Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at UC Davis
Dates
study started
completion around
Principal Investigator
by Mehrdad Abedi, MD (ucdavis)

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 University of California Health

  • Mehrdad Abedi, MD (ucdavis)
    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
ID
NCT04422912
Phase
Phase 1 Pemphigus Research Study
Study Type
Interventional
Participants
Expecting 55 study participants
Last Updated