Ignite Creation Date:
2025-12-24 @ 9:08 PM
Ignite Modification Date:
2026-01-22 @ 7:48 AM
Study NCT ID:
NCT01753804
Status:
TERMINATED
Last Update Posted:
2017-12-08
First Post:
2012-12-13
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D020388', 'term': 'Muscular Dystrophy, Duchenne'}, {'id': 'D009135', 'term': 'Muscular Diseases'}, {'id': 'D009136', 'term': 'Muscular Dystrophies'}], 'ancestors': [{'id': 'D020966', 'term': 'Muscular Disorders, Atrophic'}, {'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'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D019370', 'term': 'Observation'}], 'ancestors': [{'id': 'D008722', 'term': 'Methods'}, {'id': 'D008919', 'term': 'Investigative Techniques'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITHOUT_DNA', 'description': 'Blood sampling at 4 time points (first visit then once a year). Urinalysis sampling at 4 time points (first visit then once a year).'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'CASE_ONLY'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 269}}, 'statusModule': {'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2012-09-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-12', 'completionDateStruct': {'date': '2016-10-01', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2017-12-06', 'studyFirstSubmitDate': '2012-12-13', 'studyFirstSubmitQcDate': '2012-12-17', 'lastUpdatePostDateStruct': {'date': '2017-12-08', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2012-12-20', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2016-10-01', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': '6 minute walk distance', 'timeFrame': 'Change from visit 1 walking distance', 'description': 'Participants are asked to walk at their own preferred speed on a fixed distance for 6 minutes. Subjects are warned of the time and that they may stop earlier if they feel unable to continue. Total distance walked within 6 minutes (or until stopping) is recorded.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['DMD', 'Myopathy', 'Natural History', 'Muscular dystrophy', 'Biomarkers', 'Muscle testing'], 'conditions': ['Duchenne Muscular Dystrophy']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'http://www.biomarin.com', 'label': 'BioMarin website'}]}, 'descriptionModule': {'briefSummary': 'To characterize the natural history and progression of Duchenne Muscular Dystrophy (DMD) to help inform the design of future studies, to capture biomarkers of safety and disease progression and to provide comparative data for the development of rare exons for which formal controlled trials are not feasible.', 'detailedDescription': 'This is a prospective study. All DMD patients that fulfil the inclusion/exclusion criteria are eligible although the study is weighted towards ambulant subjects aged 3 years or older. There will be 7 study visits and subjects will be in the study for a maximum of 3 years. Visits will occur every 6 months (+/- 1 month).\n\nUp to 250 DMD subjects planned in the following categories :\n\n* 75 % ambulant subjects aged between 3 and 18 years at study entry\n* 25% non-ambulant subjects with a maximum age of 18 years at study entry\n\nSubjects will be asked to perform muscle testing assessment with a clinical evaluator, such as walking for 6 minutes, climb stairs, breathe in a tube, see how they can move their arms and legs. They will be asked questions about how they feel overall and perform daily activities. These measurements will be assessed every 6 months.\n\nUrine and blood samples will be collected once a year to measure biomarkers that will allow to have a better overview of DMD.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '18 Years', 'minimumAge': '3 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Subjects diagnosed with DMD resulting from a mutation in the DMD gene which is confirmed by a state of the art DNA diagnostic technique covering all DMD gene exons', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Diagnosis of DMD resulting from a mutation in the DMD gene confirmed by a state of the art DNA diagnostic technique covering all DMD gene exons.\n* Age 3 - 18 years\n* Willing and able to comply with protocol requirements\n* Life expectancy of at least 3 years\n* Able to give informed assent and/or consent in writing signed by the subject and/or parent(s)/legal guardian (according to local regulations)\n\nExclusion Criteria:\n\n* Current participation in a clinical study with an Investigational Medicinal Product (IMP)\n* Participation within the previous 1 month in a clinical study with an IMP'}, 'identificationModule': {'nctId': 'NCT01753804', 'briefTitle': 'A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.', 'organization': {'class': 'INDUSTRY', 'fullName': 'BioMarin Pharmaceutical'}, 'officialTitle': 'A Prospective Natural History Study of Progression of Physical Impairment, Activity Limitation and Quality of Life in Duchenne Muscular Dystrophy.', 'orgStudyIdInfo': {'id': 'PRO-DMD-01'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Study participants', 'description': 'All participants will follow the same protocol, including muscle strength and function testing, and blood and urine collection, for a maximum of 7 visits over 3 years.', 'interventionNames': ['Other: Observational study']}], 'interventions': [{'name': 'Observational study', 'type': 'OTHER', 'description': 'There is no medication or device tested in this study. This is an obversational study on the progression of the disease.', 'armGroupLabels': ['Study participants']}]}, 'contactsLocationsModule': {'locations': [{'zip': '95817', 'city': 'Sacramento', 'state': 'California', 'country': 'United States', 'facility': 'UC Davis Health System', 'geoPoint': {'lat': 38.58157, 'lon': -121.4944}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': "Cincinnati Children's Hospital Medical Center", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '43205', 'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'facility': "Nationwide Children's Hospital", 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'city': 'Buenos Aires', 'country': 'Argentina', 'facility': 'Hospital de Pediatria Prof Dr Juan P Garrahan', 'geoPoint': {'lat': -34.61315, 'lon': -58.37723}}, {'city': 'Ghent', 'country': 'Belgium', 'facility': 'Universitair Ziekenhuis', 'geoPoint': {'lat': 51.05, 'lon': 3.71667}}, {'city': 'Leuven', 'country': 'Belgium', 'facility': 'Universitair Ziekenhuis Leuven', 'geoPoint': {'lat': 50.87959, 'lon': 4.70093}}, {'city': 'São Paulo', 'country': 'Brazil', 'facility': 'Hospital das Clinicas da Faculdade de Medicina da USP', 'geoPoint': {'lat': -23.5475, 'lon': -46.63611}}, {'city': 'Toulouse', 'country': 'France', 'facility': 'CHU Hopital des enfants', 'geoPoint': {'lat': 43.60426, 'lon': 1.44367}}, {'city': 'Essen', 'country': 'Germany', 'facility': 'Universitaetsklinikum Essen', 'geoPoint': {'lat': 51.45657, 'lon': 7.01228}}, {'city': 'Freiburg im Breisgau', 'country': 'Germany', 'facility': 'Universitaetsklinikum Freiburg', 'geoPoint': {'lat': 47.9959, 'lon': 7.85222}}, {'city': 'Messina', 'country': 'Italy', 'facility': 'Azienda Ospedaliera Universitaria Policlinico G. Martino', 'geoPoint': {'lat': 38.19394, 'lon': 15.55256}}, {'city': 'Rome', 'country': 'Italy', 'facility': 'Policlinico Univsersitario Agostino Gemelli', 'geoPoint': {'lat': 41.89193, 'lon': 12.51133}}, {'city': 'Leiden', 'country': 'Netherlands', 'facility': 'Leids Universitair Medisch Centrum', 'geoPoint': {'lat': 52.15833, 'lon': 4.49306}}, {'city': 'Nijmegen', 'country': 'Netherlands', 'facility': 'UMC St. Radboud', 'geoPoint': {'lat': 51.8425, 'lon': 5.85278}}, {'city': 'Gothenburg', 'country': 'Sweden', 'facility': 'Drottning Silvias Barn- ochungdomssjukhus', 'geoPoint': {'lat': 57.70716, 'lon': 11.96679}}, {'city': 'Ankara', 'country': 'Turkey (Türkiye)', 'facility': 'Hacettepe University Medical Faculty', 'geoPoint': {'lat': 39.91987, 'lon': 32.85427}}], 'overallOfficials': [{'name': 'Nathalie Goemans, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'UZ Leuven, Belgium'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'BioMarin Pharmaceutical', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}