The U.K. NorthStar Clinical Network

NCT06711692 | Not Yet Recruiting DMC

• 1 other identifier: 242567
• Study Type: observational
• Target: 300
• Locations: 1 country
• Sites: 26

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.

Conditions

Duchenne Muscular Dystrophy

Keywords

DMD; Duchenne Muscular Dystrophy; NorthStar Clinical Network; Northstar; Standards of care in DMD

Outcome Measures

Primary Outcomes (1)

• Longitudinal data on motor function in Duchenne Muscular Dystrophy (DMD) patients from the childhood to the adult phases of life

  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.

  From enrolment to the end of the study, across 18 months

Secondary Outcomes (2)

• Longitudinal data on quality of life in childhood and adult patients.

  From enrolment to the end of the study, across 18 months

• Longitudinal data on quality of life in childhood and adult patients.

  From enrolment to the end of the study, across 18 months

Study Arms (1)

DMD children and adult

Paediatric and adults with DMD

Eligibility Criteria

• Age: Up to 99 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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

You may qualify if:

• 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.

You may not qualify if:

• Patients based outside of the United Kingdom.

Sponsors & Collaborators

• University College, London: lead
• Muscular Dystrophy UK: collaborator

Study Sites (26)

Aberdeen, NHS Grampian

Aberdeen, United Kingdom

Location

Belfast Health and Social Care Trust

Belfast, United Kingdom

Location

Birmingham Children's Hospital

Birmingham, United Kingdom

Location

Birmingham Community Healthcare NHS Foundation Trust

Birmingham, United Kingdom

Location

Birmingham Heartlands Hospital

Birmingham, United Kingdom

Location

University Hospitals Bristol and Weston

Bristol, United Kingdom

Location

Cambridge University Hospitals

Cambridge, United Kingdom

Location

Cardiff and Vale University Health Board

Cardiff, United Kingdom

Location

NHS Dundee - Tayside

Dundee, United Kingdom

Location

NHS Greater Glasgow and Clyde

Glasgow, United Kingdom

Location

Lancashire Teaching Hospitals NHS Foundation Trust

Lancaster, United Kingdom

Location

Leeds Teaching Hospitals NHS Trust

Leeds, United Kingdom

Location

University Hospitals of Leicester NHS Trust

Leicester, United Kingdom

Location

Alder Hey Children's Hospital Trust

Liverpool, United Kingdom

Location

Great Ormond Street Hospital for Children NHS Foundation Trust

London, WC1N 3BH, United Kingdom

Location

Evelina London Children's Hospital

London, United Kingdom

Location

Great Ormond Street Hospital for Children NHS Foundation Trust

London, United Kingdom

Location

Manchester University NHS Foundation Trust

Manchester, United Kingdom

Location

Newcastle Hospitals NHS Foundation Trust

Newcastle, United Kingdom

Location

Nottingham University Hospitals NHS Trust

Nottingham, United Kingdom

Location

The Robert Jones and Agnes Hunt Orthopaedic Hospital

Oswestry, United Kingdom

Location

Oxford University Hospitals

Oxford, United Kingdom

Location

University Hospitals Plymouth NHS Trust

Plymouth, United Kingdom

Location

Sheffield Teaching Hospitals NHS Foundation Trust

Sheffield, United Kingdom

Location

University Hospital Southampton NHS Foundation Trust

Southampton, United Kingdom

Location

Swansea Bay University Health Board

Swansea, United Kingdom

Location

Related Links

• Funder programme brief
• GOSH Project Outline

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Professor Francesco Muntoni, FRCPCH FMed Sci

  University College, London

  STUDY CHAIR

• Professor Giovanni Baranello, MD, PhD

  University College, London

  STUDY DIRECTOR

• Professor Michela Guglieri, MD

  Newcastle University

  STUDY DIRECTOR

• Professor Rosaline Quinlivan, MD

  University College, London

  STUDY DIRECTOR

Central Study Contacts

Dr Adnan Manzur, FRCPCH

CONTACT

+44 20 7405 9200; adnan.manzur@gosh.nhs.uk

Study Inbox

CONTACT

northstar.network@ucl.ac.uk

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 18 Months
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 8, 2024
• First Posted: December 2, 2024
• Study Start: December 1, 2024
• Primary Completion: April 30, 2025
• Study Completion: June 30, 2025
• Last Updated: December 2, 2024

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will share
• Shared Documents: CSR
• Time Frame: After the study has closed, a clinical study report, publications etc may be shared with researchers. Summary-level anonymised data can be requested, subject to approval.
• Access Criteria: Academic researchers may contact the study central research group to request collaboration, usage of assessments, summary level of data.

Locations

GB (26)