Status:
RECRUITING
Defining Outcome Measures for Behavioural and Emotional Problems in Dystrophinopathies
Lead Sponsor:
University College, London
Collaborating Sponsors:
Sarepta Therapeutics, Inc.
Conditions:
DMD
BMD
Eligibility:
MALE
7-17 years
Brief 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 ...
Detailed Description
The investigation aims to develop and to evaluate the neurophysiological and physiological response to a classical conditioning task, which is comparable to findings that have been made in the mdx dys...
Eligibility Criteria
Inclusion
- 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
Exclusion
- 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
Key Trial Info
Start Date :
September 13 2022
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
December 31 2024
Estimated Enrollment :
100 Patients enrolled
Trial Details
Trial ID
NCT06581887
Start Date
September 13 2022
End Date
December 31 2024
Last Update
September 3 2024
Active Locations (1)
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1
UCL GOS Institute of Child Health
London, United Kingdom, WC1N 1EH