A Phase 1 interventional study of CLL-1-CD70 CAR-T Cell Injection in Acute Myeloid Leukemia (AML) and Relapsed/Refractory Acute Myeloid Leukemia, sponsored by Liping Dou. Not yet recruiting at 1 site in China. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-10-27.
Sponsored by Liping Dou · Phase 1, Interventional, and Treatment
This is a single-center, open-label, single-arm, prospective, investigator-initiated dose-escalation study to evaluate the safety, tolerability, and preliminary efficacy of an autologous CLL-1/CD70 dual-target CAR-T cell injection in patients with relapsed or refractory (R/R) acute myeloid leukemia (AML; acute promyelocytic leukemia excluded). After leukapheresis for CAR-T cell manufacture, participants receive lymphodepleting conditioning (fludarabine 30 mg/m\^2/day plus cyclophosphamide 300 mg/m\^2/day on Days -5 to -3), followed by a single intravenous infusion of CLL-1-CD70 CAR-T cells. Dose escalation follows a traditional 3+3 design with planned dose levels of 0.5 x 10\^6, 1 x 10\^6, and 2 x 10\^6 CAR-T cells/kg, guided by dose-limiting toxicities (DLTs) assessed during Days 1-28 after infusion. The primary endpoint is safety and tolerability, as assessed by treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs), including cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), hematologic toxicity, organ toxicity, infections, hemophagocytic lymphohistiocytosis/macrophage activation syndrome (HLH/MAS), and tumor lysis syndrome (TLS). Efficacy will be evaluated by the composite complete remission rate (CRc, defined as the proportion of patients achieving CR, CRh, or CRi) at prespecified time points according to the European LeukemiaNet (ELN) 2022 criteria, overall survival (OS), event-free survival (EFS), duration of response (DOR), and cumulative incidence of relapse (CIR) over a 1-year follow-up period. Immune reconstitution following CAR-T cell infusion will also be assessed at prespecified time points. CAR-T cellular kinetics in peripheral blood (CAR transgene copies, Cmax, Tmax, and AUC0-28d) will also be evaluated.
This is a single-center, single-arm, open-label, prospective, investigator-initiated clinical study of an autologous CLL-1/CD70 dual-target CAR-T cell injection in adults with R/R AML (APL excluded). Autologous peripheral blood mononuclear cells are collected by leukapheresis; T cells are selected, activated, and transduced ex vivo with a lentiviral vector encoding a dual-target chimeric antigen receptor against CLL-1 and CD70, and expanded using an optimized cytokine-combination process (manufacturing cycle approximately 7-8 days). Eligible participants receive lymphodepleting conditioning with fludarabine (30 mg/m\^2/day x 3 days) and cyclophosphamide (300 mg/m\^2/day x 3 days) on Days -5 to -3, which may be adjusted per the investigator's judgment of performance status, disease burden, and tolerability, followed by a single intravenous infusion of CLL-1-CD70 CAR-T cells on Day 0. Three dose levels are planned (0.5 x 10\^6, 1 x 10\^6, and 2 x 10\^6 CAR-T cells/kg) using a traditional 3+3 escalation design; the DLT observation window is Days 1-28 after infusion. Anticipated enrollment is 12 participants (range 9-18, depending on the number of dose levels requiring expansion). The study includes a screening period (within 28 days), a treatment period, and a follow-up period of up to 12 months. The primary endpoint is safety and tolerability. Safety assessments include adverse events and serious adverse events graded per NCI CTCAE v5.0, with CRS and ICANS graded per the ASTCT consensus criteria; hematologic toxicity, infection, organ toxicity, HLH/MAS, and TLS are also monitored. Disease response is assessed by bone marrow morphology and multiparameter flow cytometry according to the ELN 2022 criteria (CRc,CR, CRh, CRi, MLFS, NR and PR), with measurable residual disease (MRD) assessed by multiparameter flow cytometry; extramedullary disease is assessed by imaging (CT/MRI/PET-CT) when present. Exploratory cellular kinetics include peripheral blood CAR transgene copies (qPCR), CAR-positive T-cell counts (flow cytometry), peak expansion (Cmax), time to peak (Tmax), and the area under the curve from Day 0 to Day 28 (AUC0-28d), with peripheral blood sampling planned on Days 4, 7, 10, 14, and 28 after infusion.
3,037 studies on the registry are indexed under Leukemia, Myeloid, Acute; 747 are open to participants now.
This study's planned enrollment of 12 is below the median of 40 across 2,561 interventional studies indexed under Leukemia, Myeloid, Acute.
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Diagnosis of AML meeting at least one of the following:
Adequate renal, hepatic, cardiac, and pulmonary function, defined as follows:
Exclusion Criteria:
Note:
After leukapheresis and lymphodepleting conditioning (fludarabine 30 mg/m\^2/day plus cyclophosphamide 300 mg/m\^2/day on Days -5 to -3), participants receive a single intravenous infusion of CLL-1-CD70 CAR-T Cell Injection at one of three planned dose levels (0.5 x 10\^6, 1 x 10\^6, or 2 x 10\^6 CAR-T cells/kg) assigned by a traditional 3+3 dose-escalation design.
Drug: CLL-1-CD70 CAR-T Cell Injection
CLL-1-CD70 CAR-T Cell Injection is an investigational autologous dual-target CAR T-cell therapy (lentiviral vector-based) directed against CLL-1 and CD70, for patients with relapsed or refractory (R/R) acute myeloid leukemia (acute promyelocytic leukemia excluded). After leukapheresis and lymphodepleting conditioning (fludarabine plus cyclophosphamide), participants receive a single intravenous infusion at protocol-specified dose levels (0.5 x 10\^6, 1 x 10\^6, or 2 x 10\^6 CAR-T cells/kg). Dose escalation follows a traditional 3+3 design and is guided by the occurrence of dose-limiting toxicities (DLTs).
Also known as: CLL1-CD70 dual-target CAR-T, autologous CLL-1/CD70 CAR-T cell injection
Safety: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Number of participants with at least one TEAE following CLL-1/CD70 CAR-T cell infusion, regardless of its relationship to treatment. TEAEs are adverse events that newly occur or worsen after infusion and include serious and non-serious events. Each participant will be counted once, irrespective of the number or type of TEAEs. Severity will be graded using NCI CTCAE v5.0, except that cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) will be graded using the ASTCT consensus criteria. Abnormal laboratory, vital sign, physical examination, and electrocardiogram findings will be included when they meet the protocol-defined adverse event criteria.
Time frame: From CAR-T cell infusion through 12 months after infusion.
Composite Complete Remission Rate (CRc)
Percentage of participants meeting the criteria for complete remission (CR), complete remission with partial hematologic recovery (CRh), or complete remission with incomplete hematologic recovery (CRi) at each prespecified assessment time point, according to the European LeukemiaNet (ELN) 2022 response criteria.
Time frame: At Months 1, 3, 6, 9, and 12 after CAR-T cell infusion.
Complete Remission (CR) Rate
Percentage of participants meeting the criteria for complete remission at each prespecified assessment time point. CR is defined as bone marrow blasts \<5%, absence of circulating blasts and extramedullary disease, an absolute neutrophil count ≥1.0 × 10\^9/L, and a platelet count ≥100 × 10\^9/L, according to the ELN 2022 response criteria.
Time frame: At Months 1, 3, 6, 9, and 12 after CAR-T cell infusion.
Complete Remission With Partial Hematologic Recovery (CRh) Rate
Percentage of participants meeting the criteria for CRh at each prespecified assessment time point. CRh is defined as meeting the morphologic criteria for CR, with an absolute neutrophil count ≥0.5 × 10\^9/L and a platelet count ≥50 × 10\^9/L, without meeting the criteria for CR, according to the ELN 2022 response criteria.
Time frame: At Months 1, 3, 6, 9, and 12 after CAR-T cell infusion.
Complete Remission With Incomplete Hematologic Recovery (CRi) Rate
Percentage of participants meeting the criteria for CRi at each prespecified assessment time point. CRi is defined as meeting the morphologic criteria for CR but with incomplete neutrophil or platelet recovery. Participants meeting the criteria for CR or CRh will not be included in this category. Response will be assessed according to the ELN 2022 criteria.
Time frame: At Months 1, 3, 6, 9, and 12 after CAR-T cell infusion.
No Response (NR) Rate
Percentage of response-evaluable participants who do not meet the criteria for CR, CRh, CRi, morphologic leukemia-free state (MLFS), or partial remission (PR) at each prespecified assessment time point. Response categories will be determined using the ELN 2022 criteria and the protocol-specified response assessment rules.
Time frame: At Months 1, 3, 6, 9, and 12 after CAR-T cell infusion.
Partial Remission (PR) Rate
Percentage of participants meeting the criteria for partial remission at each prespecified assessment time point. PR is defined as meeting all hematologic criteria for CR, with bone marrow blasts reduced to 5%-25% and a reduction of at least 50% from the pretreatment bone marrow blast percentage, according to the ELN 2022 response criteria.
Time frame: At Months 1, 3, 6, 9, and 12 after CAR-T cell infusion.
Morphologic Leukemia-Free State (MLFS) Rate
Percentage of participants meeting the criteria for MLFS at each prespecified assessment time point. MLFS is defined as bone marrow blasts \<5%, absence of circulating blasts and extramedullary disease, without requiring hematologic recovery. Bone marrow adequacy and response classification will be assessed according to the ELN 2022 criteria.
Time frame: At Months 1, 3, 6, 9, and 12 after CAR-T cell infusion.
Overall Survival (OS)
Time from CAR-T cell infusion to death from any cause, reported in months. Participants who have not died will be censored on the date they were last known to be alive.
Time frame: From CAR-T cell infusion through 12 months after infusion.
Event-Free Survival (EFS)
Time from CAR-T cell infusion to the first occurrence of a protocol-defined event: failure to achieve CR, CRh, or CRi at the protocol-specified response assessment; hematologic relapse after CR, CRh, or CRi; initiation of new anti-leukemia therapy; or death from any cause, whichever occurs first. Participants without an event will be censored on the date of their last adequate disease assessment. Time will be reported in months.
Time frame: From CAR-T cell infusion through 12 months after infusion.
Duration of Response (DOR)
Time from the first documented response to disease progression, relapse, or death from any cause, whichever occurs first, among participants achieving CR, CRh, CRi, MLFS, or PR. Duration will be reported in months. Censoring will follow the protocol-specified analysis rules.
Time frame: From the first documented response through 12 months after CAR-T cell infusion.
Cumulative Incidence of Relapse (CIR)
Cumulative probability of hematologic relapse among participants achieving CR, CRh, or CRi, measured from the first documented achievement of remission. Death without prior relapse will be treated as a competing event. The cumulative incidence will be expressed as a percentage.
Time frame: From the first documented remission through 12 months after CAR-T cell infusion.
Peripheral Blood CAR Transgene Copy Number
CAR transgene copy number in peripheral blood measured by quantitative polymerase chain reaction (qPCR) at each protocol-specified post-infusion sampling time point. Results will be reported as CAR transgene copies per microgram of genomic DNA (copies/µg genomic DNA).
Time frame: From CAR-T cell infusion through 12 months after infusion.
Maximum Observed Peripheral Blood CAR Transgene Copy Number (Cmax)
Maximum observed CAR transgene copy number in peripheral blood during the first 28 days after CAR-T cell infusion, determined from serial qPCR measurements. Cmax will be reported in copies/µg genomic DNA.
Time frame: From CAR-T cell infusion through Day 28.
Time to Maximum Observed Peripheral Blood CAR Transgene Copy Number (Tmax)
Elapsed time from CAR-T cell infusion to the maximum observed peripheral blood CAR transgene copy number during the first 28 days after infusion, determined from serial qPCR measurements. Tmax will be reported in days.
Time frame: From CAR-T cell infusion through Day 28.
Area Under the Peripheral Blood CAR Transgene Copy Number-Time Curve From Day 0 to Day 28 (AUC0-28d)
Area under the peripheral blood CAR transgene copy number-time curve from Day 0 to Day 28 after CAR-T cell infusion, calculated using the linear trapezoidal method. Results will be reported in copies·days/µg genomic DNA. Handling of the time-zero value and missing measurements will follow prespecified analysis rules.
Time frame: From CAR-T cell infusion through Day 28.
Measurable Residual Disease (MRD) Negativity Rate (Exploratory)
Percentage of participants with MRD-negative bone marrow at each prespecified post-infusion assessment time point among participants achieving CR, CRh, or CRi. MRD will be assessed by multiparameter flow cytometry with a target analytical sensitivity of 10\^-4. MRD negativity is defined as fewer than 0.01% leukemia-associated or different-from-normal immunophenotypic cells among CD45-expressing cells. Samples must meet assay quality requirements and include at least 500,000 assessed CD45-expressing cells to be evaluable for MRD negativity. Inadequate samples will not be classified as MRD-negative.
Time frame: At Day 28 and Months 2, 3, 6, 9, and 12 after CAR-T cell infusion.
Absolute Peripheral Blood CD3+ T-Cell Count
Absolute CD3+ T-cell count in peripheral blood measured by flow cytometry at each prespecified assessment time point. Results will be reported in cells/µL.
Time frame: At screening, Day -1, Day 28, and Months 2, 3, 6, 9, and 12 after CAR-T cell infusion.
Absolute Peripheral Blood CD4+ T-Cell Count
Absolute CD4+ T-cell count in peripheral blood measured by flow cytometry at each prespecified assessment time point. Results will be reported in cells/µL.
Time frame: At screening, Day -1, Day 28, and Months 2, 3, 6, 9, and 12 after CAR-T cell infusion.
Absolute Peripheral Blood CD8+ T-Cell Count
Absolute CD8+ T-cell count in peripheral blood measured by flow cytometry at each prespecified assessment time point. Results will be reported in cells/µL.
Time frame: At screening, Day -1, Day 28, and Months 2, 3, 6, 9, and 12 after CAR-T cell infusion.
Absolute Peripheral Blood CD19+ B-Cell Count
Absolute CD19+ B-cell count in peripheral blood measured by flow cytometry at each prespecified assessment time point. Results will be reported in cells/µL.
Time frame: At screening, Day -1, Day 28, and Months 2, 3, 6, 9, and 12 after CAR-T cell infusion.
Absolute Peripheral Blood CD16+CD56+ Natural Killer Cell Count
Absolute CD16+CD56+ natural killer (NK) cell count in peripheral blood measured by flow cytometry using the protocol-specified gating strategy at each prespecified assessment time point. Results will be reported in cells/µL.
Time frame: At screening, Day -1, Day 28, and Months 2, 3, 6, 9, and 12 after CAR-T cell infusion.
Serum Immunoglobulin G (IgG) Concentration
Serum immunoglobulin G (IgG) concentration measured at each prespecified assessment time point. Results will be reported in mg/dL.
Time frame: At screening, Day -1, Day 28, and Months 2, 3, 6, 9, and 12 after CAR-T cell infusion.
Serum Immunoglobulin A (IgA) Concentration
Serum immunoglobulin A (IgA) concentration measured at each prespecified assessment time point. Results will be reported in mg/dL.
Time frame: At screening, Day -1, Day 28, and Months 2, 3, 6, 9, and 12 after CAR-T cell infusion.
Serum Immunoglobulin M (IgM) Concentration
Serum immunoglobulin M (IgM) concentration measured at each prespecified assessment time point. Results will be reported in mg/dL.
Time frame: At screening, Day -1, Day 28, and Months 2, 3, 6, 9, and 12 after CAR-T cell infusion.
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