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

NCT03971799 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 52

Last updated 2025-07-14

No results posted yet for this study

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.

Conditions

  • Acute Myelogenous Leukemia

Interventions

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.

Sponsors & Collaborators

Principal Investigators

  • Nirali Shah, MD, MHSc · National Cancer Institute (NCI)

  • Richard Aplenc, MD, PhD · Children's Hospital of Philadelphia

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Year
Max Age
35 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-01-08
Primary Completion
2029-12-31
Completion
2039-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03971799 on ClinicalTrials.gov