Donor-derived CD117 CAR-T cells for relapsed or refractory acute myeloid leukemia

Donor Derived CD117 CAR-T Cells in the Treatment of Relapsed/Refractory Acute Myeloid Leukemia

This trial will try donor-derived CD117 CAR-T cells to treat patients with CD117-positive relapsed or refractory acute myeloid leukemia.

Quick facts

• Phase: Early Phase 1
• Study type: Interventional
• Enrollment: 50 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Zhejiang University (Academic / other)
• Drugs / interventions: chemotherapy, CAR-T
• Locations: 1 site (Hangzhou, Zhejiang)
• Trial ID: NCT07144020 on ClinicalTrials.gov

What this trial studies

This is a single-arm, open-label, dose-escalation trial planned to enroll 15–50 patients with CD117-positive relapsed or refractory AML. Participants receive donor-derived CD117-targeted CAR-T cells at escalating dose levels following conditioning chemotherapy. The study monitors safety, tolerability, adverse events, and preliminary anti-leukemia activity including response rates and duration of response. Procedures include donor leukapheresis for CAR-T manufacture, infusion, and serial clinical and laboratory follow-up.

Who should consider this trial

Good fit: Ideal candidates are patients whose leukemia is confirmed CD117-positive and who meet the study's relapsed or refractory AML definitions with no suitable standard therapies available.

Not a fit: Patients whose leukemia lacks CD117 expression, who have effective standard treatment options available, or who have contraindications to cellular therapy are unlikely to benefit from participation.

Why it matters

Potential benefit: If successful, this therapy could offer a new targeted cellular treatment that induces remissions in patients with relapsed or refractory AML who have limited options.

How similar studies have performed: CAR-T therapies for AML are experimental with limited early-phase human data, and donor-derived CD117-directed CAR-T is a relatively novel approach with little prior clinical evidence of success.

Eligibility criteria

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.

Where this trial is running

Hangzhou, Zhejiang

• The first affiliated hospital of medical college of zhejiang university — Hangzhou, Zhejiang, China (Recruiting)

Study contacts

• Principal investigator: He Huang, MD — First Affiliated Hospital of Zhejiang University
• Study coordinator: He Huang, MD

Conditions Acute Myeloid Leukemia; CD117 CAR-T