Anti-CD33-CLL1 CAR-T Cells (ICG415) for the Treatment of Relapsed/Refractory Acute Myeloid Leukemia (ICG415-AML-01)

June 19, 2026 updated by: iCell Gene Therapeutics

A Clinical Study to Evaluate the Safety and Efficacy of ICG415 CAR-T Cells in Adult Patients With Relapsed/Refractory Acute Myeloid Leukemia

This single-arm, open-label phase I trial evaluates the safety and tolerability of ICG415, autologous CAR-T cells targeting CD33 and CLL1, in patients with relapsed or refractory acute myeloid leukemia (AML). Subjects receive lymphodepleting chemotherapy followed by autologous CAR-T infusion. The primary goal is to assess safety and preliminary anti-leukemic efficacy in patients failing standard AML therapies.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Interventional

Enrollment (Estimated)

18

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • China
    • Jiangxi
      • Nanchang, Jiangxi, China, 330006
        • Recruiting
        • Jiangxi Provincial People's Hospital (Participating Site)
        • Contact:
      • Nanchang, Jiangxi, China, 330006
        • Recruiting
        • The First Affiliated Hospital of Nanchang University (Lead Site)
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Written informed consent approved by IRB/IEC obtained from subject or legally authorized representative prior to any screening procedures.
  2. Age ≥ 18 years and ≤ 70 years at the time of informed consent signing.
  3. 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.
  4. Bone marrow leukemic blasts positive for both CLL-1 and CD33 by flow cytometry.
  5. If circulating blasts are detectable at screening, tumor cell surface immunophenotype must be CD4 and CD8 double-negative by flow cytometry.
  6. ECOG performance status 0-2.
  7. Expected overall survival > 3 months.
  8. 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:

  1. Prior receipt of CAR-T cell therapy or other genetically modified cell therapy prior to informed consent.
  2. 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.
  3. Active uncontrolled infection: positive HBsAg/HBV-DNA, active HCV-RNA positivity, HIV positive, positive syphilis antibody, active uncontrolled EBV or CMV viremia.
  4. Unstable severe systemic disease requiring continuous medication.
  5. Grade >2 bleeding within 30 days before screening or chronic long-term anticoagulant treatment.
  6. Uncontrolled life-threatening bacterial, fungal or viral infection.
  7. Non-leukemic central nervous system organic disease or active CNS-2/CNS-3 leukemia; previously treated and resolved CNS leukemia is permitted.
  8. Concurrent other malignant tumor except cured in-situ carcinoma or malignancies with ≥5 years continuous complete remission.
  9. Live-attenuated vaccines within 30 days before screening or planned within 3 months after CAR-T infusion.
  10. Received any other investigational medicinal product within 3 months prior to ICF signature.
  11. Allogeneic hematopoietic stem cell transplantation within 6 months before screening.
  12. Pregnant or breastfeeding women.
  13. Suicidal tendency, ongoing alcohol or illicit drug dependence.
  14. Known hypersensitivity to investigational product, excipients or concomitant drugs.
  15. Any other condition judged inappropriate for trial entry by investigator.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Single Arm, Anti-CD33-CLL1 CAR-T (ICG415) for Relapsed or Refractory AML
Biological: Anti-CD33-CLL1 CAR-T cells (ICG415) following lymphodepleting fludarabine and cyclophosphamide
Anti-CD33, Anti-CLL1 Compound CAR-T Cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of Dose-Limiting Toxicities (DLTs)
Time Frame: Within 28 days after ICG415 CAR-T cell infusion
DLTs assessed according to the protocol-defined criteria.
Within 28 days after ICG415 CAR-T cell infusion
Incidence and Severity of Treatment-Emergent Adverse Events (TEAEs)
Time Frame: From first dose through 24 months post infusion
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.).
From first dose through 24 months post infusion

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Objective Response Rate (ORR) at Months 1, 3, and 6
Time Frame: Month 1, Month 3, Month 6
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.
Month 1, Month 3, Month 6
Rates of CR, CRi, and PR at Year 1 and Year 2
Time Frame: Year 1, Year 2
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.
Year 1, Year 2
Cumulative Incidence of Relapse (CIR) at Year 1 and Year 2
Time Frame: Year 1, Year 2
Cumulative incidence of disease relapse at 1 and 2 years after CAR-T cell infusion.
Year 1, Year 2
Duration of Response (DOR)
Time Frame: From first documented response to disease relapse or death from any cause, assessed up to 24 months
Time from first objective response (CR, CRi, or CRh) to relapse or death, whichever occurs first.
From first documented response to disease relapse or death from any cause, assessed up to 24 months
Progression-Free Survival (PFS)
Time Frame: From date of infusion to disease progression or death from any cause, assessed up to 24 months
Time from ICG415 infusion to disease progression (relapse or treatment failure) or death from any cause.
From date of infusion to disease progression or death from any cause, assessed up to 24 months
Overall Survival (OS)
Time Frame: From date of infusion to death from any cause, assessed up to 24 months
Time from ICG415 infusion to death from any cause.
From date of infusion to death from any cause, assessed up to 24 months
Event-Free Survival (EFS)
Time Frame: From date of infusion to any treatment failure, relapse, or death, assessed up to 24 months
Time from ICG415 infusion to any event including lack of response, disease relapse, or death from any cause.
From date of infusion to any treatment failure, relapse, or death, assessed up to 24 months
CAR-T Cell Kinetics - Persistence over Time
Time Frame: Days 0, 4, 7, 14, 21, 28; Months 2, 3, 6, 9, 12, 18, 24
Number and duration of detectable CAR-T cells in peripheral blood. Testing stops after two consecutive negative results.
Days 0, 4, 7, 14, 21, 28; Months 2, 3, 6, 9, 12, 18, 24
Cytokine Level Changes
Time Frame: Days 0, 7, 14, 21, 28; Months 2, 3, 6
Changes in serum cytokine levels including IL-6, IL-10, IL-15, TNF-α, and IFN-γ.
Days 0, 7, 14, 21, 28; Months 2, 3, 6
Peripheral Blood Lymphocyte Subset Changes
Time Frame: Days 0, 7, 14, 21, 28; Months 2, 3, 6
Changes in lymphocyte subsets including T cells, B cells, NK cells, and CD4/CD8 ratio measured by flow cytometry.
Days 0, 7, 14, 21, 28; Months 2, 3, 6
Anti-Drug Antibody (ADA) Levels
Time Frame: Day 28; Months 3, 6, 12
Incidence and titers of anti-drug antibodies (ADA) against ICG415 CAR-T cells.
Day 28; Months 3, 6, 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

iCell Gene Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 10, 2026

Primary Completion (Estimated)

July 4, 2028

Study Completion (Estimated)

July 4, 2029

Study Registration Dates

First Submitted

June 19, 2026

First Submitted That Met QC Criteria

June 19, 2026

First Posted (Actual)

June 25, 2026

Study Record Updates

Last Update Posted (Actual)

June 25, 2026

Last Update Submitted That Met QC Criteria

June 19, 2026

Last Verified

June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ICG415-001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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