Irinotecan in Treating Young Patients With Refractory or Recurrent 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: NCT00287976
Recruitment Status : Unknown
Verified June 2009 by National Cancer Institute (NCI).
Recruitment status was:  Active, not recruiting
First Posted : February 7, 2006
Last Update Posted : September 17, 2013
Information provided by:
National Cancer Institute (NCI)

Brief Summary:

RATIONALE: Drugs used in chemotherapy, such as irinotecan, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing.

PURPOSE: This phase II trial is studying how well irinotecan works in treating young patients with refractory or recurrent hepatoblastoma.

Condition or disease Intervention/treatment Phase
Liver Cancer Drug: irinotecan hydrochloride Phase 2

Detailed Description:



  • Determine the biological activity of irinotecan hydrochloride, when given on a prolonged schedule, in terms of response rate and rate of early progression, in young patients with refractory or recurrent hepatoblastoma.


  • Determine the duration of response in patients showing stable disease or an objective response (partial or complete response) to this drug.
  • Determine the time to progression and overall survival of patients treated with this drug.
  • Determine the rate of resectability in patients treated with this drug.
  • Determine the toxicity of this drug in these patients.

OUTLINE: This is a multicenter study.

Patients receive irinotecan IV over 1 hour on days 1-5 and 8-12. Treatment repeats every 21 days for up to 4 courses in the absence of disease progression or unacceptable toxicity.

Patients undergo evaluation for tumor resectability after courses 2, 3, or 4. Patients whose disease is considered resectable at any of these time points proceed to surgery.

After completion of study treatment, patients are followed periodically.

PROJECTED ACCRUAL: A total of 30 patients will be accrued for this study.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Irinotecan Single-Drug Treatment For Children With Refractory or Recurrent Hepatoblastoma
Study Start Date : April 2003
Estimated Primary Completion Date : December 2008

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Primary Outcome Measures :
  1. Best overall response (complete response and partial response)

Secondary Outcome Measures :
  1. Early progression
  2. Death
  3. Disease progression or recurrence
  4. Surgical resection (complete or incomplete)

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 20 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Diagnosis of hepatoblastoma

    • Refractory or recurrent disease

      • Failed prior first-line or second-line treatment
    • Metastatic disease allowed
  • Measurable disease, defined as ≥ 1 unidimensionally measurable lesion ≥ 20 mm by conventional techniques OR ≥ 10 mm by spiral CT scan

    • Elevated serum alpha-fetoprotein (AFP) allowed
  • No hepatocellular carcinoma


  • Lansky performance status 50-100% in patients ≤ 10 years of age OR Karnofsky performance status 50-100% in patients > 10 years of age
  • Life expectancy > 8 weeks
  • Hemoglobin > 8 g/dL
  • Absolute neutrophil count > 1,000/mm^3
  • Platelet count > 100,000/mm^3
  • Serum bilirubin ≤ 2 times normal
  • AST/ALT ≤ 2 times normal
  • Serum creatinine ≤ 3 times normal
  • Normal metabolic parameters (i.e., serum electrolytes, glucose, calcium, and phosphate)
  • Not pregnant or nursing
  • No severe uncontrolled infection or enterocolitis


  • Recovered from toxicity of prior therapy
  • No chemotherapy within 3 weeks prior to study entry
  • No prior irinotecan
  • No other concurrent anticancer therapy

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00287976

Our Lady's Hospital for Sick Children Crumlin
Dublin, Ireland, 12
Emma Kinderziekenhuis
Amsterdam, Netherlands, NL-1100 DE
United Kingdom
Birmingham Children's Hospital
Birmingham, England, United Kingdom, B4 6NH
Institute of Child Health at University of Bristol
Bristol, England, United Kingdom, BS2 8AE
Addenbrooke's Hospital
Cambridge, England, United Kingdom, CB2 2QQ
Leeds Cancer Centre at St. James's University Hospital
Leeds, England, United Kingdom, LS9 7TF
Leicester Royal Infirmary
Leicester, England, United Kingdom, LE1 5WW
Royal Liverpool Children's Hospital, Alder Hey
Liverpool, England, United Kingdom, L12 2AP
Royal London Hospital
London, England, United Kingdom, E1 1BB
Great Ormond Street Hospital for Children
London, England, United Kingdom, WC1N 3JH
Royal Manchester Children's Hospital
Manchester, England, United Kingdom, M27 4HA
Sir James Spence Institute of Child Health at Royal Victoria Infirmary
Newcastle-Upon-Tyne, England, United Kingdom, NE1 4LP
Queen's Medical Centre
Nottingham, England, United Kingdom, NG7 2UH
Oxford Radcliffe Hospital
Oxford, England, United Kingdom, 0X3 9DU
Children's Hospital - Sheffield
Sheffield, England, United Kingdom, S10 2TH
Southampton General Hospital
Southampton, England, United Kingdom, SO16 6YD
Royal Marsden - Surrey
Sutton, England, United Kingdom, SM2 5PT
Royal Belfast Hospital for Sick Children
Belfast, Northern Ireland, United Kingdom, BT12 6BE
Royal Aberdeen Children's Hospital
Aberdeen, Scotland, United Kingdom, AB25 2ZG
Royal Hospital for Sick Children
Edinburgh, Scotland, United Kingdom, EH9 1LF
Royal Hospital for Sick Children
Glasgow, Scotland, United Kingdom, G3 8SJ
Childrens Hospital for Wales
Cardiff, Wales, United Kingdom, CF14 4XW
Sponsors and Collaborators
Children's Cancer and Leukaemia Group
Study Chair: Jozsef Zsiros, MD, PhD Emma Kinderziekenhuis
OverallOfficial: Laurence Brugieres, MD Gustave Roussy, Cancer Campus, Grand Paris
OverallOfficial: Penelope Brock, MD, PhD Great Ormond Street Hospital for Children NHS Foundation Trust Identifier: NCT00287976     History of Changes
Other Study ID Numbers: CDR0000454758
First Posted: February 7, 2006    Key Record Dates
Last Update Posted: September 17, 2013
Last Verified: June 2009

Keywords provided by National Cancer Institute (NCI):
childhood hepatoblastoma
recurrent childhood liver cancer

Additional relevant MeSH terms:
Liver Neoplasms
Digestive System Neoplasms
Neoplasms by Site
Digestive System Diseases
Liver Diseases
Neoplasms, Complex and Mixed
Neoplasms by Histologic Type
Antineoplastic Agents, Phytogenic
Antineoplastic Agents
Topoisomerase I Inhibitors
Topoisomerase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action