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- Ensaio Clínico NCT07668557
Anti-CD33-CLL1 CAR-T Cells (ICG415) for the Treatment of Relapsed/Refractory Acute Myeloid Leukemia (ICG415-AML-01)
A Clinical Study to Evaluate the Safety and Efficacy of ICG415 CAR-T Cells in Adult Patients With Relapsed/Refractory Acute Myeloid Leukemia
Visão geral do estudo
Status
Condições
Descrição detalhada
Acute myeloid leukemia (AML) is an aggressive hematologic malignancy with limited treatment options for patients who are relapsed or refractory (R/R) to standard therapies. Leukemic blasts in R/R AML frequently co-express CD33 and CLL1, while sparing normal hematopoietic stem cells, making them rational targets for chimeric antigen receptor (CAR) T-cell therapy.
This phase I, single-arm, open-label study evaluates ICG415 CAR-T Cells in patients with R/R AML. After leukapheresis and ex vivo modification, patients receive a single CAR-T cell infusion following lymphodepleting chemotherapy with fludarabine and cyclophosphamide. Primary objectives are safety and tolerability, including dose-limiting toxicities (DLTs), cytokine release syndrome (CRS), and neurological events. Secondary objectives include response rate (CR/CRi/PR), minimal residual disease (MRD) negativity, progression-free survival (PFS), and overall survival (OS).
All participants will be followed for up to 24 months with regular clinical, laboratory, and imaging evaluations to monitor both treatment efficacy and potential complications.
Tipo de estudo
Inscrição (Estimado)
Estágio
- Fase 1
Contactos e Locais
Contato de estudo
- Nome: Yu Min
- Número de telefone: +86 150 7902 9006
- E-mail: 625668742@qq.com
Estude backup de contato
- Nome: Li Fei
- Número de telefone: +86 139 7003 8386
- E-mail: yx021021@sina.com
Locais de estudo
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Jiangxi
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Nanchang, Jiangxi, China, 330006
- Recrutamento
- Jiangxi Provincial People's Hospital (Participating Site)
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Contato:
- Cheng Hongbo
- Número de telefone: +86 137 0708 5405
- E-mail: 784260212@qq.com
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Nanchang, Jiangxi, China, 330006
- Recrutamento
- The First Affiliated Hospital of Nanchang University (Lead Site)
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Contato:
- Li Fei
- Número de telefone: +86 139 7003 8386
- E-mail: yx021021@sina.com
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-
Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
- Adulto
- Adulto mais velho
Aceita Voluntários Saudáveis
Descrição
Inclusion Criteria:
- Written informed consent approved by IRB/IEC obtained from subject or legally authorized representative prior to any screening procedures.
- Age ≥ 18 years and ≤ 70 years at the time of informed consent signing.
- Diagnosis of acute myeloid leukemia (AML) per 2022 WHO Classification, meeting criteria for relapsed/refractory (R/R) AML as defined in the Chinese Guidelines for the Diagnosis and Management of Relapsed/Refractory Acute Myeloid Leukemia (2023 Edition): Relapsed AML: Reappearance of leukemic blasts in peripheral blood, bone marrow blasts ≥5%, or extramedullary leukemic infiltration after complete remission (CR). Refractory AML: failure to achieve CR after two cycles of standard induction chemotherapy; early relapse within 12 months post-CR; late relapse with salvage chemotherapy resistance; ≥2 disease relapses or persistent extramedullary disease.
- Bone marrow leukemic blasts positive for both CLL-1 and CD33 by flow cytometry.
- If circulating blasts are detectable at screening, tumor cell surface immunophenotype must be CD4 and CD8 double-negative by flow cytometry.
- ECOG performance status 0-2.
- Expected overall survival > 3 months.
- Females of childbearing potential: negative serum pregnancy test and effective contraception for 1 year post-infusion. Males of reproductive potential: effective barrier contraception for 1 year post-infusion and no sperm donation within 1 year after infusion.
Exclusion Criteria:
- Prior receipt of CAR-T cell therapy or other genetically modified cell therapy prior to informed consent.
- Severe major organ dysfunction: Renal: eGFR < 50 mL/min (Cockcroft-Gault); Hepatic: ALT/AST > 3 × ULN (>5×ULN if disease-related), total bilirubin > 2 × ULN (>3×ULN for Gilbert syndrome); Cardiac: LVEF < 50%, room air SpO₂ <94%, uncontrolled severe cardiac disease.
- Active uncontrolled infection: positive HBsAg/HBV-DNA, active HCV-RNA positivity, HIV positive, positive syphilis antibody, active uncontrolled EBV or CMV viremia.
- Unstable severe systemic disease requiring continuous medication.
- Grade >2 bleeding within 30 days before screening or chronic long-term anticoagulant treatment.
- Uncontrolled life-threatening bacterial, fungal or viral infection.
- Non-leukemic central nervous system organic disease or active CNS-2/CNS-3 leukemia; previously treated and resolved CNS leukemia is permitted.
- Concurrent other malignant tumor except cured in-situ carcinoma or malignancies with ≥5 years continuous complete remission.
- Live-attenuated vaccines within 30 days before screening or planned within 3 months after CAR-T infusion.
- Received any other investigational medicinal product within 3 months prior to ICF signature.
- Allogeneic hematopoietic stem cell transplantation within 6 months before screening.
- Pregnant or breastfeeding women.
- Suicidal tendency, ongoing alcohol or illicit drug dependence.
- Known hypersensitivity to investigational product, excipients or concomitant drugs.
- Any other condition judged inappropriate for trial entry by investigator.
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
- Finalidade Principal: Tratamento
- Alocação: N / D
- Modelo Intervencional: Atribuição sequencial
- Mascaramento: Nenhum (rótulo aberto)
Armas e Intervenções
Grupo de Participantes / Braço |
Intervenção / Tratamento |
|---|---|
|
Experimental: Single Arm, Anti-CD33-CLL1 CAR-T (ICG415) for Relapsed or Refractory AML
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Anti-CD33, Anti-CLL1 Compound CAR-T Cells
|
O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Incidence of Dose-Limiting Toxicities (DLTs)
Prazo: Within 28 days after ICG415 CAR-T cell infusion
|
DLTs assessed according to the protocol-defined criteria.
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Within 28 days after ICG415 CAR-T cell infusion
|
|
Incidence and Severity of Treatment-Emergent Adverse Events (TEAEs)
Prazo: From first dose through 24 months post infusion
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Frequency and severity of TEAEs graded by NCI-CTCAE Version 5.0, including changes from baseline in vital signs, physical examination, 12-lead ECG, and clinical laboratory parameters (complete blood count, urinalysis, blood chemistry, coagulation function, etc.).
|
From first dose through 24 months post infusion
|
Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Objective Response Rate (ORR) at Months 1, 3, and 6
Prazo: Month 1, Month 3, Month 6
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ORR defined as proportion of subjects achieving complete remission (CR), complete remission with incomplete count recovery (CRi), or complete remission with partial hematological recovery (CRh) per response criteria.
|
Month 1, Month 3, Month 6
|
|
Rates of CR, CRi, and PR at Year 1 and Year 2
Prazo: Year 1, Year 2
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Proportion of subjects achieving complete remission (CR), complete remission with incomplete count recovery (CRi), and partial remission (PR) at 1 and 2 years post infusion.
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Year 1, Year 2
|
|
Cumulative Incidence of Relapse (CIR) at Year 1 and Year 2
Prazo: Year 1, Year 2
|
Cumulative incidence of disease relapse at 1 and 2 years after CAR-T cell infusion.
|
Year 1, Year 2
|
|
Duration of Response (DOR)
Prazo: From first documented response to disease relapse or death from any cause, assessed up to 24 months
|
Time from first objective response (CR, CRi, or CRh) to relapse or death, whichever occurs first.
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From first documented response to disease relapse or death from any cause, assessed up to 24 months
|
|
Progression-Free Survival (PFS)
Prazo: From date of infusion to disease progression or death from any cause, assessed up to 24 months
|
Time from ICG415 infusion to disease progression (relapse or treatment failure) or death from any cause.
|
From date of infusion to disease progression or death from any cause, assessed up to 24 months
|
|
Overall Survival (OS)
Prazo: From date of infusion to death from any cause, assessed up to 24 months
|
Time from ICG415 infusion to death from any cause.
|
From date of infusion to death from any cause, assessed up to 24 months
|
|
Event-Free Survival (EFS)
Prazo: From date of infusion to any treatment failure, relapse, or death, assessed up to 24 months
|
Time from ICG415 infusion to any event including lack of response, disease relapse, or death from any cause.
|
From date of infusion to any treatment failure, relapse, or death, assessed up to 24 months
|
|
CAR-T Cell Kinetics - Persistence over Time
Prazo: Days 0, 4, 7, 14, 21, 28; Months 2, 3, 6, 9, 12, 18, 24
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Number and duration of detectable CAR-T cells in peripheral blood.
Testing stops after two consecutive negative results.
|
Days 0, 4, 7, 14, 21, 28; Months 2, 3, 6, 9, 12, 18, 24
|
|
Cytokine Level Changes
Prazo: Days 0, 7, 14, 21, 28; Months 2, 3, 6
|
Changes in serum cytokine levels including IL-6, IL-10, IL-15, TNF-α, and IFN-γ.
|
Days 0, 7, 14, 21, 28; Months 2, 3, 6
|
|
Peripheral Blood Lymphocyte Subset Changes
Prazo: Days 0, 7, 14, 21, 28; Months 2, 3, 6
|
Changes in lymphocyte subsets including T cells, B cells, NK cells, and CD4/CD8 ratio measured by flow cytometry.
|
Days 0, 7, 14, 21, 28; Months 2, 3, 6
|
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Anti-Drug Antibody (ADA) Levels
Prazo: Day 28; Months 3, 6, 12
|
Incidence and titers of anti-drug antibodies (ADA) against ICG415 CAR-T cells.
|
Day 28; Months 3, 6, 12
|
Colaboradores e Investigadores
Patrocinador
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Real)
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
Palavras-chave
Termos MeSH relevantes adicionais
- Processos Patológicos
- Neoplasias
- Atributos da doença
- Neoplasias por Tipo Histológico
- Doenças Hematológicas
- Condições Patológicas, Sinais e Sintomas
- Doenças hemic e linfáticas
- Recorrência
- Leucemia
- Leucemia Mieloide
- Leucemia Mieloide Aguda
- Produtos químicos orgânicos
- Hidrocarbonetos
- Mustarazas de fosforamida
- Compostos de mostarda de nitrogênio
- Compostos de mostarda
- Hidrocarbonetos, halogenados
- Fosforamidas
- Compostos organofosforos
- Ciclofosfamida
Outros números de identificação do estudo
- ICG415-001
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
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