Global Registry and Trial Readiness
In the previous funding periods, GENOMIT formed a global registry initiative including the nationwide clinical networks of Germany/Austria/Switzerland (mitoNET), Italy (Mitocon), UK and USA,as well as the national center in Paris for the recruitment and standardized phenotyping of mitochondrial patients. The registry allows for both cross-sectional and longitudinal (by virtue of regular follow-up visits) data collection and analysis. Thus, it provides key groundwork to improve the number and quality of outcome measures and to design future clinical studies which will be feasible by increased trial readiness of patients. In the third funding period, the global registry will be joined by new countries, e.g. by the mitochondrial center of Japan (CCB) covering a very large population.
The primary aim of this work package is to use the global registry to define natural history and outcome measures for mitochondrial disorders and to enable the design of clinical trials.
This work package is coordinated by Friedrich-Baur-Institute, Dept. of Neurology, LMU Hospital Munich in cooperation with partners Klinikum rechts der Isar (IHG TUM-MED, Munich), Salzburger Landeskliniken (SALK), Institute Imagine (INSERM, Paris), Foundation IRCCS Neurological Institute C. Besta (Milan), University of Pisa, Newcastle University (WCMR) and Chiba Children´s Hospital (CCH, Chiban, Japan).