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.

Public Health Relevance

The conduct of pilot phase I/II clinical trials linked to mechanistic studies is critical to scientific advancement in vasculitis. 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.

Agency
National Institute of Health (NIH)
Institute
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Type
Specialized Center--Cooperative Agreements (U54)
Project #
2U54AR057319-12
Application #
8918191
Study Section
Special Emphasis Panel (ZTR1-CI-8 (01))
Program Officer
Wang, Yan Z
Project Start
2014-09-01
Project End
2019-08-31
Budget Start
2014-09-01
Budget End
2015-08-31
Support Year
12
Fiscal Year
2014
Total Cost
$105,374
Indirect Cost
$6,745
Name
University of Pennsylvania
Department
Type
DUNS #
042250712
City
Philadelphia
State
PA
Country
United States
Zip Code
19104
Bingham 3rd, Clifton O; Bartlett, Susan J; Merkel, Peter A et al. (2016) Using patient-reported outcomes and PROMIS in research and clinical applications: experiences from the PCORI pilot projects. Qual Life Res 25:2109-16
Kermani, Tanaz A; Cuthbertson, David; Carette, Simon et al. (2016) The Birmingham Vasculitis Activity Score as a Measure of Disease Activity in Patients with Giant Cell Arteritis. J Rheumatol 43:1078-84
Sy, Alice; Khalidi, Nader; Dehghan, Natasha et al. (2016) Vasculitis in patients with inflammatory bowel diseases: A study of 32 patients and systematic review of the literature. Semin Arthritis Rheum 45:475-82
Merkel, Peter A; Manion, Michele; Gopal-Srivastava, Rashmi et al. (2016) The partnership of patient advocacy groups and clinical investigators in the rare diseases clinical research network. Orphanet J Rare Dis 11:66
Tamura, Roy N; Krischer, Jeffrey P; Pagnoux, Christian et al. (2016) A small n sequential multiple assignment randomized trial design for use in rare disease research. Contemp Clin Trials 46:48-51
Pagnoux, C; Carette, S; Khalidi, N A et al. (2015) Comparability of patients with ANCA-associated vasculitis enrolled in clinical trials or in observational cohorts. Clin Exp Rheumatol 33:S-77-83
Kermani, Tanaz A; Warrington, Kenneth J; Cuthbertson, David et al. (2015) Disease Relapses among Patients with Giant Cell Arteritis: A Prospective, Longitudinal Cohort Study. J Rheumatol 42:1213-7
Hatemi, Gulen; Ozguler, Yesim; Direskeneli, Haner et al. (2015) Current Status, Goals, and Research Agenda for Outcome Measures Development in Behçet Syndrome: Report from OMERACT 2014. J Rheumatol 42:2436-41
Aydin, Sibel Zehra; Direskeneli, Haner; Sreih, Antoine et al. (2015) Update on Outcome Measure Development for Large Vessel Vasculitis: Report from OMERACT 12. J Rheumatol 42:2465-9
Dejaco, Christian; Oppl, Bastian; Monach, Paul et al. (2015) Serum biomarkers in patients with relapsing eosinophilic granulomatosis with polyangiitis (Churg-Strauss). PLoS One 10:e0121737

Showing the most recent 10 out of 59 publications