NCT06499025

Treatment of Relapsed or Refractory t(8; 21) AML With Targeted AML1-ETO Neoantigen Cytotoxic T Cells (CTL)

A Clinical Study to Evaluate the Safety and Initial Efficacy of Targeted AML1-ETO Neoantigen Cytotoxic T Cells (CTL) in the Treatment of Relapsed or Refractory t(8; 21) AML

• Status: Enrolling By Invitation
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 16 (estimated)
• Sponsor: BGI, China · Academic / Other
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

1. To evaluate the safety and tolerability of targeted AML1-ETO neoantigen cytotoxic T cells (CTL) in the treatment of relapsed or refractory acute myeloid leukemia . 2. To evaluate the effectiveness of targeted AML1-ETO neoantigen cytotoxic T cells (CTL)，by the complete response rate(CRR) and overall survival (OS) followed.

Detailed description

This is a single arm、open label and non-randomied clinical trial ，divided into dose exploration phase (Part A) and dose extension phase (Part B). Part A: Plan to enroll six subjects to evaluate the safety and tolerabilty of targeted neoantigen cytotoxic T cells (CTL),determine dose-limiting toxicity(DLT),explore the maximum tolerated dose (MTD) or the recommended dose for later clilnical studies.The DLT observation period is 28 days after the infusion of targeted neoantigen cytotoxic T cells (CTL) iniection. One dose group(total number of cells is 5×10\^7/bag) and the another one (total number of cells is 10×10\^7 /bag )is setted by the 3+3 test design. Part B: Ten subjects are planned to be enrolled in the dose-exploration phase with the recommended dose, to further evaluate the safety、tolerability and its efficacy of targeted neoantigen cytotoxic T cells (CTL) in relapsed or refractory acute myeloid leukemia.

Conditions

• AML
• t(8;21)
• Neoantigen

Interventions

Type	Name	Description
BIOLOGICAL	targeted AML1-ETO neoantigen cytotoxic T cells (CTL)	After subject screening, peripheral blood mononuclear cell #PBMC# donors matching half or more of the subject's HLA matching will undergo blood collection to prepare neoantigen cytotoxic T cells. Neoantigen cytotoxic T cells preparation is expected to be 25-30 days after blood collection. In this study, the bridging therapy will be allowed before Chemotherapy preconditioning. Chemotherapy preconditioning will be performed before neoantigen cytotoxic T cells transfusion.
DRUG	Cyclophosphamide injection	Cyclophosphamide injection will be performed in -10 to -8d before neoantigen cytotoxic T cells transfusion
DRUG	Decitabine Injection	Decitabine Injection will be performed in -12 to -8d before neoantigen cytotoxic T cells transfusion
DRUG	Liposome mitoxantrone	Liposome mitoxantrone will be performed in -9 to -8d before neoantigen cytotoxic T cells transfusion

Timeline

• Start date: 2024-02-01
• Primary completion: 2026-12-30
• Completion: 2027-12-30
• First posted: 2024-07-12
• Last updated: 2025-08-28

Locations

1 site across 1 country: China