Using arsenic trioxide with chemotherapy to treat pediatric cancer with p53 mutations
Clinical Research on Efficacy and Safety of Arsenic Trioxide Combined With Chemotherapy in p53-mutated Pediatric Cancer Patients：A Prospective，Single-arm, Multi-center Study
This study is testing if adding arsenic trioxide to chemotherapy can help children with Li-Fraumeni syndrome and p53 mutations fight their cancer more safely.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 50 (estimated) |
| Ages | N/A to 18 Years |
| Sex | All |
| Sponsor | Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University Academic / other |
| Drugs / interventions | chemotherapy, radiation |
| Locations | 1 site (Guangzhou, Guangdong) |
| Trial ID | NCT06088030 on ClinicalTrials.gov |
What this trial studies
This clinical trial is a prospective, single-arm, multi-center investigation aimed at evaluating the efficacy and safety of arsenic trioxide in combination with chemotherapy for treating pediatric cancers associated with p53 mutations. The study focuses on children diagnosed with Li-Fraumeni syndrome, a hereditary condition that significantly increases the risk of developing various cancers at a young age. Given the limitations of current treatment options, which often involve DNA-damaging therapies, this trial seeks to explore a novel approach that may restore the function of mutant p53 without causing further DNA damage. Participants will be closely monitored for treatment response and safety outcomes.
Who should consider this trial
Good fit: Ideal candidates for this study are pediatric patients under 18 years old with either germline or somatic p53 mutations.
Not a fit: Patients with critical organ failures, such as heart, brain, or kidney failure, may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could provide a safer and more effective treatment option for children with p53-mutated cancers.
How similar studies have performed: While there is limited research specifically targeting Li-Fraumeni syndrome, the use of arsenic trioxide in other cancer contexts has shown promise, suggesting potential for success in this novel application.
Eligibility criteria
Show full inclusion / exclusion criteria
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. Exclusion Criteria: Patients with one or more critical organs failure such as heart, brain, kidney failure.
Where this trial is running
Guangzhou, Guangdong
- Sun Yat-sen Memorial Hospital, Sun Yat-sen University — Guangzhou, Guangdong, China (Recruiting)
Study contacts
- Study coordinator: Yang Li, Professor
- Email: drliyang@126.com
- Phone: +8602081332456
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.