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Alkylating agents

Risk-Based Therapy for Liver Cancer

Phase 3

Waitlist Available

Led By Howard M Katzenstein

Research Sponsored by National Cancer Institute (NCI)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients must be newly diagnosed with histologically-proven hepatoblastoma

Patients must have a performance status corresponding to ECOG scores 0, 1, or 2

Must not have

Patients with stage I or II disease who do not have specimens submitted for rapid central pathology review by day 14 after initial surgical resection

Patients who have received any prior chemotherapy, currently receiving another investigational drug, or currently receiving other anticancer agents are not eligible

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 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 5 years

Awards & highlights

Pivotal Trial

No Placebo-Only Group

Summary

This trial looks at side effects and effectiveness of different treatments for newly diagnosed liver cancer in young patients.

Who is the study for?

This trial is for young patients with newly diagnosed liver cancer called hepatoblastoma. They must not have had any previous chemotherapy or other treatments for the condition, and their organ functions should meet specific criteria. Pregnant or breastfeeding females are ineligible, as well as those on certain medications like corticosteroids or anticoagulants.

What is being tested?

The study tests risk-based therapy involving surgery, various chemotherapy drugs (Dexrazoxane, Vincristine Sulfate, Fluorouracil, etc.), and potentially a liver transplant based on the stage of cancer. The aim is to see if tailoring treatment by risk group can effectively treat the cancer while minimizing side effects.

What are the potential side effects?

Possible side effects include reactions to medication such as nausea, hair loss, increased infection risk due to weakened immune system from chemotherapy drugs; surgical complications; and potential issues related to liver transplantation.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been recently diagnosed with hepatoblastoma.

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I can take care of myself and perform daily activities.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My early-stage cancer tissue wasn't reviewed within 2 weeks after surgery.

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I have not had chemotherapy, other cancer drugs, or experimental treatments recently.

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I have not had an organ transplant, am not pregnant or breastfeeding, and do not have an uncontrolled infection.

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I am not taking steroids, seizure medications, blood thinners, or ACE inhibitors.

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I have not had major surgery in the last 6 weeks.

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I am not taking strong medications that affect liver enzymes.

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I am using effective birth control.

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I have not received chemotherapy or specific treatments for hepatoblastoma before.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 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 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~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 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 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 5 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Disease Status at the End of 2 Courses of Therapy

Event-free Survival

Number of Cycles on Which Grade 3 or Higher Adverse Events Coded According to CTC AE Version 5 Were Observed

+1 more

Secondary study objectives

Feasibility of Referral for Liver Transplantation

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

5Treatment groups

Experimental Treatment

Group I: Very low-risk groupExperimental Treatment2 Interventions

Patients undergo surgery and then receive no further treatment.

Group II: Low-risk group (regimen T)Experimental Treatment5 Interventions

Patients undergo surgery and then receive adjuvant cisplatin IV over 6 hours on day 1, fluorouracil IV over 2-4 minutes on day 2, and vincristine sulfate IV over 1 minute on days 2, 9, and 16. Treatment repeats every 21 days for 2 courses in the absence of disease progression or unacceptable toxicity.

Group III: Intermediate-risk group (regimen F)Experimental Treatment8 Interventions

Patients receive C5VD chemotherapy comprising cisplatin IV over 6 hours on day 1, fluorouracil IV over 2-4 minutes on day 2, vincristine sulfate IV over 1 minute on days 2, 9, and 16, and doxorubicin hydrochloride IV over 15 minutes on days 1-2. Treatment repeats every 21 days for 6 courses in the absence of disease progression or unacceptable toxicity. Patients also undergo surgical resection after course 2 OR surgical resection or liver transplantation after course 4 of C5VD. Patients may also receive dexrazoxane IV over 5-15 minutes on days 1-2 of courses 5 and 6. (Closed to accrual as of 3/12/2012)

Group IV: High-risk group (regimen W)Experimental Treatment7 Interventions

(regimen W replaced by regimen H as of Amendment 3B) Patients receive up front VI chemotherapy comprising vincristine sulfate IV on days 1 and 8 and irinotecan hydrochloride IV over 90 minutes on days 1-5. Treatment with VI repeats every 21 days for 2 courses in the absence of disease progression or unacceptable toxicity. Patients with disease response then receive 6 courses of C5VD with 1 courses of VI in between each 2-course block. Patients with no disease response receive 6 courses of C5VD in the absence of disease progression or unacceptable toxicity.

Group V: High-risk group (regimen H)Experimental Treatment9 Interventions

Patients receive up front VIT chemotherapy comprising vincristine sulfate IV over 1 minute on days 1 and 8 and irinotecan hydrochloride IV over 90 minutes on days 1-5, and temsirolimus IV over 30 minutes on days 1 and 8. Treatment with VIT repeats every 21 days for 2 courses in the absence of disease progression or unacceptable toxicity. Patients with disease response then receive 6 courses of C5VD with 4 courses of VIT in between each 2-course block. Patients with no disease response receive 6 courses of C5VD in the absence of disease progression or unacceptable toxicity. Patients undergo tumor resection or liver transplant after course 4 of C5VD followed by 2 courses of adjuvant C5VD. Patients may also receive dexrazoxane IV over 5-15 minutes on days 1-2 of courses 5 and 6.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Cisplatin

2013

Completed Phase 3

~3120

Dexrazoxane

2016

Completed Phase 2

~80