MedPath

Cisplatin With or Without Sodium Thiosulfate in Treating Young Patients With Stage I, II, or III Childhood Liver Cancer

Phase 3
Completed
Conditions
Liver Cancer
Ototoxicity
Interventions
Registration Number
NCT00652132
Lead Sponsor
University of Birmingham
Brief Summary

RATIONALE: Drugs used in chemotherapy, such as cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Chemoprotective drugs, such as sodium thiosulfate, may protect normal cells from the side effects of chemotherapy. It is not yet known whether giving sodium thiosulfate is effective in reducing hearing damage caused by cisplatin in treating young patients with liver cancer.

PURPOSE: This randomized phase III trial is studying how well sodium thiosulfate works to decrease hearing loss caused by cisplatin in treating young patients with stage I, stage II, or stage III childhood liver cancer.

Detailed Description

OBJECTIVES:

Primary

* To assess the efficacy of sodium thiosulfate (STS) to reduce the hearing impairment caused by cisplatin chemotherapy.

Secondary

* To carefully monitor any potential impact of STS on response to cisplatin and survival.

* To assess the short- and long-term tolerability of the combination of STS and cisplatin

* To prospectively evaluate and validate biological, radiological and pathological features of standard-risk hepatoblastoma for future risk adapted management

* To investigate the effect of STS on the formation of cisplatin-DNA adducts.

* To prospectively collect patient DNA specifically for the analysis of possible genetic factors that may contribute to the development of treatment-related ototoxicity and nephrotoxicity

OUTLINE: This is a multicenter study. Patients are stratified according to country, median age (\< 15 months vs ≥ 15 months), and PRETEXT tumor classification (I vs II vs III). Patients are randomized to 1 of 2 treatment arms.

* Arm I (Neoadjuvant and adjuvant cisplatin): Patients receive cisplatin IV over 6 hours on day 1. Treatment repeats every 2 weeks for 4 courses. Patients with progressive disease after course 4 are taken off study. Patients without evidence of disease progression proceed to surgery. Beginning within 3 weeks after surgery, patients receive cisplatin IV over 6 hours on day 1. Treatment repeats every 2 weeks for 2 courses in the absence of disease progression or unacceptable toxicity.

* Arm II (Neoadjuvant and adjuvant cisplatin and sodium thiosulphate): Patients receive cisplatin IV over 6 hours and sodium thiosulphate IV over 15 minutes (beginning 6 hours after completion of cisplatin) on day 1. Treatment repeats every 2 weeks for 4 courses. Patients with progressive disease after course 4 are taken off study. Patients without evidence of disease progression proceed to surgery. Beginning within 3 weeks after surgery, patients receive cisplatin IV over 6 hours and sodium thiosulphate IV over 15 minutes (as in neoadjuvant therapy) on day 1. Treatment repeats every 2 weeks for 2 courses in the absence of disease progression or unacceptable toxicity.

Patients undergo blood collection and tumor biopsies periodically for biological and pharmacological studies consisting of biomarker analysis, gene expression profiling, IHC, proteomic analysis, and gene rearrangement analysis. Patients undergo auditory evaluations at baseline, and at the completion of study treatment or at an age of at least 3.5 years to measure ototoxicity and hearing impairment.

After completion of study treatment, patients are followed periodically for at least 5 years.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
116
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Arm II (cisplatin + STS)sodium thiosulfateNeoadjuvant and adjuvant cisplatin and sodium thiosulphate (STS): patients receive cisplatin IV over 6 hours and sodium thiosulphate IV over 15 minutes (beginning 6 hours after completion of cisplatin) on day 1. Treatment repeats every 2 weeks for 4 courses. Patients with progressive disease after course 4 are taken off study. Patients without evidence of disease progression proceed to surgery. Beginning within 3 weeks after surgery, patients receive cisplatin IV over 6 hours and sodium thiosulphate IV over 15 minutes (as in neoadjuvant therapy) on day 1. Treatment repeats every 2 weeks for 2 courses in the absence of disease progression or unacceptable toxicity.
Arm I (cisplatin)cisplatinNeoadjuvant and adjuvant cisplatin: patients receive cisplatin IV over 6 hours on day 1. Treatment repeats every 2 weeks for 4 courses. Patients with progressive disease after course 4 are taken off study. Patients without evidence of disease progression proceed to surgery. Beginning within 3 weeks after surgery, patients receive cisplatin IV over 6 hours on day 1. Treatment repeats every 2 weeks for 2 courses in the absence of disease progression or unacceptable toxicity.
Arm II (cisplatin + STS)cisplatinNeoadjuvant and adjuvant cisplatin and sodium thiosulphate (STS): patients receive cisplatin IV over 6 hours and sodium thiosulphate IV over 15 minutes (beginning 6 hours after completion of cisplatin) on day 1. Treatment repeats every 2 weeks for 4 courses. Patients with progressive disease after course 4 are taken off study. Patients without evidence of disease progression proceed to surgery. Beginning within 3 weeks after surgery, patients receive cisplatin IV over 6 hours and sodium thiosulphate IV over 15 minutes (as in neoadjuvant therapy) on day 1. Treatment repeats every 2 weeks for 2 courses in the absence of disease progression or unacceptable toxicity.
Primary Outcome Measures
NameTimeMethod
Rate of Brock grade ≥ 1 hearing lossEnd of trial treatment or at an age of 3.5 years, whichever is later

To investigate if the administration of sodium thiosulfate simultaneously with the administration of Cisplatin significantly reduces the hearing impairment

Secondary Outcome Measures
NameTimeMethod
Complete remissionEnd of trial treatment

Lack of evidence of residual disease and normal (for age) alpha-foetal protein (AFP). To establish a complete remission all of the following requirements must be fulfilled:

* No evidence of tumour intra-abdominally: negative abdominal (including hepatic) ultrasound or CT scan or Magnetic resonance imaging

* No evidence of metastases: clear chest X-ray (PA and lateral) for non-metastatic patients. (Normal lung CT scan for patients with lung metastasis at diagnosis, who are high-risk by definition and not treated according to SIOPEL 6).

* Serum AFP level either normal or compatible with age for at least 4 weeks after normalisation.

Toxicity as graded by CTCAE v 3.030 days post treatment

Adverse drug reactions are defined as adverse events, which are possibly, probably or definitely related to the trial treatment. They will be assessed according to NCI CTCAE v 3.0.

Feasibility of central audiology reviewEnd of trial treatment or at an age of 3.5 years, whichever is later

The feasibility of central review

Event-free survival (EFS)Until first event or up to 5 years

Calculated from the time of randomisation to the first of the following events: progression, relapse, secondary primary malignancy or death.

Overall survival (OS)Until event or up to 5 years

Calculated from the time of randomisation to death.

Long-term renal clearanceUntil event or up to 5 years

By clearance method either EDTA, iohexol or inulin.

Response to preoperative chemotherapyFollowing completion of preoperative chemotherapy

Defined as:

Complete response (CR):

no evidence of disease and normal serum AFP value (for age).

Partial response (PR):

any tumour volume shrinkage associated with a decreasing serum AFP value, \> 1 log below the original measurement.

Stable disease (SD):

no tumour volume change and no change, or \< 1 log fall of the serum AFP concentration.

Progressive disease (PD):

unequivocal increase in 1 or more dimensions and/or any unequivocal increase of the serum AFP concentration (three successive 1-2 weekly determinations) even without clinical (physical and/or radiological) evidence of tumour re-growth.

Complete resectionWithin 2 weeks after surgery.

Total macroscopic removal of the tumour as reported by the surgeon and pathologist. In case of any doubt the lack of residual tumour must be confirmed with imaging studies performed

Trial Locations

Locations (16)

Southampton Children's Hospital

🇬🇧

Southampton, United Kingdom

Royal Manchester Children's Hospital

🇬🇧

Manchester, England, United Kingdom

Royal Hospital for Sick Children

🇬🇧

Glasgow, Scotland, United Kingdom

The Noah's Ark Children's Hospital for Wales

🇬🇧

Cardiff, United Kingdom

John Radcliffe Hospital

🇬🇧

Oxford, United Kingdom

Bristol Royal Hospital for Childre

🇬🇧

Bristol, England, United Kingdom

Sheffield Hallam University - City Campus

🇬🇧

Sheffield, England, United Kingdom

Great Ormond Street Hospital for Children

🇬🇧

London, England, United Kingdom

Addenbrooke's Hospital

🇬🇧

Cambridge, England, United Kingdom

Royal Marsden - London

🇬🇧

London, England, United Kingdom

Royal Hospital For Sick Children

🇬🇧

Edinburgh, United Kingdom

Queen's Medical Centre

🇬🇧

Nottingham, England, United Kingdom

Royal Aberdeen Children's Hospital

🇬🇧

Aberdeen, Scotland, United Kingdom

Leicester Royal Infirmary

🇬🇧

Leicester, United Kingdom

Alder Hey Children's Hospital Trust

🇬🇧

Liverpool, United Kingdom

Birmingham Children's Hospital

🇬🇧

Birmingham, England, United Kingdom

© Copyright 2025. All Rights Reserved by MedPath