Overview
A Clinical Study on the Safety and Effectiveness of Donor Derived CD117 CAR-T Cell in the treatment of Relapsed/Refractory Acute Myeloid Leukemia
Description
This is a single-arm, open-label, dose-escalation clinical trial to evaluate the safety and efficacy of CD117 CAR-T Cell in patients with relapsed or refractory acute myeloid leukemia. It is planned to enroll 15-50 participants in this trial.
Eligibility
Inclusion Criteria:
- 1\. Patients with a histologically or immunophenotypically confirmed diagnosis of CD117-positive Acute Myeloid Leukemia (AML).
- 2\. Diagnosis must meet the 2016 WHO classification criteria for AML and fulfill the definitions for relapsed or refractory disease per the \Chinese Guidelines for the Diagnosis and Management of Relapsed/Refractory Acute Myeloid Leukemia (2017 Edition)\, with no available suitable standard therapeutic options or registered clinical trials.
- a). Relapsed AML: Defined as the reappearance of leukemic blasts in the peripheral blood, bone marrow blast count \>5% (when assessed morphologically, after excluding regenerative changes post-consolidation chemotherapy), or development of extramedullary disease after achieving a Complete Remission (CR).
- b). Refractory AML (meeting at least one criterion): Failure to achieve CR following two cycles of standard induction therapy in newly diagnosed patients; relapse within 12 months after CR following consolidation therapy; relapse beyond 12 months that fails to respond to conventional salvage chemotherapy; ≥2 relapses; or persistent extramedullary leukemia.
- 3\. Presence of \>5% bone marrow blasts (by morphology) and/or \>1% (by flow cytometric analysis).
- 4\. Total bilirubin ≤1.5 × ULN (≤51 μmol/L) ALT and AST ≤3 × ULN Serum creatinine ≤1.5 × ULN (≤176.8 μmol/L)
- 5\. Left ventricular ejection fraction (LVEF) ≥50% as assessed by echocardiography.
- 6\. Oxygen saturation ≥92% on room air.
- 7\. Life expectancy ≥3 months.
- 8\. Eastern Cooperative Oncology Group (ECOG) Performance Status of 0, 1, or 2.
- 9\. For patients of childbearing potential: Agreement to use highly effective contraception from screening, throughout the study treatment period, and for at least 6 months after the cell infusion (due to unknown risks to the fetus).
- 10\. Voluntary participation, understanding of the study procedures, and provision of written informed consent by the patient or their legally authorized representative.
Exclusion Criteria:
- 1\. Patients with the history of epilepsy or other CNS disease;
- 2\. Patients with prolonged QT interval time or severe heart disease;
- 3\. Active infection with no cure;
- 4\. Active infection of hepatitis B virus or C virus ;
- 5\. Before using any gene therapy products;
- 6\. The proiferation rate is less than 5 times response to CD3/CD28 co-stimulation signal;
- 7\. Suffering from other uncontrolled diseases that the researchers consider unsuitable for joining;
- 8\. Infected with AIDS virus;
- 9\. Any situation that researchers believe may increase the risk to the subjects or interfere with the trial results.
