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

Phase 2

Recruiting

• Conditions: Li-Fraumeni Syndrome; p53 Mutations; Pediatric Cancer
• Interventions: Drug: Arsenic trioxide

• Registration Number: NCT06088030
• Lead Sponsor: Yang Li

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.

Detailed Description

Germline mutation on tumor suppressor p53 can result in Li-Fraumeni syndrome (LFS), a hereditary condition characterized by the development of multiple cancer types, often at a young or middle age. LFS individuals face a lifetime cancer risk of up to 80-90%, with approximately half of them developing cancer by the age of 30 years. Despite the significantly i...

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study First Submitted: 2023-10-12
• Study First Submitted QC: 2023-10-12
• Study First Posted: 2023-10-18
• Study Start: 2023-12-13
• Status Verified: 2024-01
• Last Update Submitted: 2024-01-02
• Last Update Posted: 2024-01-03
• Primary Completion: 2031-12-14
• Study Completion: 2031-12-14

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

1. Pathological diagnosis basis of malignant tumor；
2. Patients not more than 18 years old;
3. 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);
4. There are measurable lesions;
5. Guardians agreed and signed informed consent.

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Exclusion Criteria

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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Arsenic trioxide combined chemotherapy	Arsenic trioxide	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.

Primary Outcome Measures

Name	Time	Method
Objective response rate	Four weeks after ATO-combined chemotherapy	Objective response rate

Secondary Outcome Measures

Name	Time	Method
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	Progression Free Survival
Overall survival	From date of randomization to death whichever the cause is, up to 3 years.	Overall survival
Incidence of Adverse Events	From date of ATO-combined chemotherapy until the date of first documented adverse event, and follow up for 3 years	Incidence of Adverse Events

Trial Locations

Locations (1)

Sun Yat-sen Memorial Hospital, Sun Yat-sen University; 🇨🇳 Guangzhou, Guangdong, China