RecruitingPhase 1Phase 2NCT04422912

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)

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


Sponsor

Cabaletta Bio

Enrollment

40 participants

Start Date

Sep 29, 2020

Study Type

INTERVENTIONAL

Conditions

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

Min Age: 18 Years

Inclusion Criteria9

  • 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
  • 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 Criteria10

  • 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
  • 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.

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Interventions

BIOLOGICALDSG3-CAART

Intravenous infusions of DSG3-CAART alone at different doses and different fractionations, with or without intravenous immunoglobulin, cyclophosphamide, and/or fludarabine.

BIOLOGICALCABA-201

Single intravenous infusion of CABA-201 at escalating doses, with or without preconditioning.


Locations(13)

Stanford University, Dept. of Dermatology

Redwood City, California, United States

UC Davis, Dept. of Dermatology

Sacramento, California, United States

Yale University

New Haven, Connecticut, United States

Northwestern University

Chicago, Illinois, United States

University of Iowa

Iowa City, Iowa, United States

Brigham and Women's Hospital

Boston, Massachusetts, United States

Mount Sinai - Icahn School of Medicine

New York, New York, United States

Columbia University

New York, New York, United States

University of North Carolina, Department of Dermatology

Chapel Hill, North Carolina, United States

University of Pennsylvania

Philadelphia, Pennsylvania, United States

UT Southwestern Medical Center, Dept. of Dermatology

Dallas, Texas, United States

MD Anderson Texas Medical Center

Houston, Texas, United States

University of Washington

Seattle, Washington, United States

View Full Details on ClinicalTrials.gov

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NCT04422912


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