Defining Outcome Measures for Behavioural and Emotional Problems in Dystrophinopathies
NCT ID: NCT06581887
Last Updated: 2024-09-03
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
100 participants
OBSERVATIONAL
2022-09-13
2024-12-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
CROSS_SECTIONAL
Study Groups
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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
Classical conditioning task
To 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.
Interventions
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Classical conditioning task
To 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.
Eligibility Criteria
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Inclusion Criteria
1. Male
2. Age range 7-17 years
3. A genetically proven diagnosis of DMD.
4. 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-).
5. Ability to consent/assent
BMD patients:
1. Male
2. Age range 7-17 years
3. A genetically proven diagnosis of BMD.
4. A genetic mutation that decreases expression of Dp427 alone (assigned to BMD Group 1), of both Dp427 and Dp140 (assigned to BMD Group 2).
5. Ability to consent/assent
Control participants:
1. Male
2. Age range 7-17 years.
3. Ability to consent/assent
Exclusion Criteria
1. Significant visual or hearing impairment
2. Specific phobias or sensory sensitivities to stimuli similar to the ones used in this study
3. Current participation in a clinical trial investigating a new drug involved in dystrophin modulation.
4. 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:
1. Significant visual or hearing impairment
2. Specific phobias or sensory sensitivities to stimuli similar to the ones used in this study
3. Any diagnosis of neurological or psychiatric condition
1. Claustrophobia
2. Pacemakers and defibrillators
3. Nerve stimulators
4. Intracranial clips
5. Intraorbital or intraocular metallic fragments
6. Cochlear implants
7. Ferromagnetic implants (e.g. thoracic implant for scoliosis)
8. Inability to lie supine during less than 45 minutes
9. Not having a general practitioner
10. Severe learning disability which will require a general anaesthetic
7 Years
17 Years
MALE
Yes
Sponsors
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Sarepta Therapeutics, Inc.
INDUSTRY
University College, London
OTHER
Responsible Party
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Principal Investigators
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Francesco Muntoni, Professor
Role: PRINCIPAL_INVESTIGATOR
University College, London
Locations
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UCL GOS Institute of Child Health
London, , United Kingdom
Countries
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Central Contacts
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Facility Contacts
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Francesco Muntoni, Professor
Role: backup
David Skuse, Professor
Role: backup
Related Links
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Duchenne clinical trial listing
Health Research Authority research summary
Other Identifiers
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302390
Identifier Type: OTHER
Identifier Source: secondary_id
21/WM/0267
Identifier Type: -
Identifier Source: org_study_id
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