Acute Myeloid Leukemia (AML)Phase 1NCT07897136
CLL-1/CD70 Dual-Target CAR-T Cell Therapy for Acute Myeloid Leukemia
A Phase 1 study testing CLL-1-CD70 CAR-T Cell Injection in people with Acute Myeloid Leukemia (AML) and Relapsed/Refractory Acute Myeloid Leukemia, run by Liping Dou at 1 site in China.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia (AML), Relapsed/Refractory Acute Myeloid Leukemia
- What is being tested?
- CLL-1-CD70 CAR-T Cell Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteChina
- Study dates
- 30 Nov 2026to 30 Nov 2029
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Voluntarily agrees to participate in the study, has signed the informed consent form, and is willing and able to comply with scheduled visits, study treatment, laboratory tests, and other study procedures.
- Diagnosis of acute myeloid leukemia (AML), excluding acute promyelocytic leukemia (APL), according to the 2022 World Health Organization (WHO) classification; target testing shows CLL-1 expression >=50% and/or CD70 expression >=50%.
- Diagnosis of AML meeting at least one of the following:
- Relapsed AML: reappearance of leukemic cells in the peripheral blood after achieving complete remission (CR), bone marrow blasts >=5% (excluding bone marrow regeneration after consolidation chemotherapy or other non-leukemic causes), or the presence of extramedullary leukemic infiltration.
- Refractory AML: newly diagnosed AML with no response after one course of standard induction therapy; relapse within 12 months after achieving CR followed by consolidation/intensification therapy; relapse more than 12 months after CR with failure to respond to conventional chemotherapy; two or more relapses; or persistent extramedullary leukemia.
- Male or female participants aged 18 years or older.
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- Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
- Estimated life expectancy of more than 3 months from the date of signing the informed consent form.
- Hemoglobin (HGB) >=60 g/L (transfusion permitted).
- Adequate renal, hepatic, cardiac, and pulmonary function, defined as follows:
- Serum creatinine <=1.5 x the upper limit of normal (ULN);
- Left ventricular ejection fraction (LVEF) >=50%;
- Oxygen saturation >90% on room air;
- Total bilirubin <=1.5 x ULN; alanine aminotransferase (ALT) and aspartate aminotransferase (AST) <=2.5 x ULN;
- No clinically significant pleural effusion, and oxygen saturation >92% on room air at baseline.
- Participants with reproductive plans must agree to use contraception before enrollment and for 6 months during/after the study. Participants must immediately notify the investigator if pregnancy occurs or is suspected.
Who cannot join
- Severe cardiac dysfunction, or left ventricular ejection fraction (LVEF) <50%.
- A history of severe pulmonary disease with impaired lung function.
- Concurrent progressive malignancy other than acute myeloid leukemia.
- Severe active infection that cannot be effectively controlled.
- Severe autoimmune disease or congenital immunodeficiency.
- Active hepatitis, defined as hepatitis B virus DNA (HBV-DNA) or hepatitis C virus RNA (HCV-RNA) levels above the lower limit of detection.
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- Human immunodeficiency virus (HIV) infection, known acquired immunodeficiency syndrome (AIDS), or syphilis infection.
- A history of severe allergic reactions to biological products, including antibiotics.
- Any other severe physical or psychiatric disorder or clinically significant laboratory abnormality that may increase the risk associated with study participation, interfere with the interpretation of study results, or, in the investigator's judgment, make the participant unsuitable for participation in the study.
- Note:
- Severe infection is defined as sepsis or an infection with an uncontrolled infectious focus. Participants may be enrolled after the infection has been adequately controlled.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
Chinese PLA General Hospital
Beijing, Beijing Municipality, 100853
Liping Dou, Dr., 86-010-66937232, lipingruirui@163.com
About this study
Official title: A Clinical Study on the Safety of Bispecific CLL-1-CD70 Dual-Target CAR-T Therapy for Acute Myeloid Leukemia
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.
Read the detailed description
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.
- Being tested
- CLL-1-CD70 CAR-T Cell Injection
- Keywords
- CLL-1, CD70, dual-target CAR-T, acute myeloid leukemia, relapsed/refractory, consolidation, lentiviral vector, dose escalation, 3+3, cytokine release syndrome, measurable, residual disease
- Medical subject headings
- Leukemia, Myeloid, Acute, Recurrence, Cytokine Release Syndrome, Neoplasm, Residual
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CLL-1-CD70 CAR-T Cell InjectionDrug
Also called CLL1-CD70 dual-target CAR-T, autologous 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).
| Study group | Role | What they receive |
|---|---|---|
| CLL-1-CD70 CAR-T Treatment Group | Experimental | 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. Receives: Drug: CLL-1-CD70 CAR-T Cell Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From CAR-T cell infusion through 12 months after infusion.
Secondary outcomes (24)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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).
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: At screening, Day -1, Day 28, and Months 2, 3, 6, 9, and 12 after CAR-T cell infusion.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2026
- Study start30 Nov 2026 (estimated)
- Primary completion30 Nov 2028 (estimated)
- Study completion30 Nov 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid Leukemia (AML)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Single-arm dose-escalation study using a traditional 3+3 design. All 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). Dose escalation is guided by dose-limiting toxicities (DLTs) assessed during Days 1-28 after infusion.
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Liping Dou
- Sponsor type
- University, hospital or other
- Collaborators
- Hebei Taihe Chunyu Biotechnology Co., Ltd
- Responsible party
- Sponsor-investigator, Liping Dou, Chinese PLA General Hospital
- Investigators
- Liping Dou, Dr., Principal investigator, Chinese PLA General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07897136
- Sponsor's protocol ID
- Th-Z030
- Status last verified
- Oct 2026
- First submitted
- 13 Oct 2026
- Last updated
- 27 Oct 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Clinical Study on the Safety of Bispecific CLL-1-CD70 Dual-Target CAR-T Therapy for Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT07897136
- Acronym / sponsor ID
- Th-Z030
- Keywords
- CLL-1, CD70, dual-target CAR-T, acute myeloid leukemia, relapsed/refractory, consolidation, lentiviral vector, dose escalation
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Liping DouUniversity, hospital or other
- Collaborators
- Hebei Taihe Chunyu Biotechnology Co., Ltd
- Study start
- 30 Nov 2026 (estimated)
- Primary completion
- 30 Nov 2028 (estimated)
- Study completion
- 30 Nov 2029 (estimated)
- First posted
- 27 Oct 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Single-arm dose-escalation study using a traditional 3+3 design. All 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). Dose escalation is guided by dose-limiting toxicities (DLTs) assessed during Days 1-28 after infusion.
| Arm | Type | Receives |
|---|---|---|
| CLL-1-CD70 CAR-T Treatment Group | Experimental | Drug: CLL-1-CD70 CAR-T Cell InjectionAfter 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- Voluntarily agrees to participate in the study, has signed the informed consent form, and is willing and able to comply with scheduled visits, study treatment, laboratory tests, and other study procedures.
- Diagnosis of acute myeloid leukemia (AML), excluding acute promyelocytic leukemia (APL), according to the 2022 World Health Organization (WHO) classification; target testing shows CLL-1 expression >=50% and/or CD70 expression >=50%.
- Diagnosis of AML meeting at least one of the following:
- Male or female participants aged 18 years or older.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
Key exclusion criteria (11 listed)
- Severe cardiac dysfunction, or left ventricular ejection fraction (LVEF) <50%.
- A history of severe pulmonary disease with impaired lung function.
- Concurrent progressive malignancy other than acute myeloid leukemia.
- Severe active infection that cannot be effectively controlled.
- Severe autoimmune disease or congenital immunodeficiency.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CLL-1-CD70 CAR-T Cell InjectionAlso: CLL1-CD70 dual-target CAR-T, autologous CLL-1/CD70 CAR-T cell injectionCLL-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). | Drug | CLL-1-CD70 CAR-T Treatment Group |
Primary outcome
Safety: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time frame: From CAR-T cell infusion through 12 months after infusion.
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.
24 secondary outcomes
Composite Complete Remission Rate (CRc)
Time frame: At Months 1, 3, 6, 9, and 12 after CAR-T cell infusion.
Complete Remission (CR) Rate
Time frame: At Months 1, 3, 6, 9, and 12 after CAR-T cell infusion.
Complete Remission With Partial Hematologic Recovery (CRh) Rate
Time frame: At Months 1, 3, 6, 9, and 12 after CAR-T cell infusion.
Complete Remission With Incomplete Hematologic Recovery (CRi) Rate
Time frame: At Months 1, 3, 6, 9, and 12 after CAR-T cell infusion.
No Response (NR) Rate
Time frame: At Months 1, 3, 6, 9, and 12 after CAR-T cell infusion.
Partial Remission (PR) Rate
Time frame: At Months 1, 3, 6, 9, and 12 after CAR-T cell infusion.
Morphologic Leukemia-Free State (MLFS) Rate
Time frame: At Months 1, 3, 6, 9, and 12 after CAR-T cell infusion.
Overall Survival (OS)
Time frame: From CAR-T cell infusion through 12 months after infusion.
Event-Free Survival (EFS)
Time frame: From CAR-T cell infusion through 12 months after infusion.
Duration of Response (DOR)
Time frame: From the first documented response through 12 months after CAR-T cell infusion.
Cumulative Incidence of Relapse (CIR)
Time frame: From the first documented remission through 12 months after CAR-T cell infusion.
Peripheral Blood CAR Transgene Copy Number
Time frame: From CAR-T cell infusion through 12 months after infusion.
Maximum Observed Peripheral Blood CAR Transgene Copy Number (Cmax)
Time frame: From CAR-T cell infusion through Day 28.
Time to Maximum Observed Peripheral Blood CAR Transgene Copy Number (Tmax)
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)
Time frame: From CAR-T cell infusion through Day 28.
Measurable Residual Disease (MRD) Negativity Rate (Exploratory)
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
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
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
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
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
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
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
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
Time frame: At screening, Day -1, Day 28, and Months 2, 3, 6, 9, and 12 after CAR-T cell infusion.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Liping Dou, Dr. principal investigator
Chinese PLA General Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CLL-1-CD70 CAR-T Cell Injection in people with Acute Myeloid Leukemia (AML) and Relapsed/Refractory Acute Myeloid Leukemia, run by Liping Dou at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 27 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Meng Li, Dr. (+86-13552095979, lipingruirui@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) (time frame: From CAR-T cell infusion through 12 months after infusion.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Oct 2026.