Duchenne Muscular DystrophyNSNCT06711692
The U.K. NorthStar Clinical Network
An observational study of Duchenne Muscular Dystrophy, run by University College, London at 26 sites in United Kingdom.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 99 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- How many people
- 300 participants
- Where
- 26 sitesUnited Kingdom
- Study dates
- 1 Dec 2024to 30 Jun 2025
- How long will I take part?
- 18 Months
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 99 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
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
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
26 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Study Inbox contact
26 sites in 1 country
United Kingdom26 sites
Aberdeen, NHS Grampian
Aberdeen
Elma Stephen, MD, +44845 456 6000
elma.stephen@nhs.scot
Belfast Health and Social Care Trust
Belfast
Sandya Tirupathi, MD, +44 028 9504 0100, Sandya.Tirupathi@belfasttrust.hscni.net
Sandya Tirupathi, MD
Birmingham Children's Hospital
Birmingham
Tracy Willis, MD, +44 121 333 9999, tracey.willis1@nhs.net
Tracey Willis, MD
Birmingham Community Healthcare NHS Foundation Trust
Birmingham
Muhammad Chughtai, MD, 0121 466 6000, muhammad.chughtai1@nhs.net
Birmingham Heartlands Hospital
Birmingham
Deepak pParasuraman, MD, +44121 424 2000, deepak.parasuraman@uhb.nhs.uk
Zoya Alhaswani, MD, Zoya.Alhaswani@uhb.nhs.uk
University Hospitals Bristol and Weston
Bristol
Anirban Majumdar, MD, +44 117 923 0000, Anirban.majumdar@uhbw.nhs.uk
Anirban Majumdar, MD
Cambridge University Hospitals
Cambridge
Deepa Krishnakumar, MD, 01223 805000, deepa.krishnakumar@nhs.net
Gautam Ambegaonkar, MD
Cardiff and Vale University Health Board
Cardiff
Kate Skone, MD, 029 2074 7747, Kate.Skone@wales.nhs.uk
Anurag Saxena, MD, Anurag.Saxena@wales.nhs.uk
NHS Dundee - Tayside
Dundee
Gavin Cobb, MD, 01382 660111, Gavin.Cobb@nhs.scot
Gemma Robertson, MD
NHS Greater Glasgow and Clyde
Glasgow
Jarod Wong, MD, 0141 347 8468, Jarod.Wong@glasgow.ac.uk
Iain Horrocks, MD
Lancashire Teaching Hospitals NHS Foundation Trust
Lancaster
Christian DeGoede, MD, 01772 716565, christian.degoede@lthtr.nhs.uk
Amy Parkes, MD, amy.parkes@thtr.nhs.uk
Leeds Teaching Hospitals NHS Trust
Leeds
Anne-Marie Childs, MD, 0113 2433 144, anne-marie.childs@nhs.net
Anne-Marie Childs, MD
University Hospitals of Leicester NHS Trust
Leicester
Dhinesh Baskaran, MD, +44 (0)116 252 2522., dhinesh.baskaran@uhl-tr.nhs.uk
Dhinesh Baskaran, MD
Alder Hey Children's Hospital Trust
Liverpool
Rajesh Karuvattil, MD, 0151 228 4811, rajesh.karuvattil@alderhey.nhs.uk
Rajesh Madhu, MD
Great Ormond Street Hospital for Children NHS Foundation Trust
London, WC1N 3BH
Clinical Investigator, MD, adnan.manzur@gosh.nhs.uk
Evelina London Children's Hospital
London
Elizabeth Wraige, MD, 020 7188 7188, Elizabeth.wraige@gstt.nhs.uk
Vasantha Gowda, MD
Great Ormond Street Hospital for Children NHS Foundation Trust
London
Adnan Manzur, MD, 020 7405 9200, adnan.manzur@gosh.nhs.uk
Adnan Manzur, MD
Manchester University NHS Foundation Trust
Manchester
Imelda Hughes, MD, 0161 276 1234, Imelda.hughes@mft.nhs.uk
Imelda Hughes, MD
Newcastle Hospitals NHS Foundation Trust
Newcastle
Nottingham University Hospitals NHS Trust
Nottingham
Neuro Admin, MD, paedneuroadmin@nuh.nhs.uk
Krupa Torne, MD
The Robert Jones and Agnes Hunt Orthopaedic Hospital
Oswestry
Richa Kulshrestha, MD, 01691 404000, richakulshrestha@nhs.net
Richa Kulshrestha, MD
Oxford University Hospitals
Oxford
Richard Tarver, 0300 304 7777, childneuromuscular@ouh.nhs.uk
Sithara Ramdas, MD
University Hospitals Plymouth NHS Trust
Plymouth
Richard Tomlinson, MD, 01752 202082, richard.tomlinson@nhs.net
Heather Jarvis, MD
Sheffield Teaching Hospitals NHS Foundation Trust
Sheffield
Sheffield Admin Neuromuscular, 0114 271 1900, scn-tr.sheffield.nmtherapy@nhs.net
Min Ong, MD
University Hospital Southampton NHS Foundation Trust
Southampton
Marjorie Illingworth, MD, 023 8077 7222, marjorie.illingworth@uhs.nhs.uk
Marjorie Illingworth, MD
Swansea Bay University Health Board
Swansea
Kate Skone, MD, 01639 683344, Kate.Skone@wales.nhs.uk
Kate Skone
About this study
Official title: Natural History and Standards of Care in Duchenne Muscular Dystrophy - the U.K. NorthStar Clinical Network.
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.
Read more
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.
Read the 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.
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- DMD, Duchenne Muscular Dystrophy, NorthStar Clinical Network, Northstar, Standards of care in DMD
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| DMD children and adult | Paediatric and adults with DMD |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measure: Duchenne Muscular Dystrophy Quality of Life Measure (DMD-QoL). The DMD-QoL has a hierarchical (or 'higher-order') factor structure, with 3 lower-order factors (physical, social, and psychological) and 1 higher-order factor (overall quality of life [QoL], comprised of the 3 lower-order factors). Higher scores represent a more positive QoL (overall or within each subscale).
Measured: From enrolment to the end of the study, across 18 months
Longitudinal data on quality of life in childhood and adult patients.
Measure: The Quality of Life in Genetic Neuromuscular Disease Questionnaire (QoL-gNMD). The QoL-gNMD domain is measured on a T score metric i.e. a normal distribution with a mean of 50 and a standard deviation of 10.
High values represent good quality of life.
Measured: From enrolment to the end of the study, across 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Dec 2024
- Study start1 Dec 2024 (estimated)
- Primary completion30 Apr 2025 (estimated)
- Study completion30 Jun 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by University College, LondonThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 300 participants (estimated)
- Target duration
- 18 Months
Sponsor and investigators
- Lead sponsor
- University College, London
- Sponsor type
- University, hospital or other
- Collaborators
- Muscular Dystrophy UK
- Responsible party
- Sponsor
- Investigators
- Professor Francesco Muntoni, FRCPCH FMed Sci, Study chair, University College, LondonProfessor Giovanni Baranello, MD, PhD, Study director, University College, LondonProfessor Michela Guglieri, MD, Study director, Newcastle UniversityProfessor Rosaline Quinlivan, MD, Study director, University College, London
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06711692
- Sponsor's protocol ID
- 242567
- Acronym
- NS
- Status last verified
- Nov 2024
- First submitted
- 8 Nov 2024
- Last updated
- 2 Dec 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Fully anonymised summary-level data can be made available on request for collaborations. The data dictionary is currently publicly available.
Study at a glance
From the registry record, last updated by the sponsor on 2 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Natural History and Standards of Care in Duchenne Muscular Dystrophy - the U.K. NorthStar Clinical Network.
- ClinicalTrials.gov ID
- NCT06711692
- Acronym / sponsor ID
- NS / 242567
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- DMD, Duchenne Muscular Dystrophy, NorthStar Clinical Network, Northstar, Standards of care in DMD
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- University College, LondonUniversity, hospital or other
- Collaborators
- Muscular Dystrophy UK
- Study start
- 1 Dec 2024 (estimated)
- Primary completion
- 30 Apr 2025 (estimated)
- Study completion
- 30 Jun 2025 (estimated)
- First posted
- 2 Dec 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 18 Months
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| DMD children and adult | – | –Paediatric and adults with DMD |
Who may qualify
- Ages
- Up to 99 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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
Key inclusion criteria (1 listed)
- 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.
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Longitudinal data on motor function in Duchenne Muscular Dystrophy (DMD) patients from the childhood to the adult phases of life
Time frame: From enrolment to the end of the study, across 18 months
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.
2 secondary outcomes
Longitudinal data on quality of life in childhood and adult patients.
Time frame: From enrolment to the end of the study, across 18 months
Longitudinal data on quality of life in childhood and adult patients.
Time frame: From enrolment to the end of the study, across 18 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Dec 2024.
- Funder programme briefLink provided by the sponsor.
- GOSH Project OutlineLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Fully anonymised summary-level data can be made available on request for collaborations. The data dictionary is currently publicly available.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Study Inbox contact
Professor Francesco Muntoni, FRCPCH FMed Sci study chair
University College, London
Professor Giovanni Baranello, MD, PhD study director
University College, London
Professor Michela Guglieri, MD study director
Newcastle University
26 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Duchenne Muscular Dystrophy, run by University College, London at 26 sites in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 2 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Dr Adnan Manzur, FRCPCH (+44 20 7405 9200 ext. 5849, adnan.manzur@gosh.nhs.uk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Longitudinal data on motor function in Duchenne Muscular Dystrophy (DMD) patients from the childhood to the adult phases of life (time frame: From enrolment to the end of the study, across 18 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Dec 2024.