Acute Myeloid LeukemiaPhase 1/2NCT06006403
Safety and Efficacy of CD123-targeted CAR-NK for Relapsed/Refractory Acute Myeloid Leukemia or Blastic Plasmacytoid Dendritic Cell Neoplasm
A Phase 1/2 study testing CD123 targeted CAR-NK cells in people with Acute Myeloid Leukemia and Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN), run by Chongqing Precision Biotech Co., Ltd at 1 site in China.
Trial summary
This study is a single-arm, open-label, dose-escalating + dose-expansion clinical study, aiming to evaluate the safety and efficacy of targeting CD123 CAR-NK cell preparations in Relapsed/refractory acute myeloid leukemia (AML) or blastocytic plasmacytoid dendritic cell neoplasm (BPDCN). Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Acute Myeloid Leukemia, Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN), Relapse Leukemia
- What is being tested?
- CD123 targeted CAR-NK cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteChina
- Study dates
- 28 Mar 2024to 31 Dec 2025
- Phase
- Phase 1/2
- 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 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Gender is not limited, age 18-75 years old (including the threshold value);
- The expression of CD123 in tumor cells was detected by flow cytometry.
- Patients with relapsed/refractory AML or BPDCN diagnosed with CD123 positive: 1) AML: a. Recurrent: After complete response (CR), the recurrence of leukemia cells in peripheral blood or bone marrow original cells ≥5% (except for other reasons such as bone marrow regeneration after consolidation chemotherapy) or the occurrence of extramedullary leukemia cell infiltration; b. Refractory: refers to those who have failed to receive 2 courses of treatment with standard protocols; Patients recurrence within 12 months after CR with consolidation and intensive treatment; Recurrence after 12 months but failed to respond to conventional chemotherapy; 2 or more relapses; Extramedullary leukemia persists;
- 2) BPDCN: has failed to receive guidelines-recommended salvage therapy or is unable to tolerate current therapy, and has persistent or recurrent disease in any of the peripheral blood, bone marrow, lymph nodes, spleen, skin lesions, or other site lesions.
- 4. Expected survival time is more than 12 weeks;
- 5. ECOG 0-2 points (Appendix 2);
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- 6. No serious mental disorders; The functions of important organs are basically normal:
- Cardiac function: echocardiography indicated cardiac ejection fraction ≥50%, and no obvious abnormality was found in electrocardiogram;
- Renal function: serum creatinine ≤2.0×ULN;
- Liver function: ALT and AST ≤ 3.0×ULN;
- Total bilirubin and alkaline phosphatase ≤ 2.0×ULN (Gilbert syndrome ≤ 3.0×ULN);
- Blood oxygen saturation \> 92%.
- 7. The patient or his/her guardian agrees to participate in the clinical trial and signs the ICF, indicating that he/she understands the purpose and procedure of the clinical trial and is willing to participate in the study.
Who cannot join
- Prior to screening, the following anti-tumor therapies were received: chemotherapy, targeted therapy, or other investigational drug treatment within 14 days or at least 5 half-lives (whichever is shorter), except in cases where disease progression has been confirmed after treatment;
- had a cerebrovascular accident or seizure within 6 months before signing the ICF;
- There is an active or uncontrolled infection that requires systemic treatment within 1 week prior to screening;
- suffering from any of the following heart diseases:
- New York Heart Association (NYHA) Stage III or IV congestive heart failure;
- Had myocardial infarction or coronary artery bypass grafting (CABG) within ≤6 months before enrollment;
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- A history of clinically significant ventricular arrhythmia, or unexplained syncope (other than those caused by vasovagal or dehydration);
- History of severe non-ischemic cardiomyopathy;
- combined with active hepatitis B;
- Combined with active autoimmune diseases, long-term immunosuppressive therapy is required;
- have other malignancies, except for adequately treated cervical carcinoma in situ, basal cell or squamous cell skin cancer, local prostate cancer after radical surgery, and ductal carcinoma in situ after radical surgery;
- Had received live attenuated vaccine within 4 weeks prior to screening;
- Women who are pregnant or breastfeeding, and male or female subjects who plan to have a family within 1 year after receiving CAR T cell transfusion;
- Circumstances deemed unsuitable for participation in the study by other researchers.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Clinical Study of Targeting CD123 Chimeric Antigen Receptor Natural Killer Cells (CAR-NK) in the Treatment of Relapsed/Refractory Acute Myeloid Leukemia or Blastic Plasmacytoid Dendritic Cell Neoplasm
This study is a single-arm, open-label, dose-escalating + dose-expansion clinical study, aiming to evaluate the safety and efficacy of targeting CD123 CAR-NK cell preparations in Relapsed/refractory acute myeloid leukemia (AML) or blastocytic plasmacytoid dendritic cell neoplasm (BPDCN). The pharmacokinetic characteristics of CAR-NK cell preparations for the treatment of patients with Relapsed/refractory acute myeloid leukemia or blastocytic plasmacytoid dendritic cell neoplasm were obtained and the recommended dose.
Read the detailed description
According to the different disease type, it is divided into two subgroups: AML and BPDCN. Each subgroup includes a dose exploration stage (Part A) and a dose expansion stage (Part B). 3 patients were explored, starting from the low-dose group, and in the dose expansion phase, the safety and efficacy were further verified according to the safe recommended dose obtained in the dose exploration phase.
- Conditions
- Acute Myeloid Leukemia, Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN), Relapse Leukemia, Refractory Leukemia
- Being tested
- CD123 targeted CAR-NK cells
- Medical subject headings
- Leukemia, Myeloid, Acute, Blastic Plasmacytoid Dendritic Cell Neoplasm, Leukemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CD123 targeted CAR-NK cellsBiological
Administration method: intravenous infusion； Subjects will receive conditioning therapy by Fludarabine and Cyclophosphamide before cell infusion.
| Study group | Role | What they receive |
|---|---|---|
| Acute Myeloid Leukemia | Experimental | Infusion of CD123-targeted CAR-NK cells by dose of 1-10x10^6 cells/kg Receives: Biological: CD123 targeted CAR-NK cells |
| Blastic Plasmacytoid Dendritic Cell Neoplasm | Experimental | Infusion of CD123-targeted CAR-NK cells by dose of 1-10x10^6 cells/kg Receives: Biological: CD123 targeted CAR-NK cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the safety of CAR-NK cell preparations in the treatment of Relapsed/Refractory Acute Myeloid Leukemia or Blastic Plasmacytoid Dendritic Cell Neoplasm[Safety and Tolerability]
The incidence of adverse events after CD123 CAR-NK cell infusion was assessed by the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE, version 5.0)
Measured: 1 month
To evaluate the efficacy of CAR-NK cell preparations in the treatment of Relapsed/Refractory Acute Myeloid Leukemia or Blastic Plasmacytoid Dendritic Cell Neoplasm[Effectiveness]
Objective response rate at 4W±7D and 3M±7D after CAR-NK infusion (Objective response rate includes CR, CRi)
Measured: 1month,3months
Secondary outcomes (4)
AUCS of CD123 CAR-NK cells [Cell dynamics]
AUCS is defined as the area under the curve in 90 days
Measured: 3 months
CMAX of CD123 CAR-NK cells [Cell dynamics]
CMAX is defined as the highest concentration of CEA CAR-T cells expanded in peripheral blood
Measured: 3 months
TMAX of CD123 CAR-NK cells[Cell dynamics]
TMAX is defined as the time to reach the highest concentration
Measured: 3 months
Pharmacodynamics of CD123 CAR-NK cells[Cell dynamics]
The degree of clearance of malignant cells in peripheral blood was detected by blood smear at each time point，and the concentration level of serum cytoplasmic factors such as CRP and IL-6 were detected by ELISA at each time point
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Aug 2023
- Study start28 Mar 2024
- Primary completion25 Sep 2025
- Study completion31 Dec 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Chongqing Precision Biotech Co., Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Shanxi Bethune Hospital
- Responsible party
- Sponsor
- Investigators
- Jia Wei, M.D, Principal investigator, Shanxi Bethune Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06006403
- Sponsor's protocol ID
- PBC050
- Status last verified
- Aug 2023
- First submitted
- 17 Aug 2023
- Last updated
- 5 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study of Targeting CD123 Chimeric Antigen Receptor Natural Killer Cells (CAR-NK) in the Treatment of Relapsed/Refractory Acute Myeloid Leukemia or Blastic Plasmacytoid Dendritic Cell Neoplasm
- ClinicalTrials.gov ID
- NCT06006403
- Acronym / sponsor ID
- PBC050
- Conditions
- Acute Myeloid Leukemia, Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN), Relapse Leukemia, Refractory Leukemia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Chongqing Precision Biotech Co., LtdIndustry (pharma, biotech, devices)
- Collaborators
- Shanxi Bethune Hospital
- Study start
- 28 Mar 2024 (actual)
- Primary completion
- 25 Sep 2025 (actual)
- Study completion
- 31 Dec 2025 (actual)
- First posted
- 23 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 study is a single-arm, open-label, dose-escalating + dose-expansion clinical study, aiming to evaluate the safety and efficacy of targeting CD123 CAR-NK cell preparations in Relapsed/refractory acute myeloid leukemia (AML) or blastocytic plasmacytoid dendritic cell neoplasm (BPDCN). The pharmacokinetic characteristics of CAR-NK cell preparations for the treatment of patients with Relapsed/refractory acute myeloid leukemia or blastocytic plasmacytoid dendritic cell neoplasm were obtained and the recommended dose.
Detailed description
According to the different disease type, it is divided into two subgroups: AML and BPDCN. Each subgroup includes a dose exploration stage (Part A) and a dose expansion stage (Part B). 3 patients were explored, starting from the low-dose group, and in the dose expansion phase, the safety and efficacy were further verified according to the safe recommended dose obtained in the dose exploration phase.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Acute Myeloid Leukemia | Experimental | Biological: CD123 targeted CAR-NK cellsInfusion of CD123-targeted CAR-NK cells by dose of 1-10x10^6 cells/kg |
| Blastic Plasmacytoid Dendritic Cell Neoplasm | Experimental | Biological: CD123 targeted CAR-NK cellsInfusion of CD123-targeted CAR-NK cells by dose of 1-10x10^6 cells/kg |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Gender is not limited, age 18-75 years old (including the threshold value);
- The expression of CD123 in tumor cells was detected by flow cytometry.
- Patients with relapsed/refractory AML or BPDCN diagnosed with CD123 positive: 1) AML: a. Recurrent: After complete response (CR), the recurrence of leukemia cells in peripheral blood or bone marrow original cells ≥5% (except for other reasons such as bone marrow regeneration after consolidation chemotherapy) or the occurrence of extramedullary leukemia cell infiltration; b. Refractory: refers to those who have failed to receive 2 courses of treatment with standard protocols; Patients recurrence within 12 months after CR with consolidation and intensive treatment; Recurrence after 12 months but failed to respond to conventional chemotherapy; 2 or more relapses; Extramedullary leukemia persists;
- 2) BPDCN: has failed to receive guidelines-recommended salvage therapy or is unable to tolerate current therapy, and has persistent or recurrent disease in any of the peripheral blood, bone marrow, lymph nodes, spleen, skin lesions, or other site lesions.
- 4. Expected survival time is more than 12 weeks;
Key exclusion criteria (14 listed)
- Prior to screening, the following anti-tumor therapies were received: chemotherapy, targeted therapy, or other investigational drug treatment within 14 days or at least 5 half-lives (whichever is shorter), except in cases where disease progression has been confirmed after treatment;
- had a cerebrovascular accident or seizure within 6 months before signing the ICF;
- There is an active or uncontrolled infection that requires systemic treatment within 1 week prior to screening;
- suffering from any of the following heart diseases:
- combined with active hepatitis B;
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 |
|---|---|---|
| CD123 targeted CAR-NK cellsAdministration method: intravenous infusion； Subjects will receive conditioning therapy by Fludarabine and Cyclophosphamide before cell infusion. | Biological | Acute Myeloid Leukemia, Blastic Plasmacytoid Dendritic Cell Neoplasm |
Primary outcome
To evaluate the safety of CAR-NK cell preparations in the treatment of Relapsed/Refractory Acute Myeloid Leukemia or Blastic Plasmacytoid Dendritic Cell Neoplasm[Safety and Tolerability]
Time frame: 1 month
The incidence of adverse events after CD123 CAR-NK cell infusion was assessed by the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE, version 5.0)
To evaluate the efficacy of CAR-NK cell preparations in the treatment of Relapsed/Refractory Acute Myeloid Leukemia or Blastic Plasmacytoid Dendritic Cell Neoplasm[Effectiveness]
Time frame: 1month,3months
Objective response rate at 4W±7D and 3M±7D after CAR-NK infusion (Objective response rate includes CR, CRi)
4 secondary outcomes
AUCS of CD123 CAR-NK cells [Cell dynamics]
Time frame: 3 months
CMAX of CD123 CAR-NK cells [Cell dynamics]
Time frame: 3 months
TMAX of CD123 CAR-NK cells[Cell dynamics]
Time frame: 3 months
Pharmacodynamics of CD123 CAR-NK cells[Cell dynamics]
Time frame: 3 months
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 5 Aug 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.
Jia Wei, M.D principal investigator
Shanxi Bethune Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing CD123 targeted CAR-NK cells in people with Acute Myeloid Leukemia and Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN), run by Chongqing Precision Biotech Co., Ltd 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 13 inclusion and 14 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 5 Aug 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
To evaluate the safety of CAR-NK cell preparations in the treatment of Relapsed/Refractory Acute Myeloid Leukemia or Blastic Plasmacytoid Dendritic Cell Neoplasm[Safety and Tolerability] (time frame: 1 month). 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 5 Aug 2026.