Defining Outcome Measures for Behavioural and Emotional Problems in Dystrophinopathies

Status: Recruiting
Location: See location...
Intervention Type: Behavioral
Study Type: Observational
SUMMARY

Study aims to develop and to evaluate the neurophysiological and physiological response to a classical conditioning task.To better understand how Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD) impacts mental health and how to assess it. Participants invited to complete questionnaires about behaviour, cognitive function and social interactions, complete computer tasks and have an optional MRI brain scan,

Eligibility
Participation Requirements
Sex: Male
Minimum Age: 7
Maximum Age: 17
Healthy Volunteers: t
View:

• DMD patients:

∙ Male

‣ Age range 7-17 years

‣ A genetically proven diagnosis of DMD.

‣ A genetic mutation that abrogates expression of Dp427 alone (assigned in DMD Group 1: Dp427-/Dp140+) or both Dp427 and Dp140 (assigned to DMD Group 2: Dp427-/Dp140-).

‣ Ability to consent/assent

• BMD patients:

⁃ Male

⁃ Age range 7-17 years

⁃ A genetically proven diagnosis of BMD.

⁃ A genetic mutation that decreases expression of Dp427 alone (assigned to BMD Group 1), of both Dp427 and Dp140 (assigned to BMD Group 2).

⁃ Ability to consent/assent

• Control participants:

⁃ Male

⁃ Age range 7-17 years.

⁃ Ability to consent/assent

Locations
Other Locations
United Kingdom
UCL GOS Institute of Child Health
RECRUITING
London
Contact Information
Primary
Anna Kolesnik, Dr
braindmd@ucl.ac.uk
44 (0) 20 7905 2600
Backup
Natasha Aslam, MSc
natasha.aslam@ucl.ac.uk
44 (0) 20 7905 2600
Time Frame
Start Date: 2022-09-13
Estimated Completion Date: 2024-12-31
Participants
Target number of participants: 100
Treatments
Cohort
* Social Responsiveness Scale (SRS - II)~* ADHD Rating scale (5th edition) (ADHD - RS V)~* Child Behaviour Checklist (CBCL)~* Revised Children's Anxiety and Depression Scale (RCADS-P)~* Behaviour Rating Inventory of Executive Function (BRIEF - P)~* Wechsler Intelligence test for children version V (WISC-V)~* Autism Diagnostic Observation Schedule (ADOS)~* Behavioural Assessment of the Dysexecutive Syndrome for Children (BADS-C)~* Psychophysiological task: A task previously used to measure the emotional response of patients through the usage of visual and auditory aversive stimuli. The emotional response will be measured through skin conductance, heartrate, pupil responses.~* Purdue Pegboard~* A subset of patients will be also asked to undergo an MRI brain scan
Sponsors
Leads: University College, London
Collaborators: Sarepta Therapeutics, Inc.

This content was sourced from clinicaltrials.gov