Defining Outcome Measures for Behavioural and Emotional Problems in Dystrophinopathies

ConditionsDMDBMD
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexMale
Age7-17
SponsorUniversity College, London

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

Qualifiers

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 design

Treatments tested in this trial

  • Classical conditioning task

Treatment groups

100 Participants
are divided into 1 treatment group

Sponsors and collaborators

University College, London

Lead sponsor

Sarepta Therapeutics, Inc.

Collaborator