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

ConditionsPediatric CancerLi-Fraumeni Syndromep53 Mutations
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
AgeUp to 18
SponsorYang Li

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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;

Disqualifiers

None

Trial design

Design model

Single group

Treatments tested in this trial

  • 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.

Treatment groups

50 Participants
are divided into 1 treatment group
Group A: Arsenic trioxide combined chemotherapyExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Objective response rate

Objective response rate

Time frame
Four weeks after ATO-combined chemotherapy

Secondary outcomes

1

Progression Free Survival

Progression Free Survival

Time frame
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
2

Overall survival

Overall survival

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

Incidence of Adverse Events

Incidence of Adverse Events

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

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Yang Li

Lead sponsor

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

Sponsor institution

Ruijin Hospital

Collaborator

Source ClinicalTrials.gov
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