Adding Of Naxitamb In Induction Therapy For High Risk Neuroblastoma

Adding Of Naxitamb In Induction Therapy For High Risk Neuroblastoma: A Prospective, Single-Arm Clinical Study

To explore the efficacy of adding anti-GD2 immunotherapy (Naxitamab) at the stage of induction chemotherapy for newly diagnosed high risk neuroblastoma patients. To investigate wether anding Naxitamab in the induction phase will improve the response rate at the end of induction therapy and further improve the overall survival rate.

Study Overview

• Status: Recruiting
• Conditions: Neuroblastoma; GD2 Antibody (Naxitamab)
• Intervention / Treatment: Drug: Addition of anti-GD2 immunotherapy (Naxitamab) at the stage of induction chemotherapy

• Study Type: Observational
• Enrollment (Estimated): 64

Contacts and Locations

• Study Contact: Name: Wenyue Si, Bachelor; Phone Number: +86-(020)-38367270; Email: feky@gwcmc.org

Study Locations

• China
  • Guangdong
    • Guanzhou, Guangdong, China, 510000
      • Recruiting
      • Guangzhou Women and Children's Medical Center
      • Contact: Wenyue Si, Bachelor; Phone Number: +86-(020)-38767270; Email: feky@gwcmc.org

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Newly Diagnosed High risk Neuroblastoma

Description

Inclusion Criteria:

Neuroblastoma patients who meet certain criteria are eligible for enrollment in the following stages of diagnosis:

1. Children with newly diagnosed stage 4 neuroblastoma according to the International Neuroblastoma Staging System (INSS) who meet the following criteria are eligible for enrollment: i. Age > 18 months (> 547 days) regardless of biological characteristics; or ii. Age 12-18 months (365-547 days), with one of the following three unfavorable biological characteristics (MYCN amplification, pathological type of poor histopathological prognosis, and/or DNA index = 1); or iii. MYCN amplification (MYCN signal increase > 4 times compared to reference signal) regardless of age or other biological characteristics.
2. Children with newly diagnosed stage 3 INSS neuroblastoma who meet the following criteria are eligible for enrollment: i. MYCN amplification (MYCN signal increase > 4 times compared to reference signal) regardless of age or other biological characteristics; or ii. Age > 18 months (> 547 days), with pathological type of poor histopathological prognosis regardless of MYCN status.
3. Children with newly diagnosed stage 2A/2B INSS neuroblastoma with MYCN amplification (MYCN signal increase > 4 times compared to reference signal) regardless of age or other biological characteristics. The subject must be aged ≤ 21 years at the time of initial diagnosis, and must be aged > 12 months at the time of enrollment.

Exclusion criteria:

Infants less than 1 year old, those aged 12-18 months, INSS stage 4, and all INSS stage 3 patients with favorable biological characteristics (i.e., non-amplified MYCN, good pathological histopathological prognosis, and DNA index >1) are not eligible.

Subjects who have received immunosuppressive treatment (excluding local steroids) within the last 4 weeks prior to enrollment. Subjects who are currently receiving any investigational drug.

Any other medical condition that, in the opinion of the investigator, may interfere with the interpretation of results or affect the subject's ability to provide informed consent, the legal guardian's ability to provide informed consent, and the subject's cooperation and participation in the study, including but not limited to malabsorption syndrome, mental illness, or substance abuse. Subjects with significant comorbidities (any serious medical condition unrelated to cancer or its treatment that is not covered by the detailed exclusion criteria and is expected to interfere with the investigation drug(s) action or significantly increase the severity of the trial treatment toxicity)

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
High risk Neuroblastoma	Drug: Addition of anti-GD2 immunotherapy (Naxitamab) at the stage of induction chemotherapy; Addition of anti-GD2 immunotherapy (Naxitamab) at the stage of induction chemotherapy

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The number of the patients of VGPR or CR at the end of induction therapy	Measured by cross-sectional computed tomography (CT) or magnetic resonance imaging (MRI) imaging and/or by methyl iodobenzylguanidine (MIBG) or positron emission tomography (PET) scans and bone marrow (BM) responses, evaluated according to the 1993 International Neuroblastoma Response Criteria (INRC), and compared with relevant historical control groups.	5 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Objective Response Rate (ORR)	According to the International Neuroblastoma Response Criteria (INRC) of 1993 and 2017, the proportion of complete or partial responses was calculated in newly diagnosed high-risk group patients with neuroblastoma who received standard induction therapy. The disease status was evaluated by CT or MRI and/or MIBG or PET scans and bone marrow aspiration. After two courses of treatment, the proportion of complete or partial responses was calculated in newly diagnosed high-risk group patients with neuroblastoma who received standard induction therapy after two courses of treatment were completed.	5 years
The number of days a patient survives	The overall survival (OS) of patients receiving standard-induced chemotherapy and GD2 antibody combination therapy.	3 years after the last patient was enrolled
The number of days the patient survive without relapse or progression	The event-free survival (EFS) of patients receiving standard-induced chemotherapy and GD2 antibody combination therapy.	3 years after the last patient was enrolled
The objective response rate (ORR) after 6 cycles of treatment.	The objective response rate (ORR) after 6 cycles of treatment.	5 years
The number of patients with adverse events related to treatment	The number of patients with adverse events related to treatment	5 years

Collaborators and Investigators

• Sponsor: Guangzhou Women and Children's Medical Center

Study record dates

Study Major Dates

• Study Start (Actual): May 6, 2024
• Primary Completion (Estimated): December 31, 2029
• Study Completion (Estimated): December 31, 2032

Study Registration Dates

• First Submitted: August 18, 2024
• First Submitted That Met QC Criteria: August 24, 2024
• First Posted (Actual): August 28, 2024

Study Record Updates

• Last Update Posted (Actual): August 28, 2024
• Last Update Submitted That Met QC Criteria: August 24, 2024
• Last Verified: August 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Neuroectodermal Tumors, Primitive; Neuroectodermal Tumors, Primitive, Peripheral; Neuroblastoma; Physiological Effects of Drugs; Immunologic Factors; Immunomodulating Agents
• Other Study ID Numbers: neuroblastoma 2024

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No