Sirolimus as Secondary Therapy in Chronic Graft-Versus-Host Disease Not Responding To Prior Treatment
23 de maio de 2017 atualizado por: Paul Carpenter, Fred Hutchinson Cancer Center
A Phase II Clinical Trial to Evaluate the Safety and Efficacy of Sirolimus for Secondary Treatment of Chronic Graft-versus-Host Disease
This phase II trial studies the side effects and how well sirolimus works as secondary therapy in treating patients with chronic graft-versus-host disease (GVHD) that did not respond to prior treatment.
Sirolimus may be an effective treatment for chronic GVHD
Visão geral do estudo
Status
Concluído
Condições
Intervenção / Tratamento
Descrição detalhada
PRIMARY OBJECTIVES:
I. To assess the safety of sirolimus administered at a dose which provides steady-state, whole blood trough levels of 5-10 ng/mL in patients with chronic GVHD.
II. To determine whether administration of sirolimus provides benefit for patients with chronic GVHD that has not responded adequately to previous systemic treatment.
OUTLINE:
Patients receive sirolimus orally (PO) once daily (QD). Patients continue to receive prednisone and cyclosporine or tacrolimus at the discretion of the managing physician.
After completion of study treatment, patients are followed up periodically.
Tipo de estudo
Intervencional
Inscrição (Real)
44
Estágio
- Fase 2
Contactos e Locais
Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.
Locais de estudo
-
-
Washington
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Seattle, Washington, Estados Unidos, 98109
- Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium
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Critérios de participação
Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.
Critérios de elegibilidade
Idades elegíveis para estudo
- Filho
- Adulto
- Adulto mais velho
Aceita Voluntários Saudáveis
Não
Gêneros Elegíveis para o Estudo
Tudo
Descrição
Inclusion Criteria:
Biopsy-confirmed diagnosis of clinical extensive chronic GVHD with inadequate response to previous treatment and where secondary systemic therapy is indicated because of
- Clinical progression of signs and symptoms of chronic GVHD in a previously involved organ, or
- Development of signs and symptoms of chronic GVHD in a previously uninvolved organ, or
- Absence of improvement after 3 months of primary treatment, or
- Continued need for treatment with prednisone at doses >= 1.0 mg/kg/day for more than 2 months, without qualification for type of donor, graft or conditioning regimen
- Patient or guardian able and willing to provide informed consent
- Stated willingness to use contraception in women of child-bearing potential (Food and Drug Administration [FDA] requirement)
- Stated willingness of the patient to comply with study procedures and reporting requirements
- Stated willingness of the physician most involved in management of chronic GVHD (the "managing physician,") to comply with study procedures and reporting requirements
Exclusion Criteria:
- Fungal or viral infection with no radiographic evidence of improvement during continued appropriate antimicrobial therapy
- Cytomegalovirus (CMV) antigenemia unresponsive to antiviral therapy
- Active disseminated varicella zoster virus (VZV) infection with persistent non-crusted lesions
- Inability to tolerate oral medications
- Absolute neutrophil count (ANC) < 1500/uL
- Platelet count < 50,000/uL
- Persistent or recurrent malignancy, including histopathologic evidence of myeloma or lymphoma; patients with breakpoint cluster region-abelson (bcr/abl) detected by polymerase chain reaction (PCR) assay as the only evidence of persistent chronic myeloid leukemia may be enrolled
- Pregnancy
- Known history of hypersensitivity to sirolimus
Plano de estudo
Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.
Como o estudo é projetado?
Detalhes do projeto
- Finalidade Principal: Cuidados de suporte
- Alocação: N / D
- Modelo Intervencional: Atribuição de grupo único
- Mascaramento: Nenhum (rótulo aberto)
Armas e Intervenções
Grupo de Participantes / Braço |
Intervenção / Tratamento |
|---|---|
|
Experimental: Sirolimus
Study participants receive sirolimus added once daily to their baseline combination therapy of prednisone plus either cyclosporine or tacrolimus at the discretion of the managing physician.
Treatment other than cyclosporine (or tacrolimus) and prednisone must be discontinued when administration of sirolimus is started.
Topical therapy, including psoralen and UVA irradiation (PUVA), glucocorticoid creams, topical tacrolimus, oral beclomethasone, topical azathioprine and ophthalmic glucocorticoids may be given at the discretion of the managing physician in consultation with the transplant center.
|
Dado PO
Outros nomes:
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O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
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Number of Participants Experiencing Treatment Success
Prazo: Approximately 7 years
|
Defined as the absence of any immunosuppressive treatment, including sirolimus, with resolution of all reversible manifestations of chronic GVHD and no additional systemic therapy.
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Approximately 7 years
|
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Number of Participants Experiencing Treatment Failure
Prazo: Approximately 7 years
|
Defined as the initiation of additional systemic therapy, development of bronchiolitis obliterans, or death from causes other than recurrent malignancy during primary treatment for chronic GVHD, whichever occurs first.
|
Approximately 7 years
|
|
Number of Participants Needing Additional Systemic Therapy
Prazo: Approximately 7 years
|
Includes any intervention intended to control chronic GVHD through an immunosuppressive effect from oral or parenteral administration of any systemic medication not originally given under auspices of this protocol.
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Approximately 7 years
|
|
Number of Participants With Recurrent Malignancy
Prazo: Approximately 7 years
|
Defined as clinical or histopathologic evidence demonstrating the presence of any malignancy considered as the indication for transplant.
Recurrent malignancy will also be defined as any post-transplant intervention not routinely used to prevent the development of overt recurrence, prompted by laboratory evidence of persisting malignant cells but without clinical or histopathologic evidence of recurrence.
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Approximately 7 years
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Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Proportion of Patients Who Discontinue Administration of Sirolimus Because of Toxicity
Prazo: Approximately 7 years
|
Approximately 7 years
|
|
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Proportion With Infections Categorized by Organism
Prazo: Approximately 7 years
|
Approximately 7 years
|
|
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Secondary Malignancies
Prazo: Up to 7 years
|
Proportion of participants who developed at least one secondary malignancy by 7 years
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Up to 7 years
|
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Duration of Treatment With Prednisone
Prazo: Approximately 7 years
|
Approximately 7 years
|
|
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Probability of Survival Without Recurrent Malignancy
Prazo: Approximately 7 years
|
Kaplan-Meier estimate assessed at 7 years for probability of survival without recurrent malignancy.
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Approximately 7 years
|
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Probability of Overall Survival
Prazo: Approximately 7 years
|
Kaplan-Meier estimate assessed at 7 years
|
Approximately 7 years
|
|
Probability of Cumulative Incidence of Death Without Recurrent Malignancy
Prazo: Approximately 7 years
|
Analyzed with recurrent malignancy as a competing risk factor.
Assessed at 7 years.
|
Approximately 7 years
|
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Probability of Cumulative Incidence of Recurrent Malignancy
Prazo: Approximately 7 years
|
Analyzed with death as a competing risk factor.
Assessed at 7 years.
|
Approximately 7 years
|
Colaboradores e Investigadores
É aqui que você encontrará pessoas e organizações envolvidas com este estudo.
Patrocinador
Colaboradores
Datas de registro do estudo
Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.
Datas Principais do Estudo
Início do estudo
1 de abril de 2002
Conclusão Primária (Real)
1 de julho de 2009
Conclusão do estudo (Real)
10 de junho de 2010
Datas de inscrição no estudo
Enviado pela primeira vez
8 de março de 2004
Enviado pela primeira vez que atendeu aos critérios de CQ
9 de março de 2004
Primeira postagem (Estimativa)
10 de março de 2004
Atualizações de registro de estudo
Última Atualização Postada (Real)
20 de junho de 2017
Última atualização enviada que atendeu aos critérios de controle de qualidade
23 de maio de 2017
Última verificação
1 de maio de 2017
Mais Informações
Termos relacionados a este estudo
Termos MeSH relevantes adicionais
Outros números de identificação do estudo
- 1706.00
- NCI-2011-01817 (Identificador de registro: CTRP (Clinical Trial Reporting Program))
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
NÃO
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