Ignite Creation Date:
2025-12-25 @ 2:19 AM
Ignite Modification Date:
2026-01-01 @ 6:25 AM
Study NCT ID:
NCT04433234
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-09-19
First Post:
2020-06-12
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D020388', 'term': 'Muscular Dystrophy, Duchenne'}], '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': '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': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 8}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2020-06-30', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-09', 'completionDateStruct': {'date': '2027-01-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-09-18', 'studyFirstSubmitDate': '2020-06-12', 'studyFirstSubmitQcDate': '2020-06-12', 'lastUpdatePostDateStruct': {'date': '2025-09-19', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2020-06-16', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2027-01-31', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence of Adverse Events (AEs)', 'timeFrame': 'From first injection to after the last injection of DS-5141b in this study (within approximately 2 years)'}, {'measure': 'Change in distance walked during 6-minute walk test (6MWT)', 'timeFrame': 'Every 3 months (within approximately 2 years)'}, {'measure': 'Change in time to stand (TTSTAND)', 'timeFrame': 'Every 3 months (within approximately 2 years)'}, {'measure': 'Change in time in Timed up and go test', 'timeFrame': 'Every 3 months (within approximately 2 years)'}, {'measure': 'Change in time in 10-meter Run/Walk test', 'timeFrame': 'Every 3 months (within approximately 2 years)'}, {'measure': 'Change in score in the North Star Ambulatory Assessment (NSAA)', 'timeFrame': 'Every 3 months (within approximately 2 years)'}, {'measure': 'Change in score in the Performance of Upper Limb (PUL)', 'timeFrame': 'Every 3 months (within approximately 2 years)'}, {'measure': 'Change in Left Ventricular Ejection Fraction percentage (LVEF %)', 'timeFrame': 'Every 6 months (within approximately 2 years)'}, {'measure': 'Change in in Forced Vital Capacity (FVC) (percent predicted)', 'timeFrame': 'Every 6 months (within approximately 2 years)'}, {'measure': 'Change in Muscle Strength Measured by Quantitative Muscle Strength Assessment', 'timeFrame': 'Every 3 months (within approximately 2 years)'}], 'secondaryOutcomes': [{'measure': 'Concentration of DS-5141a in plasma', 'timeFrame': 'Every 3 months (within approximately 2 years)'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Duchenne muscular dystrophy', 'DMD', 'Oligonucleotides, antisense', 'Exon skipping', 'DS-5141b'], 'conditions': ['Duchenne Muscular Dystrophy']}, 'descriptionModule': {'briefSummary': 'This is a multicenter, open-label, long-term, extension, phase 2 study to evaluate the safety and efficacy of long-term treatment with DS-5141b in patients with DMD who have completed DS5141-A-J101.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '5 Years', 'genderBased': True, 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Has competed a study of DS5141-A-J101\n\nExclusion Criteria:\n\n* Significant safety issues in a study of DS5141-A-J101\n* Patient who does not consent to use appropriate contraception\n* Patient not appropriate to participant in the study as determined by the Investigator'}, 'identificationModule': {'nctId': 'NCT04433234', 'briefTitle': 'Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy', 'organization': {'class': 'INDUSTRY', 'fullName': 'Daiichi Sankyo'}, 'officialTitle': 'A Phase II, Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy', 'orgStudyIdInfo': {'id': 'DS5141-A-J201'}, 'secondaryIdInfos': [{'id': '205321', 'type': 'OTHER', 'domain': 'JapicCTI'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'DS-5141b 2.0 mg/kg', 'description': 'Participants who will receive DS-5141b 2.0 mg/kg once weekly.', 'interventionNames': ['Drug: DS-5141b']}, {'type': 'EXPERIMENTAL', 'label': 'DS-5141b 6.0 mg/kg', 'description': 'Participants who will receive DS-5141b 6.0 mg/kg once weekly.', 'interventionNames': ['Drug: DS-5141b']}], 'interventions': [{'name': 'DS-5141b', 'type': 'DRUG', 'otherNames': ['Renadirsen'], 'description': 'Administered via subcutaneous injection once weekly', 'armGroupLabels': ['DS-5141b 2.0 mg/kg', 'DS-5141b 6.0 mg/kg']}]}, 'contactsLocationsModule': {'locations': [{'zip': '187-8551', 'city': 'Tokyo', 'state': 'Kodaira-Shi', 'country': 'Japan', 'facility': 'National Center of Neurology and Psychiatry', 'geoPoint': {'lat': 35.6895, 'lon': 139.69171}}, {'zip': '650-0017', 'city': 'Hyōgo', 'country': 'Japan', 'facility': 'Kobe University Hospital', 'geoPoint': {'lat': 43.36667, 'lon': 144.43333}}], 'overallOfficials': [{'name': 'Clinical Study Leader', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Daiichi Sankyo Co., Ltd.'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Daiichi Sankyo Co., Ltd.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}