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UNKNOWNN/A

Defining Outcome Measures for Behavioural and Emotional Problems in Dystrophinopathies

Sponsor: University College, London

NCT ID: NCT06581887

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Health checks and monitoring — no treatment given
Type of study
Observational (no treatment given)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
Classical conditioning task (behavioural)
How long the study runs
Study runs about 27 months (dates as stated)
About the drug or intervention
Classical conditioning task — behavioural: 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).
Patient visit burden
Not specified by the sponsor
Type of study
Observing health over time
Ages
7 Years to 17 Years
Who
Male
Number of participants
100
Started
2022-09-13
Last checked
2024-08

Plain English Summary

What is this study?

  • • Testing a new treatment for dmd
  • • Clinical study - 100 participants
  • • Study aims to develop and to evaluate the neurophysiological and physiological response to a classical conditioning task

Who can take part?

  • • Ages 7 Years to 17 Years
  • • Diagnosed with dmd
  • • Male only

Where?

  • • London - UCL GOS Institute of Child Health

This is a simplified summary. Always discuss with your doctor before making any decisions.

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,

More detail

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 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. At the end of the study, the objective is to deliver a comprehensive test battery that is suitable for use in a trial of AON delivery to improve brain function.

DMDBMD

How this trial compares with your answers

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What we know so far

Condition· Matched your search
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Still need:

  • • Tell us your age for better matching
  • • Tell us your sex for better matching

Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 7 Years - 17 Years
  • Who can join: Male only

What the study is looking for

  • ✓DMD patients:
  • ✓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...
  • ✓Ability to consent/assent

Who cannot take part

  • ✗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.
  • ✗Control participants:
See the full criteria
Inclusion Criteria: * DMD patients: 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: * DMD \& BMD patients: 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 General exclusion criteria for MRI: 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

Where Is This Study? (1 UK site)

UCL GOS Institute of Child Health

London WC1N 1EH, United Kingdom

Recruiting
Site contact (verified)
Anna Kolesnik, PhDbraindmd@ucl.ac.uk
Natasha Aslam, MScnatasha.aslam@ucl.ac.uk

How to Get in Touch

Anna Kolesnik, Dr

Sponsor contact

CONTACT

44 (0) 20 7905 2600 braindmd@ucl.ac.uk

Natasha Aslam, MSc

Sponsor contact

CONTACT

44 (0) 20 7905 2600 natasha.aslam@ucl.ac.uk
Data sourced from ClinicalTrials.gov · Last verified: 2024-08