TriangleNCT03363373 · CITED IN SOURCE DOCUMENTS
Naxitamab for High-Risk Neuroblastoma Patients With Primary Refractory Disease or Incomplete Response to Salvage Treatment in Bone and/or Bone Marrow
An NCI or FDA source cites this study. A citation does not establish that it applies to an individual diagnosis.
- Phase
- PHASE2
- Status at capture
- ACTIVE NOT RECRUITING
- Registry last update
- 2026-07-30
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
Open the original ClinicalTrials.gov record → · Download preserved record
What did the study report?
Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.
No posted result sections were captured. Registered plans do not establish that a treatment works.
Who could take part
- eligibility Criteria
- 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
- healthy Volunteers
- false
- minimum Age
- 1 Year
- sex
- ALL
- std Ages
- CHILD
- ADULT
- OLDER_ADULT
Treatment arms and interventions
- arm Groups
- description
- Each investigational cycle is started with 5 days of 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 until CR or PR 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. After end of treatment patients will enter a long-term follow up for up to 3 years after end of treatment visit.
- intervention Names
- Biological: GM-CSF + Naxitamab
- label
- GM-CSF + Naxitamab
- type
- EXPERIMENTAL
- interventions
- arm Group Labels
- GM-CSF + Naxitamab
- description
- Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) and Humanized IgG1 monoclonal GD2 antibody
- name
- GM-CSF + Naxitamab
- type
- BIOLOGICAL
Study design
- allocation
- NA
- intervention Model
- SINGLE_GROUP
- intervention Model Description
- Patients will receive cycles of GM-CSF and Naxitamab every 4 weeks up to a total of 101 weeks. Safety and efficacy will be investigated with short-term follow-up at minimum 4 weeks after last treatment and with long-term follow-up for up to 3 years after end of treatment visit.
- masking Info
- masking
- NONE
- primary Purpose
- TREATMENT
Enrollment
- count
- 122
- type
- ESTIMATED
Registered outcome plans (not posted results)
- primary Outcomes
- description
- Overall objective response rate (ORR) during the Naxitamab treatment period that will be centrally assessed according to the International Neuroblastoma Response Criteria (INRC) modified with 123I-MIBG criteria and following the use of 18F FDG-PET for MIBG non-avid lesions.
- measure
- Response rate during Naxitamab treatment
- time Frame
- 101 weeks
- secondary Outcomes
- description
- Safety will be evaluated by the incidence of adverse events (AE) and serious adverse events (SAEs) graded according to CTCAE, version 4.0.
- measure
- Incidence of adverse events and serious adverse events
- time Frame
- 101 weeks
- description
- Length of time from patient response to disease progression.
- measure
- Duration of Response (DoR)
- time Frame
- 101 weeks
- description
- The complete response (CR) rate is defined as the fraction of patients experiencing a CR according to International Neuroblastoma Response Criteria (INRC) criteria during the treatment period.
- measure
- Complete Response Rate
- time Frame
- 101 weeks
- description
- Calculation of maximum serum concentration of naxitamab will be calculated and summarized with descriptive statistics.
- measure
- Assessment of the maximum serum concentration (cmax) of naxitamab
- time Frame
- Pre-naxitamab dose - 552 hours
- description
- Calculation of minimum serum concentration of naxitamab will be calculated and summarized with descriptive statistics.
- measure
- Assessment of the minimum serum concentration (cmin) of naxitamab
- time Frame
- Pre-naxitamab dose - 552 hours
- description
- Calculation of clearance of naxitamab will be calculated and summarized with descriptive statistics.
- measure
- Assessment of the clearance of naxitamab
- time Frame
- Pre-naxitamab dose - 552 hours
- description
- Calculation of the volume of distribution of naxitamab will be calculated and summarized with descriptive statistics.
- measure
- Assessment of the volume of distribution of naxitamab
- time Frame
- Pre-naxitamab dose - 552 hours
- description
- Calculation of the AUC of naxitamab will be calculated and summarized with descriptive statistics.
- measure
- Assessment of the Area under the Curve (AUC) of naxitamab
- time Frame
- Pre-naxitamab dose - 552 hours
- description
- Calculation of the t½ of naxitamab will be calculated and summarized with descriptive statistics.
- measure
- Assessment of the terminal half-life (t½) of naxitamab
- time Frame
- Pre-naxitamab dose - 552 hours
- description
- ADA formation will be investigated following a multi-tiered approach: A screening confirmation-titration analysis plus a ligand binding assay to examine a potential neutralizing effect of anti-naxitamab antibodies.
- measure
- Assessment of anti-drug antibody (ADA) formation
- time Frame
- Pre-naxitamab dose - 552 hours
- description
- 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
- measure
- Intravenous (IV) opioid use (cycle 1)
- time Frame
- 6 hours
- description
- 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
- measure
- Intravenous (IV) opioid use (all cycles)
- time Frame
- 101 weeks
Full 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.
Source references
Source notices and limitations
Discovery and provenance
- release id
- ctgov-registry-7ad944f6deaf1f5cd6122126
- edge id
- nct-edge-cbb4754c901b965f81b0
- requested nct id
- NCT03363373
- primary nct id
- NCT03363373
- source kind
- fda_spl_label
- source record id
- 5878035d-081a-1653-e063-6394a90af965
- source file sha256
- bbaf8e3e49a06d91fa717d034adb1798932735c99e42d8d769cf5f9eb31d345e
- payload sha256
- ce1579b4305c602224a9b00a0348dfe4c4befd9862fa6ae76a9a0bd9c0b25aef
- payload
- application numbers
- BLA761171
- clinical indication matching status
- not_inferred
- end exclusive
- true
- id
- nct-edge-cbb4754c901b965f81b0
- link basis
- explicit_NCT_identifier_in_acquired_source
- nct id
- NCT03363373
- occurrences
- context exact
- apsed disease in the bone or bone marrow, Study 201 and Study 12-230. Study 201 The efficacy of DANYELZA in combination with GM-CSF was evaluated in Study 201 (NCT03363373), a multicenter open-label, single arm trial, in a subpopulation of patients who had refractory or relapsed high-risk neuroblastoma in the bone or bone marrow
- end
- 357
- exact text
- NCT03363373
- start
- 346
- offset unit
- unicode_code_points
- source file
- /tmp/cancer-full-research/indications/raw/labels-00300.json
- source file sha256
- bbaf8e3e49a06d91fa717d034adb1798932735c99e42d8d769cf5f9eb31d345e
- source json pointer
- /results/68/clinical_studies/0
- source kind
- fda_spl_label
- source record id
- 5878035d-081a-1653-e063-6394a90af965
- source string basis
- parsed_JSON_string_unmodified
- source string sha256
- 8bdf5e3b4588201d7a934c14c201b26bbcbe6936b7a05f3c8fda80f65dcc9ed3
- spl effective time
- 20260807
- spl set id
- b0f5ee96-369f-4cb9-a783-3bfa70fe8f90
Preserved source evidence · Not medical advice