Clinical Trial: Risk-Based Therapy in Treating Younger Patients With Newly Diagnosed Liver Cancer

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Treatment of Children With All Stages of Hepatoblastoma With Temsirolimus (NSC#683864) Added to High Risk Stratum Treatment

Brief Summary: This phase III trial studies the side effects and how well risk-based therapy works in treating younger patients with newly diagnosed liver cancer. Surgery, chemotherapy drugs (cancer fighting medicines), and when necessary, liver transplant, are the main current treatments for hepatoblastoma. The stage of the cancer is one factor used to decide the best treatment. Treating patients according to the risk group they are in may help get rid of the cancer, keep it from coming back, and decrease the side effects of chemotherapy.

Detailed Summary:

PRIMARY OBJECTIVES:

I. To estimate the event-free survival (EFS) in children with stage I (non-pure fetal histology [PFH], non-small cell undifferentiated [SCU]) and stage II (non-SCU) hepatoblastoma treated with surgical resection followed by 2 cycles of cisplatin, fluorouracil, and vincristine sulfate (C5V).

II. To determine the feasibility and toxicity of adding doxorubicin (doxorubicin hydrochloride) to the chemotherapy regimen of C5V for children with intermediate-risk hepatoblastoma.

III. To estimate the response rate to vincristine (vincristine sulfate), irinotecan (irinotecan hydrochloride), and temsirolimus in previously untreated children with high-risk, metastatic hepatoblastoma.

IV. To determine whether timely (between diagnosis and end of second cycle of chemotherapy) consultation with a treatment center with surgical expertise in major pediatric liver resection and transplant can be achieved in 70% of patients with potentially unresectable hepatoblastoma.

V. To foster the collection of tumor tissue and biologic samples to facilitate translational research and to provide data that may aid in risk-adapted approaches for subsequent clinical trials.

SECONDARY OBJECTIVES:

I. To estimate the EFS of patients with stage I PFH treated with surgery alone. II. To determine whether orthotopic liver transplantation (OLT) can be accomplished after successful referral and completion of 4 cycles of initial chemotherapy.

III. To estimate the 2-year EFS for patients once identified as candidates for possible OLT, the 2-year EFS for
Sponsor: National Cancer Institute (NCI)

Current Primary Outcome:

  • Disease status at the end of 2 courses of therapy [ Time Frame: Up to 42 days ]
  • Event-free survival [ Time Frame: Time from patient enrollment to progression, treatment failure, death from any cause, diagnosis of a second malignant neoplasm, or last follow-up, assessed up to 7 years ]
    Estimated by the method of Kaplan and Meier.
  • Incidence of adverse events graded according to Common Terminology Criteria for Adverse Events version 4.0 [ Time Frame: Up to 7 years ]
    All grade 3 or 4 or greater non-hematological toxicities as well as any toxicity that requires submission of an Cancer Therapy Evaluation Program Adverse Event Reporting System report will be reported while the patient is on protocol therapy. The frequency of each toxicity type will be quantified as the percent of reporting periods on which the toxicity of the relevant grade is reported.
  • Rate of death [ Time Frame: Possibly, probably or likely related to systemic chemotherapy assessed up to 7 years ]
    The estimated on-protocol-therapy death rate and its 95% confidence interval will be reported as the cumulative incidence of on-treatment death and the 95% confidence intervals at four and six month.


Original Primary Outcome:

  • Survival rate
  • Disease status at the end of 2 courses of therapy


Current Secondary Outcome: Feasibility of referral for liver transplantation [ Time Frame: Up to 7 years ]

A patient for whom referral is considered appropriate who receives a consultation after enrollment will be considered a success with respect to feasibility.


Original Secondary Outcome: Feasibility of referral for liver transplantation

Information By: National Cancer Institute (NCI)

Dates:
Date Received: September 18, 2009
Date Started: September 2009
Date Completion:
Last Updated: May 12, 2017
Last Verified: May 2017