AML, AdultPhase 1NCT05987696
Natural Killer(NK) Cell Therapy in Acute Myeloid Leukemia
A Phase 1 study testing CD33/CLL1 dual CAR-NK cell, Cyclophosphamid and Fludarabine in people with AML, Adult and Minimal Residual Disease, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
This is a phase 1, first-in-human (FIH), open-label, multicohort study to evaluate the safety, tolerability and preliminary efficacy of iPSC NK cells in patients with relapsed/refractory AML or AML Minimal Residual Disease (MRD). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- AML, Adult, Minimal Residual Disease
- What is being tested?
- CD33/CLL1 dual CAR-NK cell, Cyclophosphamid and Fludarabine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteChina
- Study dates
- 31 Jul 2023to 17 Jan 2025
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Provision of signed and dated informed consent form (ICF).
- ≥18 years old.
- Eastern Cooperative Oncology Group (ECOG) performance status ≤1 and life expectancy greater than 12 weeks.
- Diagnosis of r/r AML (Cohort 1 and 2) or AML MRD (Cohort 3).
- Cohort 1: Both CLL1 and CD33 expression are positive in AML blasts; Cohort 2: The expression of CD33 in AML blast is positive.
- Adequate organ and marrow function, as defined below:
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- Blood creatinine (Cr) ≤ 2 x ULN or calculated creatinine clearance (Cockcroft-Gault formula) ≥ 50 mL/min;
- Total bilirubin (TBIL) ≤ 2 x the ULN;
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3 x ULN;
- International normalized ratio (INR) and activated partial thromboplastin time (aPTT) ≤ 1.5 x ULN;
- Females of childbearing potential must have a negative serum pregnancy test.
- Donor specific antibody (DSA) is negative: MFI <= 2000.
Who cannot join
- Allergic to drug used in this study.
- Subjects received any antitumor therapy as follows, prior to first NK infusion:
- Systemic steroid therapy within 3 days (except physiological replacement therapy);
- Systemic antitumor therapy within 2 weeks or at least 5 half-lives, whichever is less;
- Radiotherapy within 4 weeks;
- Donor lymphocyte infusion within 6 weeks;
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- Intrathecal treatment within 1 week;
- CAR-T therapy, CAR-NK therapy, or any other genetically modified cell therapy product within 6 months;
- History of allogeneic stem cell transplantation.
- Received the vaccine within 4 weeks prior to the first infusion and/or expected to require vaccination from the study period to 12 weeks after the last infusion.
- Active central nervous system Leukemia.
- Acute Promyelocytic Leukemia (APL).
- History of other malignant tumors, except for those who have achieved complete remission more than 5 years after radical treatment without any signs of recurrence.
- Active autoimmune diseases.
- History of central nervous system disease or meningeal involvement such as epilepsy, paralysis, aphasia, stroke, etc.
- Serious cardiovascular and cerebrovascular diseases:
- Severe heart rhythm or conduction abnormalities, corrected QT interval (QTc)≥480 ms;
- Acute coronary syndrome, congestive heart failure, aortic dissection, stroke, or other grade 3 or higher cardiovascular and cerebrovascular events within 6 months prior to first infusion;
- New York Heart Association (NYHA) class II or above congestive heart failure or left ventricular ejection fraction (LVEF) <50% in color Doppler echocardiography;
- Hypertension that cannot be controlled by drug.
- Active pulmonary infection; SpO2 ≤90%; Pulmonary embolism, chronic obstructive pulmonary disease, or interstitial lung disease.
- Uncontrolled bacterial, fungal, or viral infection. Known HIV infection, active Hepatitis B (HBV) or Hepatitis C (HCV) infection.
- History of substance abuse.
- Toxicity induced by previous therapy not recovered to ≤ grade 2(NCI-CTCAE v5.0).
- Large surgical treatment within 4 weeks prior to first infusion, not including diagnostic biopsy.
- Pregnant/breastfeeding women.
- Investigator-assessed presence of any medical or social issues that are likely to interfere with study conduct or may cause increased risk to subject.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase I Study to Evaluate the Safety and Efficacy of NK Cell Therapy in Acute Myeloid Leukemia (AML).
This is a phase 1, first-in-human (FIH), open-label, multicohort study to evaluate the safety, tolerability and preliminary efficacy of iPSC NK cells in patients with relapsed/refractory AML or AML Minimal Residual Disease (MRD).
- Conditions
- AML, Adult, Minimal Residual Disease
- Being tested
- CD33/CLL1 dual CAR-NK cell, Cyclophosphamid, Fludarabine, Cytarabine, CD33 CAR-NK cell
- Medical subject headings
- Leukemia, Myeloid, Acute, Neoplasm, Residual
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CD33/CLL1 dual CAR-NK cellDrug
NK cell therapy
CyclophosphamidDrug
Lympho-conditioning Agent
FludarabineDrug
Lympho-conditioning Agent
CytarabineDrug
Lympho-conditioning Agent
CD33 CAR-NK cellDrug
NK cell therapy
super NK cellDrug
NK cell therapy
| Study group | Role | What they receive |
|---|---|---|
| CD33/CLL1 dual CAR-NK cell | Experimental | CLL1/CD33 dual CAR-NK cell therapy in Adult subjects with r/r AML Receives: Drug: CD33/CLL1 dual CAR-NK cell; Drug: Cyclophosphamid; Drug: Fludarabine; Drug: Cytarabine |
| CD33 CAR-NK cell | Experimental | CD33 CAR-NK cell therapy in Adult subjects with r/r AML Receives: Drug: Cyclophosphamid; Drug: Fludarabine; Drug: Cytarabine; Drug: CD33 CAR-NK cell |
| super NK cell | Experimental | super NK cell therapy in Adult subjects with AML MRD Receives: Drug: Cyclophosphamid; Drug: Fludarabine; Drug: Cytarabine; Drug: super NK cell |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Measured: 28 Days from first dose of iPSC NK cell infusion
Incidence of subjects with Dose Limiting Toxicities within each dose level cohort
Measured: 28 Days from first dose of iPSC NK cell infusion
Secondary outcomes (6)
Overall Response Rate(ORR)
Measured: Up to approximately 2 years after last dose of iPSC NK cell infusion
MRD negative rate
Measured: 28 Days from first dose of iPSC NK cell infusion
Event-free survival
Measured: Up to approximately 2 years after last dose of iPSC NK cell infusion]
Relapse-free survival
Measured: Up to approximately 2 years after last dose of iPSC NK cell infusion
Overall survival (OS)
Measured: Up to approximately 2 years after last dose of iPSC NK cell infusion
Determination of the pharmacokinetics (PK) of iPSC NK cells in peripheral blood
The PK of iPSC NK in peripheral blood will be reported as the relative percentage of product DNA versus patient DNA (% chimerism) measured from blood samples at the specified time points.
Measured: Up to approximately 2 years after last dose of iPSC NK cell infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Aug 2023
- Study start31 Jul 2023
- Primary completion17 Jan 2025
- Study completion17 Jan 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AML, AdultStudies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe sponsor's other registered studies.
- 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Hangzhou Qihan Biotech Co., Ltd.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05987696
- Sponsor's protocol ID
- IIT2023028
- Status last verified
- May 2024
- First submitted
- 24 Jul 2023
- Last updated
- 11 Feb 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 11 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Study to Evaluate the Safety and Efficacy of NK Cell Therapy in Acute Myeloid Leukemia (AML).
- ClinicalTrials.gov ID
- NCT05987696
- Acronym / sponsor ID
- IIT2023028
- Conditions
- AML, Adult, Minimal Residual Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Collaborators
- Hangzhou Qihan Biotech Co., Ltd.
- Study start
- 31 Jul 2023 (actual)
- Primary completion
- 17 Jan 2025 (actual)
- Study completion
- 17 Jan 2025 (actual)
- First posted
- 14 Aug 2023 (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 is a phase 1, first-in-human (FIH), open-label, multicohort study to evaluate the safety, tolerability and preliminary efficacy of iPSC NK cells in patients with relapsed/refractory AML or AML Minimal Residual Disease (MRD).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| CD33/CLL1 dual CAR-NK cell | Experimental | Drug: CD33/CLL1 dual CAR-NK cell, Drug: Cyclophosphamid, Drug: Fludarabine, Drug: CytarabineCLL1/CD33 dual CAR-NK cell therapy in Adult subjects with r/r AML |
| CD33 CAR-NK cell | Experimental | Drug: Cyclophosphamid, Drug: Fludarabine, Drug: Cytarabine, Drug: CD33 CAR-NK cellCD33 CAR-NK cell therapy in Adult subjects with r/r AML |
| super NK cell | Experimental | Drug: Cyclophosphamid, Drug: Fludarabine, Drug: Cytarabine, Drug: super NK cellsuper NK cell therapy in Adult subjects with AML MRD |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Provision of signed and dated informed consent form (ICF).
- ≥18 years old.
- Eastern Cooperative Oncology Group (ECOG) performance status ≤1 and life expectancy greater than 12 weeks.
- Diagnosis of r/r AML (Cohort 1 and 2) or AML MRD (Cohort 3).
- Cohort 1: Both CLL1 and CD33 expression are positive in AML blasts; Cohort 2: The expression of CD33 in AML blast is positive.
Key exclusion criteria (27 listed)
- Allergic to drug used in this study.
- Subjects received any antitumor therapy as follows, prior to first NK infusion:
- History of allogeneic stem cell transplantation.
- Received the vaccine within 4 weeks prior to the first infusion and/or expected to require vaccination from the study period to 12 weeks after the last infusion.
- Active central nervous system Leukemia.
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 |
|---|---|---|
| CD33/CLL1 dual CAR-NK cellNK cell therapy | Drug | CD33/CLL1 dual CAR-NK cell |
| CyclophosphamidLympho-conditioning Agent | Drug | CD33 CAR-NK cell, CD33/CLL1 dual CAR-NK cell, super NK cell |
| FludarabineLympho-conditioning Agent | Drug | CD33 CAR-NK cell, CD33/CLL1 dual CAR-NK cell, super NK cell |
| CytarabineLympho-conditioning Agent | Drug | CD33 CAR-NK cell, CD33/CLL1 dual CAR-NK cell, super NK cell |
| CD33 CAR-NK cellNK cell therapy | Drug | CD33 CAR-NK cell |
| super NK cellNK cell therapy | Drug | super NK cell |
Primary outcome
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Time frame: 28 Days from first dose of iPSC NK cell infusion
Incidence of subjects with Dose Limiting Toxicities within each dose level cohort
Time frame: 28 Days from first dose of iPSC NK cell infusion
6 secondary outcomes
Overall Response Rate(ORR)
Time frame: Up to approximately 2 years after last dose of iPSC NK cell infusion
MRD negative rate
Time frame: 28 Days from first dose of iPSC NK cell infusion
Event-free survival
Time frame: Up to approximately 2 years after last dose of iPSC NK cell infusion]
Relapse-free survival
Time frame: Up to approximately 2 years after last dose of iPSC NK cell infusion
Overall survival (OS)
Time frame: Up to approximately 2 years after last dose of iPSC NK cell infusion
Determination of the pharmacokinetics (PK) of iPSC NK cells in peripheral blood
Time frame: Up to approximately 2 years after last dose of iPSC NK cell infusion
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Feb 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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CD33/CLL1 dual CAR-NK cell, Cyclophosphamid and Fludarabine in people with AML, Adult and Minimal Residual Disease, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site 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 12 inclusion and 27 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: finished, as of the sponsor's last update on 11 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] (time frame: 28 Days from first dose of iPSC NK 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 11 Feb 2026; AzTrials last refreshed its copy on 7 Nov 2026.