About this trial
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 criteria
This trial accepts healthy volunteersQualifiers
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-).
Disqualifiers
Significant visual or hearing impairment
Specific phobias or sensory sensitivities to stimuli similar to the ones used in this study
Current participation in a clinical trial investigating a new drug involved in dystrophin modulation.
Inability to consent (for parents/guardians or self-reporting participants aged 16 and 17) or assent. This will exclude the rare individuals with extremely severe learning disability, as the assent in these patients is impossible (or the consent in self-reporting participants aged 16 and 17).
Trial population
40 DMD, 30 BMD patients and 30 age-matched controls.
Trial design
Cohort
Cross-sectional
Treatments tested in this trial
Classical conditioning task
BehavioralTo evaluate the neurophysiological and physiological response to a classical conditioning task, which is comparable to findings that have been made in the mdx dystrophic mouse (deficient in Dp427). The investigators will assess correlations between the specific DMD/BMD genotype and susceptibility to conditioning, as well as the relationship between conditioning and behavioural/emotional characteristics of the syndrome.
Treatment groups
Trial outcomes
Primary outcomes
Group differences between DMD, BMD and controls in the initial aversive unconditioned stimulus.
Following an emotional response task, an interim analysis will be done after the first 30 patients have been tested (10 DMD, 10 BMD, 10 controls). A favourable outcome will demonstrate a difference in the emotional response of these groups. Groups will complete questionnaires, an emotional response task, and a fine motor assessment.
Secondary outcomes
To observe any difference between and within BMD, DMD and control groups in regard to learning, habituation and extinction
This will be measured by analysis measuring any difference between and within groups (10 BMD, 10 DMD and 10 control). Physiological responses generated by the task will be measured, along with neural imaging.
Sponsors and contacts
Click on the lead sponsor to view all of their trials.
University College, London
Lead sponsor
Sarepta Therapeutics, Inc.
Collaborator