DuchenneConnect Patient-Report Registry Infrastructure Project

Details and Information

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Areas of Focus: Duchenne and Becker muscular dystrophy

Population Characteristics: Pediatric onset including Adults with DBMD and women who are carriers

Organization: Parent Project Muscular Dystrophy

Contact: [email protected]  or 888-520-8675

Principal Investigator: Ann Lucas, MS, CGC

Co-Principal Investigator: Holly Peay, PhD, MS CGC

About DuchenneConnect

DuchenneConnect (DCN) is a program of Parent Project Muscular Dystrophy (PPMD), the largest and most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne and Becker muscular dystrophy (DBMD). DBMD is a set of rare, progressive neuromuscular disorders that cause progressive muscle weakness and early death. Onset is typically in the pediatric years.

DuchenneConnect is a self-report registry that began collecting data in 2007 as a community resource to facilitate and speed research in DBMD. The fact that DCN is a program of an advocacy organization has strongly influenced the registry’s goals, processes, and governance. Patient and caregiver engagement is at the heart of advocacy organization and is deeply ingrained in our culture. We currently have close to 4,000 registrants in DCN from over 100 countries.

DCN has successfully achieved Phase I goals of patient and caregiver engagement and governance, enhanced data collection, and rigorous but streamlined informed consent. We have improved recruitment with a continued focus on outreach to underserved communities. Our data is “analysis-ready” in compliance with the Common Data Model. DCN successfully engaged our communities to identify resource priorities, prioritize topic domains, assess face validity of existing or purposively developed PROs, and rapidly contact our registrants for purposes of recruitment into clinical trials and research studies. In addition, DCN is developing robust, mutually beneficial relationships with CDRNs and PPRNS where the needs and priorities of patients and caregivers are actualized through collaborative research and decision support tools.

New opportunities in Phase II include the integration of PPMD’s Certified Duchenne Care Centers (CDCCs). These CDCCs represent a robust, engaged network of expert clinicians who are proven partners in addressing community-identified research priorities. DCN is also implementing a community-engaged request for proposals, which will contribute to the identification of research priorities, the development of multi-stakeholder research groups, the leveraging of PPMD resources to support protocol and proposal development, DCN mentoring, and avenues to sustainability funding. DCN will continue to contribute to PCORnet as a PPRN that is directly accountable to patients and caregivers; as a network with years of collaborative research experience with academic, industry, and patients and families; in our emerging capacity to integrate electronic health record data; and most importantly as an engaged network responsive to the needs of our stakeholders. DCN is anticipating an unparalleled opportunity to answer questions of high importance and identify and address unmet needs related to our progressive, fatal pediatric disorder. We continue to value the PCORnet philosophy and experience and anticipate continued efforts to engage fully with our esteemed network partners.

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Last updated on August 25, 2016