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Observational Prolonged Trial In Myotonic dystrophy type 1 to Improve QoL-Standards, a Target Identification Collaboration

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.

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
Address
Houtlaan 4
6525 XZ Nijmegen
Netherlands
Activity type
Higher or Secondary Education Establishments
EU contribution
€ 1 344 550,40
Administrative Contact
Maarten Van Langen (Mr.)

Participants (8)

UNIVERSITY OF NEWCASTLE UPON TYNE
United Kingdom
EU contribution
€ 464 326
Address
Kings Gate
NE1 7RU Newcastle Upon Tyne
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Airey Fiona (Mrs.)
LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN
Germany
EU contribution
€ 263 200,80
Address
Geschwister Scholl Platz 1
80539 Muenchen
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Benedikt Schoser (Prof.)
ASSISTANCE PUBLIQUE HOPITAUX DE PARIS
France
EU contribution
€ 356 800,80
Address
3 Avenue Victoria
75000 Paris
Activity type
Research Organisations
Administrative Contact
Guillaume Bassez (Dr.)
UNIVERSITY OF GLASGOW
United Kingdom
EU contribution
€ 85 000
Address
University Avenue
G12 8QQ Glasgow
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Joe Galloway (Mr.)
UNIVERSITY OF DUNDEE
United Kingdom
EU contribution
€ 225 678
Address
Nethergate
DD1 4HN Dundee
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Zoe Kidd (Ms.)
CATT-SCI LIMITED
United Kingdom
EU contribution
€ 33 126
Address
Brampton Close 14
NN8 5XG Wellingborough
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Michael Catt (Mr.)
Concentris Research Management GmbH
Germany
EU contribution
€ 190 000
Address
Ludwigstr. 4
D-82256 Fürstenfeldbruck
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Ameli Schwalber (Dr.)
THE UNIVERSITY COURT OF THE UNIVERSITY OF ABERDEEN
United Kingdom
EU contribution
€ 37 318
Address
King's College Regent Walk
AB24 3FX Aberdeen
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Kerry Kidd (Mrs.)