A phase 1/2, open-label, safety and dosing study of autologous CART cells (desmoglein 3 chimeric autoantibody receptor T cells \[DSG3-CAART\] or CD19-specific Chimeric Antigen Receptor T cells \[CABA-201\]) in subjects with active, pemphigus vulgaris
Confirmed diagnosis of mPV by prior or screening biopsy and prior positive anti- DSG3 antibody ELISA
mPV inadequately managed by at least one standard immunosuppressive therapies
Active mPV at screening
Anti-DSG3 antibody ELISA positive at screening
Active cutaneous lesions associated with PV that indicates mucocutaneous rather than mucosal-dominant disease
Rituximab in last 12 months unless PV symptoms have recently worsened or anti-DSG3 antibody titers have recently increased
Prednisone > 0.25mg/kg/day
Other autoimmune disorder requiring immunosuppressive therapies
Sequential
DSG3-CAART
Biological/VaccineIntravenous infusions of DSG3-CAART alone at different doses and different fractionations, with or without intravenous immunoglobulin, cyclophosphamide, and/or fludarabine.
CABA-201
Biological/VaccineSingle intravenous infusion of CABA-201 at escalating doses, with or without preconditioning.
Adverse events, including Dose Limit Toxicity
Incidence of adverse events that are related to DSG3-CAART therapy
For CABA-201 Sub-study: To evaluate adverse events reported by subjects
Incidence and severity of AEs
Percent of CAAR-transduced cells
Percent of total cells for infusion that are CAAR-transduced cells by flow cytometry
Total DSG3-CAART positive cells
Total DSG3-CAART positive cells for each manufacturing run by flow cytometry
Cellular kinetics profile of DSG3-CAART
Cellular kinetics profile of DSG3-CAART assessed by quantitative polymerase chain reaction
Change in DSG3 autoantibody titer
Change in DSG3 autoantibody titer by ELISA compared to pre-infusion visit
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