CAR T Cell TherapyPhase 1NCT07538713
Functionally Optimized CD33 CAR-T Cell Therapy Targeting Recurrent/Refractory Acute Myeloid Leukemia
A Phase 1 study testing Functionally optimized CD33 CAR-T in people with CAR T Cell Therapy and CD33 Positive Acute Myelogenous Leukemia, run by Qi deng.
Trial summary
Relapsed/refractory acute myeloid leukemia (R/R AML) currently lacks effective CAR-T therapeutic agents due to the absence of tumor-specific target antigens. Most AML-associated antigens are expressed on normal hematopoietic stem/progenitor cells (HSPCs) and healthy tissues, increasing the risk of on-target off-tumor… Read more about this study
- Who is it for?
- Ages 14 to 75 years
- Conditions the study is for
- CAR T Cell Therapy, CD33 Positive Acute Myelogenous Leukemia
- What is being tested?
- Functionally optimized CD33 CAR-T
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- No sites listed yet
- Study dates
- 1 Jun 2026to 31 May 2028
- 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 14 to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subjects diagnosed with refractory/recurrent acute myeloid leukemia (excluding M3) who meet any of the following criteria:
- Relapse: Recurrence of leukemia cells in peripheral blood or ≥5% blast cells in bone marrow after complete remission (CR) of AML (excluding other causes such as bone marrow regeneration following consolidation chemotherapy), or extramedullary leukemia infiltration.
- Refractory: First-time cases unresponsive to two cycles of standard therapy; relapse within 12 months after consolidation therapy following CR; relapse after 12 months without response to conventional chemotherapy; two or more relapses; persistent extramedullary leukemia.
- During enrollment screening, bone marrow flow cytometry must demonstrate a CD33+ expression rate of ≥80% in leukemia cells and/or pathological immunohistochemical confirmation of CD33+ extramedullary lesions.
- Estimated survival duration exceeding 3 months as of the date of informed consent signing.
- Participants with Eastern Cooperative Oncology Group (ECOG) physical status scores ranging from 0 to 2.
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- Age range of 14 years ≤ ≤ 75 years, inclusive, with no gender restriction.
- Hemoglobin (HGB) level ≥70 g/L with transfusion capability.
- Liver/kidney function and cardiopulmonary function meeting the following criteria:
- Creatinine ≤1.5×ULN;
- Left ventricular ejection fraction ≥50%;
- Blood oxygen saturation>90%;
- Total bilirubin ≤1.5×ULN; ALT and AST ≤2.5×ULN.
- Acceptance of autologous CART cells with peripheral blood tumor burden ≤ 30%;
- The subject or guardian understands and signs the informed consent form.
Who cannot join
- Presence of one of the following cardiac criteria:
- Atrial fibrillation;
- Myocardial infarction (MI) within the past 12 months;
- Prolonged QT syndrome or secondary QT prolongation as determined by the investigator;
- Echocardiographic left ventricular systolic fraction (LVSF) <30% or left ventricular ejection fraction (LVEF) <50%;
- Clinically significant pericardial effusion; New York Heart Association (NYHA) class III or IV heart failure (confirmed by echocardiography within 12 months after treatment).
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- Active graft-versus-host disease (GVHD).
- History of severe pulmonary dysfunction.
- Concurrent other progressive malignancies.
- Concurrent severe or persistent infections that cannot be effectively controlled.
- Concurrent severe autoimmune diseases or congenital immunodeficiency.
- Active hepatitis (HBV-DNA ≥ 500 IU/ml with abnormal liver function or HCV antibody [HCV-Ab] positivity, HCV-RNA exceeding the detection limit of analytical methods with abnormal liver function).
- Human immunodeficiency virus (HIV) infection or syphilis infection.
- History of severe allergic reactions to biological products (including antibiotics).
- Presence of central nervous system disorders such as uncontrolled epilepsy, cerebrovascular ischemia/hemorrhage, dementia, or cerebellar diseases.
- Female patients in pregnancy or lactation, or planning pregnancy within 12 months.
- Situations where investigators consider may increase subject risk or interfere with trial outcomes.
About this study
Official title: Clinical Study on the Efficacy and Safety of Functionally Optimized CD33 CAR-T Cells (FO33 CAR-T) Therapy Targeting CD33-Positive Recurrent/Refractory Acute Myeloid Leukemia
Relapsed/refractory acute myeloid leukemia (R/R AML) currently lacks effective CAR-T therapeutic agents due to the absence of tumor-specific target antigens. Most AML-associated antigens are expressed on normal hematopoietic stem/progenitor cells (HSPCs) and healthy tissues, increasing the risk of on-target off-tumor toxicity and non-neoplastic toxicity. CD33 is present on leukemic cells in over 80% of AML patients. Compared with CLL-1, CD123 and other targets, CD33 exhibits higher expression across diverse AML subtypes, reducing the risk of treatment failure and relapse caused by antigen escape and thus serving as an ideal therapeutic target for AML. However, conventional CD33-targeted CAR-T cells demonstrate suboptimal efficacy in clinical trials, accompanied by significant toxicity and inadequate in vivo expansion. To further investigate the safety and efficacy of CAR-T therapy for AML, our center has initiated a clinical trial of functionally optimized CD33 CAR-T (FO33 CAR-T) cells for R/R AML. We constructed a lentiviral CAR vector containing the CD33-targeting scFv, 4-1BB, and CD3ζ, followed by insertion of adjuvant molecule X. FO33 CAR-T cells showed superior cytotoxicity against AML cell lines and enhanced biological activity compared with conventional CD33 CAR-T cells, and exerted safe and effective antitumor effects in preclinical models. This single-center, open-label, prospective clinical trial aims to evaluate the safety and efficacy of FO33 CAR-T cells in patients with R/R AML, as well as to characterize the pharmacokinetic and pharmacodynamic (PK/PD) profiles of this therapy.
- Being tested
- Functionally optimized CD33 CAR-T
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Functionally optimized CD33 CAR-TBiological
Functionally optimized CD33 CAR-T intravenous infusion
| Study group | Role | What they receive |
|---|---|---|
| Functionally optimized CD33 CAR-T | Experimental | Based on previously reported clinical data regarding the safety and efficacy of CAR-T cell infusion in AML trials, as well as ethical considerations for benefit-risk assessment aimed at protecting subject safety, the initial infusion doses in this trial were set as follows: Dose 1: 0.5×10⁶ (±30%) CAR-T cells/kg, Dose 2: 1×10⁶ (±30%) CAR-T cells/kg, and Dose 3: 2×10⁶ (±30%) CAR-T cells/kg. Receives: Biological: Functionally optimized CD33 CAR-T |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate the Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability) of Functionally optimized CD33 CAR-T cell therapy in relapsed/refractory B Cell Acute Myeloid Leukemia
the incidence and severity of immune therapy related toxic reactions (irAEs)
Measured: up to one month after the CAR-T infusion
Evaluate the Complete Response rate of Functionally optimized CD33 CAR-T cell therapy in relapsed/refractory B Cell Acute Myeloid Leukemia
Complete Response rate on M1 and M3
Measured: one month and three month after the CAR-T infusion
Secondary outcomes (2)
Cell pharmacokinetics Dynamic indicators
CAR-T/T% by flow cytometry
Measured: Day7, Day10, Day14, Day28 after the CAR-T infusion
long-term efficacy
Overall Survival
Measured: up to one year after the CAR-T infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Apr 2026
- Study start1 Jun 2026 (estimated)
- Primary completion31 May 2028 (estimated)
- Study completion31 May 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CAR T Cell TherapyStudies 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
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Qi deng
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Qi deng, Tianjin First Central Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07538713
- Sponsor's protocol ID
- XBAP2025-13
- Status last verified
- Apr 2026
- First submitted
- 13 Apr 2026
- Last updated
- 23 Apr 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study on the Efficacy and Safety of Functionally Optimized CD33 CAR-T Cells (FO33 CAR-T) Therapy Targeting CD33-Positive Recurrent/Refractory Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT07538713
- Acronym / sponsor ID
- XBAP2025-13
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- Qi dengUniversity, hospital or other
- Study start
- 1 Jun 2026 (estimated)
- Primary completion
- 31 May 2028 (estimated)
- Study completion
- 31 May 2028 (estimated)
- First posted
- 20 Apr 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.
Relapsed/refractory acute myeloid leukemia (R/R AML) currently lacks effective CAR-T therapeutic agents due to the absence of tumor-specific target antigens. Most AML-associated antigens are expressed on normal hematopoietic stem/progenitor cells (HSPCs) and healthy tissues, increasing the risk of on-target off-tumor toxicity and non-neoplastic toxicity. CD33 is present on leukemic cells in over 80% of AML patients. Compared with CLL-1, CD123 and other targets, CD33 exhibits higher expression across diverse AML subtypes, reducing the risk of treatment failure and relapse caused by antigen escape and thus serving as an ideal therapeutic target for AML. However, conventional CD33-targeted CAR-T cells demonstrate suboptimal efficacy in clinical trials, accompanied by significant toxicity and inadequate in vivo expansion. To further investigate the safety and efficacy of CAR-T therapy for AML, our center has initiated a clinical trial of functionally optimized CD33 CAR-T (FO33 CAR-T) cells for R/R AML. We constructed a lentiviral CAR vector containing the CD33-targeting scFv, 4-1BB, and CD3ζ, followed by insertion of adjuvant molecule X. FO33 CAR-T cells showed superior cytotoxicity against AML cell lines and enhanced biological activity compared with conventional CD33 CAR-T cells, and exerted safe and effective antitumor effects in preclinical models. This single-center, open-label, prospective clinical trial aims to evaluate the safety and efficacy of FO33 CAR-T cells in patients with R/R AML, as well as to characterize the pharmacokinetic and pharmacodynamic (PK/PD) profiles of this therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Functionally optimized CD33 CAR-T | Experimental | Biological: Functionally optimized CD33 CAR-TBased on previously reported clinical data regarding the safety and efficacy of CAR-T cell infusion in AML trials, as well as ethical considerations for benefit-risk assessment aimed at protecting subject safety, the initial infusion doses in this trial were set as follows: Dose 1: 0.5×10⁶ (±30%) CAR-T cells/kg, Dose 2: 1×10⁶ (±30%) CAR-T cells/kg, and Dose 3: 2×10⁶ (±30%) CAR-T cells/kg. |
Who may qualify
- Ages
- 14 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (15 listed)
- Subjects diagnosed with refractory/recurrent acute myeloid leukemia (excluding M3) who meet any of the following criteria:
- During enrollment screening, bone marrow flow cytometry must demonstrate a CD33+ expression rate of ≥80% in leukemia cells and/or pathological immunohistochemical confirmation of CD33+ extramedullary lesions.
- Estimated survival duration exceeding 3 months as of the date of informed consent signing.
- Participants with Eastern Cooperative Oncology Group (ECOG) physical status scores ranging from 0 to 2.
- Age range of 14 years ≤ ≤ 75 years, inclusive, with no gender restriction.
Key exclusion criteria (17 listed)
- Presence of one of the following cardiac criteria:
- Active graft-versus-host disease (GVHD).
- History of severe pulmonary dysfunction.
- Concurrent other progressive malignancies.
- Concurrent severe or persistent infections that cannot be effectively controlled.
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 |
|---|---|---|
| Functionally optimized CD33 CAR-TFunctionally optimized CD33 CAR-T intravenous infusion | Biological | Functionally optimized CD33 CAR-T |
Primary outcome
Evaluate the Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability) of Functionally optimized CD33 CAR-T cell therapy in relapsed/refractory B Cell Acute Myeloid Leukemia
Time frame: up to one month after the CAR-T infusion
the incidence and severity of immune therapy related toxic reactions (irAEs)
Evaluate the Complete Response rate of Functionally optimized CD33 CAR-T cell therapy in relapsed/refractory B Cell Acute Myeloid Leukemia
Time frame: one month and three month after the CAR-T infusion
Complete Response rate on M1 and M3
2 secondary outcomes
Cell pharmacokinetics Dynamic indicators
Time frame: Day7, Day10, Day14, Day28 after the CAR-T infusion
long-term efficacy
Time frame: up to one year after the CAR-T 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 23 Apr 2026.
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Functionally optimized CD33 CAR-T in people with CAR T Cell Therapy and CD33 Positive Acute Myelogenous Leukemia, run by Qi deng.
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 15 inclusion and 17 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 23 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate the Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability) of Functionally optimized CD33 CAR-T cell therapy in relapsed/refractory B Cell Acute Myeloid Leukemia (time frame: up to one month after the CAR-T 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 23 Apr 2026; AzTrials last refreshed its copy on 28 Oct 2026.