Work Packages

WP01 Cognitive Behavioural Therapy (Lead: Hans Knoop, Radboud University Medical Centre, Nijmegen)

This team will work to develop a cognitive behavioural therapy protocol aimed at reducing fatigue and optimising interaction with others. The efficiency of the protocol will be tested on 114 patients with DM1 at locations in the Netherlands, Germany, France and United Kingdom. The ultimate goal of this team is to produce evidence based clinical guidelines aimed at improving quality of life in DM1 patients.

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WP02 Exercise Therapy (Lead: Mike Trenell, Newcastle University)

This team plans to design and deliver a support pathway implanting exercise therapy to improve quality of life in DM1 patients. These guidelines will be made available for use by patients and professionals to reduce fatigue and increase muscle strength. In addition this team will assess the cardiac implications of exercise therapy in DM1.

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WP03 Clinical outcome measures, effectiveness and adverse events (Lead: Guillaume Bassez, Assistance Publique-Hopitaux de Paris)

This team is working to assess the effectiveness of cognitive behavioural therapy and exercise therapy described in WP01 and WP02. They will also be monitoring to make sure no adverse events, or problems occur during these treatments. In addition they will establish the most effective outcome measures for use in future clinical trials.

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WP04 Cardiac Investigations (Lead: Mike Trenell, Newcastle University)

This group will work to define a standard operating procedure for the cardiac screening of patients with DM1. They will also support the team working on exercise therapy to understand the implications of this on cardiac function.

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WP05 Biomarkers and bioinformatics (Lead: Jeffrey Glennon, Radboud University Medical Centre, Nijmegen.)

This team aims to produce a biomarker profile from blood serum and urine samples before and after therapies; using next generation sequencing to assess miRNAs and mRNA levels. They will combine these findings with those of DNA (WP07) and data from other groups using the latest bioinformatics tools to create a “genetic network” in which to identify novel therapeutic targets, and create treatment outcome profiles. This will be compared to the machine learning approach used in WP08.

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WP06 Magnetic Resonance of Muscle (Lead: Arend Heerschap, Radboud University Medical Centre, Nijmegen)

This group will use magnetic resonance technology to perform measurements on about 100 patients before and after therapy. This information will be analysed and compared with controls looking for baseline biomarker values and changes caused by the treatments.

 

WP07 Genetic Analysis (Lead: Darren Monckton, University of Glasgow)

This team will look to increase the understanding of the genetic mechanism of DM1. They will confirm the molecular diagnosis of all patients involved in OPTIMISTIC. They will identify patients who have variant repeats within the CTG expansion. The group will also look to further develop a prognostic tool that can be used to better predict the course of DM1.

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WP08 Machine Learning (Lead: Tom Heskes, Radboud University Medical Centre, Nijmegen)

This team are concerned with the bringing together of all data in order to accurately assess quality of life in DM1 patients. They will develop novel machine learning algorithms to combine and handle the data collected in the project and use it to explain the findings.

 

WP09 Clinical trial coordination, standardisation and training (Lead: Shaun Treweek, University of Dundee)

This group are responsible for the pre-trial set-up and establishing the documentation and approvals that are needed to ensure the project can go ahead successfully, in addition they will be active in the recruitment and governance of the trial itself. They will also be in charge of ensuring the end of the trial is completed correctly and that all participants are informed of the outcome.

 

WP10 Dissemination, business development and ethics (Lead: Jeffrey Glennon, Radboud University Medical Centre, Nijmegen)

This group is involved in communicating with the scientific and general public about the project; ensuring that information and results are disseminated effectively. Furthermore this team will communicate with the external advisory board and patient association committee. This team is also in charge of protecting the intellectual property generated in the project.

 

WP11 Project Management (Lead: Ameli Schwalber, concentris)

Effective project management is a central element of successful research. This is because large research projects entail a lot of administrative work. The management WP makes sure that the project achieves its objectives and delivers in time, budget and quality its milestones and deliverables. It is furthermore concerned with communication, reporting, meeting organisation, financial management and intellectual property rights. The responsibility for project management lies primarily with the coordinator who is supported by concentris.