Acute Myeloid Leukemia (AML)Phase 1ICG415-AML-01NCT07668557
Anti-CD33-CLL1 CAR-T Cells (ICG415) for the Treatment of Relapsed/Refractory Acute Myeloid Leukemia
A Phase 1 study testing Anti-CD33-CLL1 CAR-T cells (ICG415) following lymphodepleting fludarabine and cyclophosphamide in people with Acute Myeloid Leukemia (AML), run by iCell Gene Therapeutics at 2 sites in China.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Acute Myeloid Leukemia (AML)
- What is being tested?
- Anti-CD33-CLL1 CAR-T cells (ICG415) following lymphodepleting fludarabine and cyclophosphamide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 2 sitesChina
- Study dates
- 10 Jun 2026to 4 Jul 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 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Written informed consent approved by IRB/IEC obtained from subject or legally authorized representative prior to any screening procedures.
- Age ≥ 18 years and ≤ 70 years at the time of informed consent signing.
- 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.
- Bone marrow leukemic blasts positive for both CLL-1 and CD33 by flow cytometry.
- If circulating blasts are detectable at screening, tumor cell surface immunophenotype must be CD4 and CD8 double-negative by flow cytometry.
- ECOG performance status 0-2.
- Expected overall survival > 3 months.
- 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.
Who cannot join
- Prior receipt of CAR-T cell therapy or other genetically modified cell therapy prior to informed consent.
- 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.
- Active uncontrolled infection: positive HBsAg/HBV-DNA, active HCV-RNA positivity, HIV positive, positive syphilis antibody, active uncontrolled EBV or CMV viremia.
- Unstable severe systemic disease requiring continuous medication.
- Grade >2 bleeding within 30 days before screening or chronic long-term anticoagulant treatment.
- Uncontrolled life-threatening bacterial, fungal or viral infection.
Show all 15Show fewer
- Non-leukemic central nervous system organic disease or active CNS-2/CNS-3 leukemia; previously treated and resolved CNS leukemia is permitted.
- Concurrent other malignant tumor except cured in-situ carcinoma or malignancies with ≥5 years continuous complete remission.
- Live-attenuated vaccines within 30 days before screening or planned within 3 months after CAR-T infusion.
- Received any other investigational medicinal product within 3 months prior to ICF signature.
- Allogeneic hematopoietic stem cell transplantation within 6 months before screening.
- Pregnant or breastfeeding women.
- Suicidal tendency, ongoing alcohol or illicit drug dependence.
- Known hypersensitivity to investigational product, excipients or concomitant drugs.
- Any other condition judged inappropriate for trial entry by investigator.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
China2 sites, 2 recruiting
- Recruiting
Jiangxi Provincial People's Hospital (Participating Site)
Nanchang, Jiangxi, 330006
Cheng Hongbo, +86 137 0708 5405, 784260212@qq.com
- Recruiting
The First Affiliated Hospital of Nanchang University (Lead Site)
Nanchang, Jiangxi, 330006
Li Fei, +86 139 7003 8386, yx021021@sina.com
About this study
Official title: 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.
Read the 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.
- Conditions
- Acute Myeloid Leukemia (AML)
- Being tested
- Anti-CD33-CLL1 CAR-T cells (ICG415) following lymphodepleting fludarabine and cyclophosphamide
- Keywords
- Acute Myeloid Leukemia, AML, Leukemia, Acute Leukemia, Myeloid Leukemia, CAR-T, Cellular Therapy, CD33-CLL1 CAR-T, Relapsed or Refractory, R/R AML
- Medical subject headings
- Leukemia, Myeloid, Acute, Leukemia, Leukemia, Myeloid, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Anti-CD33-CLL1 CAR-T cells (ICG415) following lymphodepleting fludarabine and cyclophosphamideBiological
Anti-CD33, Anti-CLL1 Compound CAR-T Cells
| Study group | Role | What they receive |
|---|---|---|
| Single Arm, Anti-CD33-CLL1 CAR-T (ICG415) for Relapsed or Refractory AML | Experimental | Not described Receives: Biological: Anti-CD33-CLL1 CAR-T cells (ICG415) following lymphodepleting fludarabine and cyclophosphamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Dose-Limiting Toxicities (DLTs)
DLTs assessed according to the protocol-defined criteria.
Measured: Within 28 days after ICG415 CAR-T cell infusion
Incidence and Severity of Treatment-Emergent Adverse Events (TEAEs)
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.).
Measured: From first dose through 24 months post infusion
Secondary outcomes (11)
Objective Response Rate (ORR) at Months 1, 3, and 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.
Measured: Month 1, Month 3, Month 6
Rates of CR, CRi, and PR at Year 1 and 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.
Measured: Year 1, Year 2
Cumulative Incidence of Relapse (CIR) at Year 1 and Year 2
Cumulative incidence of disease relapse at 1 and 2 years after CAR-T cell infusion.
Measured: Year 1, Year 2
Duration of Response (DOR)
Time from first objective response (CR, CRi, or CRh) to relapse or death, whichever occurs first.
Measured: From first documented response to disease relapse or death from any cause, assessed up to 24 months
Progression-Free Survival (PFS)
Time from ICG415 infusion to disease progression (relapse or treatment failure) or death from any cause.
Measured: From date of infusion to disease progression or death from any cause, assessed up to 24 months
Overall Survival (OS)
Time from ICG415 infusion to death from any cause.
Measured: From date of infusion to death from any cause, assessed up to 24 months
Event-Free Survival (EFS)
Time from ICG415 infusion to any event including lack of response, disease relapse, or death from any cause.
Measured: From date of infusion to any treatment failure, relapse, or death, assessed up to 24 months
CAR-T Cell Kinetics - Persistence over Time
Number and duration of detectable CAR-T cells in peripheral blood. Testing stops after two consecutive negative results.
Measured: Days 0, 4, 7, 14, 21, 28; Months 2, 3, 6, 9, 12, 18, 24
Cytokine Level Changes
Changes in serum cytokine levels including IL-6, IL-10, IL-15, TNF-α, and IFN-γ.
Measured: Days 0, 7, 14, 21, 28; Months 2, 3, 6
Peripheral Blood Lymphocyte Subset Changes
Changes in lymphocyte subsets including T cells, B cells, NK cells, and CD4/CD8 ratio measured by flow cytometry.
Measured: Days 0, 7, 14, 21, 28; Months 2, 3, 6
Anti-Drug Antibody (ADA) Levels
Incidence and titers of anti-drug antibodies (ADA) against ICG415 CAR-T cells.
Measured: Day 28; Months 3, 6, 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jun 2026
- Study start10 Jun 2026
- Primary completion4 Jul 2028 (estimated)
- Study completion4 Jul 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
- Sequential
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- iCell Gene Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07668557
- Sponsor's protocol ID
- ICG415-001
- Acronym
- ICG415-AML-01
- Status last verified
- Jun 2026
- First submitted
- 19 Jun 2026
- Last updated
- 25 Jun 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 25 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Clinical Study to Evaluate the Safety and Efficacy of ICG415 CAR-T Cells in Adult Patients With Relapsed/Refractory Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT07668557
- Acronym / sponsor ID
- ICG415-AML-01 / ICG415-001
- Conditions
- Acute Myeloid Leukemia (AML)
- Keywords
- Acute Myeloid Leukemia, AML, Leukemia, Acute Leukemia, Myeloid Leukemia, CAR-T, Cellular Therapy, CD33-CLL1 CAR-T
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- iCell Gene TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 10 Jun 2026 (actual)
- Primary completion
- 4 Jul 2028 (estimated)
- Study completion
- 4 Jul 2029 (estimated)
- First posted
- 25 Jun 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 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.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single Arm, Anti-CD33-CLL1 CAR-T (ICG415) for Relapsed or Refractory AML | Experimental | Biological: Anti-CD33-CLL1 CAR-T cells (ICG415) following lymphodepleting fludarabine and cyclophosphamide |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Written informed consent approved by IRB/IEC obtained from subject or legally authorized representative prior to any screening procedures.
- Age ≥ 18 years and ≤ 70 years at the time of informed consent signing.
- 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.
- Bone marrow leukemic blasts positive for both CLL-1 and CD33 by flow cytometry.
- If circulating blasts are detectable at screening, tumor cell surface immunophenotype must be CD4 and CD8 double-negative by flow cytometry.
Key exclusion criteria (15 listed)
- Prior receipt of CAR-T cell therapy or other genetically modified cell therapy prior to informed consent.
- 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.
- Active uncontrolled infection: positive HBsAg/HBV-DNA, active HCV-RNA positivity, HIV positive, positive syphilis antibody, active uncontrolled EBV or CMV viremia.
- Unstable severe systemic disease requiring continuous medication.
- Grade >2 bleeding within 30 days before screening or chronic long-term anticoagulant treatment.
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 |
|---|---|---|
| Anti-CD33-CLL1 CAR-T cells (ICG415) following lymphodepleting fludarabine and cyclophosphamideAnti-CD33, Anti-CLL1 Compound CAR-T Cells | Biological | Single Arm, Anti-CD33-CLL1 CAR-T (ICG415) for Relapsed or Refractory AML |
Primary outcome
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.
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.).
11 secondary outcomes
Objective Response Rate (ORR) at Months 1, 3, and 6
Time frame: Month 1, Month 3, Month 6
Rates of CR, CRi, and PR at Year 1 and Year 2
Time frame: Year 1, Year 2
Cumulative Incidence of Relapse (CIR) at Year 1 and Year 2
Time frame: 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
Progression-Free Survival (PFS)
Time frame: 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
Event-Free Survival (EFS)
Time frame: 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
Cytokine Level Changes
Time frame: 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
Anti-Drug Antibody (ADA) Levels
Time frame: Day 28; Months 3, 6, 12
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 25 Jun 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.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Anti-CD33-CLL1 CAR-T cells (ICG415) following lymphodepleting fludarabine and cyclophosphamide in people with Acute Myeloid Leukemia (AML), run by iCell Gene Therapeutics at 2 sites 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 8 inclusion and 15 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: looking for participants now, as of the sponsor's last update on 25 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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 Yu Min (+86 150 7902 9006, 625668742@qq.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Dose-Limiting Toxicities (DLTs) (time frame: Within 28 days after ICG415 CAR-T cell infusion). The study lists 2 primary outcomes in all. 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 25 Jun 2026.