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The U.K. NorthStar Clinical N...

The U.K. NorthStar Clinical Network

Last Updated: 2024-12-02

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NOT_YET_RECRUITING

Total Enrollment

300 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-12-01

Study Completion Date

2025-06-30

Brief Summary

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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

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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.

Conditions

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Duchenne Muscular Dystrophy

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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DMD children and adult

Paediatric and adults with DMD

No interventions assigned to this group

Eligibility Criteria

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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.

Maximum Eligible Age

99 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Professor Francesco Muntoni, FRCPCH FMed Sci

Role: STUDY_CHAIR

University College, London

Professor Giovanni Baranello, MD, PhD

Role: STUDY_DIRECTOR

University College, London

Professor Michela Guglieri, MD

Role: STUDY_DIRECTOR

Newcastle University

Professor Rosaline Quinlivan, MD

Role: STUDY_DIRECTOR

University College, London

Reach out to these primary contacts for questions about participation or study logistics.

Dr Adnan Manzur, FRCPCH

Role: CONTACT

[email protected]

+44 20 7405 9200 ext. 5849

Study Inbox

Role: CONTACT

[email protected]

Facility Contacts

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Elma Stephen, MD

Role: primary

+44121 424 2000

Role: primary

[email protected]

029 2074 7747

Role: primary

[email protected]

01772 716565

Role: primary

Role: backup

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242567

Identifier Type: -

Identifier Source: org_study_id

The U.K. NorthStar Clinical Network

Study Results

Basic Information

Brief Summary

Detailed Description

Conditions

Study Design

Study Groups

DMD children and adult

Eligibility Criteria

Inclusion Criteria

Exclusion Criteria

Sponsors

Muscular Dystrophy UK

University College, London

Responsible Party

Principal Investigators

Professor Francesco Muntoni, FRCPCH FMed Sci

Professor Giovanni Baranello, MD, PhD

Professor Michela Guglieri, MD

Professor Rosaline Quinlivan, MD

Locations

Aberdeen, NHS Grampian

Belfast Health and Social Care Trust

Birmingham Children's Hospital

Birmingham Community Healthcare NHS Foundation Trust

Birmingham Heartlands Hospital

University Hospitals Bristol and Weston

Cambridge University Hospitals

Cardiff and Vale University Health Board

NHS Dundee - Tayside

NHS Greater Glasgow and Clyde

Lancashire Teaching Hospitals NHS Foundation Trust

Leeds Teaching Hospitals NHS Trust

University Hospitals of Leicester NHS Trust

Alder Hey Children's Hospital Trust

Great Ormond Street Hospital for Children NHS Foundation Trust

Evelina London Children's Hospital

Great Ormond Street Hospital for Children NHS Foundation Trust

Manchester University NHS Foundation Trust

Newcastle Hospitals NHS Foundation Trust

Nottingham University Hospitals NHS Trust

The Robert Jones and Agnes Hunt Orthopaedic Hospital

Oxford University Hospitals

University Hospitals Plymouth NHS Trust

Sheffield Teaching Hospitals NHS Foundation Trust

University Hospital Southampton NHS Foundation Trust

Swansea Bay University Health Board

Countries

Central Contacts

Dr Adnan Manzur, FRCPCH

Study Inbox

Facility Contacts

Elma Stephen, MD

Elma Stephen, MD

Sandya Tirupathi, MD

Sandya Tirupathi, MD

Tracy Willis, MD

Tracey Willis, MD

Muhammad Chughtai, MD

Deepak pParasuraman, MD

Zoya Alhaswani, MD

Deepak Parasuraman, MD

Anirban Majumdar, MD

Anirban Majumdar, MD

Deepa Krishnakumar, MD

Gautam Ambegaonkar, MD

Kate Skone, MD

Anurag Saxena, MD

Kate Skone, MD

Gavin Cobb, MD

Gemma Robertson, MD

Jarod Wong, MD

Iain Horrocks, MD

Christian DeGoede, MD

Amy Parkes, MD

Christian DeGoede, MD

Anne-Marie Childs, MD

Anne-Marie Childs, MD

Dhinesh Baskaran, MD