Experimental CAR T-Cell therapy takes on tough childhood leukemia
NCT ID NCT07464951
First seen Jun 27, 2026 · Last updated Jul 08, 2026 · Updated 2 times
Summary
This early-phase trial is testing a new treatment called CART123 for children and young adults with acute myeloid leukemia (AML) that has come back or not responded to standard therapy. CART123 cells are specially engineered immune cells designed to find and kill leukemia cells. Some participants will also receive a drug called ruxolitinib to see if it helps the therapy work better. The main goals are to check safety and find the best dose.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- CART123 cells (a type of immune cell therapy targeting CD123) and ruxolitinib (a JAK inhibitor drug)
- What this could lead to
- If successful, this could lead to a new treatment option for children and young adults with hard-to-treat acute myeloid leukemia (AML).
- What could go wrong
- This is an early Phase 1 trial with only 30 participants, so it is primarily testing safety and dosing. The therapy may not work well, and there are risks of serious side effects like cytokine release syndrome or organ damage.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for ACUTE MYELOID LEUKEMIA (AML) are added.
By submitting, you agree to our Terms of use
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
-
Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a new pill boost the power of an existing leukemia treatment?
- Can a new drug combo outperform standard care for a tough blood cancer?
- Can donor immune cells fight leukemia relapse after transplant?
- Can a liposomal chemo combo outsmart tough leukemias?
- Massive AML trial aims to find best chemo and transplant protection for younger patients
- New drug cocktail aims to stop leukemia return after transplant