- ICH GCP
- Registro de ensaios clínicos dos EUA
- Ensaio Clínico NCT03748927
Natural History Study of Children and Adults With Fibrolamellar Hepatocellular Carcinoma
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.
Visão geral do estudo
Status
Condições
Descrição detalhada
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.
Tipo de estudo
Contactos e Locais
Locais de estudo
-
-
Maryland
-
Bethesda, Maryland, Estados Unidos, 20892
- National Institutes of Health Clinical Center
-
-
Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
Aceita Voluntários Saudáveis
Gêneros Elegíveis para o Estudo
Método de amostragem
População do estudo
Descrição
- 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:
None
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
- Modelos de observação: Coorte
- Perspectivas de Tempo: Prospectivo
Coortes e Intervenções
Grupo / Coorte |
---|
1/ Cohort 1
Subjects with FL-HCC.
|
O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
---|---|---|
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 recurre...
Prazo: 10 years
|
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
|
10 years
|
Colaboradores e Investigadores
Patrocinador
Publicações e links úteis
Links úteis
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Real)
Conclusão Primária (Real)
Conclusão do estudo (Real)
Datas de inscrição no estudo
Enviado pela primeira vez
Enviado pela primeira vez que atendeu aos critérios de CQ
Primeira postagem (Real)
Atualizações de registro de estudo
Última Atualização Postada (Real)
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última verificação
Mais Informações
Termos relacionados a este estudo
Palavras-chave
Termos MeSH relevantes adicionais
Outros números de identificação do estudo
- 190021
- 19-C-0021
Essas informações foram obtidas diretamente do site clinicaltrials.gov sem nenhuma alteração. Se você tiver alguma solicitação para alterar, remover ou atualizar os detalhes do seu estudo, entre em contato com register@clinicaltrials.gov. Assim que uma alteração for implementada em clinicaltrials.gov, ela também será atualizada automaticamente em nosso site .