- ICH GCP
- Registro de ensayos clínicos de EE. UU.
- Ensayo 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
Descripción general del estudio
Estado
Condiciones
Intervención / Tratamiento
Descripción detallada
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 estudio
Inscripción (Estimado)
Fase
- Fase 1
Contactos y Ubicaciones
Estudio Contacto
- Nombre: Yu Min
- Número de teléfono: +86 150 7902 9006
- Correo electrónico: 625668742@qq.com
Copia de seguridad de contactos de estudio
- Nombre: Li Fei
- Número de teléfono: +86 139 7003 8386
- Correo electrónico: yx021021@sina.com
Ubicaciones de estudio
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Jiangxi
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Nanchang, Jiangxi, Porcelana, 330006
- Reclutamiento
- Jiangxi Provincial People's Hospital (Participating Site)
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Contacto:
- Cheng Hongbo
- Número de teléfono: +86 137 0708 5405
- Correo electrónico: 784260212@qq.com
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Nanchang, Jiangxi, Porcelana, 330006
- Reclutamiento
- The First Affiliated Hospital of Nanchang University (Lead Site)
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Contacto:
- Li Fei
- Número de teléfono: +86 139 7003 8386
- Correo electrónico: yx021021@sina.com
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
- Adulto
- Adulto Mayor
Acepta Voluntarios Saludables
Descripción
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.
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Tratamiento
- Asignación: N / A
- Modelo Intervencionista: Asignación Secuencial
- Enmascaramiento: Ninguno (etiqueta abierta)
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
|---|---|
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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
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¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Incidence of Dose-Limiting Toxicities (DLTs)
Periodo de tiempo: Within 28 days after ICG415 CAR-T cell infusion
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DLTs assessed according to the protocol-defined criteria.
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Within 28 days after ICG415 CAR-T cell infusion
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Incidence and Severity of Treatment-Emergent Adverse Events (TEAEs)
Periodo de tiempo: 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.).
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From first dose through 24 months post infusion
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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Objective Response Rate (ORR) at Months 1, 3, and 6
Periodo de tiempo: 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.
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Month 1, Month 3, Month 6
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Rates of CR, CRi, and PR at Year 1 and Year 2
Periodo de tiempo: 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
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Cumulative Incidence of Relapse (CIR) at Year 1 and Year 2
Periodo de tiempo: Year 1, Year 2
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Cumulative incidence of disease relapse at 1 and 2 years after CAR-T cell infusion.
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Year 1, Year 2
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Duration of Response (DOR)
Periodo de tiempo: From first documented response to disease relapse or death from any cause, assessed up to 24 months
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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
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Progression-Free Survival (PFS)
Periodo de tiempo: From date of infusion to disease progression or death from any cause, assessed up to 24 months
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Time from ICG415 infusion to disease progression (relapse or treatment failure) or death from any cause.
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From date of infusion to disease progression or death from any cause, assessed up to 24 months
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Overall Survival (OS)
Periodo de tiempo: From date of infusion to death from any cause, assessed up to 24 months
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Time from ICG415 infusion to death from any cause.
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From date of infusion to death from any cause, assessed up to 24 months
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Event-Free Survival (EFS)
Periodo de tiempo: From date of infusion to any treatment failure, relapse, or death, assessed up to 24 months
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Time from ICG415 infusion to any event including lack of response, disease relapse, or death from any cause.
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From date of infusion to any treatment failure, relapse, or death, assessed up to 24 months
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CAR-T Cell Kinetics - Persistence over Time
Periodo de tiempo: 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.
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Days 0, 4, 7, 14, 21, 28; Months 2, 3, 6, 9, 12, 18, 24
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Cytokine Level Changes
Periodo de tiempo: Days 0, 7, 14, 21, 28; Months 2, 3, 6
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Changes in serum cytokine levels including IL-6, IL-10, IL-15, TNF-α, and IFN-γ.
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Days 0, 7, 14, 21, 28; Months 2, 3, 6
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Peripheral Blood Lymphocyte Subset Changes
Periodo de tiempo: Days 0, 7, 14, 21, 28; Months 2, 3, 6
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Changes in lymphocyte subsets including T cells, B cells, NK cells, and CD4/CD8 ratio measured by flow cytometry.
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Days 0, 7, 14, 21, 28; Months 2, 3, 6
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Anti-Drug Antibody (ADA) Levels
Periodo de tiempo: Day 28; Months 3, 6, 12
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Incidence and titers of anti-drug antibodies (ADA) against ICG415 CAR-T cells.
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Day 28; Months 3, 6, 12
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Colaboradores e Investigadores
Patrocinador
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Actual)
Finalización primaria (Estimado)
Finalización del estudio (Estimado)
Fechas de registro del estudio
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Actual)
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Más información
Términos relacionados con este estudio
Palabras clave
Términos MeSH relevantes adicionales
- Procesos Patológicos
- Neoplasias
- Atributos de la enfermedad
- Neoplasias por tipo histológico
- Enfermedades hematológicas
- Condiciones Patológicas, Signos y Síntomas
- Enfermedades hemic y linfáticas
- Reaparición
- Leucemia
- Leucemia Mieloide
- Leucemia Mieloide Aguda
- Químicos orgánicos
- Hidrocarburos
- Mostaza de fosforamida
- Compuestos de mostaza de nitrógeno
- Compuestos de mostaza
- Hidrocarburos, halogenados
- Fosforamidas
- Compuestos organofosforados
- Ciclofosfamida
Otros números de identificación del estudio
- ICG415-001
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
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