Safety and Efficacy of CD123-Targeted CAR-T Therapy for Relapsed/Refractory Acute Myeloid Leukemia

PBC016

This is a single arm study to evaluate the efficacy and safety of CD123-targeted CAR-T cells therapy for patients with relapsed/refractory Acute Myeloid Leukemia.

2019-12-01

2023-12-31

2024-07-01

Recruiting

Early phase I

40

Inclusion Criteria: 1. Signed written informed consent; 2. Diagnose as relapsed /refractory acute myeloid leukemia, and meet one of the following conditions: 1. Failed to standard chemotherapy regimens; 2. Relapse after complete remission, high-risk and / or refractory patients ; 3. Relapse after hematopoietic stem cell transplantation; 3. Evidence for cell membrane CD123 expression; 4. All genders, ages: 3 to 75 years； 5. The expect time of survive is above 12 weeks; 6. KPS\>60； 7. No serious mental disorders ; 8. Left ventricular ejection fraction ≥50% 9. Sufficient hepatic function defined by ALT/AST≤3 x ULN and bilirubin≤2 x ULN; 10. Sufficient renal function defined by creatinine clearance≤2 x ULN; 11. Sufficient pulmonary function defined by indoor oxygen saturation≥92%; 12. With single or venous blood collection standards, and no other cell collection contraindications; 13. Ability and willingness to adhere to the study visit schedule and all protocol requirements. Exclusion Criteria: 1. Have received CAR-T therapy or other genetically modified cell therapy before screening； 2. Participated in other clinical research within 1 month before screening； 3. Have received the following anti-tumor treatment before screening: Have received chemotherapy, targeted therapy or other experimental drug treatment within 4 weeks, except those who have confirmed disease progression after treatment; 4. Live attenuated vaccine within 4 weeks before screening; 5. Convulsion or stoke within past 6 months; 6. Previous history of other malignancy; 7. Presence of uncontrolled active infection; 8. Subjects with positive HBsAg or HBcAb positive and peripheral blood HBV DNA titer is higher than the lower limit of detection of the research institution; HCV antibody positive and peripheral blood HCV RNA titer is higher than the lower limit of detection of the research institution; HIV antibody positive; syphilis primary screening antibody positive; 9. Pregnant or breasting-feeding women; 10. Any situation that investigators regard not suitable for attending in this study or may affect the data analysis.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 40, 'type': 'ESTIMATED'}}

2023-04-18