Relapsed/Refractory Acute Myeloid Leukemia (AML)Phase 1NCT07841002
CLL-1-Targeted In Vivo CAR-T Cell Immunotherapy for Relapsed/Refractory Acute Myeloid Leukemia
A Phase 1 study testing SL1CC Injection in people with Relapsed/Refractory Acute Myeloid Leukemia (AML) and Acute Myeloid Leukemia (AML), run by Liping Dou at 1 site in China.
Trial summary
This is a single-center, open-label, single-arm, phase 1 dose-escalation study to evaluate the safety, tolerability, preliminary efficacy, and cellular kinetics of SL1CC Injection, an in vivo CAR-T therapy targeting CLL-1, in patients with relapsed or refractory acute myeloid leukemia (R/R AML; acute promyelocytic… Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Relapsed/Refractory Acute Myeloid Leukemia (AML), Acute Myeloid Leukemia (AML)
- What is being tested?
- SL1CC 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
- 1 Oct 2026to 30 Sep 2030
- Phase
- Phase 1
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 to 75 years
- 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 relapsed or refractory acute myeloid leukemia (AML), excluding acute promyelocytic leukemia (APL), according to the 2022 World Health Organization (WHO) classification, 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 two courses 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.
- CLL-1 expression ≥50% on AML blasts, confirmed by flow cytometry on bone marrow or peripheral blood samples at the local certified laboratory.
- Patients with targetable mutations (e.g., FLT3, IDH1/2, NPM1, or KMT2A rearrangement) must have received, be intolerant to, or be ineligible for the corresponding approved targeted therapy.
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- Recovered from acute toxicities of prior antileukemic therapy to ≤ Grade 1 (CTCAE v6.0) before SL1CC infusion, except for alopecia and hematologic abnormalities attributable to the underlying disease.
- Male or female participants aged 18 to 75 years, inclusive.
- 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.
- Adequate renal, hepatic, cardiac, and pulmonary function, defined as follows:
- Creatinine clearance (estimated by the Cockcroft-Gault formula) ≥60 mL/min or serum creatinine ≤1.5 × the upper limit of normal (ULN);
- Serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST)≤2.5×upper limit of normal (ULN), total bilirubin≤1.5×ULN;
- Cardiac ejection fraction≥50%, no pericardial effusion on echocardiography, and no significant abnormalities on electrocardiogram (ECG);
- No clinically significant pleural effusion, and oxygen saturation >92% on room air at baseline.
- Participants of reproductive potential must agree to use highly effective contraception before enrollment and for at least 12 months after SL1CC infusion. A negative serum pregnancy test is required for women of childbearing potential at screening. Participants must immediately notify the investigator if pregnancy occurs or is suspected.
Who cannot join
- The patient has severe cardiac dysfunction, or left ventricular ejection fraction (LVEF) <50%, or a history within the 12 months prior to enrollment of myocardial infarction, coronary angioplasty or coronary stent implantation, unstable angina, clinically significant arrhythmia, or other severe cardiovascular diseases.
- A history of severe pulmonary disease associated 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 B or hepatitis C infection, 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; has received a live vaccine within 4 weeks, or is anticipated to require a live vaccine during the study period.
- A history of severe allergic reactions to biological products, including antibiotics.
- Prior allogeneic hematopoietic stem cell transplantation with persistent acute graft-versus-host disease (GVHD) after discontinuation of immunosuppressive therapy for at least 1 month.
- Prior treatment with any gene therapy product or any in vivo CAR-T therapy, or known pre-existing neutralizing immunity to the lentiviral vector.
- Active central nervous system (CNS) leukemia or leptomeningeal involvement not controlled after adequate intrathecal therapy; cerebrospinal fluid examination is required during screening.
- The patient has severe autoimmune disease, congenital immunodeficiency or active autoimmune disease requiring ongoing systemic immunosuppressive treatment, or is currently within the washout period after having received medications that may affect CAR-T cell immunotherapy, or is anticipated to require medications during the study period that may affect CAR-T cell immunotherapy treatment, or active GVHD requiring systemic therapy.
- Pregnancy or breastfeeding (lactation).
- The patient has other significant uncontrolled comorbidities that may affect protocol compliance or interpretation of results.
- The investigator judges that the patient is unlikely to complete all visits and procedures required by the study protocol (including medium- and long-term follow-up visits), such as insufficient willingness of the patient and their family members to participate in the study, refusal to participate, inability of the patient to fully cooperate with the study arrangements, or inadequate compliance on the part of the patient and their family members.
- 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.
Li-Ping Dou, Dr contact
About this study
Official title: A Clinical Study on the Safety of CLL-1-Targeted In Vivo CAR-T-Cell Immunotherapy for Relapsed/Refractory Acute Myeloid Leukemia
This is a single-center, open-label, single-arm, phase 1 dose-escalation study to evaluate the safety, tolerability, preliminary efficacy, and cellular kinetics of SL1CC Injection, an in vivo CAR-T therapy targeting CLL-1, in patients with relapsed or refractory acute myeloid leukemia (R/R AML; acute promyelocytic leukemia excluded). SL1CC Injection is administered as a single intravenous infusion without lymphodepleting conditioning. Dose escalation follows a traditional 3+3 design with planned dose levels of 1 × 10^9, 3 × 10^9, and 6 × 10^9 transducing units (TU), guided by the occurrence of dose-limiting toxicities (DLTs). Treatment-emergent adverse events and serious adverse events, including cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), hematologic toxicity, organ toxicity, and other immune therapy-related toxicities, will be assessed. Preliminary antitumor activity, assessed by composite complete remission (CR/CRi) and measurable residual disease (MRD) status according to the European LeukemiaNet (ELN) 2022 criteria, and the expansion and persistence of CAR-T cells in peripheral blood will also be evaluated.
Read the detailed description
This is a single-center, single-arm, open-label, phase 1 dose-escalation study of SL1CC Injection in adults with R/R AML. SL1CC is an in vivo CAR-T product in which a lentiviral vector (LVV) encoding a CLL-1-specific chimeric antigen receptor is administered intravenously to transduce the patient's endogenous T cells in situ; no leukapheresis, ex vivo manufacturing, or lymphodepleting conditioning is required. Three dose levels are planned (1 × 10^9, 3 × 10^9, and 6 × 10^9 TU) using a traditional 3+3 escalation design, and dose-limiting toxicity (DLT) is assessed during the first 28 days after a single infusion. Anticipated enrollment is 12 participants (range 9-18, depending on the number of dose levels requiring expansion). The study includes a screening period, a treatment (infusion) period, and a follow-up period of up to 24 months. Safety assessments include adverse events and serious adverse events graded per NCI CTCAE v6.0, with CRS and ICANS graded per the ASTCT consensus criteria; hematologic recovery, infection, organ toxicity, and viral shedding (blood, saliva, and urine; qPCR) are also monitored. Disease response is assessed by bone marrow morphology according to the ELN 2022 criteria (CR, CRi, MLFS, and PR), together with MRD measured by multiparameter flow cytometry. Exploratory cellular kinetics include peripheral blood CAR transgene copies (qPCR), CAR-positive T-cell counts, Cmax, Tmax, and AUC0-28d. Long-term safety monitoring for potential insertional mutagenesis associated with integrating lentiviral vectors is planned in accordance with applicable gene therapy guidance.
- Being tested
- SL1CC Injection
- Keywords
- in vivo CAR-T, CLL-1, acute myeloid leukemia, relapsed/refractory, 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.
SL1CC InjectionDrug
SL1CC Injection is an investigational in vivo CLL-1-targeted CAR T-cell therapy for patients with relapsed or refractory acute myeloid leukemia. Participants will receive SL1CC Injection by intravenous infusion at protocol-specified dose levels. Dose escalation will follow a traditional 3+3 design and will be guided by the occurrence of dose-limiting toxicities (DLTs).
| Study group | Role | What they receive |
|---|---|---|
| SL1CC Treatment Group | Experimental | single intravenous infusion of SL1CC Injection at one of three planned dose levels (1 × 10^9, 3 × 10^9, or 6 × 10^9 TU) assigned by a traditional 3+3 dose-escalation design; no lymphodepleting conditioning is administered. Additional dose cohorts can be added based on safety. Receives: Drug: SL1CC Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and Severity of Adverse Events and Serious Adverse Events
The number, percentage, severity, and relatedness of adverse events (AEs) and serious adverse events (SAEs) occurring from SL1CC infusion through the 12-month safety observation period, including cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), hematologic toxicity, organ toxicity, infection, and other immunotherapy-related toxicities. AEs are graded per NCI CTCAE v6.0; CRS and ICANS are graded per the ASTCT consensus criteria.
Measured: From SL1CC infusion through 12 months after treatment (primary safety observation period); long-term follow-up continues through 24 months
Incidence of Dose-Limiting Toxicities (DLTs)
The number and percentage of participants who experience dose-limiting toxicities (DLTs) during the protocol-defined DLT observation period (Days 0-28) after a single intravenous infusion of SL1CC Injection. DLT definitions are specified in the protocol.
Measured: From Day 0 through Day 28 after a single SL1CC infusion (protocol-defined DLT observation window)
Secondary outcomes (12)
Objective Response Rate at Prespecified Follow-up Time Points
The objective response rate (ORR) at 1, 2, 3, 6, 9, 12, 18, and 24 months after treatment, defined as the proportion of participants who achieve a complete remission (CR), CR with incomplete hematologic recovery (CRi), morphologic leukemia-free state (MLFS), or partial remission (PR), as defined by the European LeukemiaNet (ELN) 2022 recommendations for AML.
Measured: At 1, 2, 3, 6, 9, 12, 18, and 24 months after SL1CC infusion
Complete Response Rate
The proportion of participants who achieve composite complete remission (CRc; CR + CRi) at each prespecified assessment time point, per the ELN 2022 criteria.
Measured: At 1, 2, 3, 6, 9, 12, 18, and 24 months after SL1CC infusion
Partial Remission (PR) Rate
The proportion of participants who achieve partial remission (PR) at each prespecified assessment time point, per the ELN 2022 criteria.
Measured: At 1, 2, 3, 6, 9, 12, 18, and 24 months after SL1CC infusion
Overall Survival
Overall survival (OS), defined as the time from SL1CC infusion to death from any cause. Participants who are alive at the last follow-up will be censored on the date of their last known survival status.
Measured: From SL1CC infusion through 24 months after treatment
Progression-Free Survival
Progression-free survival (PFS), defined as the time from SL1CC infusion to relapse after CR/CRi, disease progression, or death from any cause, whichever occurs first, using the ELN 2022 criteria for relapse and progressive disease. Participants without an event will be censored at the date of the last disease assessment.
Measured: From SL1CC infusion through 24 months after treatment
Event-Free Survival
Event-free survival (EFS), defined as the time from SL1CC infusion to the occurrence of a protocol-defined event, including failure to achieve CR/CRi at the protocol-defined response assessment, relapse after CR/CRi, initiation of new anti-leukemia therapy, or death from any cause, whichever occurs first, as defined by the ELN 2022 criteria. Participants without an event will be censored at the date of their last follow-up.
Measured: From SL1CC infusion through 24 months after treatment
Measurable Residual Disease (MRD) Negativity Rate
The proportion of participants with MRD negativity in bone marrow assessed by multiparameter flow cytometry (sensitivity of at least 10^-4) among participants who achieve CR/CRi, at each prespecified assessment time point.
Measured: At 1, 2, 3, 6, 9, 12, 18, and 24 months after SL1CC infusion
CAR Transgene Copy Number in Peripheral Blood (qPCR)
CAR transgene copy number in peripheral blood measured by quantitative PCR (qPCR)
Measured: From Day 0 through 24 months after SL1CC infusion
CAR-Positive T-Cell Counts in Peripheral Blood (Flow Cytometry)
Number of CAR-positive T cells in peripheral blood measured by flow cytometry
Measured: From Day 0 through 24 months after SL1CC infusion
Peak Expansion of CAR-T Cells (Cmax)
Peak level of CAR transgene expansion in peripheral blood
Measured: From Day 0 through Day 28 after SL1CC infusion
Time to Peak Expansion (Tmax)
Time from infusion to peak CAR transgene expansion
Measured: From Day 0 through Day 28 after SL1CC infusion
AUC from Day 0 to Day 28 (AUC0-28d)
Area under the curve of peripheral blood CAR transgene levels from Day 0 to Day 28
Measured: From Day 0 through Day 28 after SL1CC infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Sep 2026
- Study start1 Oct 2026 (estimated)
- Primary completion31 Oct 2028 (estimated)
- Study completion30 Sep 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Relapsed/Refractory 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
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Liping Dou
- Sponsor type
- University, hospital or other
- Collaborators
- Hebei Senlang Biotechnology Inc., Ltd.
- Responsible party
- Sponsor-investigator, Liping Dou, Chinese PLA General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07841002
- Sponsor's protocol ID
- 2026-748
- Status last verified
- Oct 2026
- First submitted
- 18 Sep 2026
- Last updated
- 2 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No