CD33KO-HSPC Infusion Followed by CART-33 Infusion(s) for Refractory/Relapsed AML
- Conditions
- Leukemia, Myeloid, Acute
- Registration Number
- NCT05945849
- Lead Sponsor
- University of Pennsylvania
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 16
Inclusion Criteria:<br><br> 1. Male or female 18 years of age or older<br><br> 2. Subjects with AML unlikely to be cured with currently available therapies<br><br> 1. AML that has not achieved a complete remission or morphologic leukemia free<br> state by ELN criteria; partial remission or refractory disease (including<br> primary refractory) are eligible; OR:<br><br> 2. AML relapsed following allogeneic stem cell transplantation (including MDS<br> evolved to AML post-allogeneic stem cell transplantation). Note: morphologic<br> relapse is not required; persistent/recurrent disease-associated molecular,<br> phenotypic or cytogenetic abnormalities (measurable residual disease, MRD) at<br> any time after allogeneic HCT is eligible; OR:<br><br> 3. Subjects with relapsed disease after prior transplant must be off systemic<br> immunosuppression for at least 1 month at the time of enrollment.<br><br> 3. Subjects must have a suitable stem cell donor.<br><br> 4. Satisfactory organ function<br><br> 1. Creatinine clearance > 40 ml/min<br><br> 2. ALT/AST must be = 5x upper limit of normal unless related to disease and < 20 x<br> upper limit of normal if related to disease<br><br> 3. Direct bilirubin < 2.0 mg/dl, unless subject has Gilbert's syndrome (= 3.0<br> mg/dL)<br><br> 5. Left ventricular ejection fraction = 40% as confirmed by echocardiogram or MUGA<br><br> 6. DLCO > 45% predicted<br><br> 7. ECOG performance status 0-1<br><br> 8. Written informed consent is given<br><br> 9. Subjects of reproductive potential must agree to use acceptable birth control<br> methods<br><br>Exclusion Criteria:<br><br> 1. Pregnant or lactating (nursing) women<br><br> 2. Active hepatitis B or hepatitis C or HIV infection<br><br> 3. Concurrent use of systemic steroids or immunosuppressant medications<br><br> 4. Any uncontrolled active medical disorder that would preclude participation as<br> outlined<br><br> 5. Subjects with signs or symptoms indicative of CNS involvement.<br><br> 6. Known history of allergy or hypersensitivity to study product excipients (human<br> serum albumin, DMSO, and Dextran 40)<br><br> 7. Class III/IV cardiovascular disability according to New York Heart Association<br> Classification<br><br> 8. Subjects with a known history or prior diagnosis of optic neuritis or other<br> immunologic or inflammatory disease affecting the central nervous system, and<br> unrelated to leukemia or previous leukemia treatment.<br><br> 9. Subjects with clinically apparent arrhythmia, or arrhythmias that are not stable on<br> medical management, within 2 weeks of the screening/enrollment visit.
Not provided
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Manufacturing feasibility;Occurrence of dose-limiting toxicities related to CD33KO-HSPC;Occurrence of dose-limiting toxicities related to CART-33
- Secondary Outcome Measures
Name Time Method Efficacy of CD33KO-HSPC;Efficacy of at least 1 dose of CART-33;Overall Survival (OS);Progression free survival (PFS);Duration of Response (DOR)