Ignite Creation Date:
2025-12-24 @ 3:49 PM
Ignite Modification Date:
2026-01-02 @ 6:04 AM
Study NCT ID:
NCT06711692
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-12-02
First Post:
2024-11-08
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
The U.K. NorthStar Clinical Network
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D020388', 'term': 'Muscular Dystrophy, Duchenne'}], 'ancestors': [{'id': 'D009136', 'term': 'Muscular Dystrophies'}, {'id': 'D020966', 'term': 'Muscular Disorders, Atrophic'}, {'id': 'D009135', 'term': 'Muscular Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 300}, 'targetDuration': '18 Months', 'patientRegistry': True}, 'statusModule': {'overallStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2024-12-01', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-11', 'completionDateStruct': {'date': '2025-06-30', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2024-11-26', 'studyFirstSubmitDate': '2024-11-08', 'studyFirstSubmitQcDate': '2024-11-26', 'lastUpdatePostDateStruct': {'date': '2024-12-02', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2024-12-02', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-04-30', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Longitudinal data on motor function in Duchenne Muscular Dystrophy (DMD) patients from the childhood to the adult phases of life', 'timeFrame': 'From enrolment to the end of the study, across 18 months', 'description': '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.'}], 'secondaryOutcomes': [{'measure': 'Longitudinal data on quality of life in childhood and adult patients.', 'timeFrame': 'From enrolment to the end of the study, across 18 months', 'description': "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)."}, {'measure': 'Longitudinal data on quality of life in childhood and adult patients.', 'timeFrame': 'From enrolment to the end of the study, across 18 months', 'description': '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.\n\nHigh values represent good quality of life.'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['DMD', 'Duchenne Muscular Dystrophy', 'NorthStar Clinical Network', 'Northstar', 'Standards of care in DMD'], 'conditions': ['Duchenne Muscular Dystrophy']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'https://www.musculardystrophyuk.org/research/current-projects/northstar-programme/', 'label': 'Funder programme brief'}, {'url': 'https://www.gosh.nhs.uk/wards-and-departments/departments/clinical-specialties/dubowitz-neuromuscular-centre-dnc-information-parents-and-visitors/dubowitz-neuromuscular-centre/northstar-project/', 'label': 'GOSH Project Outline'}]}, 'descriptionModule': {'briefSummary': "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.\n\nThe primary objective of the study is to collect longitudinal data on motor and respiratory function in DMD patients from childhood to adulthood.\n\nThe 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.\n\nParticipants 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.", 'detailedDescription': '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.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '99 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': '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', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* 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.\n\nExclusion Criteria:\n\n* 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.\n* Patients based outside of the United Kingdom.'}, 'identificationModule': {'nctId': 'NCT06711692', 'acronym': 'NS', 'briefTitle': 'The U.K. NorthStar Clinical Network', 'organization': {'class': 'OTHER', 'fullName': 'University College, London'}, 'officialTitle': 'Natural History and Standards of Care in Duchenne Muscular Dystrophy - the U.K. NorthStar Clinical Network.', 'orgStudyIdInfo': {'id': '242567'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'DMD children and adult', 'description': 'Paediatric and adults with DMD'}]}, 'contactsLocationsModule': {'locations': [{'city': 'Aberdeen', 'country': 'United Kingdom', 'contacts': [{'name': 'Elma Stephen, MD', 'role': 'CONTACT', 'phone': '+44845 456 6000'}, {'role': 'CONTACT', 'email': 'elma.stephen@nhs.scot'}, {'name': 'Elma Stephen, MD', 'role': 'CONTACT'}], 'facility': 'Aberdeen, NHS Grampian', 'geoPoint': {'lat': 57.14369, 'lon': -2.09814}}, {'city': 'Belfast', 'country': 'United Kingdom', 'contacts': [{'name': 'Sandya Tirupathi, MD', 'role': 'CONTACT', 'email': 'Sandya.Tirupathi@belfasttrust.hscni.net', 'phone': '+44 028 9504 0100'}, {'name': 'Sandya Tirupathi, MD', 'role': 'CONTACT'}], 'facility': 'Belfast Health and Social Care Trust', 'geoPoint': {'lat': 54.59682, 'lon': -5.92541}}, {'city': 'Birmingham', 'country': 'United Kingdom', 'contacts': [{'name': 'Tracy Willis, MD', 'role': 'CONTACT', 'email': 'tracey.willis1@nhs.net', 'phone': '+44 121 333 9999'}, {'name': 'Tracey Willis, MD', 'role': 'CONTACT'}], 'facility': "Birmingham Children's Hospital", 'geoPoint': {'lat': 52.48142, 'lon': -1.89983}}, {'city': 'Birmingham', 'country': 'United Kingdom', 'contacts': [{'name': 'Muhammad Chughtai, MD', 'role': 'CONTACT', 'email': 'muhammad.chughtai1@nhs.net', 'phone': '0121 466 6000'}], 'facility': 'Birmingham Community Healthcare NHS Foundation Trust', 'geoPoint': {'lat': 52.48142, 'lon': -1.89983}}, {'city': 'Birmingham', 'country': 'United Kingdom', 'contacts': [{'name': 'Deepak pParasuraman, MD', 'role': 'CONTACT', 'email': 'deepak.parasuraman@uhb.nhs.uk', 'phone': '+44121 424 2000'}, {'name': 'Zoya Alhaswani, MD', 'role': 'CONTACT', 'email': 'Zoya.Alhaswani@uhb.nhs.uk'}, {'name': 'Deepak Parasuraman, MD', 'role': 'CONTACT'}], 'facility': 'Birmingham Heartlands Hospital', 'geoPoint': {'lat': 52.48142, 'lon': -1.89983}}, {'city': 'Bristol', 'country': 'United Kingdom', 'contacts': [{'name': 'Anirban Majumdar, MD', 'role': 'CONTACT', 'email': 'Anirban.majumdar@uhbw.nhs.uk', 'phone': '+44 117 923 0000'}, {'name': 'Anirban Majumdar, MD', 'role': 'CONTACT'}], 'facility': 'University Hospitals Bristol and Weston', 'geoPoint': {'lat': 51.45523, 'lon': -2.59665}}, {'city': 'Cambridge', 'country': 'United Kingdom', 'contacts': [{'name': 'Deepa Krishnakumar, MD', 'role': 'CONTACT', 'email': 'deepa.krishnakumar@nhs.net', 'phone': '01223 805000'}, {'name': 'Gautam Ambegaonkar, MD', 'role': 'CONTACT'}], 'facility': 'Cambridge University Hospitals', 'geoPoint': {'lat': 52.2, 'lon': 0.11667}}, {'city': 'Cardiff', 'country': 'United Kingdom', 'contacts': [{'name': 'Kate Skone, MD', 'role': 'CONTACT', 'email': 'Kate.Skone@wales.nhs.uk', 'phone': '029 2074 7747'}, {'name': 'Anurag Saxena, MD', 'role': 'CONTACT', 'email': 'Anurag.Saxena@wales.nhs.uk'}, {'name': 'Kate Skone, MD', 'role': 'CONTACT'}], 'facility': 'Cardiff and Vale University Health Board', 'geoPoint': {'lat': 51.48, 'lon': -3.18}}, {'city': 'Dundee', 'country': 'United Kingdom', 'contacts': [{'name': 'Gavin Cobb, MD', 'role': 'CONTACT', 'email': 'Gavin.Cobb@nhs.scot', 'phone': '01382 660111'}, {'name': 'Gemma Robertson, MD', 'role': 'CONTACT'}], 'facility': 'NHS Dundee - Tayside', 'geoPoint': {'lat': 56.46913, 'lon': -2.97489}}, {'city': 'Glasgow', 'country': 'United Kingdom', 'contacts': [{'name': 'Jarod Wong, MD', 'role': 'CONTACT', 'email': 'Jarod.Wong@glasgow.ac.uk', 'phone': '0141 347 8468'}, {'name': 'Iain Horrocks, MD', 'role': 'CONTACT'}], 'facility': 'NHS Greater Glasgow and Clyde', 'geoPoint': {'lat': 55.86515, 'lon': -4.25763}}, {'city': 'Lancaster', 'country': 'United Kingdom', 'contacts': [{'name': 'Christian DeGoede, MD', 'role': 'CONTACT', 'email': 'christian.degoede@lthtr.nhs.uk', 'phone': '01772 716565'}, {'name': 'Amy Parkes, MD', 'role': 'CONTACT', 'email': 'amy.parkes@thtr.nhs.uk'}, {'name': 'Christian DeGoede, MD', 'role': 'CONTACT'}], 'facility': 'Lancashire Teaching Hospitals NHS Foundation Trust', 'geoPoint': {'lat': 54.04649, 'lon': -2.79988}}, {'city': 'Leeds', 'country': 'United Kingdom', 'contacts': [{'name': 'Anne-Marie Childs, MD', 'role': 'CONTACT', 'email': 'anne-marie.childs@nhs.net', 'phone': '0113 2433 144'}, {'name': 'Anne-Marie Childs, MD', 'role': 'CONTACT'}], 'facility': 'Leeds Teaching Hospitals NHS Trust', 'geoPoint': {'lat': 53.79648, 'lon': -1.54785}}, {'city': 'Leicester', 'country': 'United Kingdom', 'contacts': [{'name': 'Dhinesh Baskaran, MD', 'role': 'CONTACT', 'email': 'dhinesh.baskaran@uhl-tr.nhs.uk', 'phone': '+44 (0)116 252 2522.'}, {'name': 'Dhinesh Baskaran, MD', 'role': 'CONTACT'}], 'facility': 'University Hospitals of Leicester NHS Trust', 'geoPoint': {'lat': 52.6386, 'lon': -1.13169}}, {'city': 'Liverpool', 'country': 'United Kingdom', 'contacts': [{'name': 'Rajesh Karuvattil, MD', 'role': 'CONTACT', 'email': 'rajesh.karuvattil@alderhey.nhs.uk', 'phone': '0151 228 4811'}, {'name': 'Rajesh Madhu, MD', 'role': 'CONTACT'}], 'facility': "Alder Hey Children's Hospital Trust", 'geoPoint': {'lat': 53.41058, 'lon': -2.97794}}, {'zip': 'WC1N 3BH', 'city': 'London', 'country': 'United Kingdom', 'contacts': [{'name': 'Clinical Investigator, MD', 'role': 'CONTACT', 'email': 'adnan.manzur@gosh.nhs.uk'}], 'facility': 'Great Ormond Street Hospital for Children NHS Foundation Trust', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'city': 'London', 'country': 'United Kingdom', 'contacts': [{'name': 'Elizabeth Wraige, MD', 'role': 'CONTACT', 'email': 'Elizabeth.wraige@gstt.nhs.uk', 'phone': '020 7188 7188'}, {'name': 'Vasantha Gowda, MD', 'role': 'CONTACT'}], 'facility': "Evelina London Children's Hospital", 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'city': 'London', 'country': 'United Kingdom', 'contacts': [{'name': 'Adnan Manzur, MD', 'role': 'CONTACT', 'email': 'adnan.manzur@gosh.nhs.uk', 'phone': '020 7405 9200'}, {'name': 'Adnan Manzur, MD', 'role': 'CONTACT'}], 'facility': 'Great Ormond Street Hospital for Children NHS Foundation Trust', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'city': 'Manchester', 'country': 'United Kingdom', 'contacts': [{'name': 'Imelda Hughes, MD', 'role': 'CONTACT', 'email': 'Imelda.hughes@mft.nhs.uk', 'phone': '0161 276 1234'}, {'name': 'Imelda Hughes, MD', 'role': 'CONTACT'}], 'facility': 'Manchester University NHS Foundation Trust', 'geoPoint': {'lat': 53.48095, 'lon': -2.23743}}, {'city': 'Newcastle', 'country': 'United Kingdom', 'facility': 'Newcastle Hospitals NHS Foundation Trust', 'geoPoint': {'lat': 54.21804, 'lon': -5.88979}}, {'city': 'Nottingham', 'country': 'United Kingdom', 'contacts': [{'name': 'Neuro Admin, MD', 'role': 'CONTACT', 'email': 'paedneuroadmin@nuh.nhs.uk', 'phoneExt': '0115 948 554'}, {'name': 'Krupa Torne, MD', 'role': 'CONTACT'}], 'facility': 'Nottingham University Hospitals NHS Trust', 'geoPoint': {'lat': 52.9536, 'lon': -1.15047}}, {'city': 'Oswestry', 'country': 'United Kingdom', 'contacts': [{'name': 'Richa Kulshrestha, MD', 'role': 'CONTACT', 'email': 'richakulshrestha@nhs.net', 'phone': '01691 404000'}, {'name': 'Richa Kulshrestha, MD', 'role': 'CONTACT'}], 'facility': 'The Robert Jones and Agnes Hunt Orthopaedic Hospital', 'geoPoint': {'lat': 52.86195, 'lon': -3.05497}}, {'city': 'Oxford', 'country': 'United Kingdom', 'contacts': [{'name': 'Richard Tarver', 'role': 'CONTACT', 'email': 'childneuromuscular@ouh.nhs.uk', 'phone': '0300 304 7777'}, {'name': 'Sithara Ramdas, MD', 'role': 'CONTACT'}], 'facility': 'Oxford University Hospitals', 'geoPoint': {'lat': 51.75222, 'lon': -1.25596}}, {'city': 'Plymouth', 'country': 'United Kingdom', 'contacts': [{'name': 'Richard Tomlinson, MD', 'role': 'CONTACT', 'email': 'richard.tomlinson@nhs.net', 'phone': '01752 202082'}, {'name': 'Heather Jarvis, MD', 'role': 'CONTACT'}], 'facility': 'University Hospitals Plymouth NHS Trust', 'geoPoint': {'lat': 50.37153, 'lon': -4.14305}}, {'city': 'Sheffield', 'country': 'United Kingdom', 'contacts': [{'name': 'Sheffield Admin Neuromuscular', 'role': 'CONTACT', 'email': 'scn-tr.sheffield.nmtherapy@nhs.net', 'phone': '0114 271 1900'}, {'name': 'Min Ong, MD', 'role': 'CONTACT'}], 'facility': 'Sheffield Teaching Hospitals NHS Foundation Trust', 'geoPoint': {'lat': 53.38297, 'lon': -1.4659}}, {'city': 'Southampton', 'country': 'United Kingdom', 'contacts': [{'name': 'Marjorie Illingworth, MD', 'role': 'CONTACT', 'email': 'marjorie.illingworth@uhs.nhs.uk', 'phone': '023 8077 7222'}, {'name': 'Marjorie Illingworth, MD', 'role': 'CONTACT'}], 'facility': 'University Hospital Southampton NHS Foundation Trust', 'geoPoint': {'lat': 50.90395, 'lon': -1.40428}}, {'city': 'Swansea', 'country': 'United Kingdom', 'contacts': [{'name': 'Kate Skone, MD', 'role': 'CONTACT', 'email': 'Kate.Skone@wales.nhs.uk', 'phone': '01639 683344'}, {'name': 'Kate Skone', 'role': 'CONTACT'}], 'facility': 'Swansea Bay University Health Board', 'geoPoint': {'lat': 51.62079, 'lon': -3.94323}}], 'centralContacts': [{'name': 'Dr Adnan Manzur, FRCPCH', 'role': 'CONTACT', 'email': 'adnan.manzur@gosh.nhs.uk', 'phone': '+44 20 7405 9200', 'phoneExt': '5849'}, {'name': 'Study Inbox', 'role': 'CONTACT', 'email': 'northstar.network@ucl.ac.uk'}], 'overallOfficials': [{'name': 'Professor Francesco Muntoni, FRCPCH FMed Sci', 'role': 'STUDY_CHAIR', 'affiliation': 'University College, London'}, {'name': 'Professor Giovanni Baranello, MD, PhD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'University College, London'}, {'name': 'Professor Michela Guglieri, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Newcastle University'}, {'name': 'Professor Rosaline Quinlivan, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'University College, London'}]}, 'ipdSharingStatementModule': {'infoTypes': ['CSR'], 'timeFrame': '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.', 'ipdSharing': 'YES', 'description': 'Fully anonymised summary-level data can be made available on request for collaborations. The data dictionary is currently publicly available.', 'accessCriteria': 'Academic researchers may contact the study central research group to request collaboration, usage of assessments, summary level of data.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University College, London', 'class': 'OTHER'}, 'collaborators': [{'name': 'Muscular Dystrophy UK', 'class': 'UNKNOWN'}], 'responsibleParty': {'type': 'SPONSOR'}}}}