Actively Recruiting
Phase 1 Study of Lentivirally Transduced T Cells Engineered to Contain Anti-CD33 Linked to TCRζ And 4-1BB Signaling Domains Combined With CD33KO-HSPC in Refractory or Relapsed Acute Myeloid Leukemia
Led by University of Pennsylvania · Updated on 2025-06-29
16
Participants Needed
1
Research Sites
782 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating a new treatment approach for adults with refractory or relapsed acute myeloid leukemia (AML). This phase 1 study explores combining a modified stem cell transplant with engineered chimeric antigen receptor (CAR) T cells designed to recognize and attack AML cells. The modified transplant makes healthy bone marrow cells "invisible" to the CAR T cells to improve safety during treatment. Participants receive a stem cell transplant using donor blood-forming stem cells that have been gene-edited to remove a protein called CD33. After the transplanted stem cells begin producing healthy blood cells, patients receive one to three infusions of CAR T cells from the same donor. These CAR T cells are engineered to target CD33, a protein found on leukemia cells and healthy bone marrow cells. During the study, participants will be monitored for manufacturing feasibility and dose-limiting toxicities over several months. Researchers will assess treatment safety, efficacy, overall and progression-free survival, and duration of response up to 15 years. Assessments include regular clinical evaluations, laboratory tests, and imaging to track health, treatment effects, and leukemia progression. Participants will be followed closely throughout the study and beyond for up to 20 years.
CONDITIONS
Brief Title
CD33KO-HSPC Infusion Followed by CART-33 Infusion(s) for Refractory/Relapsed AML
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female 18 years of age or older
- Diagnosis of AML unlikely to be cured with current therapies
- AML not in complete remission or with relapsed disease after transplant
- Off systemic immunosuppression for at least 1 month if relapsed after prior transplant
- Suitable stem cell donor available
- Creatinine clearance greater than 40 ml/min
- ALT/AST less than or equal to 5 times upper limit of normal unless disease-related
- Direct bilirubin less than 2.0 mg/dl (or less than or equal to 3.0 mg/dl with Gilbert's syndrome)
- Left ventricular ejection fraction 40% or higher
- DLCO greater than 45% predicted
- ECOG performance status 0 or 1
- Written informed consent provided
- Agreement to use acceptable birth control if of reproductive potential
You will not qualify if you...
- Pregnant or lactating women
- Active hepatitis B, hepatitis C, or HIV infection
- Concurrent use of systemic steroids or immunosuppressant medications
- Uncontrolled active medical disorders that preclude participation
- Signs or symptoms of CNS leukemia involvement
- Known allergy or hypersensitivity to study product excipients
- Class III or IV cardiovascular disability by New York Heart Association Classification
- History of optic neuritis or other central nervous system inflammatory diseases unrelated to leukemia
- Clinically apparent or unstable arrhythmias within 2 weeks before enrollment
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 6 months
Participants receive a stem cell transplant with gene-edited cells to make the therapy safer, followed by 1 to 3 infusions of CAR T-cells designed to target leukemia cells.
Trial Site Locations
Total: 1 location
1
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
Research Team
A
Abramson Cancer Center Clinical Trials Service
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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