CD123-targeted CAR-NK therapy for relapsed/refractory AML or BPDCN
Clinical Study of CD123 Targeting Chimeric Antigen Receptor NK Cells (CAR-NK) in the Treatment of Relapse/refractory Acute Myeloid Leukemia (AML)or Blastic Plasmacytoid Dendritic Cell Neoplasm(BPDCN)
This study is testing a new treatment using special immune cells to see if it can help people with relapsed or hard-to-treat acute myeloid leukemia or blastic plasmacytoid dendritic cell neoplasm.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 30 (estimated) |
| Ages | 18 Years to 75 Years |
| Sex | All |
| Sponsor | Chongqing Precision Biotech Co., Ltd Industry-sponsored |
| Drugs / interventions | chemotherapy |
| Locations | 1 site (Wuhan, Hubei) |
| Trial ID | NCT06690827 on ClinicalTrials.gov |
What this trial studies
This clinical trial evaluates the safety and efficacy of anti-CD123 CAR-NK cell therapy in patients with relapsed or refractory acute myeloid leukemia (AML) or blastic plasmacytoid dendritic cell neoplasm (BPDCN). It is a single-arm, open-label, dose-finding trial that includes three dose groups of CAR-positive cells. Patients will receive two infusions of CAR-NK cells, with the second dose potentially adjusted based on their condition. The primary goal is to determine the recommended dose for effective treatment.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 to 75 with CD123-positive relapsed or refractory AML or BPDCN.
Not a fit: Patients who do not express CD123 on their tumor cells or are outside the age range may not benefit from this study.
Why it matters
Potential benefit: If successful, this therapy could provide a new treatment option for patients with difficult-to-treat forms of leukemia.
How similar studies have performed: While CAR-NK therapies are a novel approach, similar studies targeting other antigens have shown promising results.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria:
1. Patients of any gender, aged between 18 and 75 years (inclusive);
2. Positive expression of CD123 on tumor cells detected by flow cytometry;
3. Patients with a confirmed diagnosis of CD123-positive relapsed/refractory AML or BPDCN:
(1) For AML patients：
* Relapsed refers to the reappearance of leukemic cells in peripheral blood after complete remission (CR), or ≥5% blasts in bone marrow (excluding other reasons such as bone marrow regeneration after consolidation chemotherapy), or the presence of leukemic cell infiltration outside the marrow;
* Refractory refers to patients who have not responded to two courses of standard treatment; patients who have relapsed within 12 months after CR and consolidation/intensification therapy; patients who have relapsed after 12 months but have not responded to conventional chemotherapy; patients with two or more relapses; patients with persistent extramedullary leukemia;
(2) For BPDCN patients: Patients who have not responded to or cannot tolerate the recommended salvage treatment according to guidelines, and have persistent or recurrent disease in any of the following: peripheral blood, bone marrow, lymph nodes, spleen, skin lesions, or other sites.
4\. Expected survival time of more than 12 weeks;
5\. ECOG score of 0-2 (Appendix 2);
6\. No severe mental disorders;
7\. Basic normal function of important organs:
1. Blood routine: white blood cells \>1.0×109/L, neutrophils \>0.5×109/L, lymphocytes \>0.5×109/L, platelets \>50×109/L;
2. Cardiac function: echocardiography indicates a left ventricular ejection fraction ≥50%, and no significant abnormalities on electrocardiogram;
3. Renal function: serum creatinine ≤2.0×ULN;
4. Liver function: ALT and AST ≤3.0×ULN (for patients with liver invasion
* 5.0×ULN);
5. Total bilirubin ≤2.0×ULN (for patients with Gilbert's syndrome ≤3.0×ULN);
6. Blood oxygen saturation \>92%. 8. The patient or their legal guardian agrees to participate in this clinical trial and signs the ICF, indicating their understanding of the purpose and procedures of the clinical trial and willingness to participate in the study.
Exclusion Criteria:
1. Presence of active central nervous system invasion during screening;
2. Receipt of anti-tumor therapies prior to screening, including chemotherapy, targeted therapy, or other experimental drug treatments within 14 days or at least 5 half-lives (whichever is shorter), except for those who have confirmed disease progression after treatment;
3. Occurrence of cerebrovascular accident or epileptic seizure within 6 months prior to screening;
4. Presence of active or uncontrolled infection requiring systemic treatment within 1 week prior to screening;
5. Presence of any of the following cardiac diseases:
1. Congestive heart failure at New York Heart Association (NYHA) class III or IV;
2. Myocardial infarction or coronary artery bypass grafting (CABG) within 6 months prior to enrollment;
3. Clinically significant ventricular arrhythmia, or history of unexplained syncope (excluding cases caused by vasovagal or dehydration);
4. History of severe non-ischemic cardiomyopathy;
6. Combination with active autoimmune diseases requiring long-term immunosuppressive therapy;
7. Presence of other malignancies, except for adequately treated carcinoma in situ of the cervix, basal cell or squamous cell carcinoma of the skin, localized prostate cancer after radical surgery, and ductal carcinoma in situ after radical surgery.
8. Receipt of live attenuated vaccines within 4 weeks prior to screening;
9. Pregnant or breastfeeding women, as well as male or female subjects who plan to have children within 1 year after receiving CAR-NK cell infusion;
10. Other conditions that the investigator deems unsuitable for participation in the study.
Where this trial is running
Wuhan, Hubei
- Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology. — Wuhan, Hubei, China (Recruiting)
Study contacts
- Study coordinator: Wei Jia, Professor
- Email: jiawei@tjh.tjmu.edu.cn
- Phone: +86 13986102084
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.