Ignite Creation Date:
2025-12-24 @ 11:46 PM
Ignite Modification Date:
2026-02-25 @ 9:40 PM
Study NCT ID:
NCT05906251
Status:
TERMINATED
Last Update Posted:
2025-09-04
First Post:
2023-06-07
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Gene Transfer Study to Evaluate the Safety, Tolerability and Efficacy of SRP-6004 in Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2B/R2 (LGMD2B/R2, Dysferlin [DYSF] Related)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D049288', 'term': 'Muscular Dystrophies, Limb-Girdle'}, {'id': 'C535899', 'term': 'Limb-girdle muscular dystrophy, type 2B'}], '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'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 2}}, 'statusModule': {'whyStopped': 'Study is being terminated due to a business decision.', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2023-05-22', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-08', 'completionDateStruct': {'date': '2025-06-13', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-08-27', 'studyFirstSubmitDate': '2023-06-07', 'studyFirstSubmitQcDate': '2023-06-07', 'lastUpdatePostDateStruct': {'date': '2025-09-04', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2023-06-15', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-06-13', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)', 'timeFrame': 'Baseline up to Month 60'}], 'secondaryOutcomes': [{'measure': 'Change from Baseline in Percent of Normal DYSF Protein Expression as Measured by Western Blot', 'timeFrame': 'Baseline, Day 90 and Month 15'}, {'measure': 'Change from Baseline in Percent of Normal DYSF Protein Expression as Measured by Immunofluorescence (IF) Fiber Intensity', 'timeFrame': 'Baseline, Day 90 and Month 15'}, {'measure': 'Change from Baseline in Percent of Normal DYSF Protein Expression as Assessed by IF Percent DYSF Positive Fibers (PPF: DYSF)', 'timeFrame': 'Baseline, Day 90 and Month 15'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Ambulatory', 'Gene-Delivery', 'LGMD2B/R2', 'Dysferlin', 'LGMD'], 'conditions': ['Limb Girdle Muscular Dystrophy']}, 'descriptionModule': {'briefSummary': 'The primary purpose of this study is to evaluate the safety of SRP-6004 administered by intravenous (IV) infusion in ambulatory participants with LGMD2B/R2 (DYSF related).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT'], 'maximumAge': '50 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic DYSF Deoxyribonucleic acid (DNA) gene mutations as documented prior to screening visits.\n* Participants must be ambulatory per protocol specified criteria.\n* Ability to cooperate with motor assessment testing.\n* Has accessible and intact lower and upper extremity musculature for biopsy.\n* Have adeno-associated virus rhesus serotype 74 (rAAVrh74) antibody titers \\< 1:400 (that is, not elevated) as determined by enzyme-linked immunosorbent assay (ELISA).\n\nExclusion Criteria:\n\n* Exposure to gene therapy, investigational medication, or other protocol-specified treatment within the protocol specified time limits.\n* Abnormality in protocol-specified diagnostic evaluations or laboratory tests.\n* Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer.\n\nNote: Other inclusion or exclusion criteria could apply.'}, 'identificationModule': {'nctId': 'NCT05906251', 'briefTitle': 'A Gene Transfer Study to Evaluate the Safety, Tolerability and Efficacy of SRP-6004 in Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2B/R2 (LGMD2B/R2, Dysferlin [DYSF] Related)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Sarepta Therapeutics, Inc.'}, 'officialTitle': 'An Open-Label, Systemic Gene Transfer Study to Evaluate the Safety, Tolerability, and Efficacy of SRP 6004 Administered by Systemic Infusion in Ambulatory Subjects With Limb Girdle Muscular Dystrophy Type 2B/R2 (LGMD2B/R2, Dysferlin Related)', 'orgStudyIdInfo': {'id': 'SRP-6004-102'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'SRP-6004', 'description': 'Participants will receive single IV infusion of SRP-6004 on Day 1.', 'interventionNames': ['Genetic: SRP-6004']}], 'interventions': [{'name': 'SRP-6004', 'type': 'GENETIC', 'otherNames': ['rAAVrh74.MHCK7.DYSF.DV'], 'description': 'Single IV infusion of SRP-6004', 'armGroupLabels': ['SRP-6004']}]}, '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'}}}}