• Longitudinal Study of the Natural History of Duchenne Muscular Dystrophy (DMD)

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ClinicalTrials.gov ID

NCT06711692

Lead Sponsor

University College, LondonOTHER

Overall Status

Not yet recruiting

Study Type

Observational

Enrollment

300Estimated

Last Update Posted

Dec 2, 2024Actual

Start Date

Dec 1, 2024Estimated

Primary Completion Date

Apr 30, 2025Estimated

Completion Date

Jun 30, 2025Estimated

Official Title

Natural History and Standards of Care in Duchenne Muscular Dystrophy - the U.K. NorthStar Clinical Network.

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.

• Duchenne Muscular Dystrophy

Ages Eligible for Study

Child (0-17)Adult (18-64)Older Adult (65+)

Sexes Eligible for Study

Male

Accepts Healthy Volunteers

No

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.

Observational Model

Cohort

Time Perspective

Prospective

Lead Sponsor

University College, LondonOTHER

Collaborators

Muscular Dystrophy UKUNKNOWN

Sandya.Tirupathi@belfasttrust.hscni.net+44 028 9504 0100

tracey.willis1@nhs.net+44 121 333 9999

deepak.parasuraman@uhb.nhs.uk+44121 424 2000

deepa.krishnakumar@nhs.net01223 805000

Kate.Skone@wales.nhs.uk029 2074 7747

Gavin.Cobb@nhs.scot01382 660111

Jarod.Wong@glasgow.ac.uk0141 347 8468

anne-marie.childs@nhs.net0113 2433 144

Elizabeth.wraige@gstt.nhs.uk020 7188 7188

childneuromuscular@ouh.nhs.uk0300 304 7777

Kate.Skone@wales.nhs.uk01639 683344