Ignite Creation Date:
2025-12-24 @ 10:06 PM
Ignite Modification Date:
2025-12-25 @ 7:43 PM
Study NCT ID:
NCT00428935
Status:
COMPLETED
Last Update Posted:
2013-02-05
First Post:
2007-01-26
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D020388', 'term': 'Muscular Dystrophy, Duchenne'}, {'id': 'D009136', 'term': 'Muscular Dystrophies'}], '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': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 6}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2006-03'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2013-02', 'completionDateStruct': {'date': '2010-07', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2013-02-04', 'studyFirstSubmitDate': '2007-01-26', 'studyFirstSubmitQcDate': '2007-01-26', 'lastUpdatePostDateStruct': {'date': '2013-02-05', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2007-01-30', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2009-03', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants with Adverse Events as a Measure of Safety and Tolerability', 'timeFrame': 'followed for 2 years post injection', 'description': 'Physical Exams assessing major organ systems and safety labs (GGT, Bilirubin, Glucose, Amylase, CBC/Diff, AFP, Platelets, PT/PTT, Creatinine, Electrolytes, Total protein, Alkaline phosphatase, and Urinalysis)'}], 'secondaryOutcomes': [{'measure': 'mini-dystrophin gene expression at the site of gene transfer', 'timeFrame': '90 days post injection'}, {'measure': 'Maximal Volume Isometric Contraction Testing as a measure of muscle strength', 'timeFrame': 'out to 2 years post injection'}]}, 'conditionsModule': {'keywords': ['Duchenne', 'Muscle', 'Muscular Dystrophy', 'Gene Therapy', 'Dystrophin', 'Adeno-Associated Virus', 'AAV'], 'conditions': ['Duchenne Muscular Dystrophy']}, 'referencesModule': {'references': [{'pmid': '12215266', 'type': 'BACKGROUND', 'citation': "Watchko J, O'Day T, Wang B, Zhou L, Tang Y, Li J, Xiao X. Adeno-associated virus vector-mediated minidystrophin gene therapy improves dystrophic muscle contractile function in mdx mice. Hum Gene Ther. 2002 Aug 10;13(12):1451-60. doi: 10.1089/10430340260185085."}, {'pmid': '11095710', 'type': 'BACKGROUND', 'citation': 'Wang B, Li J, Xiao X. Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model. Proc Natl Acad Sci U S A. 2000 Dec 5;97(25):13714-9. doi: 10.1073/pnas.240335297.'}, {'pmid': '20925545', 'type': 'RESULT', 'citation': "Mendell JR, Campbell K, Rodino-Klapac L, Sahenk Z, Shilling C, Lewis S, Bowles D, Gray S, Li C, Galloway G, Malik V, Coley B, Clark KR, Li J, Xiao X, Samulski J, McPhee SW, Samulski RJ, Walker CM. Dystrophin immunity in Duchenne's muscular dystrophy. N Engl J Med. 2010 Oct 7;363(15):1429-37. doi: 10.1056/NEJMoa1000228."}, {'pmid': '22068425', 'type': 'RESULT', 'citation': 'Bowles DE, McPhee SW, Li C, Gray SJ, Samulski JJ, Camp AS, Li J, Wang B, Monahan PE, Rabinowitz JE, Grieger JC, Govindasamy L, Agbandje-McKenna M, Xiao X, Samulski RJ. Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector. Mol Ther. 2012 Feb;20(2):443-55. doi: 10.1038/mt.2011.237. Epub 2011 Nov 8.'}], 'seeAlsoLinks': [{'url': 'http://www.ccri.net', 'label': "Columbus Children's Research Institute"}, {'url': 'http://www.askbio.com', 'label': 'Asklepios BioPharmaceutical Inc.'}, {'url': 'http://www.mdausa.org', 'label': 'Muscular Dystrophy Association'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to determine the safety of a miniature dystrophin gene in the treatment of progressive muscle weakness due to Duchenne Muscular Dystrophy (DMD).', 'detailedDescription': "This phase I randomized double blind dose escalation study investigates the safety and efficacy of the mini-dystrophin gene transferred to the biceps muscle for Duchenne muscular dystrophy patients, ages 5 to 12 years of age, using a recombinant adeno-associated virus. Eligible participants must have a known dystrophin gene mutation and may be concurrently treated with corticoid steroids. The mini-dystrophin gene or a placebo agent (normal saline or empty viral capsids) are injected directly into both biceps muscles while under conscious sedation. Following the gene transfer, patients are admitted to the hospital for 48 hours of observation followed by weekly outpatient visits at the Columbus Children's Hospital Neuromuscular Clinic. A bilateral muscle biopsy is preformed following 6 weeks with long term follow up will consisting of bi-annual visits for the next 2 years."}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD'], 'maximumAge': '15 Years', 'minimumAge': '5 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Known null mutation of the Dystrophin gene\n* Male age of 5 years or older\n* If taking corticosteroids, must have dose unchanged for the past 3 months\n* Serum creatine kinase elevation greater than 10x normal value (established by Children's Hospital)\n* Progressive, symmetrical proximal muscle weakness of arms and legs\n\nExclusion Criteria:\n\n* Unable to cooperate for muscle strength testing\n* Joint contractures that prohibit muscle strength testing\n* Concomitant illness\n* Individuals predisposed to excessive vagal responses (bradyarrhythmia or hypotension)\n* Controlled substance abuse"}, 'identificationModule': {'nctId': 'NCT00428935', 'briefTitle': 'Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy', 'organization': {'class': 'OTHER', 'fullName': "Nationwide Children's Hospital"}, 'officialTitle': 'Phase 1 Clinical Trial of rAAV2.5-CMV-mini-Dystrophin Gene Vector in Duchenne Muscular Dystrophy', 'orgStudyIdInfo': {'id': 'CCRI IRB05-00118'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Low Dose', 'description': 'Low dose cohort - 2.0E10 vg/kg', 'interventionNames': ['Biological: rAAV2.5-CMV-minidystrophin (d3990)']}, {'type': 'EXPERIMENTAL', 'label': 'High Dose', 'description': 'High Dose - 1.0E11 vg/kg', 'interventionNames': ['Biological: rAAV2.5-CMV-minidystrophin (d3990)']}], 'interventions': [{'name': 'rAAV2.5-CMV-minidystrophin (d3990)', 'type': 'BIOLOGICAL', 'description': 'Recombinant adeno-associated virus (AAV) carrying a truncated human dystrophin gene (mini-dystrophin) expressed from a cytomegalovirus (CMV) promoter.', 'armGroupLabels': ['High Dose', 'Low Dose']}]}, 'contactsLocationsModule': {'locations': [{'zip': '43205', 'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'facility': "Columbus Children's Hospital", 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}], 'overallOfficials': [{'name': 'Jerry R. Mendell, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "Nationwide Children's Hospital"}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': "Nationwide Children's Hospital", 'class': 'OTHER'}, 'collaborators': [{'name': 'AskBio Inc', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Director Center for Gene Therapy', 'investigatorFullName': 'Jerry R. Mendell', 'investigatorAffiliation': "Nationwide Children's Hospital"}}}}