Open-label Study to Determine the Maximum Tolerated Dose of DSG3-CAART in Mucosal-dominant PV Patients (mPV)

About the study

Mucosal-dominant pemphigus vulgaris (mPV) is a B-cell mediated autoimmune disorder in which painful blisters are formed on the mucosal membrane, including the mouth, nose, throat, eyelids, anus, and genitals. This phase 1 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 mPV who are inadequately managed by standard therapies. DSG3-CAART may potentially lead to complete and durable remission of disease.

Who can take part

You may be eligible to participate in the study if you meet the following criteria:

INCLUSION CRITERIA

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

EXCLUSION CRITERIA

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 6 months
Absolute lymphocyte count < 1,000/µL at screening