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

Part of paid clinical trials in Philadelphia, Pennsylvania.

Sponsor
University of Pennsylvania
Study ID
NCT05945849
Phase
PHASE1
Status
Recruiting
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Conditions

  • Leukemia, Myeloid, Acute

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • CD33KO-HSPC; CART33 — BIOLOGICAL
    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.

Study Details

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.

Key Dates

Start date
Feb 23, 2024
Status verified
Jun 2025
Primary completion
Feb 23, 2029
Completion
Feb 23, 2044

Study Design

Enrollment
16 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: CD33KO-HSPC followed by CART33
    All subjects will receive CD33KO-HSPC, followed by 1-3 CART-33 infusions

Primary Outcome Measure

Manufacturing feasibility [ Time Frame: 1 month ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
University of PennsylvaniaPhiladelphiaPennsylvania19104
Abramson Cancer Center Clinical Trials Service
[email protected]
215-349-8245

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By research site
University of Pennsylvania· Philadelphia, PA

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