NCT05945849 · University of Pennsylvania
CD33KO-HSPC Infusion Followed by CART-33 Infusion(s) for Refractory/Relapsed AML
(CART33)
What this study is about
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.
View original scientific description
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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Male or female 18 years of age or older
- Subjects with AML unlikely to be cured with currently available therapies
- 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:
- 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:
- Subjects with relapsed disease after prior transplant must be off systemic immunosuppression for at least 1 month at the time of enrollment.
- Subjects must have a suitable stem cell donor.
- Satisfactory organ function
- Creatinine clearance \> 40 ml/min
- ALT/AST must be ≤ 5x upper limit of normal unless related to disease and \< 20 x upper limit of normal if related to disease
- Direct bilirubin \< 2.0 mg/dl, unless subject has Gilbert's syndrome (≤ 3.0 mg/dL)
- Left ventricular ejection fraction ≥ 40% as confirmed by echocardiogram or MUGA
- DLCO \> 45% predicted
- ECOG performance status 0-1
- Written informed consent is given
- Subjects of reproductive potential must agree to use acceptable birth control methods
Exclusion criteria
- Pregnant or lactating (nursing) women
- Active hepatitis B or hepatitis C or HIV infection
- Concurrent use of systemic steroids or immunosuppressant medications
- Any uncontrolled active medical disorder that would preclude participation as outlined
- Subjects with signs or symptoms indicative of CNS involvement.
- Known history of allergy or hypersensitivity to study product excipients (human serum albumin, DMSO, and Dextran 40)
- Class III/IV cardiovascular disability according to New York Heart Association Classification
- Subjects with clinically apparent arrhythmia, or arrhythmias that are not stable on medical management, within 2 weeks of the screening/enrollment visit.
Where
- Philadelphia, Pennsylvania
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jul 9, 2026 · Source of record for eligibility and locations