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RecruitingLeukemia, Myeloid, Acute

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

Eligible age

18+ yrs

Accepts

All genders

Locations

1 state

Healthy volunteers

No

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About this study

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.

Sponsor: University of Pennsylvania

You may qualify if…

  • ✓ 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

You may not qualify if…

  • ✕ 1. Pregnant or lactating (nursing) women
  • ✕ 2. Active hepatitis B or hepatitis C or HIV infection
  • ✕ 3. Concurrent use of systemic steroids or immunosuppressant medications
  • ✕ 4. Any uncontrolled active medical disorder that would preclude participation as outlined
  • ✕ 5. Subjects with signs or symptoms indicative of CNS involvement.
  • ✕ 6. Known history of allergy or hypersensitivity to study product excipients (human serum albumin, DMSO, and Dextran 40)
  • ✕ 7. Class III/IV cardiovascular disability according to New York Heart Association Classification
  • ✕ 8. Subjects with clinically apparent arrhythmia, or arrhythmias that are not stable on medical management, within 2 weeks of the screening/enrollment visit.

Where it's recruiting

Pennsylvania

Philadelphia

Source: ClinicalTrials.gov · NCT05945849 · last updated 2026-07-09

CD33KO-HSPC Infusion Followed by CART-33 Infusion(s) for Refractory/Re · TrialPath