CLL-1-Targeted In Vivo CAR-T Cell Immunotherapy for Relapsed/Refractory Acute Myeloid Leukemia

NCT07841002 | Not Yet Recruiting Phase 1 DMC

• 1 other identifier: 2026-748
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief 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 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.

Conditions

Relapsed/Refractory Acute Myeloid Leukemia (AML); Acute Myeloid Leukemia (AML)

Keywords

in vivo CAR-T; CLL-1; acute myeloid leukemia; relapsed/refractory; lentiviral vector; dose escalation; 3+3; cytokine release syndrome; measurable residual disease

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  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

  At 1, 2, 3, 6, 9, 12, 18, and 24 months after SL1CC infusion

• Complete Response Rate

  At 1, 2, 3, 6, 9, 12, 18, and 24 months after SL1CC infusion

• Partial Remission (PR) Rate

  At 1, 2, 3, 6, 9, 12, 18, and 24 months after SL1CC infusion

• Overall Survival

  From SL1CC infusion through 24 months after treatment

• Progression-Free Survival

  From SL1CC infusion through 24 months after treatment

Study Arms (1)

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.

Drug: SL1CC Injection

Interventions

SL1CC Injection DRUG

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).

SL1CC Treatment Group

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Liping Dou: lead
• Hebei Senlang Biotechnology Inc., Ltd.: collaborator

Study Sites (1)

Chinese PLA General Hospital, Beijing, Beijing 100853

Beijing, China

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Recurrence; Cytokine Release Syndrome; Neoplasm, Residual

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Systemic Inflammatory Response Syndrome; Inflammation; Shock; Neoplastic Processes

Central Study Contacts

Ran Zhang

CONTACT

86-13810373716; lipingruirui@163.com

Li-Ping Dou, Dr

CONTACT

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Chief Physician, Professor, and Director of the Department of Hematology

Study Record Dates

• First Submitted: September 18, 2026
• First Posted: September 25, 2026
• Study Start: October 1, 2026
• Primary Completion (Estimated): October 31, 2028
• Study Completion (Estimated): September 30, 2030
• Last Updated: October 2, 2026

Record last verified: 2026-10

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)