Experimental Cisplatin and Combination Chemotherapy in Children and Young Adults With Hepatoblastoma or Liver Cancer After Surgery

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Age: -1 to 30 years old
Gender:
Any
Healthy Volunteers: No
Keywords: Hepatic Malignancy, hepatocellular carcinoma, hepatoblastoma, pediatric cancer
Type: Drug study, Phase [/2/,/ /3/]
Target:
5 Participants
Investigator:
Description
This research study is for people diagnosed with very low risk hepatoblastoma.

Hepatoblastoma (HB) is a type of cancer that occurs in the liver. HB is considered very low risk when it is surgically removed at diagnosis. The term risk refers to the chance of the cancer coming back after treatment.

Treatment for very low risk HB depends on how the cancer cells look under a microscope (histology).

One type of histology is called well-differentiated fetal (WDF) histology. This usually requires surgery to remove the tumor, followed by close monitoring until signs or symptoms appear or change.

Patients who do not have WDF histology also have surgery to remove the tumor. After surgery, these patients have chemotherapy (cancer fighting medicine) to kill any cancer cells that are left.

A common treatment is a combination of 3 chemotherapy drugs called C5V:
- cisplatin
- 5-fluorouracil
- vincristine

Because hepatoblastoma is rare, there is limited research in this area. In this study, multiple research groups in Europe and Japan will combine efforts to study the treatment of patients with HB.

Researchers want to find out if we can improve the treatment for subjects with very low risk HB. This study looks at how well cisplatin alone (without 5-fluorouracil and vincristine) works when it is given to children and young adults with very low risk HB after surgery. Success with giving cisplatin alone has been observed by European researchers. However, giving cisplatin without 5-fluorouracil and vincristine is experimental in North America.

This study also looks at whether we can shorten the chemotherapy treatment given after surgery, from 4 cycles to 2 cycles. Shortening chemotherapy after surgery is also experimental. By removing 2 drugs and shortening chemotherapy, we may be able to reduce side effects during treatment or in the future.
This study requires

The study team will make sure you qualify to take part in the study. Qualified participants will receive detailed information about the study. This may include a list of study-related tests and procedures.

Who can participate

Inclusion criteria:

  • Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1, or 2; use Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
  • Patients must be newly diagnosed with histologically-proven primary pediatric hepatic malignancies including hepatoblastoma or hepatocellular carcinoma, except as noted below; note that rapid central pathology review is required in some cases; please note: all patients with histology as assessed by the institutional pathologist consistent with pure small cell undifferentiated (SCU) HB will be required to have testing for INI1/SMARCB1 by immunohistochemistry (IHC) according to the practices at the institution
  • Patients with histology consistent with pure SCU must have positive INI1/SMARCB1 staining
  • In emergency situations when a patient meets all other eligibility criteria and has had baseline required observations, but is too ill to undergo a biopsy safely, the patient may be enrolled without a biopsy
  • Clinical situations in which emergent treatment may be indicated include, but are not limited to, the following circumstances:
  • Anatomic or mechanical compromise of critical organ function by tumor (e.g., respiratory distress/failure, abdominal compartment syndrome, urinary obstruction, etc.)
  • Uncorrectable coagulopathy
  • For a patient to maintain eligibility for AHEP1531 when emergent treatment is given, the following must occur:
  • The patient must have a clinical diagnosis of hepatoblastoma, including an elevated alphafetoprotein (AFP), and must meet all AHEP1531 eligibility criteria at the time of emergent treatment
  • Patient must be enrolled on AHEP1531 prior to initiating protocol therapy; a patient will be ineligible if any chemotherapy is administered prior to AHEP1531 enrollment
  • Note: If the patient receives AHEP1531 chemotherapy emergently PRIOR to undergoing a diagnostic biopsy, pathologic review of material obtained in the future during either biopsy or surgical resection must either confirm the diagnosis of hepatoblastoma or not reveal another pathological diagnosis to be included in the analysis of the study aims
  • Patients may have had surgical resection of the hepatic malignancy prior to enrollment; all other anti-cancer therapy for the current liver lesion is prohibited
  • Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or
  • A serum creatinine based on age/gender as follows:
  • Age: maximum serum creatinine (mg/dL)
  • 1 month to < 6 months: 0.4 (male and female)
  • 6 months to < 1 year: 0.5 (male and female)
  • 1 to < 2 years: 06 (male and female)
  • 2 to < 6 years: 0.8 (male and female)
  • 6 to < 10 years: 1 (male and female)
  • 10 to < 13 years: 1.2 (male and female)
  • 13 to < 16 years: 1.5 (male), 1.4 (female)
  • = 16 years: 1.7 (male), 1.4 (female)

  • Total bilirubin =< 5 x upper limit of normal (ULN) for age
  • Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase [SGOT])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) < 10x upper limit of normal (ULN) for age
  • Shortening fraction of >= 27% by echocardiogram (for patients on doxorubicin-containing regimens [Groups C, D, E, and F]), or
  • Ejection fraction of >= 50% by radionuclide angiogram (for patients on doxorubicin-containing regimens (Groups C, D, E, and F)
  • Normal pulmonary function tests (including diffusion capacity of the lung for carbon monoxide [DLCO]) if there is clinical indication for determination (e.g. dyspnea at rest, known requirement for supplemental oxygen); for patients who do not have respiratory symptoms or requirement for supplemental oxygen, pulmonary function tests (PFTs) are NOT required
  • All patients and/or their parents or legal guardians must sign a written informed consent
  • All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met

Exclusion criteria:

  • Prior chemotherapy or tumor directed therapy (i.e. radiation therapy, biologic agents, local therapy (embolization, radiofrequency ablation, and laser); therefore, patients with a pre-disposition syndrome who have a prior malignancy are not eligible
  • Patients who are currently receiving another investigational drug
  • Patients who are currently receiving other anticancer agents
  • Patients with uncontrolled infection
  • Patients who previously received a solid organ transplant
  • This criteria apply ONLY to patients who will receive chemotherapy (all groups other than Group E1):
  • Female patients who are pregnant; a pregnancy test is required for female patients of childbearing potential
  • Lactating females who plan to breastfeed their infants
  • Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation
  • Note for Group F: patients of childbearing potential should use effective birth control during treatment with sorafenib and for at least 2 weeks after stopping treatment
Benefits and risks of participating
BENEFITS:

We cannot promise any benefits to you as a result of taking part in this research study.


RISKS:

The study doctor will discuss the risks associated with taking part in this research study.
Compensation
You will not be paid to take part in this research study.
Resources
Schedule
Study duration and period
People in this study will receive treatment for about 3-6 months. After treatment, we would like to continue to find out about your health every year for up to 10 years after you enter this study.
Recruitment period
From July 13, 2018
Location
UC Davis Comprehensive Cancer Center
4501 X Street
Sacramento, CA 95817
Research Topic
Conditions:
  • Childhood Hepatocellular Carcinoma
  • Childhood Malignant Liver Neoplasm
  • Elevated Alpha-Fetoprotein
  • Hepatoblastoma
  • SMARCB1 Gene Mutation

Have any questions or want to learn more? Leave your contact details below and the research team will reach out to you.


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