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ACTIVE NOT RECRUITING

NCT05942599

PHASE1

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

Sponsor: Great Ormond Street Hospital for Children NHS Foundation Trust

View on ClinicalTrials.gov

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.

Official title: Phase 1 Study of Base Edited CAR T Cells Against AML: Deep Conditioning Ahead of Allogeneic Stem Cell Transplantation

Key Details

Gender

All

Age Range

6 Months - 16 Years

Study Type

INTERVENTIONAL

Enrollment

Start Date

2023-07-21

Completion Date

2026-06-01

Last Updated

2026-01-14

Healthy Volunteers

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

Inclusion Criteria: * Male or female patients * Age ranging between 6 months and \<16 years Medical and therapeutic criteria * Relapsed AML ahead of scheduled allogeneic haematopoietic stem cell transplantation (allo-SCT). * Morphologically confirmed with leukemic blasts in the bone marrow (\>5%) or a quantifiable MRD by multiparameter flow cytometry and/or quantitative polymerase chain reaction (\>10-4) * CD33+ leukaemia associated immunophenotype (LAIP) on \>95% of blasts * Eligible and fit for allogeneic hematopoietic stem cells transplantation with suitable donor available * Estimated life expectancy ≥ 12 weeks * Lansky (age \< 16 years at the time of assent/consent) or performance status ≥ 70; * Eastern Cooperative Oncology Group ECOG performance status \< 2. Exclusion Criteria: * Patients/parents unwilling to undergo follow-up for 15 years * Foreseeable poor compliance to the study procedures * Evidence of disease progression after cytoreduction * Uncontrollable CNS leukaemia or neurological symptoms defined as CNS grade 3 (per * National Comprehensive Cancer Network guidelines) * Absence of suitable HLA matched or mismatched donor * Weight \< 6kgs * Presence of donor-specific anti-HLA antibodies directed against BE-CAR33 * GvHD requiring systemic therapy * Systemic steroid therapy prednisolone \>0.5mg/kg/day * Known hypersensitivity to test materials or related compounds * Active bacterial, fungal or viral infections not controlled by standard of care anti- microbial or anti-viral treatment. Uncontrolled bacteraemia/ fungaemia is defined as the ongoing detection of bacteria/fungus on blood cultures despite antibiotic or anti-fungal therapy. Uncontrolled viraemia is defined as rising viral loads on two consecutive occasions despite antiviral therapy. * Risk of pregnancy or non-compliance with contraception (if applicable). Girls of childbearing potential must have been tested negative in a pregnancy test within 14 days prior to inclusion. * Lactating female participants unwilling to stop breastfeeding * Prior CAR T cell therapy known to be associated with ≥Grade 3 cytokine release syndrome (CRS) or ≥Grade 3 drug-related CNS toxicity

Locations (1)

Great Ormond Street Hospital for Children

London, United Kingdom