Gene-Edited super cells take on childhood leukemia
NCT ID NCT05942599
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial tests a new treatment for children aged 6 months to 16 years whose acute myeloid leukemia has returned. The therapy uses donor immune cells that have been gene-edited with a technique called base editing to better target and kill leukemia cells. The main goal is to check safety and see if the cells can clear the cancer before a bone marrow transplant.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Base-edited CAR T cells (BE CAR-33) made from donor white blood cells
- What this could lead to
- If it works, this could offer a way to clear leukemia before a bone marrow transplant, potentially preventing the cancer from coming back.
- What could go wrong
- This is a very early, small phase 1 trial with only 10 children. The therapy may cause severe side effects like cytokine release syndrome or graft-versus-host disease, and it may not work for everyone.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Great Ormond Street Hospital for Children
London, WC1N3JH, United Kingdom