Arsenic Trioxide Combined With Chemotherapy for the Treatment of p53-mutated Pediatric Cancer

NCT06088030 | Recruiting Phase 2

• 1 other identifier: SYS-202309
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

This prospective, single-arm, multi-center clinical trial aims to explore and evaluate the efficacy and safety of arsenic trioxide combined with chemotherapy for pediatric cancer with p53 mutation.

Conditions

Pediatric Cancer; Li-Fraumeni Syndrome; p53 Mutations

Keywords

Arsenic Trioxide

Outcome Measures

Primary Outcomes (1)

• Objective response rate

  Objective response rate

  Four weeks after ATO-combined chemotherapy

Secondary Outcomes (3)

• Progression Free Survival

  From the date of patients enrolled to the date of of first documented progression or date of death from any cause, whichever came first, assessed up to 3 years

• Overall survival

  From date of randomization to death whichever the cause is, up to 3 years.

• Incidence of Adverse Events

  From date of ATO-combined chemotherapy until the date of first documented adverse event, and follow up for 3 years

Study Arms (1)

Arsenic trioxide combined chemotherapy

EXPERIMENTAL

Patients with p53-mutated pediatric cancer should initially undergo the corresponding first-line chemotherapy regimen. If the patient is evaluated as PD/SD, arsenic trioxide (ATO) will be administered in conjunction with previous conventional chemotherapy on the third day of each treatment cycle.

Drug: Arsenic trioxide

Interventions

Arsenic trioxide DRUG

Patients should be treated with the corresponding first-line chemotherapy regimen first, for example: Neuroblastoma: CAV (cyclophosphamide, pinarubicin, vincristine), PVP (cisplatin, etoposide) ,CT (cyclophosphamide, topotecan).If patients was evaluted as PD/SD after treatments, arsenic trioxide (ATO) will be administered 0.18mg/kg per day over six hours IV daily for ten days in combination with previous chemotherapy regimen on the third day of each treatment cycle. Other pediatric tumors with TP53 mutations not mentioned above will have similar treatment regimens. If the efficacy of the conventional standard chemotherapy regimen is evaluated as PD/SD, then the next course of treatment will be combined with ATO on the basis of the standard chemotherapy regimen.

Also known as: ATO

Arsenic trioxide combined chemotherapy

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Pathological diagnosis basis of malignant tumor；
• Patients not more than 18 years old;
• Patient has either germline or somatic p53 mutations, which was shown to be partially/completely restored to function by ATO in in vitro experiments (http://www.rescuep53.net);
• There are measurable lesions;
• Guardians agreed and signed informed consent.

You may not qualify if:

• Patients with one or more critical organs failure such as heart, brain, kidney failure.

Sponsors & Collaborators

• Yang Li: lead
• Ruijin Hospital: collaborator

Study Sites (1)

Sun Yat-sen Memorial Hospital, Sun Yat-sen University

Guangzhou, Guangdong, 510120, China

RECRUITING

MeSH Terms

Conditions

Neoplasms; Li-Fraumeni Syndrome

Interventions

Arsenic Trioxide

Condition Hierarchy (Ancestors)

Neoplastic Syndromes, Hereditary; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; DNA Repair-Deficiency Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Arsenicals; Inorganic Chemicals; Oxides; Oxygen Compounds

Study Officials

• Yang Li, Professor

  Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University

  STUDY CHAIR

Central Study Contacts

Yang Li, Professor

CONTACT

+8602081332456; drliyang@126.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: October 12, 2023
• First Posted: October 18, 2023
• Study Start: December 13, 2023
• Primary Completion (Estimated): December 14, 2031
• Study Completion (Estimated): December 14, 2031
• Last Updated: January 3, 2024

Record last verified: 2024-01

Locations

CN (1)