Clinical trial · Interventional
Combination Therapy of Antibody Hu3F8 With Granulocyte- Macrophage Colony Stimulating Factor (GM-CSF) in Patients With Relapsed/Refractory High-Risk Neuroblastoma
Phase I/II Study of Combination Therapy of Antibody Hu3F8 With Granulocyte- Macrophage Colony Stimulating Factor (GM-CSF) in Patients With Relapsed/Refractory High-Risk Neuroblastoma
NCT01757626CI-TRIAL-00100370active not recruitingPhase 1 / Phase 2ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
The purpose of this study is to find out if an antibody called Humanized 3F8 (Hu3F8) combined with granulocyte- macrophage colony stimulating factor (GM-CSF) is safe for treating neuroblastoma.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Neuroblastoma | Neuroblastoma | ONTOLOGY_EXACT | 0.90 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Hu3F8 With GM-CSF | Biological | — | UNRESOLVED |
Design
Arms and outcomes
Arms (2)
- type
- EXPERIMENTAL
- label
- Hu3F8 with GM-CSF
- description
- The phase I single arm trial assesses escalating doses of iv hu3F8 (days 1, 3, 5) in the presence of sc GM-CSF (day -4 through 5). These 3 doses of hu3F8 and 10 days of GM-CSF constitute a treatment cycle. The expansion phase II single arm trial assesses the anti-NB activity of hu3F8+GM-CSF.in 3 groups of patients: Group 1 patients have primary refractory disease (no prior relapse but incomplete response to treatment) in BM as documented by histology and/or 123I-MIBG scan. Group 2 patients are in ≥2nd CR and at high risk for another relapse. Group 3 patients have secondary refractory disease (prior relapse and incomplete response to retrieval therapy) in BM as documented by histology and/or 123I-MIBG scan. Ph II: Groups 1 \& 3 pts can continue to get cycles every 1-2 months for up to 24 months from study enrollment or until they receive 5 cycles after a major response (CR or PR) is achieved.
- interventionNames
- Biological: Hu3F8 With GM-CSF
- type
- EXPERIMENTAL
- label
- expansion phase II single arm trial
- description
- Group 1 patients have primary refractory disease (no prior relapse but incomplete response to treatment) in BM as documented by histology and/or 123\^I-MIBG scan. Group 2 patients are in \>2nd CR/VGPR and at high risk for another relapse. Group 3 patients have secondary refractory disease (prior relapse and incomplete response to retrieval therapy) in BM as documented by histology and/or 123\^I-MIBG scan. GM-CSF can be omitted if patients have a history of an allergy to GM-CSF or develop an allergic reaction to GM-CSF after initiating therapy while on the protocol.
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 1 Year
Show eligibility criteria text
Inclusion Criteria: * Diagnosis of NB as defined by a) histopathology (confirmed by the MSKCC Department of Pathology), or b) BM metastases or MIBG-avid lesion(s) plus high urine catecholamine levels. * Patients must have high-risk NB (including MYCNamplified stage 2/3/4/4S of any age and MYCN-nonamplified stage 4 in patients greater than 18 months of age) AND: * Phase I: Patients must have refractory or relapsed NB, resistant to standard therapy\*. \*For NB, standard therapy includes intensive induction chemotherapy, followed by a variety of consolidation or salvage therapies, depending on response. * Phase II: Patients must have primary or secondary refractory disease in BM, defined as morphologic evidence of NB in BM and/or abnormal 123I-MIBG uptake in osteomedullary sites, OR patients patients in ≥ 2nd CR patients are in ≥2nd CR * Patients must be older than 1 year of age. * Prior treatment with murine and humanized 3F8 is allowed. Patients with prior m3F8, hu3F8, ch14.18 or hu14.18 treatment must have a negative HAHA antibody titer. Human anti-mouse antibody (HAMA) positivity is allowed. * White blood cell count ≥1000/ul (phase I only) * Absolute neutrophil count ≥500/ul (phase I only) * Absolute lymphocyte count ≥500/ul (phase I only) * Platelet count ≥25,000/ul (phase I only) * No chemotherapy or immunotherapy for a minimum of three weeks prior to start of hu3F8 * Women of child-bearing potential must be willing to practice an effective method of birth control while on treatment * Signed informed consent indicating awareness of the investigational nature of this program. Exclusion Criteria: * Existing major organ dysfunction \> grade 2, with the exception of hearing loss and hematologic toxicity (defined as suppression of all subtypes of WBCs, RBCs, and platelets). * Active life-threatening infection. * Pregnant women or women who are breast-feeding. * Inability to comply with protocol requirements, including PK studies and genetic studies (phase I only) * History of allergy to mouse proteins. * Positive human anti-hu3F8 antibody (HAHA) titer
References
Publications (4)
- DERIVEDKushner BH, Modak S, White C, Basu EM, Roberts SS, Mauguen A, Cheung NV. A phase II trial of naxitamab plus stepped-up dosing of GM-CSF for patients with high-risk neuroblastoma in second or later complete remission. Int J Cancer. 2026 Jun 15;158(12):3232-3240. doi: 10.1002/ijc.70374. Epub 2026 Feb 11. PMID 41670398
- DERIVEDKushner BH, Modak S, Mauguen A, Basu EM, Roberts SS, Cheung NV. Naxitamab plus stepped-up dosing of granulocyte-macrophage colony-stimulating factor for primary refractory high-risk neuroblastoma: results of a phase I/II trial. J Hematol Oncol. 2025 Nov 26;18(1):114. doi: 10.1186/s13045-025-01770-7. PMID 41299684
- DERIVEDKushner BH, Cheung IY, Modak S, Basu EM, Roberts SS, Cheung NK. Humanized 3F8 Anti-GD2 Monoclonal Antibody Dosing With Granulocyte-Macrophage Colony-Stimulating Factor in Patients With Resistant Neuroblastoma: A Phase 1 Clinical Trial. JAMA Oncol. 2018 Dec 1;4(12):1729-1735. doi: 10.1001/jamaoncol.2018.4005. PMID 30326045
- DERIVEDCheung IY, Kushner BH, Modak S, Basu EM, Roberts SS, Cheung NV. Phase I trial of anti-GD2 monoclonal antibody hu3F8 plus GM-CSF: Impact of body weight, immunogenicity and anti-GD2 response on pharmacokinetics and survival. Oncoimmunology. 2017 Jul 31;6(11):e1358331. doi: 10.1080/2162402X.2017.1358331. eCollection 2017. PMID 29147617