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- Ensaio Clínico NCT00077389
Intensive Neoadjuvant Chemotherapy in Treating Young Patients Undergoing Surgical Resection for High-Risk Hepatoblastoma
Intensified Pre-Operative Chemotherapy And Radical Surgery For High Risk Hepatoblastoma
RATIONALE: Drugs used in chemotherapy work in different ways to stop tumor cells from dividing so they stop growing or die. Giving chemotherapy drugs before surgery may shrink the tumor so that it can be removed.
PURPOSE: This phase II trial is studying how well neoadjuvant chemotherapy works in treating young patients who are undergoing surgical resection for high-risk hepatoblastoma.
Visão geral do estudo
Status
Condições
Descrição detalhada
OBJECTIVES:
Primary
- Determine the efficacy and short-term toxicity of intensified neoadjuvant chemotherapy in children with high-risk hepatoblastoma undergoing surgical resection.
- Increase the rate of complete surgical resection in these patients by fully implementing liver transplantation as a valid treatment option for tumor removal when partial liver resection or other surgical options remain unfeasible even after extensive preoperative chemotherapy.
- Determine, prospectively, the role of this regimen in rendering unresectable tumors resectable in these patients.
- Determine the accuracy of initial imaging in predicting the surgical options (after treatment with this regimen) for patients presenting with unresectable disease.
Secondary
- Determine the overall survival and event-free survival of patients treated with this regimen (with an acceptable overall toxicity).
- Determine the toxicity of this regimen in these patients.
- Determine the response rate in patients treated with this regimen.
- Determine whether response to this regimen, defined by the modified RECIST criteria, can be used for better monitoring of response in these patients.
- Determine whether a fall in alpha-fetoprotein during this neoadjuvant regimen can be used as a prognostic factor in these patients.
- Determine, prospectively, radiological, surgical, and pathological characteristics of the tumor that might identify possible novel factors that might influence treatment choice and outcome in these patients.
OUTLINE: This is an open-label, multicenter study.
- Intensified neoadjuvant chemotherapy: Patients receive cisplatin IV over 24 hours on days 1, 8, 15, 29, 36, 43, 57, and 64; and doxorubicin IV over 1 hour OR over 24 hours on days 8, 9, 36, 37, 57, and 58. Patients determined to have resectable disease proceed to surgery.
Patients determined to have unresectable disease after neoadjuvant chemotherapy receive additional neoadjuvant chemotherapy comprising carboplatin IV over 1 hour on days 1 and 22 and doxorubicin IV over 1 hour OR over 24 hours on days 1, 2, 3, 22, 23, and 24.
Treatment continues in the absence of unacceptable toxicity.
- Surgery: Patients determined to have resectable disease undergo complete resection and possibly liver transplantation.
- Adjuvant chemotherapy*: Patients who undergo complete surgical resection receive carboplatin IV over 1 hour on day 1 and doxorubicin IV over 1 hour OR over 24 hours on days 1 and 2. Treatment repeats every 3 weeks for a total of 3 courses.
NOTE: *Patients who received additional neoadjuvant chemotherapy for unresectable disease do not receive adjuvant chemotherapy.
Patients are followed every 2-3 months for 2 years, every 3 months for 1 year, and then every 6 months for 2 years.
Peer Reviewed and Funded or Endorsed by Cancer Research UK
PROJECTED ACCRUAL: A total of 23-57 patients will be accrued for this study within 2 years.
Tipo de estudo
Inscrição (Antecipado)
Estágio
- Fase 2
Contactos e Locais
Locais de estudo
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Villejuif, França, F-94805
- Institut Gustave Roussy
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Amsterdam, Holanda, NL-1100 DE
- Emma Kinderziekenhuis
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Dublin, Irlanda, 12
- Our Lady's Hospital for Sick Children Crumlin
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England
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Birmingham, England, Reino Unido, B4 6NH
- Birmingham Children's Hospital
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Bristol, England, Reino Unido, BS2 8AE
- Institute of Child Health at University of Bristol
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Cambridge, England, Reino Unido, CB2 2QQ
- Addenbrooke's Hospital
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Leeds, England, Reino Unido, LS9 7TF
- Leeds Cancer Centre at St. James's University Hospital
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Leicester, England, Reino Unido, LE1 5WW
- Leicester Royal Infirmary
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Leicester, England, Reino Unido, LE1 6TH
- Children's Cancer and Leukaemia Group
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Liverpool, England, Reino Unido, L12 2AP
- Royal Liverpool Children's Hospital, Alder Hey
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London, England, Reino Unido, W1T 3AA
- Middlesex Hospital
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London, England, Reino Unido, WC1N 3JH
- Great Ormond Street Hospital for Children
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Manchester, England, Reino Unido, M27 4HA
- Royal Manchester Children's Hospital
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Newcastle-Upon-Tyne, England, Reino Unido, NE1 4LP
- Sir James Spence Institute of Child Health at Royal Victoria Infirmary
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Nottingham, England, Reino Unido, NG7 2UH
- Queen's Medical Centre
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Oxford, England, Reino Unido, 0X3 9DU
- Oxford Radcliffe Hospital
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Sheffield, England, Reino Unido, S10 2TH
- Children's Hospital - Sheffield
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Southampton, England, Reino Unido, SO16 6YD
- Southampton General Hospital
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Sutton, England, Reino Unido, SM2 5PT
- Royal Marsden - Surrey
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Northern Ireland
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Belfast, Northern Ireland, Reino Unido, BT12 6BE
- Royal Belfast Hospital for Sick Children
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Scotland
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Aberdeen, Scotland, Reino Unido, AB25 2ZG
- Royal Aberdeen Children's Hospital
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Edinburgh, Scotland, Reino Unido, EH9 1LF
- Royal Hospital for Sick Children
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Glasgow, Scotland, Reino Unido, G3 8SJ
- Royal Hospital for Sick Children
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Wales
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Cardiff, Wales, Reino Unido, CF14 4XW
- Childrens Hospital for Wales
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Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
Aceita Voluntários Saudáveis
Gêneros Elegíveis para o Estudo
Descrição
DISEASE CHARACTERISTICS:
- Histologically confirmed hepatoblastoma
High-risk disease, meeting criteria for at least 1 of the following:
- Tumor involving all 4 hepatic sections
- Evidence of abdominal extrahepatic disease
- Presence of metastases
- Alpha-fetoprotein < 100 ng/mL at diagnosis
- Must have had a prior diagnostic biopsy within the past 15 days
- No recurrent disease
PATIENT CHARACTERISTICS:
Age
- Under 18
Performance status
- Not specified
Life expectancy
- Not specified
Hematopoietic
- Not specified
Hepatic
- AST and/or ALT ≤ 3 times normal
Renal
- Glomerular filtration rate ≥ 60 mL/min
Cardiovascular
- Shortening fraction ≥ 29% OR
- Ejection fraction ≥ 40%
Other
- Not pregnant
- Negative pregnancy test
- No pre-existing clinically relevant bilateral hearing loss
- No other condition that would preclude study participation
PRIOR CONCURRENT THERAPY:
Biologic therapy
- Not specified
Chemotherapy
- No prior chemotherapy
Endocrine therapy
- Not specified
Radiotherapy
- Not specified
Surgery
- Not specified
Other
- No prior therapy for hepatoblastoma
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
- Finalidade Principal: Tratamento
- Mascaramento: Nenhum (rótulo aberto)
O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
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Rate of complete remission after completion of study therapy
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Medidas de resultados secundários
Medida de resultado |
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Sobrevida geral
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Sobrevivência livre de eventos
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Taxa de ressecção completa
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Response rate to preoperative chemotherapy
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Rate of grade 2 cardiac and renal, grade 3 otological, and grade 4 nonhematological toxicity as assessed during and after completion of study therapy
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Colaboradores e Investigadores
Patrocinador
Investigadores
- Cadeira de estudo: Margaret Childs, Children's Cancer and Leukaemia Group
Publicações e links úteis
Publicações Gerais
- Zsiros J, Brugieres L, Brock P, Roebuck D, Maibach R, Zimmermann A, Childs M, Pariente D, Laithier V, Otte JB, Branchereau S, Aronson D, Rangaswami A, Ronghe M, Casanova M, Sullivan M, Morland B, Czauderna P, Perilongo G; International Childhood Liver Tumours Strategy Group (SIOPEL). Dose-dense cisplatin-based chemotherapy and surgery for children with high-risk hepatoblastoma (SIOPEL-4): a prospective, single-arm, feasibility study. Lancet Oncol. 2013 Aug;14(9):834-42. doi: 10.1016/S1470-2045(13)70272-9. Epub 2013 Jul 4.
- Weeda VB, Murawski M, McCabe AJ, Maibach R, Brugieres L, Roebuck D, Fabre M, Zimmermann A, Otte JB, Sullivan M, Perilongo G, Childs M, Brock P, Zsiros J, Plaschkes J, Czauderna P, Aronson DC. Fibrolamellar variant of hepatocellular carcinoma does not have a better survival than conventional hepatocellular carcinoma--results and treatment recommendations from the Childhood Liver Tumour Strategy Group (SIOPEL) experience. Eur J Cancer. 2013 Aug;49(12):2698-704. doi: 10.1016/j.ejca.2013.04.012. Epub 2013 May 15.
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo
Datas de inscrição no estudo
Enviado pela primeira vez
Enviado pela primeira vez que atendeu aos critérios de CQ
Primeira postagem (Estimativa)
Atualizações de registro de estudo
Última Atualização Postada (Estimativa)
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
- Doenças do aparelho digestivo
- Neoplasias por Tipo Histológico
- Neoplasias
- Neoplasias por local
- Neoplasias do Aparelho Digestivo
- Doenças do Fígado
- Neoplasias Complexas e Mistas
- Neoplasias Hepáticas
- Hepatoblastoma
- Mecanismos Moleculares de Ação Farmacológica
- Inibidores Enzimáticos
- Agentes Antineoplásicos
- Inibidores da Topoisomerase II
- Inibidores da Topoisomerase
- Antibióticos, Antineoplásicos
- Carboplatina
- Doxorrubicina
- Doxorrubicina lipossomal
Outros números de identificação do estudo
- CDR0000350221
- SIOP-SIOPEL-4
- EU-20336
- CCLG-LT-2004-09
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