NCT05942599Active, not recruitingPhase 1
Base Edited CAR T Cells Against AML: Deep Conditioning Ahead of Allogeneic Stem Cell Transplantation
Phase 1 Study of Base Edited CAR T Cells Against AML: Deep Conditioning Ahead of Allogeneic Stem Cell Transplantation
Source: ClinicalTrials.gov · Last updated 14 Jan 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 10 (estimated)
- First submitted
- 4 Jul 2023
- Start date
- 21 Jul 2023
- Primary completion date
- 1 Jun 2026
- Lead sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
- Diseases
- Acute myeloid leukemia, Childhood cancers
- NCT number
- NCT05942599
What you commit to
- Study type: Interventional · Phase 1
- Planned participants: 10
- From 21 Jul 2023 to the primary completion date, 1 Jun 2026
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Presence of donor-specific anti-HLA antibodies directed against BE-CAR33
- GvHD requiring systemic therapy
- Systemic steroid therapy prednisolone \>0.5mg/kg/day
- 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.
- Prior CAR T cell therapy known to be associated with ≥Grade 3 cytokine release syndrome (CRS) or ≥Grade 3 drug-related CNS toxicity
Other illness
- Evidence of disease progression after cytoreduction
- National Comprehensive Cancer Network guidelines)
- 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.
Other
- Patients/parents unwilling to undergo follow-up for 15 years
- Foreseeable poor compliance to the study procedures
- Uncontrollable CNS leukaemia or neurological symptoms defined as CNS grade 3 (per
- Absence of suitable HLA matched or mismatched donor
- Weight \< 6kgs
- Lactating female participants unwilling to stop breastfeeding
Who the study is looking for
- 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.
Age in the registry: 6 months to 16 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Great Ormond Street Hospital for Children Coordinator of this site? Confirm its status | London | United Kingdom | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.