Following children and adults with Duchenne muscular dystrophy over time in the UK
Records how Duchenne muscular dystrophy changes over time in UK patients.
Plain-language title generated from the registry record
Registry title: The U.K. NorthStar Clinical Network
Key facts
- Where
- Aberdeen, Belfast, Birmingham, Bristol, Cambridge, Cardiff, Dundee, Glasgow, Lancaster, Leeds, Leicester, Liverpool, London, Manchester, Newcastle, Nottingham, Oswestry, Oxford, Plymouth, Sheffield, Southampton, and Swansea, United KingdomSee the 25 sites
Where in United Kingdom
- Aberdeen, NHS GrampianAberdeen, United KingdomNo longer taking part
- Belfast Health and Social Care TrustBelfast, United KingdomNo longer taking part
- Birmingham Children's HospitalBirmingham, United KingdomNo longer taking part
- Birmingham Community Healthcare NHS Foundation TrustBirmingham, United KingdomNo longer taking part
- Birmingham Heartlands HospitalBirmingham, United KingdomNo longer taking part
- University Hospitals Bristol and WestonBristol, United KingdomNo longer taking part
- Cambridge University HospitalsCambridge, United KingdomNo longer taking part
- Cardiff and Vale University Health BoardCardiff, United KingdomNo longer taking part
- NHS Dundee - TaysideDundee, United KingdomNo longer taking part
- NHS Greater Glasgow and ClydeGlasgow, United KingdomNo longer taking part
- Lancashire Teaching Hospitals NHS Foundation TrustLancaster, United KingdomNo longer taking part
- Leeds Teaching Hospitals NHS TrustLeeds, United KingdomNo longer taking part
- University Hospitals of Leicester NHS TrustLeicester, United KingdomNo longer taking part
- Alder Hey Children's Hospital TrustLiverpool, United KingdomNo longer taking part
- Evelina London Children's HospitalLondon, United KingdomNo longer taking part
- Great Ormond Street Hospital For Children NHS Foundation TrustLondon, United KingdomNo longer taking part
- Manchester University NHS Foundation TrustManchester, United KingdomNo longer taking part
- Newcastle Hospitals NHS Foundation TrustNewcastle, United KingdomNo longer taking part
- Nottingham University Hospitals NHS TrustNottingham, United KingdomNo longer taking part
- The Robert Jones and Agnes Hunt Orthopaedic HospitalOswestry, United KingdomNo longer taking part
- Oxford University HospitalsOxford, United KingdomNo longer taking part
- University Hospitals Plymouth NHS TrustPlymouth, United KingdomNo longer taking part
- Sheffield Teaching Hospitals NHS Foundation TrustSheffield, United KingdomNo longer taking part
- University Hospital Southampton NHS Foundation TrustSouthampton, United KingdomNo longer taking part
- Swansea Bay University Health BoardSwansea, United KingdomNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- University College, London
- Trial code
- NCT06711692
- Record updated
- 2 December 2024
- Original description
The goal of this natural history study is to capture the natural history of Duchenne Muscular Dystrophy (DMD) in children and adults in the United Kingdom. Children and adults with DMD will be invited to join.Read full original descriptionShow less
Natural History and Standards of Care in Duchenne Muscular Dystrophy - the U.K. NorthStar Clinical Network.
The goal of this natural history study is to capture the natural history of Duchenne Muscular Dystrophy (DMD) in children and adults in the United Kingdom. Children and adults with DMD will be invited to join.
The primary objective of the study is to collect longitudinal data on motor and respiratory function in DMD patients from childhood to adulthood.
The secondary objectives of the study include collection of longitudinal data on other aspects of natural history on DMD, including respiratory, cardiac and endocrine complications, neurodiversity (cognitive impairment, neuro-behavioural disorders such as ADHD and autism), changes to bone density and occurrence of fractures, changes to puberty, incidence of scoliosis, unplanned hospital admissions, and quality of life. The study will also collect information on ethnicity.
Participants will attend an annual or bi-annual neuromuscular clinic, and will have a series of assessments and questionnaires with the study team. These include: key medical data, physiotherapy data, respiratory assessments, Quality of Life questionnaires, and DMD questionnaires. Following assessments and questionnaire completion, data is input into the study's tailor-made National Neuromuscular Database.
Inclusion: All patients with genetically confirmed diagnosis of Duchenne Muscular Dystrophy in the United Kingdom. Recruitment will also be possible in cases in whom the DMD diagnosis is made after a muscle biopsy even if the dystrophin gene variant is still being investigated.
Exclusion: Involvement in clinical trials is not an exclusion criterion nor having had surgical procedures, as this is an observational research study. The regular clinical data of patients in clinical trials will be acquired as part of the clinical follow up; Patients based outside of the United Kingdom.