MedTrace Logo

Base Edited CAR T Cells Against AML: Deep Conditioning Ahead of Allogeneic Stem Cell Transplantation

NCT05942599 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2026-01-14

No results posted yet for this study

Summary

In this phase 1 clinical trial, the investigators are testing an experimental medicine in children aged 6 months up to 16 years with acute myeloid leukaemia (AML), which has come back (relapsed). The new product is made from white blood cells (T cells) collected from a healthy donor and changed so they can kill leukaemia cells. These 'ready-made' CAR T cells have been made using a new technique called Base Editing to modify their DNA code and have been given the code name 'BE CAR-33'. This technique allows them to work after chemotherapy and also disarms them to prevent effects against normal cells. The main purpose of this study is to assess the safety of the 'BE CAR-33' therapy and to see if ready-made CAR T cells can get rid of Acute Myeloid Leukaemia ahead of a planned bone marrow transplant that will hopefully prevent the leukaemia from returning.

Conditions

  • Relapsed Acute Myeloid Leukaemia

Interventions

BIOLOGICAL

Cryopreserved BE CAR33 T Cells (BE752TBTTBCAR33PBL)

Single-dose intravenous infusion (weight-based dosing) of a banded dose of CAR33+ T cells/kg. BECAR33 Total duration of treatment: 28 days follow up: 12 months

Sponsors & Collaborators

  • Wellcome Trust

    collaborator OTHER

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    lead OTHER

Principal Investigators

  • Waseem Qasim, Prof · Great Ormond Street Hospital

Study Design

Allocation
NA
Purpose
OTHER
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Months
Max Age
16 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-07-21
Primary Completion
2026-06-01
Completion
2026-06-01

Countries

  • United Kingdom

Study Locations

More Related Trials

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 NCT05942599 on ClinicalTrials.gov