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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00077389
Recruitment Status : Unknown
Verified December 2009 by National Cancer Institute (NCI).
Recruitment status was:  Active, not recruiting
First Posted : February 12, 2004
Last Update Posted : June 24, 2014
Information provided by:
National Cancer Institute (NCI)

February 10, 2004
February 12, 2004
June 24, 2014
January 2004
Not Provided
Rate of complete remission after completion of study therapy
Not Provided
Complete list of historical versions of study NCT00077389 on Archive Site
  • Complete resection rate
  • Response rate to preoperative chemotherapy
  • Rate of grade 2 cardiac and renal, grade 3 otological, and grade 4 nonhematological toxicity as assessed during and after completion of study therapy
  • Overall survival
  • Event-free survival
Not Provided
Not Provided
Not Provided
Intensive Neoadjuvant Chemotherapy in Treating Young Patients Undergoing Surgical Resection for High-Risk Hepatoblastoma
Intensified Pre-Operative Chemotherapy And Radical Surgery For High Risk Hepatoblastoma

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.



  • 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.


  • 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.

Phase 2
Masking: None (Open Label)
Primary Purpose: Treatment
Liver Cancer
  • Drug: carboplatin
  • Drug: cisplatin
  • Drug: doxorubicin hydrochloride
  • Procedure: adjuvant therapy
  • Procedure: conventional surgery
  • Procedure: neoadjuvant therapy
Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Unknown status
Not Provided
Not Provided
Not Provided


  • 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



  • Under 18

Performance status

  • Not specified

Life expectancy

  • Not specified


  • Not specified


  • AST and/or ALT ≤ 3 times normal


  • Glomerular filtration rate ≥ 60 mL/min


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


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


Biologic therapy

  • Not specified


  • No prior chemotherapy

Endocrine therapy

  • Not specified


  • Not specified


  • Not specified


  • No prior therapy for hepatoblastoma
Sexes Eligible for Study: All
up to 17 Years   (Child)
Contact information is only displayed when the study is recruiting subjects
France,   Ireland,   Netherlands,   United Kingdom
Not Provided
Not Provided
Not Provided
Not Provided
University of Leicester
Not Provided
Study Chair: Margaret Childs Children's Cancer and Leukaemia Group
National Cancer Institute (NCI)
December 2009

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP