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Double-Barreled attack: Gene-Edited stem cells and CAR T-Cells take on tough leukemia

NCT ID NCT05945849

Recruiting now Disease control Sponsor: University of Pennsylvania Source: ClinicalTrials.gov ↗

First seen Jul 09, 2026 · Last updated Jul 10, 2026 · Updated 1 time

Summary

This trial tests a two-part treatment for people with acute myeloid leukemia (AML) that has not responded to standard therapy. First, patients receive a stem cell transplant from a healthy donor, but the stem cells are gene-edited to hide from the second treatment: specially engineered CAR T-cells designed to seek and destroy leukemia cells. The goal is to make the transplant safer and more effective by protecting healthy bone marrow from the CAR T-cells.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
gene-edited stem cells (CD33KO-HSPC) and CAR T-cells (CART33)
What this could lead to
If successful, this approach could offer a new treatment option for people with hard-to-treat acute myeloid leukemia by making stem cell transplants safer and more effective.
What could go wrong
This is an early phase 1 trial with only 16 participants, so safety and effectiveness are not yet proven. There are risks from the stem cell transplant and CAR T-cell therapy, including serious side effects.

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.

acute myeloid leukemia

As listed by the trial registrant

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

LEUKEMIA, MYELOID, ACUTE

Contacts and locations

Study contacts

  • Contact

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

Locations

  • University of Pennsylvania

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

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

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