Intensive Neoadjuvant Chemotherapy in Treating Young Patients Undergoing Surgical Resection for High-Risk Hepatoblastoma

NCT ID: NCT00077389

Last Updated: 2014-06-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE2

Total Enrollment

57 participants

Study Classification

INTERVENTIONAL

Study Start Date

2004-01-31

Brief Summary

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RATIONALE: Drugs used in chemotherapy work in different ways to stop tumor cells from dividing so they stop growing or die. Giving chemotherapy drugs before surgery may shrink the tumor so that it can be removed.

PURPOSE: This phase II trial is studying how well neoadjuvant chemotherapy works in treating young patients who are undergoing surgical resection for high-risk hepatoblastoma.

Detailed Description

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OBJECTIVES:

Primary

* Determine the efficacy and short-term toxicity of intensified neoadjuvant chemotherapy in children with high-risk hepatoblastoma undergoing surgical resection.
* Increase the rate of complete surgical resection in these patients by fully implementing liver transplantation as a valid treatment option for tumor removal when partial liver resection or other surgical options remain unfeasible even after extensive preoperative chemotherapy.
* Determine, prospectively, the role of this regimen in rendering unresectable tumors resectable in these patients.
* Determine the accuracy of initial imaging in predicting the surgical options (after treatment with this regimen) for patients presenting with unresectable disease.

Secondary

* Determine the overall survival and event-free survival of patients treated with this regimen (with an acceptable overall toxicity).
* Determine the toxicity of this regimen in these patients.
* Determine the response rate in patients treated with this regimen.
* Determine whether response to this regimen, defined by the modified RECIST criteria, can be used for better monitoring of response in these patients.
* Determine whether a fall in alpha-fetoprotein during this neoadjuvant regimen can be used as a prognostic factor in these patients.
* Determine, prospectively, radiological, surgical, and pathological characteristics of the tumor that might identify possible novel factors that might influence treatment choice and outcome in these patients.

OUTLINE: This is an open-label, multicenter study.

* Intensified neoadjuvant chemotherapy: Patients receive cisplatin IV over 24 hours on days 1, 8, 15, 29, 36, 43, 57, and 64; and doxorubicin IV over 1 hour OR over 24 hours on days 8, 9, 36, 37, 57, and 58. Patients determined to have resectable disease proceed to surgery.

Patients determined to have unresectable disease after neoadjuvant chemotherapy receive additional neoadjuvant chemotherapy comprising carboplatin IV over 1 hour on days 1 and 22 and doxorubicin IV over 1 hour OR over 24 hours on days 1, 2, 3, 22, 23, and 24.

Treatment continues in the absence of unacceptable toxicity.

* Surgery: Patients determined to have resectable disease undergo complete resection and possibly liver transplantation.
* Adjuvant chemotherapy\*: Patients who undergo complete surgical resection receive carboplatin IV over 1 hour on day 1 and doxorubicin IV over 1 hour OR over 24 hours on days 1 and 2. Treatment repeats every 3 weeks for a total of 3 courses.

NOTE: \*Patients who received additional neoadjuvant chemotherapy for unresectable disease do not receive adjuvant chemotherapy.

Patients are followed every 2-3 months for 2 years, every 3 months for 1 year, and then every 6 months for 2 years.

Peer Reviewed and Funded or Endorsed by Cancer Research UK

PROJECTED ACCRUAL: A total of 23-57 patients will be accrued for this study within 2 years.

Conditions

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Liver Cancer

Study Design

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Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Interventions

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carboplatin

Intervention Type DRUG

cisplatin

Intervention Type DRUG

doxorubicin hydrochloride

Intervention Type DRUG

adjuvant therapy

Intervention Type PROCEDURE

conventional surgery

Intervention Type PROCEDURE

neoadjuvant therapy

Intervention Type PROCEDURE

Eligibility Criteria

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Inclusion Criteria

DISEASE CHARACTERISTICS:

* Histologically confirmed hepatoblastoma
* High-risk disease, meeting criteria for at least 1 of the following:

* Tumor involving all 4 hepatic sections
* Evidence of abdominal extrahepatic disease
* Presence of metastases
* Alpha-fetoprotein \< 100 ng/mL at diagnosis
* Must have had a prior diagnostic biopsy within the past 15 days
* No recurrent disease

PATIENT CHARACTERISTICS:

Age

* Under 18

Performance status

* Not specified

Life expectancy

* Not specified

Hematopoietic

* Not specified

Hepatic

* AST and/or ALT ≤ 3 times normal

Renal

* Glomerular filtration rate ≥ 60 mL/min

Cardiovascular

* Shortening fraction ≥ 29% OR
* Ejection fraction ≥ 40%

Other

* Not pregnant
* Negative pregnancy test
* No pre-existing clinically relevant bilateral hearing loss
* No other condition that would preclude study participation

PRIOR CONCURRENT THERAPY:

Biologic therapy

* Not specified

Chemotherapy

* No prior chemotherapy

Endocrine therapy

* Not specified

Radiotherapy

* Not specified

Surgery

* Not specified

Other

* No prior therapy for hepatoblastoma
Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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University of Leicester

OTHER

Sponsor Role lead

Principal Investigators

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Margaret Childs

Role: STUDY_CHAIR

Children's Cancer and Leukaemia Group

Locations

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Institut Gustave Roussy

Villejuif, , France

Site Status

Our Lady's Hospital for Sick Children Crumlin

Dublin, , Ireland

Site Status

Emma Kinderziekenhuis

Amsterdam, , Netherlands

Site Status

Birmingham Children's Hospital

Birmingham, England, United Kingdom

Site Status

Institute of Child Health at University of Bristol

Bristol, England, United Kingdom

Site Status

Addenbrooke's Hospital

Cambridge, England, United Kingdom

Site Status

Leeds Cancer Centre at St. James's University Hospital

Leeds, England, United Kingdom

Site Status

Leicester Royal Infirmary

Leicester, England, United Kingdom

Site Status

Children's Cancer and Leukaemia Group

Leicester, England, United Kingdom

Site Status

Royal Liverpool Children's Hospital, Alder Hey

Liverpool, England, United Kingdom

Site Status

Middlesex Hospital

London, England, United Kingdom

Site Status

Great Ormond Street Hospital for Children

London, England, United Kingdom

Site Status

Royal Manchester Children's Hospital

Manchester, England, United Kingdom

Site Status

Sir James Spence Institute of Child Health at Royal Victoria Infirmary

Newcastle upon Tyne, England, United Kingdom

Site Status

Queen's Medical Centre

Nottingham, England, United Kingdom

Site Status

Oxford Radcliffe Hospital

Oxford, England, United Kingdom

Site Status

Children's Hospital - Sheffield

Sheffield, England, United Kingdom

Site Status

Southampton General Hospital

Southampton, England, United Kingdom

Site Status

Royal Marsden - Surrey

Sutton, England, United Kingdom

Site Status

Royal Belfast Hospital for Sick Children

Belfast, Northern Ireland, United Kingdom

Site Status

Royal Aberdeen Children's Hospital

Aberdeen, Scotland, United Kingdom

Site Status

Royal Hospital for Sick Children

Edinburgh, Scotland, United Kingdom

Site Status

Royal Hospital for Sick Children

Glasgow, Scotland, United Kingdom

Site Status

Childrens Hospital for Wales

Cardiff, Wales, United Kingdom

Site Status

Countries

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France Ireland Netherlands United Kingdom

References

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Zsiros J, Brugieres L, Brock P, Roebuck D, Maibach R, Zimmermann A, Childs M, Pariente D, Laithier V, Otte JB, Branchereau S, Aronson D, Rangaswami A, Ronghe M, Casanova M, Sullivan M, Morland B, Czauderna P, Perilongo G; International Childhood Liver Tumours Strategy Group (SIOPEL). Dose-dense cisplatin-based chemotherapy and surgery for children with high-risk hepatoblastoma (SIOPEL-4): a prospective, single-arm, feasibility study. Lancet Oncol. 2013 Aug;14(9):834-42. doi: 10.1016/S1470-2045(13)70272-9. Epub 2013 Jul 4.

Reference Type DERIVED
PMID: 23831416 (View on PubMed)

Weeda VB, Murawski M, McCabe AJ, Maibach R, Brugieres L, Roebuck D, Fabre M, Zimmermann A, Otte JB, Sullivan M, Perilongo G, Childs M, Brock P, Zsiros J, Plaschkes J, Czauderna P, Aronson DC. Fibrolamellar variant of hepatocellular carcinoma does not have a better survival than conventional hepatocellular carcinoma--results and treatment recommendations from the Childhood Liver Tumour Strategy Group (SIOPEL) experience. Eur J Cancer. 2013 Aug;49(12):2698-704. doi: 10.1016/j.ejca.2013.04.012. Epub 2013 May 15.

Reference Type DERIVED
PMID: 23683550 (View on PubMed)

Other Identifiers

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SIOP-SIOPEL-4

Identifier Type: -

Identifier Source: secondary_id

EU-20336

Identifier Type: -

Identifier Source: secondary_id

CCLG-LT-2004-09

Identifier Type: -

Identifier Source: secondary_id

CDR0000350221

Identifier Type: -

Identifier Source: org_study_id

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