UPCC 02023 A Phase 1/2, Open-label, Safety and Dosing Study of Autologous CAR T 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 (ACC-RELEVANT)

Investigation of CAR T Cells in Treating Pemphigus Vulgaris

Enrolling By Invitation
18 years or above
All
Phase 1/2
40 participants needed
1 Location

Brief description of study

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

Detailed description of study

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 in two parts. The first part is the main study 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. This study is closed to enrollment.

The second part is a sub-study is being conducted to investigate if CABA-201, also called resecabtagene autoleucel, or "rese-cel", can be safely administered while achieving clinical responses without the need for preconditioning in mucosal-dominant PV (mPV) and mucocutaneous PV (mcPV) patients. This sub-study is open to enrollment.

DSG3-CAART or CABA-201 may potentially lead to complete and durable remission of disease.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: Pemphigus Vulgaris
  • Age: 18 years or above
  • Gender: All

Inclusion Criteria for DSG3-CAART: Closed to enrollment

  • Confirmed diagnosis of mPV by prior or screening biopsy and prior positive antiDSG 3 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.

This study investigates the use of autologous CAR T cells, specifically DSG3-CAART and CABA-201, in individuals with active pemphigus vulgaris (PV). This research aims to evaluate the safety and dosing of these investigational cell therapies in patients whose condition is not adequately controlled by standard treatments.

Participants in this study will receive either DSG3-CAART or CABA-201. The study is conducted in two parts: the first part, now closed, focused on determining the maximum tolerated dose of DSG3-CAART. The second part, which is open, is evaluating the safety and effectiveness of CABA-201 without preconditioning. Participants will be monitored for clinical responses and any adverse effects during the treatment.

  • Who can participate: Adults aged 18 and over with a confirmed diagnosis of pemphigus vulgaris, inadequately managed by standard immunosuppressive therapies, may be eligible. Key factors include a positive DSG3 ELISA test and active disease at screening.
  • Study details: Participants will receive an investigational cell therapy, either DSG3-CAART or CABA-201, and will be monitored for safety and clinical responses. The study aims to determine the effectiveness of these therapies without prior conditioning treatments.
Updated on 29 May 2026. Study ID: 20-0208
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