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The U.K. NorthStar Clinical Network

NCT06711692 · Status: NOT_YET_RECRUITING · Type: OBSERVATIONAL · Enrollment: 300

Last updated 2024-12-02

No results posted yet for this study

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

Sponsors & Collaborators

  • Muscular Dystrophy UK

    collaborator UNKNOWN

  • University College, London

    lead OTHER

Principal Investigators

  • Professor Francesco Muntoni, FRCPCH FMed Sci · University College, London

  • Professor Giovanni Baranello, MD, PhD · University College, London

  • Professor Michela Guglieri, MD · Newcastle University

  • Professor Rosaline Quinlivan, MD · University College, London

Eligibility

Max Age
99 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-12-01
Primary Completion
2025-04-30
Completion
2025-06-30

Countries

  • United Kingdom

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06711692 on ClinicalTrials.gov