Naxitamab for High-Risk Neuroblastoma Patients With Primary Refractory Disease or Incomplete Response to Salvage Treatment in Bone and/or Bone Marrow

Official Title

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

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

Trial Description

Primary Outcome:

• Response rate during Naxitamab treatment

Secondary Outcome:

• Incidence of adverse events and serious adverse events
• Duration of Response (DoR)
• Complete Response Rate
• Assessment of the maximum serum concentration (cmax) of naxitamab
• Assessment of the minimum serum concentration (cmin) of naxitamab
• Assessment of the clearance of naxitamab
• Assessment of the volume of distribution of naxitamab
• Assessment of the Area under the Curve (AUC) of naxitamab
• Assessment of the terminal half-life (t½) of naxitamab
• Assessment of anti-drug antibody (ADA) formation
• Intravenous (IV) opioid use (cycle 1)
• Intravenous (IV) opioid use (all cycles)
• Hospitalization days (cycle 1)
• Safety of patients with positive human anti-drug antibody (ADA)
• Number of infusions done in an outpatient setting
• Percentage of infusions done in an outpatient setting
• Incidence of adverse events and serious adverse events in ADA positive patients
• Progression Free Survival (PFS)
• Overall Survival
• Happiness and activity levels

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.

View this trial on ClinicalTrials.gov