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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
16
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.
University of Pennsylvania
Philadelphia, Pennsylvania, United States
RECRUITINGManufacturing feasibility
Proportion of subjects whose Product 1 (CD33KO-HSPC) meets release criteria.
Time frame: 1 month
Occurrence of dose-limiting toxicities related to CD33KO-HSPC
Safety of alloHSCT: occurrence of dose-limiting toxicities related to CD33KO-HSPC
Time frame: 3 months
Occurrence of dose-limiting toxicities related to CART-33
Safety of CART-33: occurrence of dose-limiting toxicities related to CART-33
Time frame: 6 months
Efficacy of CD33KO-HSPC
Proportion of subjects with hematopoietic engraftment according to standard criteria
Time frame: 1 month
Efficacy of at least 1 dose of CART-33
Proportion of subjects with residual or recurrent AML before CART-33 infusion who attain a clinical response
Time frame: 6 months
Overall Survival (OS)
Proportion of patients who are alive at 6 months and at 12 months
Time frame: 6 months, 12 months
Progression free survival (PFS)
Proportion of patients who remained in response at 6 and 12 months after attaining a response to the first CART-33 infusion. Median time to progression of AML from infusion of CART-33.
Time frame: 6 months, 12 months
Duration of Response (DOR)
Median number of months in remission. Median time to relapse in patients who receive CART-33 and attain a response.
Time frame: 15 years
Abramson Cancer Center Clinical Trials Service
CONTACT
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