Rare Bronchiectatic Diseases Consortium

Univ Of North Carolina Chapel Hill, Chapel Hill NC

Investigators

Abstract

ADMINISTRATIVE CORE ABSTRACT The mission of the Rare Bronchiectatic Diseases Consortium (RBDC) Administrative Core is to provide comprehensive and sustained administrative, logistical, and operational support for all Consortium partners to ensure the successful completion of the goals and projects associated with the RBDC. The Administrative Core’s leadership and activities will enable the successful completion of our Consortium’s proposed research projects aimed at improving our understanding of the pathophysiology of bronchiectasis in PCD, humoral immune deficiencies (HID), STAT3 hyper IgE syndrome (STAT3-HIES), and alpha-one antitrypsin deficiency (α1AT), which will lead to identifying clinical trial biomarkers/endpoints and potential therapeutic targets (treatable traits). The RBDC Administrative Core will support the missions of the Pilot/Feasibility Governance and Career Enhancement Cores, leading to future ancillary studies and the development of early-stage investigators. All this work will be conducted in close partnership with our Patient Advocacy Groups (PAGs) to assure that the voices of patients and families inform our strategy and critical decision-making. We will undertake five aims as part of this core: Aim 1: Deliver comprehensive operational and organizational administration of the RBDC, including the establishment and monitoring of standard operating procedures and policies, institutional review board proposals, resource utilization, funds allocation, contractual agreements with our partnering research sites and collaborating PAG and industry partners, monitoring of program milestones, and timely redress of potential problems and dispute resolution. Aim 2: Direct and facilitate regular, productive communication among collaborating investigators, institutions, and PAGs in a mutually supportive environment where each stakeholder has a voice during critical discussions. Aim 3: Develop and maintain productive, collaborative working relationships with investigators, PAGs, the NIH, the Office of Rare Diseases Research, the Observational Safety and Monitoring Board, and industry partners. The Core will serve as the central point of collaboration with the Rare Diseases Clinical Research Network (RDCRN) and the Data Management and Coordinating Center, assuring best practices for handling and sharing data and biospecimens. Aim 4: Collaborating with the RDCRN and PAGs, promote awareness of the purpose, progress, and meaningful outcomes of the RBDC to the clinical, scientific, and relevant patient/stakeholder communities; partner with these groups to enhance the success of the consortium and its impacts to improve the lives of patients and families. Aim 5: Establish an External Advisory Committee consisting of scientific, clinical, and patient-stakeholder representatives who will regularly provide consultation for the consortium to enhance operations and progress towards clinical trial readiness. The Administrative Core will help assure that we achieve the RBDC’s ultimate goal of enhancing our understanding of these rare diseases associated with bronchiectasis, ultimately leading to therapeutic targets and validated clinical trial readiness tools.