Clinical trial NCT00287976: Irinotecan in Treating Young Patients With Refractory or Recurrent Hepatoblastoma
Official title: Irinotecan Single-Drug Treatment For Children With Refractory or Recurrent Hepatoblastoma
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Liver Cancer
Keywords: childhood hepatoblastoma
recurrent childhood liver cancer
Lead sponsor: National Cancer Institute (NCI)
Sponsor class: NIH
First posted: 2006-02-07
Enrollment: 30
Sex: ALL
Maximum age (years): 20
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Primary purpose: TREATMENT

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.

Detailed description: OBJECTIVES:

Primary

* 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.

Secondary

* 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.

Eligibility criteria: DISEASE CHARACTERISTICS:

* 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

PATIENT CHARACTERISTICS:

* 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

PRIOR CONCURRENT THERAPY:

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

Primary outcomes:
measure: Best overall response (complete response and partial response)
Secondary outcomes:
measure: Early progression
measure: Death
measure: Disease progression or recurrence
measure: Surgical resection (complete or incomplete)
Other outcomes:
Locations:
city: Dublin
country: Ireland
facility: Our Lady's Hospital for Sick Children Crumlin
city: Amsterdam
country: Netherlands
facility: Emma Kinderziekenhuis
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital
state: England
city: Bristol
country: United Kingdom
facility: Institute of Child Health at University of Bristol
state: England
city: Cambridge
country: United Kingdom
facility: Addenbrooke's Hospital
state: England
city: Leeds
country: United Kingdom
facility: Leeds Cancer Centre at St. James's University Hospital
state: England
city: Leicester
country: United Kingdom
facility: Leicester Royal Infirmary
state: England
city: Liverpool
country: United Kingdom
facility: Royal Liverpool Children's Hospital, Alder Hey
state: England
city: London
country: United Kingdom
facility: Royal London Hospital
state: England
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children
state: England
city: Manchester
country: United Kingdom
facility: Royal Manchester Children's Hospital
state: England
city: Newcastle-Upon-Tyne
country: United Kingdom
facility: Sir James Spence Institute of Child Health at Royal Victoria Infirmary
state: England
city: Nottingham
country: United Kingdom
facility: Queen's Medical Centre
state: England
city: Oxford
country: United Kingdom
facility: Oxford Radcliffe Hospital
state: England
city: Sheffield
country: United Kingdom
facility: Children's Hospital - Sheffield
state: England
city: Southampton
country: United Kingdom
facility: Southampton General Hospital
state: England
city: Sutton
country: United Kingdom
facility: Royal Marsden - Surrey
state: England
city: Belfast
country: United Kingdom
facility: Royal Belfast Hospital for Sick Children
state: Northern Ireland
city: Aberdeen
country: United Kingdom
facility: Royal Aberdeen Children's Hospital
state: Scotland
city: Edinburgh
country: United Kingdom
facility: Royal Hospital for Sick Children
state: Scotland
city: Glasgow
country: United Kingdom
facility: Royal Hospital for Sick Children
state: Scotland
city: Cardiff
country: United Kingdom
facility: Childrens Hospital for Wales
state: Wales
Study officials:
affiliation: Emma Kinderziekenhuis
name: Jozsef Zsiros, MD, PhD
role: STUDY_CHAIR
affiliation: Gustave Roussy, Cancer Campus, Grand Paris
name: Laurence Brugieres, MD
affiliation: Great Ormond Street Hospital for Children NHS Foundation Trust
name: Penelope Brock, MD, PhD
References: