Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at Sacramento, California and other locations
Dates
study started
completion around
Principal Investigator
by Mehrdad Abedi, MD

Description

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

Official Title

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

Details

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.

Keywords

Pemphigus Vulgaris, Pemphigus, CAAR-T Therapy, CAR-T Therapy, Desmoglein 3, Cell Therapy, Autoimmune Disease, Autoimmunity, Skin Diseases, Vesiculobullous, Immunotherapy, Adoptive, Immune System Diseases, CABA-201, Anti-CD19 CAR-T therapy, DSG3-CAART or CABA-201

Eligibility

You can join if…

Open to people ages 18 years and up

  • 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 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

You CAN'T join if...

  • 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)
  • 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
  • Investigational treatment in last 3 months

Locations

  • UC Davis, Dept. of Dermatology accepting new patients
    Sacramento California 95816 United States
  • Stanford University, Dept. of Dermatology accepting new patients
    Redwood City California 94063 United States

Lead Scientist at UC Davis

  • Mehrdad Abedi, MD
    Professor, MED: Int Med HMCTBMT, School of Medicine. Authored (or co-authored) 71 research publications

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Cabaletta Bio
Links
Sign up for this study
ID
NCT04422912
Phase
Phase 1 Pemphigus Research Study
Study Type
Interventional
Participants
Expecting 55 study participants
Last Updated