Phase II Study of Combined Chemotherapy With Arsenic Trioxide in Stage 4/M Neuroblastoma

Clinical Research on Efficacy and Safety of Combined Chemotherapy With Arsenic Trioxide in Stage 4/M Neuroblastoma：A Prospective，Single-arm, Open-label, Multi-center Study

This clinical trial aims to explore and evaluate the efficacy and safety of combined chemotherapy with arsenic trioxide for stage 4/M neuroblastoma.

Study Overview

• Status: Active, not recruiting
• Conditions: Neuroblastoma
• Intervention / Treatment: Drug: Arsenic Trioxide

Detailed Description

This study is a prospective, single-arm, open-lable, multi-center clinical trial. Children≤ 14 years old are eligible for this study if they were newly diagnosed with neuroblastoma and assessed as stage 4 according to the International Neuroblastoma Staging System (INSS) or stage M according to the International Neuroblastoma Risk Group (INRG) respectively. Patients enrolled in this study will receive combined induction chemotherapy with arsenic trioxide following an modifed protocol based on N7 and NB2004 protocols. Objective response rate (ORR) at 4 weeks after completing induction chemotherapy was defined as the main outcome and adverse events were monitored and graded in the meantime.

• Study Type: Interventional
• Enrollment (Actual): 80
• Phase: Phase 2

Contacts and Locations

Study Locations

• China
  • Guangdong
    • Guangzhou, Guangdong, China, 510120
      • Sun Yat-sen Memorial Hospital, Sun Yat-sen University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 14 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Untreated Stage 4/M neuroblastoma patients according to the International Neuroblastoma Staging System（INSS) or the International Neuroblastoma Risk Group (INRG) staging system;
2. Patients not more than 14 years old;
3. There are measurable lesions;
4. Guardians agreed and signed informed consent.

Exclusion Criteria:

1. Patients who had suffered from other tumors and received chemotherapy or abdominal radiotherapy.
2. Patients with one or more critical organs failure such as heart, brain, kidney failure.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: ATO-combined chemotherapy; Patients receive combined induction chemotherapy with arsenic trioxide.	Drug: Arsenic Trioxide; Arsenic trioxide（ATO） is administered 0.16mg/kg per day over eight hours IV daily for ten days. Patients will receive ATO alone on days 1-2 and combined with conventional induction chemotherapy on days 3-10. Nine cycles at most of ATO-combined chemotherapy were applied in the whole scheme.; Other Names: ATO

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Objective Response Rate	The percentage of patients who had complete remission（CR）or partial remission（PR）after induction chemotherapy combined ATO. Per Response Evaluation Criteria In Solid Tumors Criteria (WHO criteria) for target lesions and assessed by MRI or CT: CR: All lesions completely disappear, maintained for at least 4 weeks. PR: Estimated tumor size reduction by more than 50%, maintained for at least 4 weeks.	Four weeks after ATO-combined induction chemotherapy

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Survival Rate	The proportion of patients who are alive from the date of randomization to 3 years, regardless of the cause of death.	3 years.
Progression Free Survival Rate	The proportion of patients who have not experienced progression or death from any cause, whichever occurs first, within a maximum of 3 years from the date of enrollment. Treatment response was evaluated according to the WHO criteria for the efficacy of solid tumours, which was classified as complete response (CR), partial response (PR), stable disease (SD) and progressive disease (PD). PD was characterized as more than 25% increase in one or more lesions or appearance of new lesions.	3 years.
Number of Participants With Adverse Events	Adverse events are monitored and graded by the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE)	From date of ATO-combined chemotherapy until the date of first documented adverse event, and follow up for 3 years.

Collaborators and Investigators

• Sponsor: Yang Li
• Investigators: Study Chair: Yang Li, Professor, Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University

Study record dates

Study Major Dates

• Study Start (Actual): June 12, 2017
• Primary Completion (Actual): August 30, 2023
• Study Completion (Estimated): December 30, 2028

Study Registration Dates

• First Submitted: January 21, 2018
• First Submitted That Met QC Criteria: April 11, 2018
• First Posted (Actual): April 20, 2018

Study Record Updates

• Last Update Posted (Actual): May 21, 2025
• Last Update Submitted That Met QC Criteria: May 19, 2025
• Last Verified: May 1, 2025

More Information

Terms related to this study

• Keywords: Children; Arsenic Trioxide; Neuroblastoma
• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Histologic Type; Neoplasms, Glandular and Epithelial; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neuroblastoma; Antineoplastic Agents; Arsenic Trioxide
• Other Study ID Numbers: SYS-C-202007

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