Not yet recruitingOBSERVATIONAL

The U.K. NorthStar Clinical Network

This study, called the NorthStar Clinical Network, is looking at how Duchenne Muscular Dystrophy (DMD) affects people in the UK from childhood to adulthood. It's a 'natural history' study, meaning it observes how the condition progresses naturally, without testing new medicines. Researchers will collect information on patients' movement, breathing, heart health, bone strength, and how DMD impacts daily life. They also want to understand things like learning differences, puberty changes, and general well-being. This information will help doctors and scientists understand DMD better, which is crucial for developing and testing new treatments in the future.

At a glance

Status

Not yet recruiting

Sponsor

University College, London

Enrolment target

300

Start

01 Dec 2024

Estimated completion

30 Jun 2025

Duchenne Muscular Dystrophy

What is this study about?

This study, called the U.K. NorthStar Clinical Network, is all about understanding Duchenne Muscular Dystrophy (DMD) in young people and adults across the United Kingdom. It's an 'observational' study, which means researchers aren't testing any new treatments or medicines. Instead, they are carefully watching and recording how DMD changes and develops in people over time. Think of it like taking a detailed long-term photograph of the condition's journey.

The main goal is to collect information on how DMD affects things like movement (walking, standing) and breathing as people get older. But they're also looking at many other important aspects of health. This includes heart health, bone strength, hormone changes during puberty, spinal curvature (scoliosis), and how the condition might impact learning, behaviour, and overall quality of life. They will also gather information on people's background, like their ethnicity.

By gathering all this detailed information from many people with DMD, the study aims to create a much clearer picture of how the condition affects people at different stages of their lives. This knowledge is incredibly important. It helps doctors understand what to expect, and it's essential for researchers when they are trying to develop and test new medicines or therapies for DMD in the future.

Key takeaways

It's an observational study to understand Duchenne Muscular Dystrophy (DMD) over time.
No new treatments or medicines are given; it tracks how DMD progresses naturally.
Information gathered supports future research and development of new therapies.
Involves annual or bi-annual visits to a specialist clinic for assessments.
Open to males with a confirmed DMD diagnosis living in the UK.
Participation in other trials doesn't stop you from joining this one.

Who may be eligible?

This study is specifically for males in the United Kingdom who have been diagnosed with Duchenne Muscular Dystrophy (DMD). This diagnosis usually needs to be confirmed by a genetic test.

Even if the genetic test is still being processed, you might still be able to join if your diagnosis was made through a muscle biopsy.

Good news – if you're already taking part in another clinical trial or have had surgery, you can still join this study. This is because it's about observing your condition and not about giving you a new treatment. However, you can't join if you live outside the United Kingdom.

Quick self-check

Are you male?
Do you have a genetically confirmed diagnosis of Duchenne Muscular Dystrophy?
Do you live in the United Kingdom?
Are you willing to attend annual or bi-annual clinic appointments?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part, you'll visit your specialist muscle clinic once or twice a year. During these visits, the study team will carry out a series of assessments. These will include check-ups related to your movement and physiotherapy, breathing tests, and some questions about your general health and how DMD affects your daily life. The team will also record important medical details.

All the information collected from your visits and questionnaires will then be securely added to a special national database. There are no new medications involved; you'll continue with any treatments you're already receiving. The study aims to follow participants over many years to understand the long-term journey of DMD.

Potential risks and benefits

Taking part in this study won't directly treat your Duchenne Muscular Dystrophy, but it helps doctors and scientists understand the condition better, which is vital for future treatments. The visits involve standard check-ups, so there are very few risks beyond the time and effort of attending appointments and answering questions. You can choose to stop being part of the study at any time, for any reason, without it affecting your medical care.

Locations (26)

Aberdeen, NHS Grampian
Aberdeen, United Kingdom
Belfast Health and Social Care Trust
Belfast, United Kingdom
Birmingham Children's Hospital
Birmingham, United Kingdom
Birmingham Community Healthcare NHS Foundation Trust
Birmingham, United Kingdom
Birmingham Heartlands Hospital
Birmingham, United Kingdom
University Hospitals Bristol and Weston
Bristol, United Kingdom
Cambridge University Hospitals
Cambridge, United Kingdom
Cardiff and Vale University Health Board
Cardiff, United Kingdom
NHS Dundee - Tayside
Dundee, United Kingdom
NHS Greater Glasgow and Clyde
Glasgow, United Kingdom
Lancashire Teaching Hospitals NHS Foundation Trust
Lancaster, United Kingdom
Leeds Teaching Hospitals NHS Trust
Leeds, United Kingdom

+14 more sites — see the official record for the full list.

Common questions

What is a 'natural history' study?

It's a study that observes how a condition, like DMD, changes over time without giving any new treatments. It helps us understand the typical journey of the illness.

Will I receive any new treatment if I join?

No, this study is about observation, not about testing new medicines. You will continue with any standard care you usually receive.

How often do I need to visit the clinic?

You would typically need to attend your specialist muscle clinic once or twice a year for assessments.

What kind of information will be collected about me?

They'll collect details about your movement, breathing, heart health, general health, bone strength, learning, and quality of life through assessments and questionnaires.

What happens to the information collected?

All your information is securely added to a national database to help researchers better understand Duchenne Muscular Dystrophy.

How to find out more

Dr Adnan Manzur, FRCPCH

adnan.manzur@gosh.nhs.uk +44 20 7405 9200 Official trial record

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