Experimental CAR T-Cell therapy takes on tough childhood leukemia

NCT ID NCT07464951

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

acute myeloid leukemia leukemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Email: •••••@•••••

Locations

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Email: •••••@•••••