An Open Label Phase 2 A Exploratory Study of the Efficacy and Safety of Ublituximab in Patients with Pemphigus Vulgaris

Purpose: The aim of this phase 2A non-randomized, single-arm, open-label, single center clinical trial is to evaluate the efficacy and safety of a new recombinant chimeric monoclonoal antibody (Mab) against CD20 antigen named Ublituximab (UTX) in pemphigus vulgaris (PV). UTX has been used previously in oncologic indications. There is an FDA IND for use of UTX in PV. Pemphigus vulgaris a rare, painful, blistering autoimmune disease that normally occurs in people 50 years and older. PV is treated with corticosteroids and other immunosuppressive therapy; treatment is challenging, with few patients able to achieve clinical remission. Primary objective: To explore the safety and potential of UTX to achieve control of disease activity and complete remission of disease on minimal therapy, or off therapy, over 12 months. Eligibility criteria: Patients with active disease who have been previously unresponsive to conventional therapy and/or unable to use corticosteroids, who have a skin activity score of greater than or equal to 5 on the Pemphigus Disease Area Index (PDAI) and who meet diagnostic and other criteria will be eligible for the study. Up to 10 patients will be recruited from among the patients who attend the clinical practices of Drs. Werth and Payne, or who are referred to the practices by outside physicians. UTX, 450 mg, will be infused in the Penn CTRC once weekly x 4 weeks over about 3-8 hrs period. The 12 study visits will include a Screening visit, 5 Treatment visits, 5 Follow-p visits and an End of Study visit. Patients will be premedicated with appropriate meds to lower risk of infusion-related reactions. Main study interventions include physical and skin exams, ECGs, PKs, safety and translational labs. Subjects will complete about 12 study visits. Recruitment will begin about June 2016 and continue for about two years.