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Hepatoblastoma Biology Study and Tissue Bank

6 Months
21 Years
Open (Enrolling)

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Trial Information

Hepatoblastoma Biology Study and Tissue Bank

COG 9346 - Lay Summary

Hepatoblastoma Biology Study and Tissue Bank

Although tremendous improvement in the treatment of childhood cancer has resulted from the
use of clinical trials, it is clear that additional significant progress will require a
better understanding of the molecular makeup of pediatric malignancies. Hepatoblastoma, an
embryonal tumor of the liver, is rare in the general population. It occurs in approximately
1 in 50,000 children. However, families affected with Familial Adenomatous Polyposis, in
which there is a high incidence of colon cancer, have an 850 fold increase in the frequency
of Hepatoblastoma. Hepatoblastoma also occurs in association with Beckwith-Wiedemann
Syndrome. Thus the occurrence of Hepatoblastoma suggests the need for both careful
investigations into the family history of malignancies in relatives, as well as a search for
genetic alterations that may contribute to tumor development.

COG cancer researchers are trying to better understand the causes of Hepatoblastoma. They
want to develop improved methods for treatment, diagnosis and possibly even prevention. The
exact molecules or genes to be studied will change over time as progress in cancer research
is made. Not every gene or molecule known to be involved in cancer will be studied.
Researchers will choose to study only those genes or molecules which they think are most
promising. At present, the study of at least three genes is planned.

In some cases these changes may be detected in normal tissues such as liver or blood cells.
One of the genes to be studied is known as the APC gene. It is linked with colon cancer in
adults. Rarely this gene can be passed on in families which are prone to develop both colon
cancer and Hepatoblastoma. We don't know how common changes in this gene are in children
with Hepatoblastoma. This study will try and figure out if this gene is commonly changed in

In addition to studying specific genes or molecules, geneticists (doctors who specialize in
looking at genes) will look at the overall genetic content of tumors from some of the
children involved in this study. This will be done by what is known as a karyotype or
chromosome analysis. It is known that some Hepatoblasomas have extra copies of certain
chromosomes. However, we do not know what effect these extra chromosomes might have on
predicting which children will be most likely to respond to treatment. One of the goals of
the study is to figure out what biological factors within the tumors, including extra
chromosomes, might help predict which children will respond best to treatment. This will
hopefully lead to better treatment of Hepatoblastoma.

A single extra blood sample will be taken from the patient for this study. We will take no
more than 1 1/2 teaspoons of blood. This amount may be less for very small infants. We
will also take a single blood sample (1 tablespoon) from the parent.

A portion of tumor tissue that is removed from the patient's surgery or biopsy will also be
sent. No extra procedures will be done to get the tumor tissue. It will be taken during a
surgery that is needed for standard care.

Patients will also be asked to complete a questionnaire about their background. They may
also be asked to complete a follow-up questionnaire or to provide additional information in
the future.

Inclusion Criteria:

- Patients with hepatoblastoma.

Exclusion Criteria:


Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label

Outcome Measure:

To collect and bank Hepatoblastoma specimens.

Outcome Time Frame:

specimens will be banked indefinitely

Safety Issue:


Principal Investigator

Howard Katzenstein, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Children's Healthcare of Atlanta


United States: Institutional Review Board

Study ID:




Start Date:

September 2005

Completion Date:

January 2015

Related Keywords:

  • Hepatoblastoma
  • Hepatoblastoma



Children's Healthcare of Atlanta Atlanta, Georgia  30342