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
研究概览
地位
详细说明
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.
研究类型
注册 (估计的)
阶段
- 阶段1
联系人和位置
学习联系方式
- 姓名:Yu Min
- 电话号码:+86 150 7902 9006
- 邮箱:625668742@qq.com
研究联系人备份
- 姓名:Li Fei
- 电话号码:+86 139 7003 8386
- 邮箱:yx021021@sina.com
学习地点
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Jiangxi
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Nanchang、Jiangxi、中国、330006
- 招聘中
- Jiangxi Provincial People's Hospital (Participating Site)
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接触:
- Cheng Hongbo
- 电话号码:+86 137 0708 5405
- 邮箱:784260212@qq.com
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Nanchang、Jiangxi、中国、330006
- 招聘中
- The First Affiliated Hospital of Nanchang University (Lead Site)
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接触:
- Li Fei
- 电话号码:+86 139 7003 8386
- 邮箱:yx021021@sina.com
-
-
参与标准
资格标准
适合学习的年龄
- 成人
- 年长者
接受健康志愿者
描述
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.
学习计划
研究是如何设计的?
设计细节
- 主要用途:治疗
- 分配:不适用
- 介入模型:顺序分配
- 屏蔽:无(打开标签)
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
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实验性的: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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研究衡量的是什么?
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Incidence of Dose-Limiting Toxicities (DLTs)
大体时间: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)
大体时间: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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次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Objective Response Rate (ORR) at Months 1, 3, and 6
大体时间: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
大体时间: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
大体时间: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)
大体时间: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)
大体时间: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
|
|
Overall Survival (OS)
大体时间:From date of infusion to death from any cause, assessed up to 24 months
|
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)
大体时间: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
大体时间: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
大体时间: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
大体时间: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
大体时间:Day 28; Months 3, 6, 12
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Incidence and titers of anti-drug antibodies (ADA) against ICG415 CAR-T cells.
|
Day 28; Months 3, 6, 12
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合作者和调查者
研究记录日期
研究主要日期
学习开始 (实际的)
初级完成 (估计的)
研究完成 (估计的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
与本研究相关的术语
其他相关的 MeSH 术语
其他研究编号
- ICG415-001
计划个人参与者数据 (IPD)
计划共享个人参与者数据 (IPD)?
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