The U.K. NorthStar Clinical Network
- Conditions
- Duchenne Muscular Dystrophy
- Registration Number
- NCT06711692
- Lead Sponsor
- University College, London
- Brief Summary
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.
- Detailed Description
This study will make use of the existing NorthStar network to collect a wider range of clinically relevant data from paediatric and adult DMD patients, to describe more completely the natural history of the disease in different domains and across different life stages and disease phases. Data will be collected using assessment techniques and outcome measures appropriate to the major phases of the disease, and which are in some cases not currently part of the standard of care for DMD in the UK. The National Neuromuscular Database will continue to be used to collect and store these data. This study will allow the network to continue to describe the current natural history in DMD prospectively, with more robust subject ascertainment and completeness. This will help meet the current need for contemporary natural history data for the evaluation of new therapies, and further research in DMD.
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- Male
- Target Recruitment
- 300
- 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.
- 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.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Longitudinal data on motor function in Duchenne Muscular Dystrophy (DMD) patients from the childhood to the adult phases of life From enrolment to the end of the study, across 18 months Measure: NorthStar Ambulatory Assessment (NSAA). a scale from 0 (unable), 1 (completes independently but with modifications), and 2 (completed without compensation). Total score 0 - 34 with a higher score denoting a higher level of function.
- Secondary Outcome Measures
Name Time Method Longitudinal data on quality of life in childhood and adult patients. From enrolment to the end of the study, across 18 months Measure: The Quality of Life in Genetic Neuromuscular Disease Questionnaire (QoL-gNMD). The QoL-gNMD domain is measured on a T score metric i.e. a normal distribution with a mean of 50 and a standard deviation of 10.
High values represent good quality of life.
Related Research Topics
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Trial Locations
- Locations (25)
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
University Hospitals of Leicester NHS Trust
🇬🇧Leicester, United Kingdom
Alder Hey Children's Hospital Trust
🇬🇧Liverpool, United Kingdom
Great Ormond Street Hospital for Children NHS Foundation Trust
🇬🇧London, United Kingdom
Evelina London Children's Hospital
🇬🇧London, United Kingdom
Manchester University NHS Foundation Trust
🇬🇧Manchester, United Kingdom
Newcastle Hospitals NHS Foundation Trust
🇬🇧Newcastle, United Kingdom
Nottingham University Hospitals NHS Trust
🇬🇧Nottingham, United Kingdom
The Robert Jones and Agnes Hunt Orthopaedic Hospital
🇬🇧Oswestry, United Kingdom
Oxford University Hospitals
🇬🇧Oxford, United Kingdom
University Hospitals Plymouth NHS Trust
🇬🇧Plymouth, United Kingdom
Sheffield Teaching Hospitals NHS Foundation Trust
🇬🇧Sheffield, United Kingdom
University Hospital Southampton NHS Foundation Trust
🇬🇧Southampton, United Kingdom
Swansea Bay University Health Board
🇬🇧Swansea, United Kingdom