NeuroblastomaPhase 2NCT03363373
Naxitamab for High-Risk Neuroblastoma Patients With Primary Refractory Disease or Incomplete Response to Salvage Treatment in Bone and/or Bone Marrow
A Phase 2 study testing GM-CSF + Naxitamab in people with Neuroblastoma, run by Y-mAbs Therapeutics at 20 sites in 9 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- GM-CSF + Naxitamab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 122 participants
- Where
- 20 sitesin 9 countries
- Study dates
- 3 Apr 2018to Apr 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
20 sites in 9 countries.
20 sites in 9 countries
Spain4 sites
Hospital Sant Joan de Déu
Barcelona, 08950
Hospital Infantil Universitario Niño Jesús
Madrid, 28009
Hospital Universitario Virgen Del Rocío
Seville
Hospital Universitario y Politécnico La Fe
Valencia, 46026
Germany3 sites
University Medical Center Hamburg-Eppendorf
Hamburg
Johannes Gutenberg-Universität
Mainz
University Hospital Regensburg
Regensburg
Italy3 sites
Giannina Gaslini Hospital
Genoa, Genoa, 16147
Fondazione IRCCS Istituto Nazionale dei Tumori
Milan, Milan, 20133
Ospedale Pediatrico Bambino Gesù
Rome
United Kingdom3 sites
The Royal Glasgow Children's Hospital
Glasgow, G51 4TF
Leeds General Infirmary
Leeds, LS1 3EX
University Hospital Southampton
Southampton, SO16 6YD
United States2 sites
Riley Hospital for Children
Indianapolis, Indiana, 46202
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Hong Kong2 sites
Hong Kong Children's Hospital
Hong Kong
Queen Mary Hospital
Hong Kong
About this study
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
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
Read the 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.
- Conditions
- Neuroblastoma
- Being tested
- GM-CSF + Naxitamab
- Keywords
- Antibody, Neuroblastoma, Pediatric, Adult
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GM-CSF + NaxitamabBiological
Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) and Humanized IgG1 monoclonal GD2 antibody
| Study group | Role | What they receive |
|---|---|---|
| GM-CSF + Naxitamab | Experimental | 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. Receives: Biological: GM-CSF + Naxitamab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Response rate during Naxitamab treatment
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.
Measured: 101 weeks
Secondary outcomes (20)
Incidence of adverse events and serious adverse events
Safety will be evaluated by the incidence of adverse events (AE) and serious adverse events (SAEs) graded according to CTCAE, version 4.0.
Measured: 101 weeks
Duration of Response (DoR)
Length of time from patient response to disease progression.
Measured: 101 weeks
Complete Response Rate
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.
Measured: 101 weeks
Assessment of the maximum serum concentration (cmax) of naxitamab
Calculation of maximum serum concentration of naxitamab will be calculated and summarized with descriptive statistics.
Measured: Pre-naxitamab dose - 552 hours
Assessment of the minimum serum concentration (cmin) of naxitamab
Calculation of minimum serum concentration of naxitamab will be calculated and summarized with descriptive statistics.
Measured: Pre-naxitamab dose - 552 hours
Assessment of the clearance of naxitamab
Calculation of clearance of naxitamab will be calculated and summarized with descriptive statistics.
Measured: Pre-naxitamab dose - 552 hours
Assessment of the volume of distribution of naxitamab
Calculation of the volume of distribution of naxitamab will be calculated and summarized with descriptive statistics.
Measured: Pre-naxitamab dose - 552 hours
Assessment of the Area under the Curve (AUC) of naxitamab
Calculation of the AUC of naxitamab will be calculated and summarized with descriptive statistics.
Measured: Pre-naxitamab dose - 552 hours
Assessment of the terminal half-life (t½) of naxitamab
Calculation of the t½ of naxitamab will be calculated and summarized with descriptive statistics.
Measured: Pre-naxitamab dose - 552 hours
Assessment of anti-drug antibody (ADA) formation
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.
Measured: Pre-naxitamab dose - 552 hours
Intravenous (IV) opioid use (cycle 1)
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
Measured: 6 hours
Intravenous (IV) opioid use (all cycles)
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
Measured: 101 weeks
Hospitalization days (cycle 1)
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
Measured: 4 weeks
Safety of patients with positive human anti-drug antibody (ADA)
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
Measured: 101 weeks
Number of infusions done in an outpatient setting
Number of infusions done in an outpatient setting
Measured: 101 weeks
Percentage of infusions done in an outpatient setting
Percentage of infusions done in an outpatient setting
Measured: 101 weeks
Incidence of adverse events and serious adverse events in ADA positive patients
Safety will be evaluated by the incidence of adverse events (AE) and serious adverse events (SAEs) graded according to CTCAE, version 4.0 in ADA positive patients.
Measured: 101 weeks
Progression Free Survival (PFS)
PFS, defined as the time from the first 1st infusion of naxitamab until progressive disease or death, whichever comes first
Measured: 5 years
Overall Survival
The interval from the date of first dose of Naxitamab until the date of death due to any cause.
Measured: 5 years
Happiness and activity levels
Happiness and activity levels will be measured over time and assessed by caretaker
Measured: 39 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Dec 2017
- Study start3 Apr 2018
- Primary completion30 Jun 2026
- Study completionApr 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeuroblastomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- 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
- None (open label)
- Enrolment
- 122 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Y-mAbs Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03363373
- Sponsor's protocol ID
- 201
- Status last verified
- Jun 2026
- First submitted
- 6 Nov 2017
- Last updated
- 30 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 30 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03363373
- Acronym / sponsor ID
- 201
- Conditions
- Neuroblastoma
- Keywords
- Antibody, Neuroblastoma, Pediatric, Adult
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 122 participants (estimated)
- Lead sponsor
- Y-mAbs TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 3 Apr 2018 (actual)
- Primary completion
- 30 Jun 2026 (actual)
- Study completion
- Apr 2028 (estimated)
- First posted
- 6 Dec 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- 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.
| Arm | Type | Receives |
|---|---|---|
| GM-CSF + Naxitamab | Experimental | Biological: GM-CSF + NaxitamabEach 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. |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GM-CSF + NaxitamabGranulocyte-Macrophage Colony Stimulating Factor (GM-CSF) and Humanized IgG1 monoclonal GD2 antibody | Biological | GM-CSF + Naxitamab |
Primary outcome
Response rate during Naxitamab treatment
Time frame: 101 weeks
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.
20 secondary outcomes
Incidence of adverse events and serious adverse events
Time frame: 101 weeks
Duration of Response (DoR)
Time frame: 101 weeks
Complete Response Rate
Time frame: 101 weeks
Assessment of the maximum serum concentration (cmax) of naxitamab
Time frame: Pre-naxitamab dose - 552 hours
Assessment of the minimum serum concentration (cmin) of naxitamab
Time frame: Pre-naxitamab dose - 552 hours
Assessment of the clearance of naxitamab
Time frame: Pre-naxitamab dose - 552 hours
Assessment of the volume of distribution of naxitamab
Time frame: Pre-naxitamab dose - 552 hours
Assessment of the Area under the Curve (AUC) of naxitamab
Time frame: Pre-naxitamab dose - 552 hours
Assessment of the terminal half-life (t½) of naxitamab
Time frame: Pre-naxitamab dose - 552 hours
Assessment of anti-drug antibody (ADA) formation
Time frame: Pre-naxitamab dose - 552 hours
Intravenous (IV) opioid use (cycle 1)
Time frame: 6 hours
Intravenous (IV) opioid use (all cycles)
Time frame: 101 weeks
Hospitalization days (cycle 1)
Time frame: 4 weeks
Safety of patients with positive human anti-drug antibody (ADA)
Time frame: 101 weeks
Number of infusions done in an outpatient setting
Time frame: 101 weeks
Percentage of infusions done in an outpatient setting
Time frame: 101 weeks
Incidence of adverse events and serious adverse events in ADA positive patients
Time frame: 101 weeks
Progression Free Survival (PFS)
Time frame: 5 years
Overall Survival
Time frame: 5 years
Happiness and activity levels
Time frame: 39 days
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jul 2026.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
20 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing GM-CSF + Naxitamab in people with Neuroblastoma, run by Y-mAbs Therapeutics at 20 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 30 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in Spain, Germany, Italy, United Kingdom, United States and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Response rate during Naxitamab treatment (time frame: 101 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Jul 2026.