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There are limited options for treatment of relapse/refractory acute myeloid leukemia (AML). CD123 CAR-T cells may have an attractive and permanent effect on anti-tumor. This study purpose to estimate the safety and efficiency of CD123 CAR-T cells to patients with relapse/refractory AML.
CD123 is expressed on most myeloid leukemia cells so it is a ideal target for CAR-T. Some researches have revealed that CD123 is a marker of leukemia stem cells, which indicates that the eradication of CD123 cells may prevent relapse of leukemia. In this study, investigators will evaluate the safety and efficacy of CAR-T targeting CD123 in patients with Acute Myelocytic Leukemia. The primary goal is safety and efficiency assessment, including adverse events and disease status after treatment.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| CD123 CAR-T cells treat | Experimental | Patients will be be treated with CD123 CAR-T cells |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CD123 CAR-T cells | Biological | CD123 CAR-T cell therapy |
|
| Measure | Description | Time Frame |
|---|---|---|
| Adverse events that related to treatment | Therapy-related adverse events will be recorded and assessed according to the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE, Version 5.0). | 2 years |
| The response rate of CD123 CAR-T treatment in patients with relapse/refractory AML that treatment by CD123 CAR-T cells therapy | The response rate of CD123 CAR-T treatment will be recorded and assessed according to the National Comprehensive Cancer Network Guideline. | 2 years |
| Measure | Description | Time Frame |
|---|---|---|
| Cellular kinetics of CD123 CAR-T in Blood | In vivo (peripheral blood) rate and quantity of CD123 CAR-T cells were determined by means of flow cytometry and qPCR | 2 years |
| Cellular kinetics of CD123 CAR-T in Bone marrow |
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Inclusion Criteria:
Signed written informed consentï¼›
Diagnose as Relapsed/Refractory AML, and meet one of the following conditions:
Evidence for cell membrane CD123 expression;
KPS>60;
The expect time of survive is above 3 months;
Ages: 2 to 75 years;
All genders;
The patients that diagnosis as high risks, relapse/refractory or inconformity criteria to other therapyï¼›
No serious mental disorders;
Left ventricular ejection fraction ≥40%;
Sufficient hepatic function defined by ALT/AST<5 x ULN and bilirubin≤34.2μmol/L;
Sufficient renal function defined by creatinine clearance <220μmol/L;
Sufficient pulmonary function defined by indoor oxygen saturation≥92%;
No other illness may conflict with the protocol (e.g. autoimmune diseases, immune deficiency and organ transplantationï¼›
Ability and willingness to adhere to the study visit schedule and all protocol requirements.
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Zhi Yang, PhD | Contact | 86-13206140093 | yangzhi@precision-biotech.com |
| Name | Affiliation | Role |
|---|---|---|
| Sanbin Wang, MD | 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China | Principal Investigator |
| Cheng Qian, PhD | Chongqing University Cancer Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| 920th Hospital of Joint Logistics Support Force | Recruiting | Kunming | Yunnan | China |
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| ID | Term |
|---|---|
| D007938 | Leukemia |
| D007951 | Leukemia, Myeloid |
| D015470 | Leukemia, Myeloid, Acute |
| ID | Term |
|---|---|
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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In vivo (bone marrow) rate and quantity of CD123 CAR-T cells were determined by means of flow cytometry and qPCR
| 2 years |
| Cellular kinetics of CD123 positive cells in Bone marrow | In vivo (bone marrow) rate and quantity of CD123 positive cells were determined by means of flow cytometry | 1 years |
| Duration of Response (DOR) of CD123 CAR-T treatment in patients with refractory/relapsed AML | DOR will be assessed from the first assessment of CR or CRi to the first assessment of recurrence or progression of the disease or death from any cause (censored) | 2 years |
| Progress-free survival(PFS) of CD123 CAR-T treatment in patients with refractory/relapsed AML | PFS will be assessed from the first CAR-T cell infusion to death from any cause or the first assessment of progression (censored) | 2 years |
| Overall survival(OS) of CD123 CAR-T treatment in patients with refractory/relapsed AML | OS will be assessed from the first CAR-T cell infusion to death from any cause (censored) | 2 years |