Status:
TERMINATED
Donor-Derived Anti-CD33 CAR T Cell Therapy (VCAR33) in Patients With Relapsed or Refractory AML After Allogeneic Hematopoietic Cell Transplant
Lead Sponsor:
Vor Biopharma
Conditions:
Leukemia, Myeloid, Acute
Eligibility:
All Genders
18+ years
Phase:
PHASE1
PHASE2
Brief Summary
This is a Phase 1/2, multicenter, open-label, first-in-human (FIH) study of donor-derived anti-CD33 Chimeric Antigen Receptor (CAR) T cell therapy (VCAR33) in patients with relapsed or refractory Acut...
Detailed Description
CD33 is a preferential target for AML CAR T cell therapy due to its surface expression on the majority (\>80%) of AML blasts and due to the extensive prior clinical experience demonstrating safety and...
Eligibility Criteria
Inclusion
- Patients aged ≥18 years
- Patients must have CD33+ AML in relapse or refractory after alloHCT
- Patients must be a recipient of an 8/8 (A, B, C, DRB1) HLA-matched related or unrelated donor alloHCT. Patients previously transplanted with VOR33 in the VBP101 study who have R/R AML may also be considered.
- Disease status at the time of enrollment:
- Arm A/Morphologic disease: Defined as ≥ 5% blasts (bone marrow) post-HCT
- Arm B/MRD positive: \< 5% blasts (bone marrow) with minimal residual disease of at least 0.1% CD33+ leukemia cells by flow cytometry
- Performance status: ECOG 0 or 1
- Patient must have adequate organ function as defined by:
- Cardiac: Left ventricular ejection fraction (LVEF) ≥ 45% or fractional shortening ≥ 28%
- Pulmonary: Baseline oxygen saturation \> 92% on room air at rest
- Hepatic: Total bilirubin \< 3x institutional upper limit of normal (ULN) (except in case of patients with documented Gilbert's disease \< 5x ULN) and aspartate aminotransferase (AST/SGOT)/alanine aminotransferase (ALT/SGPT) \< 5x institutional ULN
- Renal: Serum creatinine must be ≤ 1.2x institutional ULN or creatinine clearance ≥ 60 mL/min for patients with creatinine levels above institutional normal
- Original alloHCT donor is available and willing to undergo apheresis
Exclusion
- Patients who have undergone more than one alloHCT
- Patients who have undergone alloHCT with a mismatched unrelated donor, haploidentical donor, or with umbilical cord blood as the stem cell source
- Patients who will be less than 100 days post-alloHCT at the time of VCAR33 infusion.
- Patients with any history of Grade III or IV acute GVHD or severe chronic GVHD unless approved by the Sponsor Medical Monitor
- Patients with evidence of ongoing active acute or chronic GVHD and are taking systemic immunosuppressive agents (\> 10 mg daily of prednisone equivalent or other GVHD-directed treatment, including extracorporeal photopheresis). Patients with Grade 1 acute GVHD limited to the skin or mild chronic GVHD limited to the eyes, mouth, or skin controlled with only topical therapy are eligible.
- Patients with active CNS disease. A lumbar puncture is not required to exclude CNS disease in the absence of clinical signs or symptoms suggesting CNS disease.
- Patients with the following prior therapy:
- DLI within 28 days prior to enrollment
- Prior treatment with any CAR T cell therapy product
- Patients with active or uncontrolled viral, bacterial, or fungal infection
- Patients with a history of a human immunodeficiency virus (HIV) infection or acute or chronic active hepatitis B or C infection
- Patients with a history of malignancy other than nonmelanoma skin cancer or carcinoma in situ (e.g., cervix, bladder, or breast) unless disease free for at least 3 years after the last definitive therapy
- Female patients of childbearing potential who are pregnant or breastfeeding
Key Trial Info
Start Date :
December 11 2023
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 28 2025
Estimated Enrollment :
38 Patients enrolled
Trial Details
Trial ID
NCT05984199
Start Date
December 11 2023
End Date
May 28 2025
Last Update
July 29 2025
Active Locations (14)
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1
University of California San Diego Moores Cancer Center
La Jolla, California, United States, 92093
2
Stanford Cancer Institute
Stanford, California, United States, 94305
3
Miami Cancer Institute
Miami, Florida, United States, 33176
4
Winship Cancer Institute Emory University
Atlanta, Georgia, United States, 30322