A Phase 2 interventional study of Naxitamab monotherapy and GM-CSF in Neuroblastoma, sponsored by Sun Yat-sen University. Recruiting at 1 site in China. Open to participants aged 12 Months and older. Per ClinicalTrials.gov, last updated 2025-06-17.
Sponsored by Sun Yat-sen University · Phase 2, Interventional, and Treatment
This is an prospective study to evaluate the safety and efficacy of naxitamab monotherapy or combined with chemotherapy or combined with chemotherapy and checkpoint inhibitor in the treatment of pediatric high-risk and refractory/relapsed neuroblastoma in Sun Yat-sen University Cancer Center.
Patients with high risk neuroblastoma who obtain CR after chemotherapy combined with surgery, radiotherapy and/or hematopoietic stem cell transplantation received axitamab and GM-CSF. Patients with high-risk neuroblastoma treated by chemotherapy combined with surgery, radiotherapy and or hematopoietic stem cell transplantation patients with tumor residual or progression during treatment (refractory) received naxitamab and GM-CSF in combination with irinotecan and temozolomide or naxitamab and GM-CSF in combination with irinotecan and temozolomide and PD-1 antibody.
625 studies on the registry are indexed under Neuroblastoma; 122 are open to participants now.
This study's planned enrollment of 120 is above the median of 32 across 475 interventional studies indexed under Neuroblastoma.
Browse Neuroblastoma studies →Sun Yat-sen University is the lead sponsor of 1,644 studies on the registry; 602 are open to participants now.
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1)Confirmed diagnosis of high-risk NB 2)1 year of age or above 3)Patient or parent/guardian must provide written informed consent to participate 4)If patient is sexually active, the patient agrees to use effective contraception 5)Confirmed negative urine pregnancy test for sexually active female of child-bearing potential (post-menarche)
Exclusion Criteria:
Inadequate pulmonary function defined as evidence of dyspnea at rest, exercise intolerance, and/or chronic oxygen requirement. In addition, room air pulse oximetry \< 94% and/or abnormal pulmonary function tests if these assessments are clinically indicated
Applicable for treatment with naxitamab in combination with GM-CSF only:
Suitable for patients with high risk neuroblastoma who obtain CR after chemotherapy combined with surgery, radiotherapy and/or hematopoietic stem cell transplantation. The treatment cycle is repeated every 4 weeks for a total of 5 courses, and discontinuation of nasetuzumab and GM-CSF should be considered if disease progression or unacceptable toxicity occurs.
Drug: Naxitamab monotherapy · Drug: GM-CSF
Suitable for high-risk group neuroblastoma treated by chemotherapy combined with surgery, radiotherapy and or hematopoietic stem cell transplantation patients with tumor residual or progression during treatment (refractory); Patients who relapse after initial treatment. Repeat every 3 weeks until tumor progression, patient withdrawal, or toxicity becomes intolerable, up to 8 procedures.
Drug: Irinotecan · Drug: Temozolomide · Drug: Naxitamab in combination therapy · Drug: GM-CSF with combination regimen
Suitable for high-risk group neuroblastoma treated by chemotherapy combined with surgery, radiotherapy and or hematopoietic stem cell transplantation patients with tumor residual or progression during treatment (refractory); Patients who relapse after initial treatment. Repeat every 3 weeks until tumor progression, patient withdrawal, or toxicity becomes intolerable, up to 8 procedures.
Drug: Irinotecan · Drug: Temozolomide · Drug: Naxitamab in combination therapy · Drug: GM-CSF with combination regimen · Drug: Sintilimab
Naxitamab is administered on days 1, 3, and 5
Also known as: hu3F8
Each treatment cycle is 28 days and is started with five days (days -4 to 0) of GM-CSF administered at 250 mcg/m2/day in advance of the start of naxitamab infusion. GM-CSF is thereafter administered at 500 mcg/m2/day on days 1 to 5.
Also known as: Recombinant Human Granulocyte/Macrophage Colony-stimulating Factor
Each HITS treatment cycle is 21 days. Irinotecan intravenously (IV) at 50 mg/m2/day will be administered from Day 1-5 concurrently with temozolomide orally at 150 mg/m2/day or 100 mg/m2/day.
Also known as: DNA topoisomerase I inhibitor
Each HITS treatment cycle is 21 days. Irinotecan intravenously (IV) at 50 mg/m2/day will be administered from Day 1-5 concurrently with temozolomide orally at 150 mg/m2/day or 100 mg/m2/day.
Also known as: Temozolomide for Injection
Naxitamab 2.25mg/kg IV will be administered on Days 2, 4, 8 and 10.
Also known as: hu3F8
GM-CSF 250 mcg/m2/day will be administered subcutaneously on Days 6-10.
Also known as: Recombinant Human Granulocyte/Macrophage Colony-stimulating Factor
Sintilimab was administerd with 3mg/kg (max 200mg) on day 11 every 3 weeks.
Also known as: PD-1 antibody
ORR
The proportion of patients who achieved CR or PR
Time frame: from start of naxitamab treatment to 1.5 years after EOT
DCR
The proportion of patients who achieved CR or PR or SD
Time frame: from start of naxitamab treatment to 1.5 years after EOT
EFS
The time from from start of naxitamab treatment to disease progression, recurrence
Time frame: from start of naxitamab treatment to 1.5 years after EOT
OS
The time from from start of naxitamab treatment to death or loss of follow-up
Time frame: from start of naxitamab treatment to 1.5 years after EOT
Plan to share: No
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