The TREAT-NMD Care and Trial Site Registry: A powerful tool for clinical research on neurodegenerative and neuromuscular diseases

<p>Established in 2007 in the scope of the TREAT-NMD project (EU-funded Network of Excellence, FP6) to identify possible trial sites for rare neuromuscular diseases, the Care and Trial Site Registry (CTSR) collects information on personnel and the experience of the study team, facilities and equipment, as well as patient numbers per disease and age group.</p> <p>Within the CARE-NMD project (2010-2013, funded by DG Sanco) the CTSR was extended with Duchenne-specific care questions and used to evaluate current clinical practice in different European countries.</p> <p>In September 2013, the CTSR expanded to cover the field of rare neurodegenerative diseases as a branch of NeurOmics (FP7, 2012-2017) and now encompasses 32 rare diseases.</p> <p>The CTSR is an online self-registration database hosted by the University Medical Center Freiburg, Germany. This allows swift self-registration and update of information by any web browser and regardless of geographic location. Once registered, data is entered into online forms organised by topic categories such as Patient Cohort, Diagnostic Tools, Personnel and Experience, Equipment, Care Settings and Research and Education. The quality of the data is ensured by regularly contacting the sites and asking them to update their records, usually when an official enquiry has been submitted to the CTSR.</p> <p>As of September 2013 the CTSR contained 280 centres in 44 countries with an overall count of 41,500 reported patients.</p> <p>Fourteen official requests for site identification, often in combination with TREAT-NMD patient registry information, were received between 2009 and 2013: Ten were trial feasibiltiy enquiries from major industrial firms and four enquiries from academia..</p> <p>As an ideal complement to patient registries, the CTSR provides a powerful infrastructure for feasibility and trial site selection by pharmaceutical industry and investigators, as well as improving clinical networking in rare diseases.</p