Natural History Study of Children and Adults With Fibrolamellar Hepatocellular Carcinoma
| Status: | Not yet recruiting |
|---|---|
| Conditions: | Liver Cancer, Liver Cancer, Cancer, Cancer, Cancer |
| Therapuetic Areas: | Oncology |
| Healthy: | No |
| Age Range: | 1 - 99 |
| Updated: | 4/6/2019 |
| Start Date: | April 10, 2019 |
| End Date: | December 31, 2028 |
| Contact: | Donna M Hrones, C.R.N.P. |
| Email: | donna.mabry@nih.gov |
| Phone: | (240) 858-3155 |
A Natural History Study of Children and Adults With Fibrolamellar Hepatocellular Carcinoma
Background:
Fibrolamellar Hepatocellular Carcinoma (FL-HCC) is a rare liver cancer. It usually occurs in
young people who have no history of liver disease. Currently the only effective treatment
option is surgery that removes the tumor and part of the liver. Researchers want to study the
course of the disease to learn more about it.
Objective:
To collect samples from people with FL-HCC to learn more about the disease and help develop
new treatments.
Eligibility:
People any age with FL-HCC
Design:
Participants must be enrolled on another NIH protocol.
Participants will have at least 1 study visit. They will have:
- A medical and cancer history
- A physical exam
- A review of their symptoms and their ability to do normal activities
- Tests to produce images of the body. They may have a scan (CT) that uses a small amount
of radiation. Or they may have a scan (MRI) that uses a magnetic field. These will
examine the chest, abdomen, and pelvis.
- Blood tests
Researchers will study previous tumor samples if they are available.
If participants come to NIH for visits on other studies, data will be collected about their
disease, tests, treatments, and responses. Tumor tissue will be collected if participants are
having it taken for a procedure.
All other participants will be contacted to collect this data. They will be contacted once a
month for 1 year and 2 times a year after that.
Participants will be asked to contact researchers when their health changes. They may come in
for more tests.
Fibrolamellar Hepatocellular Carcinoma (FL-HCC) is a rare liver cancer. It usually occurs in
young people who have no history of liver disease. Currently the only effective treatment
option is surgery that removes the tumor and part of the liver. Researchers want to study the
course of the disease to learn more about it.
Objective:
To collect samples from people with FL-HCC to learn more about the disease and help develop
new treatments.
Eligibility:
People any age with FL-HCC
Design:
Participants must be enrolled on another NIH protocol.
Participants will have at least 1 study visit. They will have:
- A medical and cancer history
- A physical exam
- A review of their symptoms and their ability to do normal activities
- Tests to produce images of the body. They may have a scan (CT) that uses a small amount
of radiation. Or they may have a scan (MRI) that uses a magnetic field. These will
examine the chest, abdomen, and pelvis.
- Blood tests
Researchers will study previous tumor samples if they are available.
If participants come to NIH for visits on other studies, data will be collected about their
disease, tests, treatments, and responses. Tumor tissue will be collected if participants are
having it taken for a procedure.
All other participants will be contacted to collect this data. They will be contacted once a
month for 1 year and 2 times a year after that.
Participants will be asked to contact researchers when their health changes. They may come in
for more tests.
Background:
- Fibrolamellar hepatocellular carcinoma (FL-HCC) is a rare hepatocellular carcinoma
accounting for 0.5-9% of primary liver cancer in various case series, which is usually
not associated with elevated serum alpha fetoprotein (AFP) levels, is enriched in
younger age groups, and is not associated with underlying liver disease.
- Reports on the characteristics of patients with FL-HCC as well as predictors of
recurrence and survival are scarce, largely due to the rarity of this tumor. The only
potentially curative treatment option for FL-HCC patients who have resectable disease is
surgery: either liver resection (LR) or liver transplantation (LT). However, disease
recurrence after complete surgical resection is high, ranging from 33-100%. The clinical
outcome of patients with unresectable disease is suboptimal with median survival of less
than 12 months and no patient surviving beyond 5 years.
- The role of neoadjuvant and adjuvant therapies, including systemic chemotherapy, remains
poorly defined and has been reported to have only a modest or no therapeutic effect.
Platinum-based chemotherapy in pediatric patients with FL-HCC resulted a partial
response in 31% of patients on imaging but a 3-year survival of only 22%. To date no
targeted therapy has been shown to be of any value in this disease.
- The natural history of FL-HCC varies greatly with most patients surviving only months
while others can live with the disease for years. While one cannot exclude an immune or
other host component as responsible for the diverse clinical courses, it is also
possible that there may be a genetic basis for this phenomenon. A novel somatic
recurrent 400 kb deletion on the short arm of chromosome 19, giving rise to an in-frame
DNAJB1 PRKACA gene fusion was found in FL-HCC. mTOR signaling is significantly activated
in FL-HCC compared to other liver malignancies. A bio-specimen repository will be a
major step towards more comprehensive studies of this very rare and unusual tumor and
allow us to begin to characterize subgroups within the disease.
- Patients with rare tumors seek expert advice in the management of their care. A natural
history study would establish a more formal mechanism for such referrals, while allowing
the systematic collection of epidemiologic data as well as much needed tumor samples.
Objective:
- Characterize the natural history of Fibrolamellar hepatocellular carcinoma (FL-HCC),
including clinical presentation, family history, patterns of disease progression, response or
lack of response to therapeutic interventions, disease recurrence and overall survival.
Eligibility:
- Subjects of all ages with histologically or cytologically proven FL-HCC.
Design:
- Participants will undergo a comprehensive study entry evaluation including clinical
phenotyping and imaging of tumor sites. Computed tomography scans of the thorax, abdomen
and pelvis may be performed if clinically indicated; occasionally, magnetic resonance
imaging may be performed for the visualization of lesions in the liver, spine, or other
anatomic sites if clinically indicated.
- Medical histories will be documented, and participants will be followed throughout the
course of their illnesses, with attention to patterns of disease recurrence and
progression, response to therapies and duration of responses. As part of this natural
history study, growth rates of target tumor lesions will also be calculated throughout
the course of the disease whenever available.
- Blood and tumor samples will be obtained at study entry and while on study.
- Fibrolamellar hepatocellular carcinoma (FL-HCC) is a rare hepatocellular carcinoma
accounting for 0.5-9% of primary liver cancer in various case series, which is usually
not associated with elevated serum alpha fetoprotein (AFP) levels, is enriched in
younger age groups, and is not associated with underlying liver disease.
- Reports on the characteristics of patients with FL-HCC as well as predictors of
recurrence and survival are scarce, largely due to the rarity of this tumor. The only
potentially curative treatment option for FL-HCC patients who have resectable disease is
surgery: either liver resection (LR) or liver transplantation (LT). However, disease
recurrence after complete surgical resection is high, ranging from 33-100%. The clinical
outcome of patients with unresectable disease is suboptimal with median survival of less
than 12 months and no patient surviving beyond 5 years.
- The role of neoadjuvant and adjuvant therapies, including systemic chemotherapy, remains
poorly defined and has been reported to have only a modest or no therapeutic effect.
Platinum-based chemotherapy in pediatric patients with FL-HCC resulted a partial
response in 31% of patients on imaging but a 3-year survival of only 22%. To date no
targeted therapy has been shown to be of any value in this disease.
- The natural history of FL-HCC varies greatly with most patients surviving only months
while others can live with the disease for years. While one cannot exclude an immune or
other host component as responsible for the diverse clinical courses, it is also
possible that there may be a genetic basis for this phenomenon. A novel somatic
recurrent 400 kb deletion on the short arm of chromosome 19, giving rise to an in-frame
DNAJB1 PRKACA gene fusion was found in FL-HCC. mTOR signaling is significantly activated
in FL-HCC compared to other liver malignancies. A bio-specimen repository will be a
major step towards more comprehensive studies of this very rare and unusual tumor and
allow us to begin to characterize subgroups within the disease.
- Patients with rare tumors seek expert advice in the management of their care. A natural
history study would establish a more formal mechanism for such referrals, while allowing
the systematic collection of epidemiologic data as well as much needed tumor samples.
Objective:
- Characterize the natural history of Fibrolamellar hepatocellular carcinoma (FL-HCC),
including clinical presentation, family history, patterns of disease progression, response or
lack of response to therapeutic interventions, disease recurrence and overall survival.
Eligibility:
- Subjects of all ages with histologically or cytologically proven FL-HCC.
Design:
- Participants will undergo a comprehensive study entry evaluation including clinical
phenotyping and imaging of tumor sites. Computed tomography scans of the thorax, abdomen
and pelvis may be performed if clinically indicated; occasionally, magnetic resonance
imaging may be performed for the visualization of lesions in the liver, spine, or other
anatomic sites if clinically indicated.
- Medical histories will be documented, and participants will be followed throughout the
course of their illnesses, with attention to patterns of disease recurrence and
progression, response to therapies and duration of responses. As part of this natural
history study, growth rates of target tumor lesions will also be calculated throughout
the course of the disease whenever available.
- Blood and tumor samples will be obtained at study entry and while on study.
- INCLUSION CRITERIA:
- Subjects enrolled on NCI protocol Natural History and Biospecimen Acquisition Study
for Children and Adults with Rare Solid Tumors
- Subjects of all ages with histologically or cytologically proven FL-HCC.
- Ability of subject to understand and the willingness to sign a written informed
consent document.
EXCLUSION CRITERIA:
We found this trial at
1
site
9000 Rockville Pike
Bethesda, Maryland 20892
Bethesda, Maryland 20892
301-496-2563
Phone: 888-624-1937
National Institutes of Health Clinical Center The National Institutes of Health (NIH) Clinical Center in...
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