Actively Recruiting
Defining Outcome Measures for Behavioural and Emotional Problems in Duchenne and Becker Muscular Dystrophies
Led by University College, London · Updated on 2024-09-03
100
Participants Needed
1
Research Sites
6 weeks
Total Duration
On this page
Sponsors
U
University College, London
Lead Sponsor
S
Sarepta Therapeutics, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are studying how Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD) affect mental health by evaluating neurophysiological and physiological responses to a classical conditioning task. This investigation aims to understand how different genetic types of DMD and BMD relate to emotional and behavioral characteristics. The goal is to develop a comprehensive test battery suitable for future trials targeting brain function improvement. Participants will complete questionnaires on behavior, cognitive function, and social interactions, as well as perform computer-based tasks that measure emotional responses. Some participants may also have an optional MRI brain scan. The study uses a classical conditioning task to record responses such as skin conductance, heart rate, and pupil reactions. The research includes groups of DMD, BMD, and control participants. During the study, participants will undergo various behavioral assessments including the Social Responsiveness Scale, ADHD Rating Scale, Child Behaviour Checklist, and others. The team will measure primary outcomes like differences in response to aversive stimuli among groups over an average of two years. Secondary outcomes include studying learning, habituation, and extinction patterns. Safety and participation are monitored throughout the study period, which involves multiple assessments and optional imaging.
CONDITIONS
Brief Title
Defining Outcome Measures for Behavioural and Emotional Problems in Dystrophinopathies
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male patients aged 7 to 17 years
- Genetically confirmed diagnosis of Duchenne Muscular Dystrophy (DMD) or Becker Muscular Dystrophy (BMD)
- Genetic mutation affecting Dp427 alone or both Dp427 and Dp140 for DMD and BMD groups
- Ability to provide consent or assent
- Control participants must be male, aged 7 to 17 years, and able to consent or assent
You will not qualify if you...
- Significant visual or hearing impairment
- Specific phobias or sensory sensitivities to stimuli similar to those used in the study
- Current participation in a clinical trial involving new dystrophin modulation drugs
- Inability to consent or assent, including severe learning disability preventing assent
- For controls, any diagnosis of neurological or psychiatric conditions
- MRI exclusions: claustrophobia, pacemakers, nerve stimulators, intracranial clips, metallic fragments in eyes, cochlear implants, ferromagnetic implants, inability to lie still for 45 minutes, lack of general practitioner, severe learning disability requiring general anaesthetic
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 2 years
Participants complete a series of behavioral and neurophysiological assessments including the classical conditioning task, questionnaires, cognitive tests, and some may undergo an MRI brain scan to evaluate emotional and behavioral responses.
Multiple visits depending on assessments required
Duration - Up to 2 years
Participants are observed over time to assess group differences and changes in learning, habituation, and extinction related to behavioral and emotional characteristics.
Follow-up visits as scheduled during the study period
Trial Site Locations
Total: 1 location
1
UCL GOS Institute of Child Health
London, United Kingdom, WC1N 1EH
Actively Recruiting
Research Team
A
Anna Kolesnik, Dr
N
Natasha Aslam, MSc
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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