Recruiting
Phase 1
Clinical Trial of CD123-targeted CAR-NK Therapy for Relapse/Refractory AML or BPDCN
Condition(s) studied
AML (Acute Myeloid Leukemia)BPDCN (Blastic Plasmacytoid Dendritic Cell Neoplasm)
Investigational drug(s) / intervention(s)
Anti-CD123 CAR NK cells
Anti-CD123 CAR NK cells: Each patient will receive two CAR-NK cell infusions at D0 and D7, and CAR-NK cells need to be controlled within 70 minutes from thawing to infusion completion.
Study summary
This is a clincal trial initiated by investigator to evaluate the safety and efficacy of anti-CD123 CAR-NK in the treatment of patients with relapsed/refractory acute myeloid leukemia or blastic plasma cell like dendritic cell tumors.
Eligibility
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.
Primary outcome measure(s)
- Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] — 28 dyas
The incidence of adverse events after CAR-NK cell infusion was assessed by CTCAE, version 5.0.
- Objective response rate (ORR), including complete response (CR) and partial response (PR), after CAR-NK infusion — 1month, 2 months, 3 months
- overall survival (OS) after CAR-NK infusion — 2 years
- Duration of response (DOR) after CAR-NK infusion — 2 years
- recurrence free survival (RFS) after CAR-NK infusion — 2 years
- event free survival (EFS) after CAR-NK cells infusion — 2 years
Trial sites (1)
| Facility | City | Region | Status |
| Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology. |
Wuhan |
Hubei |
Recruiting |
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