Clinical Analysis of Naxitamab (hu3F8) in the Treatment of Pediatric High Risk or Refractory/ Relapsed Neuroblastoma

NCT06013618 | Recruiting Phase 2

• 1 other identifier: 3F8-RWS
• Study Type: interventional
• Target: 120
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Neuroblastoma

Outcome Measures

Primary Outcomes (1)

• ORR

  The proportion of patients who achieved CR or PR

  from start of naxitamab treatment to 1.5 years after EOT

Secondary Outcomes (3)

• DCR

  from start of naxitamab treatment to 1.5 years after EOT

• EFS

  from start of naxitamab treatment to 1.5 years after EOT

• OS

  from start of naxitamab treatment to 1.5 years after EOT

Study Arms (3)

naxitamab and GM-CSF only

OTHER

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

naxitamab and GM-CSF in combination with irinotecan and temozolomide

OTHER

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

naxitamab and GM-CSF in combination with irinotecan and temozolomide and PD-1 antibody

OTHER

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

Interventions

Naxitamab monotherapy DRUG

Naxitamab is administered on days 1, 3, and 5

Also known as: hu3F8

naxitamab and GM-CSF only

GM-CSF DRUG

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

naxitamab and GM-CSF only

Irinotecan DRUG

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

naxitamab and GM-CSF in combination with irinotecan and temozolomide; naxitamab and GM-CSF in combination with irinotecan and temozolomide and PD-1 antibody

Temozolomide DRUG

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 and GM-CSF in combination with irinotecan and temozolomide; naxitamab and GM-CSF in combination with irinotecan and temozolomide and PD-1 antibody

Naxitamab in combination therapy DRUG

Naxitamab 2.25mg/kg IV will be administered on Days 2, 4, 8 and 10.

Also known as: hu3F8

naxitamab and GM-CSF in combination with irinotecan and temozolomide; naxitamab and GM-CSF in combination with irinotecan and temozolomide and PD-1 antibody

GM-CSF with combination regimen DRUG

GM-CSF 250 mcg/m2/day will be administered subcutaneously on Days 6-10.

Also known as: Recombinant Human Granulocyte/Macrophage Colony-stimulating Factor

naxitamab and GM-CSF in combination with irinotecan and temozolomide; naxitamab and GM-CSF in combination with irinotecan and temozolomide and PD-1 antibody

Sintilimab DRUG

Sintilimab was administerd with 3mg/kg (max 200mg) on day 11 every 3 weeks.

Also known as: PD-1 antibody

naxitamab and GM-CSF in combination with irinotecan and temozolomide and PD-1 antibody

Eligibility Criteria

• Age: 12 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• )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)

You may not qualify if:

• Significant organ toxicity
• Known or suspected allergy or hypersensitivity to anti-GD2 antibodies or to GM-CSF or its s components.
• Patient is pregnant, planning to become pregnant (while being treated with naxitamab) or is currently breastfeeding
• Patient will undergo treatment with another investigational drug, whilst being treated with naxitamab or has received another investigational drug within the 4 weeks prior to commencing treatment with naxitamab
• Patient is either eligible and able to participate in or is currently participating in an active interventional Y-mAbs sponsored clinical trial with naxitamab within the indication applied for
• Patient is unable to comply with the naxitamab treatment or has a medical condition that would potentially increase the severity of the toxicities experienced from naxitamab treatment at the discretion of the treating physician
• Left ventricular ejection fraction of \<50% by echocardiography OR other clinically relevant cardiac disorders at the discretion of the investigator
• 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:
• Patient has active progression of the NB disease
• Patient has active NB disease at primary site or soft-tissue metastasis
• Patient has known CNS metastases when initiating naxitamab treatment

Sponsors & Collaborators

• Sun Yat-sen University: lead
• Hainan General Hospital: collaborator

Study Sites (1)

Sun Yat-sen University Cancer Center

Guangzhou, Guangdong, 510060, China

RECRUITING

MeSH Terms

Conditions

Neuroblastoma

Interventions

naxitamab; Granulocyte-Macrophage Colony-Stimulating Factor; Colony-Stimulating Factors; Irinotecan; Topoisomerase I Inhibitors; Temozolomide; Injections; Combined Modality Therapy; sintilimab; spartalizumab

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Intervention Hierarchy (Ancestors)

Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors; Camptothecin; Alkaloids; Heterocyclic Compounds; Topoisomerase Inhibitors; Enzyme Inhibitors; Molecular Mechanisms of Pharmacological Action; Pharmacologic Actions; Chemical Actions and Uses; Antineoplastic Agents; Therapeutic Uses; Dacarbazine; Triazenes; Organic Chemicals; Imidazoles; Azoles; Heterocyclic Compounds, 1-Ring; Drug Administration Routes; Drug Therapy; Therapeutics

Study Officials

• Yizhuo Zhang

  SunYat Sen University Cancer Center

  STUDY CHAIR

Central Study Contacts

Yizhuo Zhang, MD

CONTACT

0087342460; zhangyzh@sysucc.org.cn

Juan Wang

CONTACT

008687342660; wangjuan@sysucc.org.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director

Study Record Dates

• First Submitted: August 19, 2023
• First Posted: August 28, 2023
• Study Start: June 19, 2023
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2027
• Last Updated: June 17, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)