A Phase 1 Study Evaluating BAFFR-targeting CAR T Cells for Patients with Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia
This phase I trial evaluates the side effects and best dose of BAFFR-CAR T cells in treating patients with B-cell Acute Lymphoblastic Leukemia that has come back (recurrent) or does not respond to treatment (refractory). T cells are infection fighting blood cells that can kill cancer cells. The T cells given in this study will come from the patient and will have a new gene put in them that makes them able to recognize BAFFR, a protein on the surface of cancer cells. These BAFFR-specific T cells may help the body's immune system identify and kill BAFFR+ cancer cells.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
24
First-in-human trial examining the safety and preliminary efficacy of BAFFR-CAR T cells in participants with r/r B-ALL
City of Hope Medical Center
Duarte, California, United States
Incidence of adverse events
Toxicity will be graded per Common Terminology Criteria for Adverse Events version 5.0, Cytokine Release Syndrome (CRS) and neurotoxicity which use the American Society for Transplantation and Cellular Therapy Consensus Criteria (ASTCT) and Graft versus Host Disease (GVHD) criteria. Toxicities will be followed from the start of lymphodepletion until the end of the study.
Time frame: Up to 1 year post treatment
Disease response
Defined as complete response \[CR\], or complete response with incomplete blood count recovery \[CRi\], or complete response with partial hematological recovery \[CRh\]. Response will be evaluated using European Leukemia Net (ELN) criteria. Rates and associated 95% binomial exact confidence limits will be estimated (CR/CRi/CRh) rate.
Time frame: Up to 1 year post treatment
Minimal residual disease (MRD)
Negative MRD is defined by malignant cells \< 0.01% by flow cytometry or clonoSEQ.
Time frame: Up to 1 year post treatment
B cell frequency
Measured by serum IgG level
Time frame: Up to 1 year post treatment
Severity of graft-versus-host disease (GVHD) in recipients of prior allogeneic hematopoietic stem cell transplantation
Defined per Keystone criteria for acute GVHD and revised National Institute of Health (NIH) consensus on grading of chronic GVHD.
Time frame: Up to 1 year post treatment.
Progression-free survival (PFS)
Kaplan-Meier product limit method with log-log transformation for the confidence interval will be used to estimate PFS.
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Time frame: From T cell infusion to the first observation of disease relapse/progression or death from any cause, whichever occurs first, assessed up to 15 years.
Overall survival (OS)
Kaplan-Meier product limit method with log-log transformation for the confidence interval will be used to estimate OS.
Time frame: From the day of BAFFR-CAR T cell infusion to death from any cause assessed, up to 15 years.