosed with high-risk neuroblastoma patients with primary refractory disease or incomplete response to salvage treatment in bone and/or bone marrow will be treated for up to 101 weeks with naxitamab and granulocyte-macrophage colony stimulating factor (GM-CSF). Patients will be followed for up to five years after first dose.
Naxitamab, also known as hu3F8 is a humanised monoclonal antibody targeting GD2
Detailed Description:
Each patient will receive treatment for up to 101 weeks following the first Naxitamab administration. After the end of trial visit, each patient will enter a long-term follow-up where they will be monitored for up to 5 years after first treatment cycle.
Each investigational cycle is started with 5 days, days -4 to 0, of Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) administered at 250 µg/m2/day in advance of the start of Naxitamab administration. GM-CSF is thereafter administered at 500 µg/m2/day on days 1 to 5. As standard treatment, Naxitamab is administered at 3 mg/kg/day on days 1, 3, and 5, totalling 9 mg/kg per cycle.
Treatment cycles are repeated every 4 weeks (±1 week) until complete response or partial response followed by 5 additional cycles every 4 weeks (±1 week). Subsequent cycles are repeated every 8 weeks (±2 weeks) through 101 weeks from first infusion at the discretion of the investigator. End of treatment will take place around 8 weeks after the last cycle and thereafter long-term follow-up will continue.
Inclusion Criteria
- Diagnosis of neuroblastoma as defined per International Neuroblastoma Response Criteria
- High-risk neuroblastoma patients with either primary refractory disease or incomplete response to salvage treatment (in both cases including stable disease, minor response and partial response) evaluable in bone and/or bone marrow.
- Life expectancy ≥ 6 months
Exclusion Criteria
- Any systemic anti-cancer therapy, including chemotherapy or immunotherapy, within 3 weeks before 1st dose of GM-CSF
- Evaluable neuroblastoma outside bone and bone marrow
- Existing major organ dysfunction > Grade 2, with the exception of hearing loss, hematological status, kidney and liver function
- Active life-threatening infection
