RecruitingOBSERVATIONAL

Defining Outcome Measures for Behavioural and Emotional Problems in Dystrophinopathies

This research wants to understand how Duchenne (DMD) and Becker (BMD) muscular dystrophies can impact thinking, feelings, and behaviour. The team is trying to find better ways to measure these effects, which is important for future studies looking at new treatments. Participants, who are boys aged 7-17 with DMD or BMD, or healthy boys of the same age, will complete questionnaires about their behaviour, thinking skills, and how they get along with others. They will also do some computer tasks that look at how their brain responds. Some participants might also have an optional brain scan (MRI). The goal is to develop a set of tests to understand these brain-related symptoms better and to help in trials of new medicines.

At a glance

Status

Recruiting

Sponsor

University College, London

Enrolment target

100

Start

13 Sep 2022

Estimated completion

31 Dec 2024

DMD BMD

What is this study about?

This study aims to understand more about how Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD) can affect a person's emotions, behaviour, and thinking skills. We know that these conditions primarily affect muscles, but they can also have an impact on the brain. This research is trying to figure out exactly how these brain effects happen and, crucially, how we can measure them accurately.

The researchers are developing and testing a specific type of computer task to see how the brain reacts. They also want to see if there's a link between a person's specific genetic change in DMD/BMD and how their brain responds, as well as how this relates to their behaviour and emotions. This is a bit like seeing how a mouse with a similar condition reacts and then trying to understand the same process in humans.

Ultimately, the goal is to create a complete set of reliable tests. These tests could then be used in future clinical trials, for example, studies looking into new medications that might help improve brain function in people with DMD or BMD. By having good ways to measure these effects, researchers can better tell if a new treatment is working.

Key takeaways

This study helps us understand how DMD and BMD affect the brain, emotions, and behaviour.
It uses questionnaires and computer tasks.
Participation might include an optional MRI brain scan.
The goal is to develop better ways to measure these effects for future research and treatments.
It helps boys aged 7-17 with DMD or BMD, and healthy boys, take part.

Who may be eligible?

This study is looking for boys between 7 and 17 years old. They need to have either a confirmed diagnosis of Duchenne Muscular Dystrophy (DMD) or Becker Muscular Dystrophy (BMD).

The study also needs healthy boys, aged 7-17, who do not have DMD or BMD, to act as a comparison group. All participants must be able to understand what the study involves and agree to take part (or assent if they are younger).

Some things might mean someone can't take part. This includes having serious problems with seeing or hearing, or strong fears or sensitivities to certain things. If a boy is already in another trial testing a new drug for muscular dystrophy, or has a severe learning disability that makes it impossible to understand and agree, they also wouldn't be able to join. For the MRI scan, certain medical devices like pacemakers or metal implants would prevent participation, as would severe claustrophobia.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you a boy aged between 7 and 17 years old?
Do you have a doctor's diagnosis of Duchenne (DMD) or Becker (BMD) muscular dystrophy, or are you a healthy boy?
Can you understand what the study involves and agree to take part?
Do you have any serious problems with your vision or hearing?
Are you already taking part in another study testing a new drug for muscular dystrophy?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you will be asked to complete several questionnaires. These will cover topics like behaviour, how you think and solve problems, and how you get along with others. You will also do some computer tasks that help the researchers understand how your brain responds. There may be an option to have a brain scan using a special machine called an MRI. The study team will explain all the details of what each part involves and how long it will take. This study does not involve taking any new medication or having any follow-up appointments after your participation.

Potential risks and benefits

The main benefit of taking part is helping researchers understand Duchenne and Becker muscular dystrophies better, which could lead to better ways to measure brain-related symptoms and ultimately help in finding future treatments. The risks are generally low, mainly involving the time commitment and potential discomfort from lying still during an optional MRI scan. There are strict safety checks for the MRI. You are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

UCL GOS Institute of Child Health
Verified postcode
London, United Kingdom· Recruiting

Common questions

What is 'classical conditioning'?

It's a type of learning where you learn to associate one thing with another, like how dogs learn to drool at the sound of a bell if they expect food to follow.

What is an MRI scan?

An MRI (Magnetic Resonance Imaging) is a safe way to take very detailed pictures of the inside of your body, including your brain, using a strong magnet and radio waves.

Will I get any medicine?

No, this study does not involve giving you any new medicines. It's about understanding and measuring how things are.

How long will I need to be involved?

The study involves a specific set of tests and questionnaires, but it's not a long-term commitment with many visits. The researchers will explain the exact time needed during the consent process.

What are Dp427 and Dp140?

These are parts of a protein called dystrophin. In Duchenne and Becker muscular dystrophy, there are problems with dystrophin, which can affect different parts of the body, including the brain.

How to find out more

Anna Kolesnik, Dr

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

Always speak to your GP or specialist before deciding to take part in a study.