Ignite Creation Date:
2025-12-24 @ 9:28 PM
Ignite Modification Date:
2025-12-29 @ 11:20 AM
Study NCT ID:
NCT01153932
Status:
COMPLETED
Last Update Posted:
2014-08-25
First Post:
2010-06-29
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Phase II Doubleblind Exploratory Study of GSK2402968 in Ambulant Subjects With Duchenne Muscular Dystrophy
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'submissionInfos': [{'releaseDate': '2017-07-23', 'unreleaseDateUnknown': True}, {'resetDate': '2018-02-09', 'releaseDate': '2017-07-24'}, {'releaseDate': '2018-02-13', 'unreleaseDate': '2018-08-15'}], 'estimatedResultsFirstSubmitDate': '2017-07-23'}}, 'conditionBrowseModule': {'meshes': [{'id': 'D009136', 'term': 'Muscular Dystrophies'}, {'id': 'D020388', 'term': 'Muscular Dystrophy, Duchenne'}], 'ancestors': [{'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': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 53}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2010-09'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2013-08', 'completionDateStruct': {'date': '2012-09', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2014-08-21', 'studyFirstSubmitDate': '2010-06-29', 'studyFirstSubmitQcDate': '2010-06-29', 'lastUpdatePostDateStruct': {'date': '2014-08-25', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2010-06-30', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2012-03', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'To assess the efficacy of 2 different dosing regimens of subcutaneous GSK2402968 administered over 24 weeks in ambulant subjects with DMD', 'timeFrame': '48 weeks'}], 'secondaryOutcomes': [{'measure': 'To assess the safety and tolerability of 2 different dosing regimens of subcutaneous GSK2402968 administered over 48 weeks in ambulant subjects', 'timeFrame': 'one year'}, {'measure': 'To assess the PK of 2 different dosing regimens of subcutaneous GSK2402968 administered over 48 weeks in ambulant subjects with DMD', 'timeFrame': '48 weeks'}, {'measure': 'To assess long term efficacy of 2 different dosing regimens of subcutaneous GSK2402968 administered over 48 weeks in ambulant subjects with DMD', 'timeFrame': 'one year'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['968', 'muscular dystrophy', 'duchenne', 'DMD'], 'conditions': ['Muscular Dystrophies']}, 'referencesModule': {'references': [{'pmid': '25209738', 'type': 'DERIVED', 'citation': 'Voit T, Topaloglu H, Straub V, Muntoni F, Deconinck N, Campion G, De Kimpe SJ, Eagle M, Guglieri M, Hood S, Liefaard L, Lourbakos A, Morgan A, Nakielny J, Quarcoo N, Ricotti V, Rolfe K, Servais L, Wardell C, Wilson R, Wright P, Kraus JE. Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study. Lancet Neurol. 2014 Oct;13(10):987-96. doi: 10.1016/S1474-4422(14)70195-4. Epub 2014 Sep 7.'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to determine whether GSK2402968 given as a continuous dose and as an intermittent dose is effective and safe in the treatment of Duchenne muscular dystrophy.', 'detailedDescription': 'This is a phase II, double-blind, exploratory, parallel-group, placebo-controlled clinical study in ambulant subjects with DMD resulting from a mutation that can be corrected by exon skipping induced by GSK2402968. The study aims to randomise 54 subjects. There will be 2 parallel cohorts. Each cohort will include subjects on GSK2402968 and matched placebo in a 2:1 ratio.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '5 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Ambulant subjects with Duchenne muscular dystrophy resulting from a mutation in the DMD gene, confirmed by a state-of-the-art DNA diagnostic technique covering all DMD gene exons, including but not limited to MLPA (Multiplex Ligation-dependent Probe Amplification), CGH (Comparative Genomic Hybridisation) or H-RMCA (High-Resolution Melting Curve Analysis), and correctable by GSK2402968-induced DMD exon 51 skipping,\n* Males, at least 5 years of age and with a life expectancy of at least 1 year\n* Able to rise from floor in ≤7 seconds (without aids/orthoses),\n* Able to complete the 6MWD test with a distance of at least 75m\n* Receiving glucocorticoids for a minimum of 6 months immediately prior to screening, with no significant change in total daily dosage or dosing regimen for a minimum of 3 months immediately prior to screening and a reasonable expectation that total daily dosage and dosing regimen will not change significantly for the duration of the study\n* QTc \\<450msec\n* On adequate contraception\n* Able to comply with and complete all protocol requirements\n\nExclusion Criteria:\n\n* any additional missing exon for DMD\n* Current of history of liver or renal disease or impairment\n* Acute illness within 4 weeks of the first dose\n* Use of prohibited meds within 6 months of fist dose\n* Current participation in any other investigational clinical trial\n* Positive hepatitis B surface antigen, hepatitis C antibody test, or human immunodeficiency virus (HIV) test at screening\n* Symptomatic cardiomyopathy\n* Children in Care'}, 'identificationModule': {'nctId': 'NCT01153932', 'briefTitle': 'Phase II Doubleblind Exploratory Study of GSK2402968 in Ambulant Subjects With Duchenne Muscular Dystrophy', 'organization': {'class': 'INDUSTRY', 'fullName': 'GlaxoSmithKline'}, 'officialTitle': 'A Phase II, Double Blind, Exploratory, Parallel-group, Placebocontrolled Clinical Study to Assess Two Dosing Regimens of GSK2402968 for Efficacy, Safety, Tolerability and Pharmacokinetics in Ambulant Subjects With Duchenne Muscular Dystrophy', 'orgStudyIdInfo': {'id': '114117'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Continuous regimen; 6mg/kg once weekly', 'description': 'Once Weekly', 'interventionNames': ['Drug: GSK2402968', 'Drug: matched placebo']}, {'type': 'EXPERIMENTAL', 'label': 'Intermittent regimen; 6mg/kg twice weekly', 'description': 'Twice weekly on 1st, 3rd and 5th weeks, once weekly on 2nd, 4th and 6th weeks, and no active drug on 7th to 10th week of each 10 week cycle', 'interventionNames': ['Drug: GSK2402968', 'Drug: matched placebo']}], 'interventions': [{'name': 'GSK2402968', 'type': 'DRUG', 'otherNames': ['968'], 'description': 'Subcutaneous injection', 'armGroupLabels': ['Continuous regimen; 6mg/kg once weekly', 'Intermittent regimen; 6mg/kg twice weekly']}, {'name': 'matched placebo', 'type': 'DRUG', 'otherNames': ['Placebo'], 'description': 'Subcutaneous injection', 'armGroupLabels': ['Continuous regimen; 6mg/kg once weekly', 'Intermittent regimen; 6mg/kg twice weekly']}]}, 'contactsLocationsModule': {'locations': [{'zip': '2145', 'city': 'Westmead', 'state': 'New South Wales', 'country': 'Australia', 'facility': 'GSK Investigational Site', 'geoPoint': {'lat': -33.80383, 'lon': 150.98768}}, {'zip': '3052', 'city': 'Parkville', 'state': 'Victoria', 'country': 'Australia', 'facility': 'GSK Investigational Site', 'geoPoint': {'lat': -37.78333, 'lon': 144.95}}, {'zip': '9000', 'city': 'Ghent', 'country': 'Belgium', 'facility': 'GSK Investigational Site', 'geoPoint': {'lat': 51.05, 'lon': 3.71667}}, {'zip': '75651', 'city': 'Paris', 'country': 'France', 'facility': 'GSK Investigational Site', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '79106', 'city': 'Freiburg im Breisgau', 'state': 'Baden-Wurttemberg', 'country': 'Germany', 'facility': 'GSK Investigational Site', 'geoPoint': {'lat': 47.9959, 'lon': 7.85222}}, {'zip': '45122', 'city': 'Essen', 'state': 'North Rhine-Westphalia', 'country': 'Germany', 'facility': 'GSK Investigational Site', 'geoPoint': {'lat': 51.45657, 'lon': 7.01228}}, {'zip': '91240', 'city': 'Jerusalem', 'country': 'Israel', 'facility': 'GSK Investigational Site', 'geoPoint': {'lat': 31.76904, 'lon': 35.21633}}, {'zip': '6525 GA', 'city': 'Nijmegen', 'country': 'Netherlands', 'facility': 'GSK Investigational Site', 'geoPoint': {'lat': 51.8425, 'lon': 5.85278}}, {'zip': '08950', 'city': 'Esplugues (Barcelona)', 'country': 'Spain', 'facility': 'GSK Investigational Site'}, {'zip': '46009', 'city': 'Valencia', 'country': 'Spain', 'facility': 'GSK Investigational Site', 'geoPoint': {'lat': 39.47391, 'lon': -0.37966}}, {'zip': '06100', 'city': 'Ankara', 'country': 'Turkey (Türkiye)', 'facility': 'GSK Investigational Site', 'geoPoint': {'lat': 39.91987, 'lon': 32.85427}}, {'zip': 'WC1N 1EH', 'city': 'London', 'country': 'United Kingdom', 'facility': 'GSK Investigational Site', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'NE1 3BZ', 'city': 'Newcastle upon Tyne', 'country': 'United Kingdom', 'facility': 'GSK Investigational Site', 'geoPoint': {'lat': 54.97328, 'lon': -1.61396}}], 'overallOfficials': [{'name': 'GSK Clinical Trials', 'role': 'STUDY_DIRECTOR', 'affiliation': 'GlaxoSmithKline'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'GlaxoSmithKline', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}, 'annotationSection': {'annotationModule': {'unpostedAnnotation': {'unpostedEvents': [{'date': '2017-07-23', 'type': 'RELEASE'}, {'type': 'UNRELEASE', 'dateUnknown': True}, {'date': '2017-07-24', 'type': 'RELEASE'}, {'date': '2018-02-09', 'type': 'RESET'}, {'date': '2018-02-13', 'type': 'RELEASE'}, {'date': '2018-08-15', 'type': 'UNRELEASE'}], 'unpostedResponsibleParty': 'GlaxoSmithKline'}}}}