RecruitingPhase 1NCT05945849

CD33KO-HSPC Infusion Followed by CART-33 Infusion(s) for Refractory/Relapsed AML

Phase 1 Study of Lentivirally Transduced T Cells Engineered to Contain Anti-CD33 Linked to TCRζ And 4-1BB Signaling Domains In Combination With CD33KO-HSPC In Subjects With Refractory Or Relapsed Acute Myeloid Leukemia


Sponsor

University of Pennsylvania

Enrollment

16 participants

Start Date

Feb 23, 2024

Study Type

INTERVENTIONAL

Conditions

Summary

The purpose of this study is to provide a new type of treatment for AML. This treatment combines a new type of stem cell transplant along with treatment using chimeric antigen receptor (CAR) T cells that have been engineered to recognize and attack your AML cells. The first treatment is a modified stem cell transplant, using blood-forming stem cells donated from a healthy donor. From the same donor, we will also make CAR T-cells, which are leukemia fighting cells, which will be given to the patient via an infusion into the vein after the transplanted stem cells have started to grow healthy blood cells. The modification of the stem cell transplant means that the healthy bone marrow cells will be "invisible" to the CAR T-cells that are trying to kill the leukemia cells.


Eligibility

Min Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This study is testing a novel two-step treatment for relapsed or refractory acute myeloid leukemia (AML) — a fast-moving blood cancer. First, patients receive specially modified stem cells that lack a protein called CD33. Then, they receive CAR-T cell therapy that targets CD33. This approach aims to destroy leukemia cells while protecting the healthy blood stem cells. **You may be eligible if...** - You are 18 or older with AML that has not responded to standard therapies or has relapsed - Your leukemia has not achieved remission, or it has returned after a prior stem cell transplant - Your overall health (ECOG 0–2) allows you to tolerate the treatment - You have adequate organ function and are off immune-suppressing medications (if post-transplant) **You may NOT be eligible if...** - You have active, uncontrolled infection - You have had a prior solid organ transplant - Your leukemia involves the brain or spinal cord - You have significant heart, lung, liver, or kidney problems Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

BIOLOGICALCD33KO-HSPC; CART33

CD33KO-HSPC: Stem cell transplant (also known as bone marrow transplant) is a common treatment used for patients with blood cancers, but for this transplant we will first modify the cells, in order to make the CAR T-cell treatment safer for when the patient receives them later. The modification is a type of gene editing - this means changing the DNA of the cells, so that a protein that the bone marrow stem cells usually show on their surface is not shown any more. This makes the bone marrow cells "invisible" to the CAR T-cells, and makes this therapy safer for the patient. The protein is called CD33. CART33: Chimeric Antigen Receptor T-cells (CART) are immune cells which are modified by adding a CAR molecule, which makes them much more efficient at finding and killing cancer cells. In this case, the CAR T-cells are programmed to target a protein called CD33, which is found on the surface of leukemia cells, and on healthy bone marrow cells.


Locations(1)

University of Pennsylvania

Philadelphia, Pennsylvania, United States

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NCT05945849


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