A Phase 2 interventional study of GM-CSF + Naxitamab in Neuroblastoma, sponsored by Y-mAbs Therapeutics. Active, not recruiting at 20 sites in 9 countries. Open to participants aged 1 Year and older. Per ClinicalTrials.gov, last updated 2026-07-30.
Sponsored by Y-mAbs Therapeutics · Phase 2, Interventional, and Treatment
Among 11 studies testing Granulocyte-Macrophage Colony-Stimulating Factor in Neuroblastoma — 2 open to participants now, 4 under way, none of them phase 3. Granulocyte-Macrophage Colony-Stimulating Factor · Neuroblastoma.
Among the 11 studies testing Granulocyte-Macrophage Colony-Stimulating Factor in Neuroblastoma, this one is the 4th largest by planned enrollment, is the only one that started in 2018, is one of 6 in phase 2, and was the 8th registered.
675 studies on the registry are indexed under Neuroblastoma; 127 are open to participants now.
This study's planned enrollment of 122 is above the median of 32 across 507 interventional studies indexed under Neuroblastoma.
Browse Neuroblastoma studies →2 of the 11 studies testing Granulocyte-Macrophage Colony-Stimulating Factor in Neuroblastoma are open now, in 2 countries. Each count lists the studies.
No approval found in the FDA or EMA files read. How studies give it is on its page →
Of the 104 Neuroblastoma studies recruiting now, 25 (24%) set no upper age limit and 74 (71%) stop at 75 or younger; 101 take children under 18; 3 take healthy volunteers.
This study: 1 Year and older · all sexes · no healthy volunteers.
The full table on the hub →11 studies; the count open now beside each. This study's row is marked.
First in 2012; this study’s year is marked. 1 started in 2018.
Median enrollment 62 across the 11 interventional studies. Of the 5 that completed or were terminated, 5 (100%) have results posted.
3 of 11 have an industry lead sponsor. The 8 most frequent:
Y-mAbs Therapeutics is the lead sponsor of 15 studies on the registry; none are open to participants now.
Of its 8 completed or terminated interventional studies of FDA-regulated products, 6 (75%) have results posted.
Y-mAbs Therapeutics has 6 studies filed under Neuroblastoma — fewer than ten, so there is no comparison to draw.
1 of the studies testing Granulocyte-Macrophage Colony-Stimulating Factor in Neuroblastoma that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT01757626Memorial Sloan Kettering Cancer CenterCombination Therapy of Antibody Hu3F8 With Granulocyte- Macrophage Colony Stimulating Factor (GM-CSF) in Patients With Relapsed/Refractory High-Risk Neuroblastoma | 2026-12 |
Among the 11 studies testing Granulocyte-Macrophage Colony-Stimulating Factor in Neuroblastoma, from their registry updates:
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
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Relapsed and refractory
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Life expectancy
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Biological: GM-CSF + Naxitamab
Drug pages: Granulocyte-Macrophage Colony-Stimulating Factor · Naxitamab
Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) and Humanized IgG1 monoclonal GD2 antibody
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.
Time frame: 101 weeks
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.
Time frame: 101 weeks
Duration of Response (DoR)
Length of time from patient response to disease progression.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Pre-naxitamab dose - 552 hours
Assessment of the clearance of naxitamab
Calculation of clearance of naxitamab will be calculated and summarized with descriptive statistics.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 101 weeks
Number of infusions done in an outpatient setting
Number of infusions done in an outpatient setting
Time frame: 101 weeks
Percentage of infusions done in an outpatient setting
Percentage of infusions done in an outpatient setting
Time frame: 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.
Time frame: 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
Time frame: 5 years
Overall Survival
The interval from the date of first dose of Naxitamab until the date of death due to any cause.
Time frame: 5 years
Happiness and activity levels
Happiness and activity levels will be measured over time and assessed by caretaker
Time frame: 39 days
Plan to share: Undecided
No publications or documents are linked to this record.
Ran from Apr 2018 to primary completion in Jun 2026, 98 months.
Of 67 phase 2 interventional studies under Neuroblastoma, half reached primary completion within 74 months of starting (the median), counting those still running; a quarter within 52 and three quarters within 99. This study's 98 months is above that.
How these figures are worked out, and which studies are left out of them.
This study is active, not recruiting, as verified in Jun 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drugs tested here: Granulocyte-Macrophage Colony-Stimulating Factor · Naxitamab
Y-mAbs Therapeutics