Ignite Creation Date:
2025-12-24 @ 2:54 PM
Ignite Modification Date:
2026-01-01 @ 10:59 AM
Study NCT ID:
NCT03652259
Status:
TERMINATED
Last Update Posted:
2025-09-10
First Post:
2018-08-28
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'C535902', 'term': 'Limb-girdle muscular dystrophy, type 2E'}, {'id': 'D049288', 'term': 'Muscular Dystrophies, Limb-Girdle'}], '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': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 6}}, 'statusModule': {'whyStopped': 'Study is being terminated due to a business decision.', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2018-10-27', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-09', 'completionDateStruct': {'date': '2025-01-14', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-09-03', 'studyFirstSubmitDate': '2018-08-28', 'studyFirstSubmitQcDate': '2018-08-28', 'lastUpdatePostDateStruct': {'date': '2025-09-10', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2018-08-29', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-01-14', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)', 'timeFrame': 'Baseline up to 7 years'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline in Quantity of Beta-Sarcoglycan (β-SG) Protein Expression at Day 60, as Measured by Western Blot', 'timeFrame': 'Baseline, Day 60', 'description': 'β-SG gene expression levels will be quantified by Western Blot and compared between pre and post muscle biopsies.'}, {'measure': 'Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunofluorescence', 'timeFrame': 'Baseline, Day 60', 'description': 'β-SG gene expression levels will be quantified by immunofluorescence and compared between pre and post muscle biopsies.'}, {'measure': 'Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunohistochemistry Percent B-SG Positive Fibers', 'timeFrame': 'Baseline, Day 60'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['limb girdle muscular dystrophy', 'LGMD2E', 'beta-sarcoglycan', 'gene transfer', 'adeno-associated virus', 'LGMDR4'], 'conditions': ['Limb-Girdle Muscular Dystrophy, Type 2E']}, 'referencesModule': {'references': [{'pmid': '38177855', 'type': 'DERIVED', 'citation': 'Mendell JR, Pozsgai ER, Lewis S, Griffin DA, Lowes LP, Alfano LN, Lehman KJ, Church K, Reash NF, Iammarino MA, Sabo B, Potter R, Neuhaus S, Li X, Stevenson H, Rodino-Klapac LR. Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results. Nat Med. 2024 Jan;30(1):199-206. doi: 10.1038/s41591-023-02730-9. Epub 2024 Jan 4.'}]}, 'descriptionModule': {'briefSummary': 'The proposed clinical trial is the first-in-human, single-center, open-label, gene delivery study of SRP-9003 (bidridistrogene xeboparvovec) in participants with LGMD2E.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '15 Years', 'minimumAge': '4 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'INCLUSION CRITERIA\n\n* Males or females of any ethnic group\n* β-SG deoxyribonucleic acid (DNA) gene mutations at both alleles\n* Weakness demonstrated based on history of difficulty in running, jumping and climbing stairs\n* A 100 meter walk/run (MWR) test result: ≥40 % of predicted for age-, height-, gender-, and weight-matched healthy controls at the screening visit\n\nEXCLUSION CRITERIA\n\n* Active viral infection based on clinical observations\n* Cardiac magnetic resonance imaging (MRI) determined left ventricular ejection fraction (LVEF) \\<40%\n* Serological evidence of human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection\n* Diagnosis of (or ongoing treatment for) an autoimmune disease\n* Abnormal laboratory values considered clinically significant\n* Concomitant illness or requirement for chronic drug treatment that, in the opinion of the Principal Investigator, creates unnecessary risks for gene transfer.\n\nOther inclusion/exclusion criteria apply.'}, 'identificationModule': {'nctId': 'NCT03652259', 'briefTitle': 'Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Sarepta Therapeutics, Inc.'}, 'officialTitle': 'A Single-Center, Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9003 Administered by Systemic Infusion in Subjects With LGMD2E (β-Sarcoglycan Deficiency)', 'orgStudyIdInfo': {'id': 'SRP-9003-101'}, 'secondaryIdInfos': [{'id': 'IRB17-00253', 'type': 'OTHER', 'domain': 'Sarepta Therapeutics'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Cohort 1: SRP-9003', 'description': 'Participants will receive a single intravenous (IV) infusion of SRP-9003 at a prespecified dose.', 'interventionNames': ['Genetic: SRP-9003']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 2: SRP-9003', 'description': 'Participants will receive a single IV infusion of SRP-9003. Dose will be determined based on the findings from Cohort 1.', 'interventionNames': ['Genetic: SRP-9003']}], 'interventions': [{'name': 'SRP-9003', 'type': 'GENETIC', 'otherNames': ['LGMD2E vector', 'bidridistrogene xeboparvovec'], 'description': 'SRP-9003 will be administered through a single systemic injection.', 'armGroupLabels': ['Cohort 1: SRP-9003', 'Cohort 2: SRP-9003']}]}, 'contactsLocationsModule': {'locations': [{'zip': '43205', 'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'facility': "Nationwide Children's Hospital", 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}], 'overallOfficials': [{'name': 'Medical Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Sarepta Therapeutics, Inc.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Sarepta Therapeutics, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}