- ICH GCP
- Registro de ensaios clínicos dos EUA
- Ensaio Clínico NCT07645469
FH-WT1-E50 TCR T Cells With Azacitidine for the Treatment of Minimal Residual Disease Positive Acute Myeloid Leukemia
Phase I Study of Autologous CD4+ and CD8+ T Cells That Have Been Transduced to Express a WT1-Specific T Cell Receptor for Treatment of MRD-Positive AML
Visão geral do estudo
Status
Condições
Intervenção / Tratamento
- Procedimento: Punção lombar
- Procedimento: Coleta de bioespécimes
- Procedimento: Tomografia por emissão de pósitrons
- Procedimento: Tomografia Computadorizada
- Procedimento: Aspiração de Medula Óssea
- Procedimento: Radiografia de tórax
- Medicamento: Azacitidina
- Procedimento: Biópsia de Medula Óssea
- Procedimento: Teste de ecocardiografia
- Medicamento: Autologous HLA-A*02:01-restricted CD4+/CD8+ Anti-WT1 TCR/CD8ab-expressing T-cells
- Procedimento: Multigated Acquisition Scan
Descrição detalhada
OUTLINE:
Patients undergo leukapheresis. 4 weeks later patients receive azacitidine intravenously (IV). At least 4 weeks after the first azacitidine infusion, patients receive azacitidine IV followed by Autologous HLA-A*02:01-restricted CD4+/CD8+ Anti-WT1 TCR/CD8ab-expressing T-cells (FHWT1-E50 TCR T) IV. If additional cells are available patients may receive a second infusion of azacitidine IV followed by FH-WT1-E50 TCR T cells IV, starting at 28 days, up to 1 year. Treatment is given in the absence of disease progression or unacceptable toxicity. Patients undergo multigated acquisition (MUGA) scan/echocardiography and chest x-ray during screening and bone marrow biopsy and aspiration and blood sample collection throughout the study. Patients may undergo lumbar puncture on study and computed tomography (CT) scan and/or positron emission tomography (PET) scan throughout the study.
After completion of study treatment, patients are followed up at day 1, 3, 7, 14, 21, 28, 56, 84, 168, 252 and 336 then every 6 months for years 1-5 the yearly until year 15.
Tipo de estudo
Inscrição (Estimado)
Estágio
- Fase 1
Contactos e Locais
Contato de estudo
- Nome: Fred Hutch Immunotherapy Intake
- Número de telefone: 206-606-4668
- E-mail: immunotherapy@fredhutch.org
Locais de estudo
-
-
Washington
-
Seattle, Washington, Estados Unidos, 98109
- Recrutamento
- Fred Hutch/University of Washington Cancer Consortium
-
Contato:
- Fred Hutch Immunotherapy Intake
- Número de telefone: 206-606-4668
- E-mail: immunotherapy@fredhutch.org
-
Investigador principal:
- Francesco Mazziotta, MD, PhD
-
-
Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
- Adulto
- Adulto mais velho
Aceita Voluntários Saudáveis
Descrição
Inclusion Criteria for Leukapheresis:
- LEUKAPHERESIS: Age 18 years or older at the time of enrollment
- LEUKAPHERESIS: Confirmed diagnosis of AML that is not M3 subtype (acute promyelocytic leukemia [APL])
- LEUKAPHERESIS: Human leukocyte antigen (HLA) type HLA-A*02:01 confirmed through HLA typing
- LEUKAPHERESIS: Tissue confirmation of WT1 expression by immunohistochemistry. Confirmation of diagnosis must be or have been performed by internal pathology review of archival biopsy material or other pathologic material at Fred Hutch/University of Washington Medical Center (UWMC)
- LEUKAPHERESIS: Capable of understanding and willing to provide informed consent
- LEUKAPHERESIS: Fertile male and female participants must be willing to use an effective contraceptive method before, during, and for at least 4 months after the last FH-WT1-E50 TCR T infusion
- LEUKAPHERESIS: Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 or Karnofsky Performance Status (KPS) ≥ 60%
- LEUKAPHERESIS: No immediate plan for allogeneic stem cell transplantation: patients must not have a planned allogeneic hematopoietic stem cell transplant (HCT) within 8 weeks of leukapheresis. Patients may still be considered for HCT in the future but must not have a scheduled transplant at the time of enrollment due to factors including, but not limited to, donor availability, performance status, comorbidities, or patient preference. Patients who subsequently become candidates for HCT (e.g., donor identified or improvement in performance status) may proceed to transplant at the discretion of the treating physician without a mandated waiting period related to study participation
- LEUKAPHERESIS: Creatinine clearance ≥ 30 ml/min by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) or 24-hour urine clearance
- LEUKAPHERESIS: Total bilirubin < 3.0 mg/dL. Participants with suspected Gilbert syndrome may be included if total bilirubin (tBili) > 3mg/dL but no other evidence of hepatic dysfunction
- LEUKAPHERESIS: Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) < 5 x upper limit of normal (ULN)
- LEUKAPHERESIS: Participants 60 years of age or older are required to have left ventricular ejection fraction (LVEF) evaluation performed within 60 days prior to enrollment. LVEF may be established with echocardiogram or MUGA scan, and left ejection fraction must be ≥ 35%. Cardiac evaluation for other participants is at the discretion of the treating physician
Inclusion Criteria at Start of Treatment:
START OF TREATMENT: Presence of Measurable Residual Disease (MRD) after induction therapy at the time of screening. Patients must have achieved a morphologic remission (marrow that is at least 10% cellular with < 5% blasts on morphologic review) with detectable MRD, regardless of incomplete recovery of neutrophil counts/less than 1,000/mm3 (CRi) or platelet counts less than 100,000/mm3 (CRp). Eligible remission-induction regimens include, but are not limited to, conventional induction chemotherapy (e.g., 7+3 or similar anthracycline/cytarabine-based regimens) and hypomethylating agent-based combinations (e.g., azacitidine/venetoclax).
MRD definition:
- MRD by flow cytometry: defined by any abnormal myeloid blasts identified by flow cytometric analysis. In addition, for patients with NPM1-mutated disease, we will incorporate a clinically validated quantitative NPM1 MRD assay performed at Fred Hutch. For patients with FLT3-ITD-mutated disease, MRD assessment will include a clinically validated FLT3-ITD assay performed at Invivoscribe.
- START OF TREATMENT: Participants must be willing to undergo serial tumor biopsies and/or aspirates for research purposes if safe and feasible at baseline (prior to first T cell infusion), 2-3 weeks after the first T cell infusion, and approximately 2 weeks +/- 1 week after the second infusion (if applicable) (these windows may vary due to manufacturing or clinical reasons). Should there be no marrow tissue that is accessible, participants will still be considered for participation, at the discretion of the investigator. Similarly, should an investigator determine that a marrow assessment cannot be performed safely for clinical reasons, those may be cancelled or rescheduled. Participants must be at least two weeks or five half lives (for small molecules) from last systemic treatment: At least 2 weeks must have passed since any: immunotherapy (for example, T cell infusions, immunomodulatory agents, interleukins, vaccines), or chemotherapy cancer treatment. At least five half-lives must have passed from treatment with small molecules or other investigational agents
- START OF TREATMENT: ECOG performance status of 0, 1, or 2 or Karnofsky Performance Status (KPS) ≥ 60%
- START OF TREATMENT: Creatinine clearance ≥ 30 ml/min by CKD-EPI or 24-hour urine clearance
- START OF TREATMENT: Total bilirubin < 3.0 mg/dL. Participants with suspected Gilbert syndrome may be included if tBili > 3mg/dL but no other evidence of hepatic dysfunction.
- START OF TREATMENT: Participants 60 years of age or older are required to have left ventricular ejection fraction (LVEF) evaluation performed within 60 days prior to enrollment. LVEF may be established with echocardiogram or MUGA scan, and left ejection fraction must be ≥ 35%. Cardiac evaluation for other participants is at the discretion of the treating physician
START OF TREATMENT: Participants with a history of chronic obstructive pulmonary disease (COPD), emphysema, or greater than 30 pack year smoking history should undergo pulmonary function tests (PFTs) and meet the following criteria:
- Forced expiratory volume in 1 second (FEV1) ≥ 50% of predicted and carbon monoxide diffusing capacity (DLCO) (corrected) ≥ 40% of predicted will be eligible
Exclusion Criteria for Leukapheresis:
- LEUKAPHERESIS: Prior solid organ transplant or allogeneic hematopoietic stem cell transplant. Kidney transplant participants will be considered on a case-by-case basis requiring discussion with principal investigator (PI). If the participant has had a kidney transplant, participant must have dialysis access, dialysis plan, supportive nephrologist, willingness to stop transplant immunosuppression, and express understanding that rejection is possible outcome. Dialysis or costs related to transplant kidney will not be supported by the study. Participants having had any other solid organ transplants will be excluded, as will those with any history of allogeneic hematopoietic stem cell transplant
Exclusion Criteria at Start of Treatment:
- START OF TREATMENT: Evidence of TET2, ASXL1 or DNMT3a mutations as sole evidence of MRD
- START OF TREATMENT: Pregnancy, breastfeeding, or expecting to conceive or father children for the duration of the trial through 4 months after last T cell infusion. Participants of childbearing potential must have a negative serum pregnancy test within the 2 weeks (14 days) preceding T cell infusion. Childbearing potential is defined as women who have not been surgically sterilized and who are not post-menopausal (free of menses for at least 1 year)
- START OF TREATMENT: Active autoimmune disease: Participants with active autoimmune disease requiring immunosuppressive therapy are excluded. Case-by-case exemptions are possible with approval by PI
- START OF TREATMENT: Unable to generate FH-WT1-E50 TCR T cells for infusions. However, if a lower than planned number of cells is available, the participant will have the option to receive the generated WT1-specific T cells
- START OF TREATMENT: Corticosteroid therapy at a systemic dose equivalent of > 0.5 mg/kg of prednisone-equivalent per day. The following treatments are permitted: intranasal, inhaled, topical, or local steroid applications; systemic corticosteroids at physiologic doses equivalent to no more than 10 mg/day prednisone; steroids as premedication for contrast dye allergy
- START OF TREATMENT: Concurrent use of other investigational anti-cancer agents
- START OF TREATMENT: Active uncontrolled infection: HIV positive participants on highly active antiretroviral therapy (HAART) with a CD4 count > 500 cells/mm3 are considered controlled, as are individuals with a history of hepatitis C who have successfully completed antiviral therapy with an undetectable viral load, and those with hepatitis B who have, per standard practice, hepatitis well-controlled on medication
- START OF TREATMENT: Uncontrolled concurrent illness: Participants may not have uncontrolled or concurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
- START OF TREATMENT: Known allergic reactions to any of the components of study treatments
- START OF TREATMENT: Other medical, social, or psychiatric factors that interfere with medical appropriateness and/or ability to comply with study, as determined by the PI
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
- Finalidade Principal: Tratamento
- Alocação: N / D
- Modelo Intervencional: Atribuição de grupo único
- Mascaramento: Nenhum (rótulo aberto)
Armas e Intervenções
Grupo de Participantes / Braço |
Intervenção / Tratamento |
|---|---|
|
Experimental: Treatment (FH-WT1-E50 TCR T cells, azacitidine)
Patients undergo leukapheresis. 4 weeks later patients receive azacitidine IV.
At least 4 weeks after the first azacitidine infusion, patients receive azacitidine IV followed by FH-WT1-E50 TCR T cells IV.
If additional cells are available patients may receive a second infusion of azacitidine IV followed by FH-WT1-E50 TCR T cells IV, starting at 28 days, up to 1 year.
Treatment is given in the absence of disease progression or unacceptable toxicity.
Patients undergo MUGA scan/echocardiography and chest x-ray during screening and bone marrow biopsy and aspiration and blood sample collection throughout the study.
Patients may undergo lumbar puncture on study and CT scan and/or PET scan throughout the study.
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Fazer punção lombar
Outros nomes:
Realizar coleta de sangue
Outros nomes:
Fazer PET scan
Outros nomes:
Fazer tomografia computadorizada
Outros nomes:
Submeta-se a aspiração de medula óssea
Faça radiografia de tórax
Outros nomes:
Dado IV
Outros nomes:
Fazer biópsia de medula óssea
Outros nomes:
Sofrer ecocardiografia
Outros nomes:
Given IV
Outros nomes:
Given MUGA scan
Outros nomes:
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O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Prazo |
|---|---|
|
Incidence of treatment related grade 3 or higher adverse events
Prazo: From first infusion up to 1 year
|
From first infusion up to 1 year
|
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Incidence of dose limiting toxicity
Prazo: From first T cell infusion, up to 28 days
|
From first T cell infusion, up to 28 days
|
Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Ability to reproducibly generate and infuse T cells at the planned dose level (feasibility)
Prazo: From baseline up to 1 year after eligibility determination
|
No formal statistical considerations will be used to evaluate this endpoint beyond a simple estimate of the proportion along with its 95% confidence interval.
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From baseline up to 1 year after eligibility determination
|
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Relapse
Prazo: At 1 and 2 years after first infusion
|
Defined as with or without measurable tumor burden prior to T cell transfer, including patients who convert to minimal residual disease-negative status during consolidation.
|
At 1 and 2 years after first infusion
|
Colaboradores e Investigadores
Patrocinador
Colaboradores
Investigadores
- Investigador principal: Francesco Mazziotta, MD, PhD, Fred Hutch/University of Washington Cancer Consortium
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Estimado)
Conclusão Primária (Estimado)
Conclusão do estudo (Estimado)
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
Termos MeSH relevantes adicionais
- Neoplasias
- Neoplasias por Tipo Histológico
- Doenças Hematológicas
- Leucemia Mieloide
- Leucemia
- Doenças hemic e linfáticas
- Leucemia Mieloide Aguda
- Produtos químicos orgânicos
- Compostos heterocíclicos, 1 anel
- Compostos heterocíclicos
- Técnicas de investigação
- Terapêutica
- Técnicas de laboratório clínico
- Técnicas e procedimentos de diagnóstico
- Diagnóstico
- Perfurações
- Procedimentos cirúrgicos, operatórios
- Técnicas citológicas
- Ácidos nucleicos, nucleotídeos e nucleosídeos
- Citodiagnóstico
- Fenômenos físicos
- Citidina
- Nucleosídeos de pirimidina
- Pirimidinas
- Técnicas de diagnóstico, cirúrgico
- Técnicas de química, analíticas
- Análise de espectro
- Compostos AZA
- Nucleosídeos
- Ribonucleosídeos
- Fenômenos eletromagnéticos
- Fenômenos magnéticos
- Técnicas de diagnóstico, neurológico
- Radiação eletromagnética
- Radiação
- Radiação, ionizando
- Azacitidina
- Biópsia
- Manipulação de amostras
- Espectroscopia de ressonância magnética
- Punção espinhal
- Raios X
Outros números de identificação do estudo
- RG1126017
- 2P01CA018029 (Concessão/Contrato do NIH dos EUA)
- 21042 (Outro identificador: Fred Hutch/University of Washington Cancer Consortium)
- NCI-2026-02251 (Identificador de registro: CTRP (Clinical Trial Reporting Program))
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
Descrição do plano IPD
Prazo de Compartilhamento de IPD
Critérios de acesso de compartilhamento IPD
Access to IPD and supporting materials will be granted to qualified researchers whose proposed use is consistent with scientifically sound secondary analyses. Requests will require submission of a research proposal, institutional affiliation, and IRB or ethics approval or exemption where applicable. Data will be shared under a data use agreement that prohibits re-identification attempts and restricts use to non-commercial research. Requests will be reviewed by a study steering committee or designated data access committee.
Data will be shared via a controlled-access repository or secure data-sharing platform, with appropriate governance and security controls.
A dedicated controlled-access data repository will be established upon study completion. The URL will be provided when the repository becomes active.
Tipo de informação de suporte de compartilhamento de IPD
- PROTOCOLO DE ESTUDO
- SEIVA
- CIF
Informações sobre medicamentos e dispositivos, documentos de estudo
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