Defining Outcome Measures for Behavioural and Emotional Problems in Dystrophinopathies

Recruiting now

Conditions studied: DMD, BMD

In brief

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,

Key facts

Study ID

NCT06581887

Run by

University College, London

People needed

100

Starts

2022-09-13

Expected to finish

2024-12-31

Last updated by the study team

2024-09-03

Who can join

Age: 7 and older, up to 17. Sex: male. Healthy volunteers: accepted.

You may qualify if…

• 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

You may not qualify if…

• DMD \& BMD patients:
• 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).
• Control participants:
• Significant visual or hearing impairment
• Specific phobias or sensory sensitivities to stimuli similar to the ones used in this study
• Any diagnosis of neurological or psychiatric condition
• General exclusion criteria for MRI:
• Claustrophobia
• Pacemakers and defibrillators
• Nerve stimulators
• Intracranial clips
• Intraorbital or intraocular metallic fragments
• Cochlear implants
• Ferromagnetic implants (e.g. thoracic implant for scoliosis)
• Inability to lie supine during less than 45 minutes
• Not having a general practitioner
• Severe learning disability which will require a general anaesthetic

Where it is running

• UCL GOS Institute of Child Health — London, United Kingdom (enrolling)

Full record on ClinicalTrials.gov

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