Clinical trial NCT06711692: The U.K. NorthStar Clinical Network
Official title: Natural History and Standards of Care in Duchenne Muscular Dystrophy - the U.K. NorthStar Clinical Network.
Study type: OBSERVATIONAL
Overall status: NOT_YET_RECRUITING
Conditions: Duchenne Muscular Dystrophy
Keywords: DMD
Duchenne Muscular Dystrophy
NorthStar Clinical Network
Northstar
Standards of care in DMD
Lead sponsor: University College, London
Sponsor class: OTHER
Collaborators: name: Muscular Dystrophy UK
First posted: 2024-12-02
Enrollment: 300
Sex: MALE
Maximum age (years): 99
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: DMD patients of all ages (both paediatric and adult) will be recruited into the study. No age limits or restrictions within the DMD population have been set for this study, as the study aims to capture the natural progression or DMD irrespective of the stage of the disease. All DMD patients in paediatric or adult clinical care will therefore be eligible to participate providing they have consented

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.

Eligibility criteria: Inclusion Criteria:

* 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 Criteria:

* 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.

Primary outcomes:
measure: Longitudinal data on motor function in Duchenne Muscular Dystrophy (DMD) patients from the childhood to the adult phases of life
timeFrame: From enrolment to the end of the study, across 18 months
Secondary outcomes:
measure: Longitudinal data on quality of life in childhood and adult patients.
timeFrame: From enrolment to the end of the study, across 18 months
measure: Longitudinal data on quality of life in childhood and adult patients.
timeFrame: From enrolment to the end of the study, across 18 months
Other outcomes:
Locations:
city: Aberdeen
country: United Kingdom
facility: Aberdeen, NHS Grampian
city: Belfast
country: United Kingdom
facility: Belfast Health and Social Care Trust
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital
city: Birmingham
country: United Kingdom
facility: Birmingham Community Healthcare NHS Foundation Trust
city: Birmingham
country: United Kingdom
facility: Birmingham Heartlands Hospital
city: Bristol
country: United Kingdom
facility: University Hospitals Bristol and Weston
city: Cambridge
country: United Kingdom
facility: Cambridge University Hospitals
city: Cardiff
country: United Kingdom
facility: Cardiff and Vale University Health Board
city: Dundee
country: United Kingdom
facility: NHS Dundee - Tayside
city: Glasgow
country: United Kingdom
facility: NHS Greater Glasgow and Clyde
city: Lancaster
country: United Kingdom
facility: Lancashire Teaching Hospitals NHS Foundation Trust
city: Leeds
country: United Kingdom
facility: Leeds Teaching Hospitals NHS Trust
city: Leicester
country: United Kingdom
facility: University Hospitals of Leicester NHS Trust
city: Liverpool
country: United Kingdom
facility: Alder Hey Children's Hospital Trust
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children NHS Foundation Trust
city: London
country: United Kingdom
facility: Evelina London Children's Hospital
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children NHS Foundation Trust
city: Manchester
country: United Kingdom
facility: Manchester University NHS Foundation Trust
city: Newcastle
country: United Kingdom
facility: Newcastle Hospitals NHS Foundation Trust
city: Nottingham
country: United Kingdom
facility: Nottingham University Hospitals NHS Trust
city: Oswestry
country: United Kingdom
facility: The Robert Jones and Agnes Hunt Orthopaedic Hospital
city: Oxford
country: United Kingdom
facility: Oxford University Hospitals
city: Plymouth
country: United Kingdom
facility: University Hospitals Plymouth NHS Trust
city: Sheffield
country: United Kingdom
facility: Sheffield Teaching Hospitals NHS Foundation Trust
city: Southampton
country: United Kingdom
facility: University Hospital Southampton NHS Foundation Trust
city: Swansea
country: United Kingdom
facility: Swansea Bay University Health Board
Study officials:
affiliation: University College, London
name: Professor Francesco Muntoni, FRCPCH FMed Sci
role: STUDY_CHAIR
affiliation: University College, London
name: Professor Giovanni Baranello, MD, PhD
role: STUDY_DIRECTOR
affiliation: Newcastle University
name: Professor Michela Guglieri, MD
role: STUDY_DIRECTOR
affiliation: University College, London
name: Professor Rosaline Quinlivan, MD
role: STUDY_DIRECTOR
References: