Researchers in Newcastle and London have opened recruitment for a new natural history study in myotonic dystrophy type 1. This is an observational study and does not involve the trial of any treatment or therapy. This study is important for doctors and researchers to understand as much as possible about myotonic dystrophy type 1 and how it affects people in different ways, this will help treatments being developed in the laboratory and be tested in clinical trials in the future.
The study will include up to 400 people with myotonic dystrophy type 1 and will involve visiting a study site twice approximately 12 months apart. Each visit will last approximately four hours and during both visits participants will be asked to complete exactly the same assessments. This will include walking tests, assessments of muscle strength and function as well as questionnaires that ask about myotonic dystrophy and how it affects them; this will include questions about pain, fatigue and quality of life. Participation in this study is entirely voluntary. Deciding to take part or not will not affect the care the participants receive.
This research has been funded by the National Institute of Health Research (NIHR), who are supporting lots of projects looking at rare conditions like myotonic dystrophy type 1. The study is being led by Professor Hanns Lochmüller and Dr Chris Turner and is called PHENODM1.
If you would like to take part you must have a diagnosis of myotonic dystrophy type 1 (DM1), be over 18 years old and able to carry out assessments involved such as walking for six minutes and completing questionnaires.
For more information or if you are interested in taking part in Newcastle at the Royal Victoria Infirmary as part of the John Walton Muscular Dystrophy Research Centre please contact Libby Wood: Elizabeth.firstname.lastname@example.org, 0191 241 8640
If you are interested in taking part in London, Queen Square at the National Hospital for Neurology and Neurosurgery please contact Louise Spiegel: Louise.Speigel@uclh.nhs.uk, 020 344 88015
Read more about myotonic dystrophy
Download the patient information sheet