Acute Myeloid Leukemia
Conditions
Keywords
CD117 CAR-T
Brief summary
A Clinical Study on the Safety and Effectiveness of Donor Derived CD117 CAR-T Cell in the treatment of Relapsed/Refractory Acute Myeloid Leukemia
Detailed 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.
Interventions
BIOLOGICALCD117 CAR T-cells
Each subject receive CD117 CAR T-cells by intravenous infusion
Sponsors
Yake Biotechnology Ltd.
Zhejiang University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Dose-limiting toxicity (DLT) | Up to 28 days after Treatment | Adverse events assessed according to NCI-CTCAE v5.0 criteria |
| Incidence of treatment-emergent adverse events (TEAEs) | Up to 2 years after Treatment | Incidence of treatment-emergent adverse events \[Safety and Tolerability\] |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Complete response (CR), and complete response with incomplete hematologic recovery (CRi) | Up to 12 weeks after CAR-T infusion | The proportion of patients with CR (complete response) /CRi (complete response with incomplete blood cell recovery) and PR (partial response). |
| Duration of remission ,DOR | Up to 1 years after CAR-T infusion | The time from CR/CRi and PR to disease relapsed or death due to disease progression after CAR-T infusion |
| Overall survival, OS | Up to 1 years after CAR-T infusion | The time from CAR-T infusion to death due to any cause |
| Leukemia-Free Survival, LFS | Up to 2 years after Treatment | The time from CAR-T infusion torecurrence or metastasis |
Countries
China
Contacts
Primary ContactHe Huang, MD
Backup ContactYongxian Hu, MD
Outcome results
None listed