A Multidimensional Profile of Dysphagia in Myotonic Dystrophy Type 1

• Research type

  Research Study

• Full title

  A Multidimensional Profile of Dysphagia in Myotonic Dystrophy Type 1 (DM1)

• IRAS ID

  317297

• Contact name

  Christina Smith

• Contact email

  christina.smith@ucl.ac.uk

• Sponsor organisation

  153848

• Duration of Study in the UK

  1 years, 5 months, 30 days

• Research summary

  This study will explore swallowing difficulties (dysphagia) caused by Myotonic Dystrophy Type 1 (DM1).

  Dysphagia can cause food, drink and saliva to travel onto the lungs. This is referred to as 'aspiration' which and can lead to pneumonia. Aspiration pneumonia is frequent and accounts for over 40% of deaths in DM1. Dysphagia often causes fear and anxiety which can lead to permanent lifestyle changes.

  A better understanding of dysphagia in DM1 will improve our assessment and treatment, and reduce its life-changing consequences.

  This research aims to examine dysphagia in people living with DM1 by studying their muscle structure and swallowing movements as well as their experience of dysphagia and their wellbeing. This will be done by:

  1. Investigating the size and structure of muscles involved in swallowing in people with and without DM1.

  2. Exploring how muscle size and structure are associated with i) swallowing function and ii) symptoms

  3. Exploring how swallowing function is associated with i) symptoms, ii) patient and caregiver wellbeing and iii) other aspects of DM1 such as walking and breathing.

  People aged 18+ with a confirmed diagnosis of DM1 will be invited to take part.
  Approximately 90 people will be recruited. They will undergo a series of tests including:

  - Ultrasound assessment of the swallowing muscles
  - An x-ray swallowing study (known as videofluoroscopy)
  - Assessment of swallowing symptoms, including questionnaires
  - Assessments of walking and breathing
  - Assessments of quality of life and wellbeing

  Approximately 20 people without DM1 will act as a control group. Primary caregivers will be invited to complete a wellbeing questionnaire.

  Data will be analysed using statistical methods and findings will be used to develop clinical practice recommendations for the assessment and treatment of dysphagia in DM1.

  The study will take place at The National Hospital for Neurology and Neurosurgery in London and will last approximately 18 months.
  

• REC name

  London - Central Research Ethics Committee

• REC reference

  23/LO/0073

• Date of REC Opinion

  3 Feb 2023

• REC opinion

  Favourable Opinion