Ignite Creation Date:
2025-12-25 @ 1:32 AM
Ignite Modification Date:
2025-12-25 @ 11:46 PM
Study NCT ID:
NCT04317794
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-07-17
First Post:
2020-03-05
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009134', 'term': 'Muscular Atrophy, Spinal'}], 'ancestors': [{'id': 'D013118', 'term': 'Spinal Cord Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D016472', 'term': 'Motor Neuron Disease'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000590926', 'term': 'nusinersen'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'CASE_ONLY'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 74}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2019-07-02', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-07', 'completionDateStruct': {'date': '2027-02-05', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-07-15', 'studyFirstSubmitDate': '2020-03-05', 'studyFirstSubmitQcDate': '2020-03-20', 'lastUpdatePostDateStruct': {'date': '2025-07-17', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2020-03-23', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2027-02-05', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)', 'timeFrame': 'Up to End of Treatment (2 Years)', 'description': 'An AE is any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An SAE is any untoward medical occurrence that at any dose results in death, places the participant at immediate risk of death, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect, or is a medically important event.'}], 'secondaryOutcomes': [{'measure': 'Change from Baseline in Hammersmith Infant Neurological Examination (HINE) Section 2 Scores', 'timeFrame': 'Baseline, Day 63 and Approximately Every 4 Months Up to End of Treatment (2 years)', 'description': 'Section 2 of the HINE is used to assess motor milestones of the participants. It is composed of 8 motor milestone categories: voluntary grasp, ability to kick in supine position, head control, rolling, sitting, crawling, standing, and walking.'}, {'measure': 'Change from Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE)', 'timeFrame': 'Baseline, Day 63 and Approximately Every 4 Months Up to End of Treatment (2 years)', 'description': 'The HFMSE is a tool used to assess motor function in children with SMA. The original 20 item Hammersmith Functional Motor Scale (HFMS) was expanded to include 13 additional items to improve sensitivity for the higher functioning ambulant population. Participants are asked to complete a specific movement and are then graded on the quality and execution of that movement. Higher scores indicate higher levels of motor ability where 0=unable, 1=performed with some assistance, and 2=unaided. The overall score is the sum of the scores for all activities.'}, {'measure': 'Time to Death', 'timeFrame': 'Up to End of Treatment (2 years)'}, {'measure': 'Number of Participants with Ventilatory Support', 'timeFrame': 'Up to End of Treatment (2 years)'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False}, 'conditionsModule': {'conditions': ['Muscular Atrophy, Spinal']}, 'descriptionModule': {'briefSummary': 'The primary objective is to evaluate the safety of nusinersen sodium injection in the postmarketing setting in Korea. The secondary objective is to evaluate the effectiveness of nusinersen sodium injection in the postmarketing setting in Korea.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Participants who are receiving or about to initiate treatment with commercial Spinraza in Korea according to the local marketing authorization (including documentation of SMA diagnosis), agree to the collection and use of data as specified in the protocol, and meet the eligibility criteria.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n* Currently receiving or about to initiate treatment with commercial Spinraza in the postmarketing setting\n* Genetic documentation of 5q-linked SMA\n\nKey Exclusion Criteria:\n\n* Hypersensitivity to the active substance or any of the excipients of Spinraza\n* Ongoing participation or participation within 6 months or 5 half-lives of the agent (whichever is longer) of enrollment in other interventional clinical trials for the treatment of SMA\n* Inability to comply with study requirements\n\nNOTE: Other protocol defined Inclusion/Exclusion criteria may apply.'}, 'identificationModule': {'nctId': 'NCT04317794', 'acronym': 'STANDARD', 'briefTitle': 'Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Biogen'}, 'officialTitle': 'A Multicenter, Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium) When Used in Routine Medical Practice in Korea', 'orgStudyIdInfo': {'id': '232SM403'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'All Participants', 'description': 'Participants who were prescribed with nusinersen sodium injection in Korea according to local marketing authorization.', 'interventionNames': ['Drug: Nusinersen Sodium Injection']}], 'interventions': [{'name': 'Nusinersen Sodium Injection', 'type': 'DRUG', 'otherNames': ['BIIB058', 'ISIS 396443', 'Spinraza'], 'description': 'Administered as specified in the treatment arm.', 'armGroupLabels': ['All Participants']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Busan', 'country': 'South Korea', 'facility': 'Pusan Natioanl University Hospital', 'geoPoint': {'lat': 35.10168, 'lon': 129.03004}}, {'city': 'Changwon', 'country': 'South Korea', 'facility': 'Samsung Changwon Hospital', 'geoPoint': {'lat': 35.22806, 'lon': 128.68111}}, {'city': 'Cheongju-si', 'country': 'South Korea', 'facility': 'Chungbuk National University Hospital', 'geoPoint': {'lat': 36.63722, 'lon': 127.48972}}, {'city': 'Daegu', 'country': 'South Korea', 'facility': 'Kyungpook National University Hospital', 'geoPoint': {'lat': 35.87028, 'lon': 128.59111}}, {'city': 'Daegu', 'country': 'South Korea', 'facility': 'Yeungnam University Hospital', 'geoPoint': {'lat': 35.87028, 'lon': 128.59111}}, {'city': 'Daejeon', 'country': 'South Korea', 'facility': 'Chungnam National University Hospital', 'geoPoint': {'lat': 36.34913, 'lon': 127.38493}}, {'city': 'Gwangju', 'country': 'South Korea', 'facility': 'Chonnam National University Hospital', 'geoPoint': {'lat': 35.15472, 'lon': 126.91556}}, {'city': 'Jeonju', 'country': 'South Korea', 'facility': 'Chonbuk National University Hospital', 'geoPoint': {'lat': 35.82194, 'lon': 127.14889}}, {'zip': '03722', 'city': 'Seoul', 'country': 'South Korea', 'facility': 'Severance Hospital', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}, {'city': 'Seoul', 'country': 'South Korea', 'facility': 'Asan Medical Center', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}, {'city': 'Seoul', 'country': 'South Korea', 'facility': 'Korea university Anam Hospital', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}, {'city': 'Seoul', 'country': 'South Korea', 'facility': 'Korea University Guro Hospital', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}, {'city': 'Seoul', 'country': 'South Korea', 'facility': 'Samsung Medical Center', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}, {'city': 'Seoul', 'country': 'South Korea', 'facility': 'Seoul Natioanl University Hospital', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}, {'city': 'Seoul', 'country': 'South Korea', 'facility': 'Severance Hospital, Yonsei University Health System', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}, {'city': 'Yangsan', 'country': 'South Korea', 'facility': 'Pusan National University Yangsan Hospital', 'geoPoint': {'lat': 35.34199, 'lon': 129.03358}}], 'overallOfficials': [{'name': 'Medical Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Biogen'}]}, 'ipdSharingStatementModule': {'url': 'https://vivli.org/', 'ipdSharing': 'YES', 'description': "In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com"}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Biogen', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}