AML (Acute Myelogenous Leukemia)Phase 1CD123-CD16-NKNCT06835140
CD123-CD16-NK Cells Immunotherapy for AML
A Phase 1 study testing Donor-derived CD123-CD16 bispecific antibody-modified NK cells in people with AML (Acute Myelogenous Leukemia) and NK Cell, run by Chunji Gao at 1 site in China.
Trial summary
The goal of this clinical trial is to evaluate the effectiveness of CD123-CD16 bispecific antibody-modified NK cells in treating patients with CD123-positive relapsed or refractory Acute Myeloid Leukemia (RR AML). It will also assess the safety of this modified NK cell therapy. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- AML (Acute Myelogenous Leukemia), NK Cell, CD123+ Acute Myeloid Leukemia
- What is being tested?
- Donor-derived CD123-CD16 bispecific antibody-modified NK cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteChina
- Study dates
- 21 Feb 2025to 31 Dec 2025
- 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
- Age: Between18 years and 70 years.
- Diagnosis and Treatment History:
- Diagnosed with Acute Myeloid Leukemia (AML) in the hospital. Has undergone multiple first-line clinical treatments and has developed resistance to current treatments. Relapse after original induction therapy failure with a predicted survival of more than three months.
- CD123 Expression:
- Flow cytometry detection shows CD123-positive AML cells.CD123 expression level is not less than 20%.
- Hospital Examination Criteria:
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- Performance Status:
- ECOG Performance Status score of 0-2 or Karnofsky Performance Status (KPS) score greater than 80.
- Donor Availability:
- Have a suitable healthy donor and agree to peripheral blood collection.
Who cannot join
- Specific AML Subtype:
- Diagnosed with Acute Promyelocytic Leukemia(APL).
- CD123 Expression:
- Flow cytometry shows CD123 negative or CD123 expression level less than 20%.
- Prior Treatment Toxicity:
- Persistent non-hematologic toxicity of grade 2 or higher related to previous treatments.
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- GVHD Requiring Immunosuppression:
- Patients requiring immunosuppressants for grade II-IV acute Graft-Versus-Host Disease (GVHD).
- Recent Steroid Treatment:
- Systemic steroid treatment within 7 days prior to first study drug treatment (excluding topical and inhaled corticosteroids or short-term prophylactic steroid treatment).
- Severe Cardiovascular and Cerebrovascular Diseases:
- Certain cardiovascular and cerebrovascular diseases within 6 months prior to first dose.
- New York Heart Association (NYHA) classification ≥3 or uncontrolled malignant arrhythmias.Other cardiovascular and cerebrovascular diseases deemed unsuitable by the investigator.
- Pregnancy and Lactation:
- Pregnant or breastfeeding women (the safety of this treatment for unborn babies is unknown).
- For female participants, pregnancy must be confirmed negative by serum or urine pregnancy test within 48 hours before infusion.
- Infections:
- Active Hepatitis B,Hepatitis C virus infection, Peripheral blood CMV-DNA ≥500 copies/mL, HIV/AIDS infection and any uncontrolled active infection.
- Allergic Reactions:
- Allergic to immunotherapy and related drugs.
- Neurological Diseases:
- Neurological diseases such as neurodegenerative diseases, primary central nervous system tumors/infections, multiple sclerosis, epilepsy, severe peripheral neuropathy, etc.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Chinese PLA General Hospital
Beijing, China, 100853
Yanqing Ma, M.D & Ph.D, +86 13120041949, mayanqing301@163.com
Chunji Gao, M.D. & Ph.D.
About this study
Official title: CD123-Targeted CD16 Antibody-Modified NK Cell Immunotherapy for Refractory/Relapsed Acute Myeloid Leukemia (R/R AML)
The goal of this clinical trial is to evaluate the effectiveness of CD123-CD16 bispecific antibody-modified NK cells in treating patients with CD123-positive relapsed or refractory Acute Myeloid Leukemia (RR AML). It will also assess the safety of this modified NK cell therapy.
The main questions： Does the infusion of CD123-CD16 bispecific antibody-modified NK cells induce remission in RR AML patients? What are the safety and potential adverse effects associated with the administration of these modified NK cells? Researchers will administer CD123-CD16 bispecific antibody-modified NK cells to RR AML patients and compare the outcomes to existing treatment options to determine efficacy and safety.
Read more
Participants will:
Undergo lymphocyte-depleting chemotherapy Fludarabine\&Cyclophosphamide from day -5 to day -3 before NK cell infusion.
Receive intravenous infusions of modified NK cells at escalating doses:
The first three patients will receive 1×10⁷ cells/kg. The next three patients will receive 2×10⁷ cells/kg. The final three patients will receive 4×10⁷ cells/kg. Have NK cell infusions administered every 96-120 hours for a total of three infusions, with each infusion completed within 10 to 15 minutes.
Undergo dose escalation with subsequent groups only after confirming the safety of the previous dose group.
Have their vital signs (temperature, heart rate, respiratory rate, blood pressure, etc.) monitored before and after each infusion.
Keep baseline data records during NK cell infusions. Participate in follow-up assessments to monitor disease remission and detect any adverse events.
This trial aims to provide new treatment options for RR AML patients by leveraging the targeted cytotoxic effects of CD123-CD16 bispecific antibody-modified NK cells to achieve disease remission.
- Being tested
- Donor-derived CD123-CD16 bispecific antibody-modified NK cells
- Keywords
- CD123-CD16 bispecific antibody, NK cell, Refractory/relapsed AML
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Donor-derived CD123-CD16 bispecific antibody-modified NK cellsDrug
Patients enrolled sequentially received varying doses of NK cell infusions. The first three patients received 1×10⁷ cells/kg, the next three received 2×10⁷ cells/kg, and the final three received 4×10⁷ cells/kg.
| Study group | Role | What they receive |
|---|---|---|
| CD123-CD16 bispecific antibody-modified NK cells for the treatment of CD123-positive R/R AML | Experimental | Patients scheduled to receive CD123-CD16-NK cell infusions undergo lymphocyte-depleting chemotherapy from day -5 to day -3. After completing preconditioning, 48 hours later, patients are intravenously infused with NK cells at a dose of 1×10⁷ cells/kg (100-150 ml volume) within 10 to 15 minutes. Infusions are administered every 96-120 hours for a total of three times. Subsequently, patients receive escalating NK cell doses of 2×10⁷ cells/kg and 4×10⁷ cells/kg. Vital signs (temperature, heart rate, respiratory rate, blood pressure, etc.) are monitored before and after infusion. Baseline data during NK cell infusion are also recorded. For each dose group, only one patient initially receives a single NK cell infusion, followed by a 3-day observation period to ensure safety before proceeding with a second infusion. If no adverse events occur in the initial dose group, the next dose group is infused accordingly, and the process continues similarly. Receives: Drug: Donor-derived CD123-CD16 bispecific antibody-modified NK cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
ORR
Overall Response Rate
Measured: Bone marrow aspiration assessments are conducted one week after each patient's treatment completion, followed by evaluations at 1 month, 3 months, and 6 months.
CR
Complete Remission Rate
Measured: Bone marrow aspiration assessments are conducted one week after each patient's treatment completion, followed by evaluations at 1 month, 3 months, and 6 months.
AE
Adverse events related to cell reinfusion (≥ Grade 3 treatment-related organ toxicity, laboratory tests, and Grade 4 hematologic toxicity, etc.) and number of participants with treatment-related AEs assessed by CTCAE v5.0
Measured: 14 days-28 days after infusion
Secondary outcomes (4)
Progressive Disease (PD)
PD parameters: content of CD123+AML cells in peripheral blood ; plasma cytokine levels at various time points;
Measured: 14 days-28 days after infusion
Stable Disease (SD)
the condition without significant progression nor significant reduction in AML cells after treatment. The disease is stable, with no significant change in the number of leukemia cells in peripheral blood or bone marrow assessed by IRC.
Measured: 14 days-28 days after infusion
Progression-free survival (PFS)
the time from cell last infusion to the first assessment of tumor progression, relapse, or death for any reason
Measured: through study completion, an average of 6-12moths
Overall survival (OS)
the time from cell last infusion to death for any reason
Measured: through study completion, an average of 6-12moths
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Feb 2025
- Study start21 Feb 2025 (estimated)
- Primary completion31 Dec 2025 (estimated)
- Study completion31 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AML (Acute Myelogenous Leukemia)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
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chunji Gao
- Sponsor type
- University, hospital or other
- Collaborators
- Chinese PLA General Hospital
- Responsible party
- Sponsor-investigator, Chunji Gao, Chinese PLA General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06835140
- Sponsor's protocol ID
- S2024-653-01
- Acronym
- CD123-CD16-NK
- Status last verified
- Feb 2025
- First submitted
- 7 Jan 2025
- Last updated
- 25 Feb 2025
- Results posted
- No
- Data monitoring committee
- Yes
- 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 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- CD123-Targeted CD16 Antibody-Modified NK Cell Immunotherapy for Refractory/Relapsed Acute Myeloid Leukemia (R/R AML)
- ClinicalTrials.gov ID
- NCT06835140
- Acronym / sponsor ID
- CD123-CD16-NK / S2024-653-01
- Keywords
- CD123-CD16 bispecific antibody, NK cell, Refractory/relapsed AML
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Chunji GaoUniversity, hospital or other
- Collaborators
- Chinese PLA General Hospital
- Study start
- 21 Feb 2025 (estimated)
- Primary completion
- 31 Dec 2025 (estimated)
- Study completion
- 31 Dec 2025 (estimated)
- First posted
- 19 Feb 2025 (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.
The goal of this clinical trial is to evaluate the effectiveness of CD123-CD16 bispecific antibody-modified NK cells in treating patients with CD123-positive relapsed or refractory Acute Myeloid Leukemia (RR AML). It will also assess the safety of this modified NK cell therapy.
The main questions： Does the infusion of CD123-CD16 bispecific antibody-modified NK cells induce remission in RR AML patients? What are the safety and potential adverse effects associated with the administration of these modified NK cells? Researchers will administer CD123-CD16 bispecific antibody-modified NK cells to RR AML patients and compare the outcomes to existing treatment options to determine efficacy and safety.
Participants will:
Undergo lymphocyte-depleting chemotherapy Fludarabine\&Cyclophosphamide from day -5 to day -3 before NK cell infusion.
Receive intravenous infusions of modified NK cells at escalating doses:
The first three patients will receive 1×10⁷ cells/kg. The next three patients will receive 2×10⁷ cells/kg. The final three patients will receive 4×10⁷ cells/kg. Have NK cell infusions administered every 96-120 hours for a total of three infusions, with each infusion completed within 10 to 15 minutes.
Undergo dose escalation with subsequent groups only after confirming the safety of the previous dose group.
Have their vital signs (temperature, heart rate, respiratory rate, blood pressure, etc.) monitored before and after each infusion.
Keep baseline data records during NK cell infusions. Participate in follow-up assessments to monitor disease remission and detect any adverse events.
This trial aims to provide new treatment options for RR AML patients by leveraging the targeted cytotoxic effects of CD123-CD16 bispecific antibody-modified NK cells to achieve disease remission.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CD123-CD16 bispecific antibody-modified NK cells for the treatment of CD123-positive R/R AML | Experimental | Drug: Donor-derived CD123-CD16 bispecific antibody-modified NK cellsPatients scheduled to receive CD123-CD16-NK cell infusions undergo lymphocyte-depleting chemotherapy from day -5 to day -3. After completing preconditioning, 48 hours later, patients are intravenously infused with NK cells at a dose of 1×10⁷ cells/kg (100-150 ml volume) within 10 to 15 minutes. Infusions are administered every 96-120 hours for a total of three times. Subsequently, patients receive escalating NK cell doses of 2×10⁷ cells/kg and 4×10⁷ cells/kg. Vital signs (temperature, heart rate, respiratory rate, blood pressure, etc.) are monitored before and after infusion. Baseline data during NK cell infusion are also recorded. For each dose group, only one patient initially receives a single NK cell infusion, followed by a 3-day observation period to ensure safety before proceeding with a second infusion. If no adverse events occur in the initial dose group, the next dose group is infused accordingly, and the process continues similarly. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Age: Between18 years and 70 years.
- Diagnosis and Treatment History:
- Diagnosed with Acute Myeloid Leukemia (AML) in the hospital. Has undergone multiple first-line clinical treatments and has developed resistance to current treatments. Relapse after original induction therapy failure with a predicted survival of more than three months.
- CD123 Expression:
- Flow cytometry detection shows CD123-positive AML cells.CD123 expression level is not less than 20%.
Key exclusion criteria (22 listed)
- Specific AML Subtype:
- Diagnosed with Acute Promyelocytic Leukemia(APL).
- CD123 Expression:
- Flow cytometry shows CD123 negative or CD123 expression level less than 20%.
- Prior Treatment Toxicity:
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 |
|---|---|---|
| Donor-derived CD123-CD16 bispecific antibody-modified NK cellsPatients enrolled sequentially received varying doses of NK cell infusions. The first three patients received 1×10⁷ cells/kg, the next three received 2×10⁷ cells/kg, and the final three received 4×10⁷ cells/kg. | Drug | CD123-CD16 bispecific antibody-modified NK cells for the treatment of CD123-positive R/R AML |
Primary outcome
ORR
Time frame: Bone marrow aspiration assessments are conducted one week after each patient's treatment completion, followed by evaluations at 1 month, 3 months, and 6 months.
Overall Response Rate
CR
Time frame: Bone marrow aspiration assessments are conducted one week after each patient's treatment completion, followed by evaluations at 1 month, 3 months, and 6 months.
Complete Remission Rate
AE
Time frame: 14 days-28 days after infusion
Adverse events related to cell reinfusion (≥ Grade 3 treatment-related organ toxicity, laboratory tests, and Grade 4 hematologic toxicity, etc.) and number of participants with treatment-related AEs assessed by CTCAE v5.0
4 secondary outcomes
Progressive Disease (PD)
Time frame: 14 days-28 days after infusion
Stable Disease (SD)
Time frame: 14 days-28 days after infusion
Progression-free survival (PFS)
Time frame: through study completion, an average of 6-12moths
Overall survival (OS)
Time frame: through study completion, an average of 6-12moths
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 Feb 2025.
- 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.
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 Donor-derived CD123-CD16 bispecific antibody-modified NK cells in people with AML (Acute Myelogenous Leukemia) and NK Cell, run by Chunji Gao 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 10 inclusion and 22 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 Feb 2025. 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?
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 Chunji Gao, M.D. (+86 13911536256, gaochunji301@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
ORR (time frame: Bone marrow aspiration assessments are conducted one week after each patient's treatment completion, followed by evaluations at 1 month, 3 months, and 6 months.). The study lists 3 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 Feb 2025.