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)

Last updated: March 14, 2025
Sponsor: Cabaletta Bio
Overall Status: Active - Recruiting

Phase

1

Condition

Epidermolysis Bullosa

Treatment

DSG3-CAART

DSG3-CAART or CABA-201

Clinical Study ID

NCT04422912
CAB-101
  • Ages > 18
  • All Genders

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 Criteria

Inclusion

Inclusion Criteria:

  • 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

  • Confirmed diagnosis of PV by prior or screening biopsy and prior positive DSG3ELISA, IIF, and/or DIF

  • PV inadequately managed by at least one standard immunosuppressive therapy

  • Active PV at screening

  • DSG3 ELISA positive at screening

Exclusion

Exclusion Criteria:

  • Active cutaneous lesions associated with PV that indicates mucocutaneous rather thanmucosal-dominant disease

  • Rituximab in last 12 months unless PV symptoms have recently worsened or anti-DSG3antibody 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 skincancer)

  • Have received rituximab or other anti-CD20 or anti-CD19 therapies in last 12 monthsunless anti-DSG3 antibody titers have recently increased or PV symptoms haverecently worsened

  • Prednisone > 0.25mg/kg/day

  • Other autoimmune disorder requiring immunosuppressive therapies

  • Investigational treatment in last 3 months

Study Design

Total Participants: 55
Treatment Group(s): 2
Primary Treatment: DSG3-CAART
Phase: 1
Study Start date:
September 29, 2020
Estimated Completion Date:
January 31, 2029

Study Description

Pemphigus vulgaris (PV) is a B-cell mediated autoimmune disorder in which painful blisters are formed on the skin or mucosal membrane, including the mouth, nose, throat, eyelids, anus, and genitals. This phase 1/2 study is being conducted to find the maximum tolerated dose and optimal fractionated infusion schedule of an investigational cell therapy, DSG3-CAART, that can be given to patients with mucosal PV who are inadequately managed by standard therapies. A sub-study will be conducted to investigate if CABA-201 can be safely administered while achieving clinical responses without the need for preconditioning in PV patients. DSG3-CAART or CABA-201 may potentially lead to complete and durable remission of disease.

Connect with a study center

  • Stanford University, Dept. of Dermatology

    Redwood City, California 94063
    United States

    Active - Recruiting

  • UC Davis, Dept. of Dermatology

    Sacramento, California 95816
    United States

    Active - Recruiting

  • Yale University

    New Haven, Connecticut 06520
    United States

    Active - Recruiting

  • Northwestern University

    Chicago, Illinois 60611
    United States

    Active - Recruiting

  • University of Iowa

    Iowa City, Iowa 52242
    United States

    Active - Recruiting

  • Brigham and Women's Hospital

    Boston, Massachusetts 02115
    United States

    Active - Recruiting

  • Columbia University

    New York, New York 10032
    United States

    Active - Recruiting

  • Mount Sinai - Icahn School of Medicine

    New York, New York 10029
    United States

    Site Not Available

  • University of North Carolina, Department of Dermatology

    Chapel Hill, North Carolina 27516
    United States

    Site Not Available

  • University of Pennsylvania

    Philadelphia, Pennsylvania 19104
    United States

    Active - Recruiting

  • UT Southwestern Medical Center, Dept. of Dermatology

    Dallas, Texas 75235
    United States

    Active - Recruiting

  • MD Anderson Texas Medical Center

    Houston, Texas 77030
    United States

    Active - Recruiting

  • University of Washington

    Seattle, Washington 98109
    United States

    Completed

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