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 (RESET-PV)
DSG3-CAART: Intravenous infusions of DSG3-CAART alone at different doses and different fractionations, with or without intravenous immunoglobulin, cyclophosphamide, and/or fludarabine.
CABA-201: Single intravenous infusion of CABA-201 at escalating doses, with or without preconditioning.
Study summary
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
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria for DSG3-CAART: Closed to enrollment
* 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
Inclusion Criteria for CABA-201 sub-study: Open to enrollment
* Age ≥18
* Confirmed diagnosis of PV by prior or screening biopsy and prior positive DSG3 ELISA, IIF, and/or DIF
* PV inadequately managed by at least one standard immunosuppressive therapy
* Active PV at screening
* DSG3 ELISA positive at screening
Exclusion Criteria:
* 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
* Investigational treatment in last 3 months
Exclusion Criteria for CABA-201 sub-study
* Have paraneoplastic pemphigus or active malignancy (not including non-melanoma skin cancer) or malignancy diagnosed within the previous 5 years
* Have received rituximab or other anti-CD20 or anti-CD19 therapies in last 12 months unless anti-DSG3 antibody titers have recently increased or PV symptoms have recently worsened
* Prednisone \> 0.25mg/kg/day
* Other autoimmune disorder requiring immunosuppressive therapies
* Treatment with any investigational agent within 4 weeks or 5 half-lives, whichever is longer.
Primary outcome measure(s)
Adverse events, including Dose Limit Toxicity — 3 months Incidence of adverse events that are related to DSG3-CAART therapy
For CABA-201 Sub-study: To evaluate adverse events reported by subjects — Up to 28 days after CABA-201 infusion Incidence and severity of AEs
Trial sites (13)
Facility
City
Region
Status
Stanford University, Dept. of Dermatology
Redwood City
California
Recruiting
UC Davis, Dept. of Dermatology
Sacramento
California
Recruiting
Yale University
New Haven
Connecticut
Recruiting
Northwestern University
Chicago
Illinois
Recruiting
University of Iowa
Iowa City
Iowa
Recruiting
Brigham and Women's Hospital
Boston
Massachusetts
Recruiting
Mount Sinai - Icahn School of Medicine
New York
New York
Withdrawn
Columbia University
New York
New York
Recruiting
University of North Carolina, Department of Dermatology
Chapel Hill
North Carolina
Withdrawn
University of Pennsylvania
Philadelphia
Pennsylvania
Recruiting
UT Southwestern Medical Center, Dept. of Dermatology
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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