NCT03971799

Study of Anti-CD33 Chimeric Antigen Receptor-Expressing T Cells (CD33CART) in Children and Young Adults With Relapsed/Refractory Acute Myeloid Leukemia

Phase 1/2 Study of Anti-CD33 Chimeric Antigen Receptor-Expressing T Cells (CD33CART) in Children and Young Adults With Relapsed/Refractory Acute Myeloid Leukemia

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 52 (estimated)
• Sponsor: Center for International Blood and Marrow Transplant Research · Network
• Sex: All
• Age: 1 Year – 35 Years
• Healthy volunteers: Not accepted

Summary

This phase 1/2 trial aims to determine the safety and feasibility of antiCD33 chimeric antigen receptor (CAR) expressing T cells (CD33CART) in children and adolescents/young adults (AYAs) with relapsed/refractory acute myeloid leukemia (AML). The trial will be done in two phases: Phase 1 will determine the maximum tolerated dose of CD33CART cells using a 3+3 trial design, with dose-escalation for autologous products separated from dose-escalation for an allogeneic arm. Phase 2 is an expansion phase designed to evaluate the rate of response to CD33CART.

Detailed description

This study consists of two phases. The objectives of Phase 1 and Phase 2 are: Phase 1: Autologous Arm: To determine the maximum tolerated dose of lentivirally transduced autologous CD33-redirected CAR-T cells (CD33CART) in children and young adults with relapsed/refractory AML Allogeneic Arm: To determine the maximum tolerated dose of lentivirally transduced allogeneic CD33-redirected CAR-T cells (ALLO-CD33CART) in children and young adults with post-HSCT relapsed/refractory AML Phase 2: To determine the percentage of recipients treated with CD33CART who achieve morphologic remission (\<5% blasts in marrow) at Day 28 post-CD33CART cell infusion

Conditions

• Acute Myelogenous Leukemia

Interventions

Type	Name	Description
BIOLOGICAL	CD33CART autologous	The treatment regimen will consist of lymphodepleting (LD) chemotherapy followed by autologous CD33CART infusion: LD option #1 (IV fludarabine 25 mg/m2/dose administered Days -4 to -2 and IV cyclophosphamide 900 mg/m2/dose on Day -2) or LD option #2 (IV fludarabine 30 mg/m2 on days -5, -4, -3, and -2; and IV cyclophosphamide 500 mg/m2 on days -3 and -2). Subjects will then proceed to allogeneic HCT or alternative therapy as clinically applicable.
BIOLOGICAL	CD33CART allogeneic	The treatment regimen will consist of lymphodepleting (LD) chemotherapy followed by allogeneic CD33CART infusion: LD option #1 (IV fludarabine 25 mg/m2/dose administered Days -4 to -2 and IV cyclophosphamide 900 mg/m2/dose on Day -2) or LD option #2 (IV fludarabine 30 mg/m2 on days -5, -4, -3, and -2; and IV cyclophosphamide 500 mg/m2 on days -3 and -2). Subjects will then proceed to allogeneic HCT or alternative therapy as clinically applicable.

Timeline

• Start date: 2020-01-08
• Primary completion: 2029-12-01
• Completion: 2039-12-01
• First posted: 2019-06-03
• Last updated: 2025-07-14

Locations

6 sites across 1 country: United States

Regulatory

• FDA-regulated drug study