Demonstration Clinical Research Project

University Of Pennsylvania, Philadelphia PA

Investigators

Linked publications & trials

Abstract

The VCRC Pilot Project Program focuses on conducting early-phase l/ll clinical trials of promising new agents for vasculitis. We have chosen this focus based on substantial unmet needs in treatment of vasculitis, the presence of well-established systems the Vasculitis Clinical Research Consortium (VCRC) has in place to efficiently initiate and conduct such studies, the strong intellectual interest and experience of the VCRC to conduct such trials, the support of vasculitis Patient Advocacy Groups and partners in the biopharmaceutical industry, and the proven track record of the VCRC to conduct pilot projects of novel therapies with linked mechanistic studies. The VCRC has an established selection process for Pilot Projects in which a proposal is presented following which if there is strong interest a detailed protocol is developed. This review involves both physicians and biostatisticians at the Data Management and Coordinating Center (DMCC) early in the project development process leading to superior protocols and appropriate prioritization. The VCRC has identified 4 pilot project areas for which there are plans to pursue further study. The initial VCRC pilot project for this grant period will investigate gevokizumab, an interieukin-1 beta anatagonist, in the treatment of complicated large vessel vasculitis (LW). Study drug will be provided by Xoma pharmaceuticals and the study will be conducted at the National Institute of Arthritis and Musculoskeletal and Skin Diseases intramural translational research program, a VCRC member. The second pilot project area involves the conduct of a multicenter open-label pilot study within the VCRC of tofacitinib, a Janus kinase inhibitor, for treatment of relapsing LW. The third pilot project focuses on the investigation of novel therapeutic approaches for cutaneous vasculitis. The fourth pilot project involves the pursuit of a novel intervention to address fatigue in vasculitis. All these studies address important unmet medical needs. The VCRC Pilot Project Program will utilize the extensive resources and infrastructure of the Consortium to conduct innovate early-phase clinical trials of promising therapies for vasculitis.