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Anti-CD33-CLL1 CAR-T Cells (ICG415) for the Treatment of Relapsed/Refractory Acute Myeloid Leukemia (ICG415-AML-01)

19 de junho de 2026 atualizado por: iCell Gene Therapeutics

A Clinical Study to Evaluate the Safety and Efficacy of ICG415 CAR-T Cells in Adult Patients With Relapsed/Refractory Acute Myeloid Leukemia

This single-arm, open-label phase I trial evaluates the safety and tolerability of ICG415, autologous CAR-T cells targeting CD33 and CLL1, in patients with relapsed or refractory acute myeloid leukemia (AML). Subjects receive lymphodepleting chemotherapy followed by autologous CAR-T infusion. The primary goal is to assess safety and preliminary anti-leukemic efficacy in patients failing standard AML therapies.

Visão geral do estudo

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

Intervencional

Inscrição (Estimado)

18

Estágio

  • Fase 1

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.

Contato de estudo

Estude backup de contato

Locais de estudo

    • Jiangxi
      • Nanchang, Jiangxi, China, 330006
        • Recrutamento
        • Jiangxi Provincial People's Hospital (Participating Site)
        • Contato:
      • Nanchang, Jiangxi, China, 330006
        • Recrutamento
        • The First Affiliated Hospital of Nanchang University (Lead Site)
        • Contato:

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

  • Adulto
  • Adulto mais velho

Aceita Voluntários Saudáveis

Não

Descrição

Inclusion Criteria:

  1. Written informed consent approved by IRB/IEC obtained from subject or legally authorized representative prior to any screening procedures.
  2. Age ≥ 18 years and ≤ 70 years at the time of informed consent signing.
  3. 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.
  4. Bone marrow leukemic blasts positive for both CLL-1 and CD33 by flow cytometry.
  5. If circulating blasts are detectable at screening, tumor cell surface immunophenotype must be CD4 and CD8 double-negative by flow cytometry.
  6. ECOG performance status 0-2.
  7. Expected overall survival > 3 months.
  8. 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:

  1. Prior receipt of CAR-T cell therapy or other genetically modified cell therapy prior to informed consent.
  2. 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.
  3. Active uncontrolled infection: positive HBsAg/HBV-DNA, active HCV-RNA positivity, HIV positive, positive syphilis antibody, active uncontrolled EBV or CMV viremia.
  4. Unstable severe systemic disease requiring continuous medication.
  5. Grade >2 bleeding within 30 days before screening or chronic long-term anticoagulant treatment.
  6. Uncontrolled life-threatening bacterial, fungal or viral infection.
  7. Non-leukemic central nervous system organic disease or active CNS-2/CNS-3 leukemia; previously treated and resolved CNS leukemia is permitted.
  8. Concurrent other malignant tumor except cured in-situ carcinoma or malignancies with ≥5 years continuous complete remission.
  9. Live-attenuated vaccines within 30 days before screening or planned within 3 months after CAR-T infusion.
  10. Received any other investigational medicinal product within 3 months prior to ICF signature.
  11. Allogeneic hematopoietic stem cell transplantation within 6 months before screening.
  12. Pregnant or breastfeeding women.
  13. Suicidal tendency, ongoing alcohol or illicit drug dependence.
  14. Known hypersensitivity to investigational product, excipients or concomitant drugs.
  15. Any other condition judged inappropriate for trial entry by investigator.

Plano de estudo

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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
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.
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
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
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
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.
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.
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
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
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

É aqui que você encontrará pessoas e organizações envolvidas com este estudo.

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 (Real)

10 de junho de 2026

Conclusão Primária (Estimado)

4 de julho de 2028

Conclusão do estudo (Estimado)

4 de julho de 2029

Datas de inscrição no estudo

Enviado pela primeira vez

19 de junho de 2026

Enviado pela primeira vez que atendeu aos critérios de CQ

19 de junho de 2026

Primeira postagem (Real)

25 de junho de 2026

Atualizações de registro de estudo

Última Atualização Postada (Real)

25 de junho de 2026

Última atualização enviada que atendeu aos critérios de controle de qualidade

19 de junho de 2026

Última verificação

1 de junho de 2026

Mais Informações

Termos relacionados a este estudo

Plano para dados de participantes individuais (IPD)

Planeja compartilhar dados de participantes individuais (IPD)?

NÃO

Informações sobre medicamentos e dispositivos, documentos de estudo

Estuda um medicamento regulamentado pela FDA dos EUA

Não

Estuda um produto de dispositivo regulamentado pela FDA dos EUA

Não

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