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Recruiting Phase 1 NCT05945849

NCT05945849 CD33KO-HSPC Infusion Followed by CART-33 Infusion(s) for Refractory/Relapsed AML

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Clinical Trial Summary
NCT ID NCT05945849
Status Recruiting
Phase Phase 1
Sponsor University of Pennsylvania
Condition Leukemia, Myeloid, Acute
Study Type INTERVENTIONAL
Enrollment 16 participants
Start Date 2024-02-23
Primary Completion 2029-02-23

Trial Parameters

Condition Leukemia, Myeloid, Acute
Sponsor University of Pennsylvania
Study Type INTERVENTIONAL
Phase Phase 1
Enrollment 16
Sex ALL
Min Age 18 Years
Max Age N/A
Start Date 2024-02-23
Completion 2029-02-23
Interventions
CD33KO-HSPC; CART33

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Brief 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 Criteria

Inclusion Criteria: 1. Male or female 18 years of age or older 2. Subjects with AML unlikely to be cured with currently available therapies 1. AML that has not achieved a complete remission or morphologic leukemia free state by ELN criteria; partial remission or refractory disease (including primary refractory) are eligible; OR: 2. AML relapsed following allogeneic stem cell transplantation (including MDS evolved to AML post-allogeneic stem cell transplantation). Note: morphologic relapse is not required; persistent/recurrent disease-associated molecular, phenotypic or cytogenetic abnormalities (measurable residual disease, MRD) at any time after allogeneic HCT is eligible; OR: 3. Subjects with relapsed disease after prior transplant must be off systemic immunosuppression for at least 1 month at the time of enrollment. 3. Subjects must have a suitable stem cell donor. 4. Satisfactory organ function 1. Creatinine clearance \> 40 ml/min 2. ALT/AST must be ≤ 5x upper limit of normal unles

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