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

Conditions

Treatment of high-risk neuroblastoma patients with primary refractory disease or incomplete response to salvage treatment in bone and/or bone marrow

Brief summary

Objective Response Rate (ORR) during the naxitamab treatment period, centrally assessed according to the International Neuroblastoma Response Criteria (INRC)

Detailed description

Safety will be evaluated by the incidence of adverse events (AEs) and serious adverse events (SAEs) graded according to Common Terminology Criteria for Adverse Events (CTCAE), version 4.0, DoR, defined as the time from first objective response (CR or partial response (PR)) to PD; data will be censored at the date of last disease evaluation before new anti-NB treatment, Complete response rate, during the naxitamab treatment period, centrally assessed according to the INRC, ORR, during the naxitamab treatment period, investigator assessed according to International Neuroblastoma Response Criteria (INRC), Assessment of the PK of naxitamab, Assessment of ADA formation, Intravenous (IV) opioid use during cycle 1 defined as total dosage of IV morphine (or equivalent opioid) administered 2 hours before infusion until 4 hours after end of infusion of naxitamab, Intravenous (IV) opioid use for each cycle during the trial defined as total dosage of IV morphine (or equivalent opioid) administered 2 hours before infusion until 4 hours after end of infusion of naxitamab, Number of hospitalization days related to naxitamab during cycle 1, defined as number of overnight stays. Hospitalizations required solely for protocol-specified assessments (e.g., PK sampling) or non-medical circumstances are excluded, Number and percentage of infusions done in an outpatient setting, In patients with positive ADA at trial inclusion, safety will be evaluated by the incidence of AEs and SAEs graded according to CTCAE, version 4.0, Happiness and activity levels measured over time assessed by caretaker, Pain during naxitamab infusion as assessed by Wong Baker- and FLACC scales

Related papers

No related papers found yet.

Interventions

DRUGHumanized IgG1 monoclonal antibody against GD2

DRUGLeukine

Eligibility

Sex/Gender

All

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Objective Response Rate (ORR) during the naxitamab treatment period, centrally assessed according to the International Neuroblastoma Response Criteria (INRC)	—

Secondary

Measure	Time frame
Safety will be evaluated by the incidence of adverse events (AEs) and serious adverse events (SAEs) graded according to Common Terminology Criteria for Adverse Events (CTCAE), version 4.0, DoR, defined as the time from first objective response (CR or partial response (PR)) to PD; data will be censored at the date of last disease evaluation before new anti-NB treatment, Complete response rate, during the naxitamab treatment period, centrally assessed according to the INRC, ORR, during the naxitamab treatment period, investigator assessed according to International Neuroblastoma Response Criteria (INRC), Assessment of the PK of naxitamab, Assessment of ADA formation, Intravenous (IV) opioid use during cycle 1 defined as total dosage of IV morphine (or equivalent opioid) administered 2 hours before infusion until 4 hours after end of infusion of naxitamab, Intravenous (IV) opioid use for each cycle during the trial defined as total dosage of IV morphine (or equivalent opioid) administered	—

Measure

Time frame

Safety will be evaluated by the incidence of adverse events (AEs) and serious adverse events (SAEs) graded according to Common Terminology Criteria for Adverse Events (CTCAE), version 4.0, DoR, defined as the time from first objective response (CR or partial response (PR)) to PD; data will be censored at the date of last disease evaluation before new anti-NB treatment, Complete response rate, during the naxitamab treatment period, centrally assessed according to the INRC, ORR, during the naxitamab treatment period, investigator assessed according to International Neuroblastoma Response Criteria (INRC), Assessment of the PK of naxitamab, Assessment of ADA formation, Intravenous (IV) opioid use during cycle 1 defined as total dosage of IV morphine (or equivalent opioid) administered 2 hours before infusion until 4 hours after end of infusion of naxitamab, Intravenous (IV) opioid use for each cycle during the trial defined as total dosage of IV morphine (or equivalent opioid) administered

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Countries

Denmark, France, Germany, Italy, Spain

Outcome results

None listed