Objective OPTIMISTIC is a proposal for a European collaborative project of doctors, scientists, relevant stakeholders (TREAT-NMD, patient organizations) and SMEs with the aim to improve clinical practice for patients suffering from a rare, inherited, and neglected disease, myotonic dystrophy type 1 (DM1). It is one of the most variable human diseases with complex, multi-systemic and progressively worsening clinical manifestations. Despite the huge impact of DM1 on the daily life of both patients and their family members, DM1 patients fail to receive the quality of healthcare that is available as they are not assertive users of the health care system.There is no cure for DM1. The aim of treatment is to relieve impairments, reduce limitations and support participation in everyday activities. Based on our DM1-specific model which shows that physical activity and experienced fatigue are main determinants of DM1 health status (Kalkman 2007), OPTIMISTIC investigates the effect of cognitive behavioural therapy (CBT) in combination with exercise training to improve functional capacity and to stimulate an active lifestyle.OPTIMISTIC compares the outcome of a treatment regimen with regular management in a multi-centre, assessor-blinded, randomized controlled trial, designed to 1) result in evidence-based clinical guidelines on exercise and cognitive behavioural therapy in DM1, 2) capture clinically meaningful changes in existing and novel outcome measures, and 3) identify both individual and composite biomarkers as surrogate treatment outcome measures that are reflective of the disease state.There is an urgent need for an European clinical trial infrastructure for DM1. High prioritization of this is required to enhance the speed of clinical development of new putative DM1 therapeutics approaching the market. OPTIMISTIC will provide this and safeguard the rapid uptake of the developed clinical guidelines ensuring improvements in DM1 care and quality of life. Programme(s) FP7-HEALTH - Specific Programme "Cooperation": Health Topic(s) HEALTH.2012.2.4.4-2 - Observational trials in rare diseases Call for proposal FP7-HEALTH-2012-INNOVATION-1 See other projects for this call Funding Scheme CP-FP - Small or medium-scale focused research project Coordinator STICHTING RADBOUD UNIVERSITEIT EU contribution € 1 344 550,40 Address HOUTLAAN 4 6525 XZ Nijmegen Netherlands See on map Region Oost-Nederland Gelderland Arnhem/Nijmegen Activity type Higher or Secondary Education Establishments Administrative Contact Maarten Van Langen (Mr.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data Participants (8) Sort alphabetically Sort by EU Contribution Expand all Collapse all UNIVERSITY OF NEWCASTLE UPON TYNE United Kingdom EU contribution € 464 326,00 Address KINGS GATE NE1 7RU Newcastle Upon Tyne See on map Region North East (England) Northumberland and Tyne and Wear Tyneside Activity type Higher or Secondary Education Establishments Administrative Contact Airey Fiona (Mrs.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN Germany EU contribution € 263 200,80 Address GESCHWISTER SCHOLL PLATZ 1 80539 Muenchen See on map Region Bayern Oberbayern München, Kreisfreie Stadt Activity type Higher or Secondary Education Establishments Administrative Contact Benedikt Schoser (Prof.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data ASSISTANCE PUBLIQUE HOPITAUX DE PARIS France EU contribution € 356 800,80 Address 55 BOULEVARD DIDEROT 75012 Paris See on map Region Ile-de-France Ile-de-France Val-de-Marne Activity type Research Organisations Administrative Contact Guillaume Bassez (Dr.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data UNIVERSITY OF GLASGOW United Kingdom EU contribution € 85 000,00 Address UNIVERSITY AVENUE G12 8QQ Glasgow See on map Region Scotland West Central Scotland Glasgow City Activity type Higher or Secondary Education Establishments Administrative Contact Joe Galloway (Mr.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data UNIVERSITY OF DUNDEE United Kingdom EU contribution € 225 678,00 Address Nethergate DD1 4HN Dundee See on map Region Scotland Eastern Scotland Angus and Dundee City Activity type Higher or Secondary Education Establishments Administrative Contact Zoe Kidd (Ms.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data CATT-SCI LIMITED United Kingdom EU contribution € 33 126,00 Address BRAMPTON CLOSE 14 NN8 5XG WELLINGBOROUGH See on map Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Administrative Contact Michael Catt (Mr.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data CONCENTRIS RESEARCH MANAGEMENT GMBH Germany EU contribution € 190 000,00 Address Ludwigstr. 4 82256 FURSTENFELDBRUCK See on map Region Bayern Oberbayern Fürstenfeldbruck Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Administrative Contact Ameli Schwalber (Dr.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data THE UNIVERSITY COURT OF THE UNIVERSITY OF ABERDEEN United Kingdom EU contribution € 37 318,00 Address KING'S COLLEGE REGENT WALK AB24 3FX ABERDEEN See on map Region Scotland North Eastern Scotland Aberdeen City and Aberdeenshire Activity type Higher or Secondary Education Establishments Administrative Contact Kerry Kidd (Mrs.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data