Ignite Creation Date:
2025-12-24 @ 9:06 PM
Ignite Modification Date:
2025-12-27 @ 7:15 PM
Study NCT ID:
NCT04010604
Status:
RECRUITING
Last Update Posted:
2019-09-23
First Post:
2019-07-01
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Registered Cohort Study on SMA
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'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 2000}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2019-07-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2019-09', 'completionDateStruct': {'date': '2049-12-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2019-09-19', 'studyFirstSubmitDate': '2019-07-01', 'studyFirstSubmitQcDate': '2019-07-03', 'lastUpdatePostDateStruct': {'date': '2019-09-23', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2019-07-08', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2039-12-31', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'The time to death', 'timeFrame': 'From date of enrollment until the date of death from any cause, assessed up to 20years'}, {'measure': 'The correlation of genotype and phenotype', 'timeFrame': 'From date of enrollment until the date of death from any cause, assessed up to 20years', 'description': 'Genotype is defined by survival motor neuron (SMN) 2 copy number(s) and phenotype is defined by clinical types and characteristics.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Spinal Muscular Atrophy']}, 'descriptionModule': {'briefSummary': 'Spinal muscular atrophy (SMA) is an autosomal recessive disease that causes progressive muscle wasting and weakness due to loss of motor neurons in the spinal cord. This is a registered cohort of spinal muscular atrophy (SMA) type I，II and III in China. This study will provide further insights into the clinical course of SMA including overall survival, demographic characteristics, motor function, respiratory support, feeding and nutritional support, growth and development. The correlation of genotype and phenotype will be conducted.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '70 Years', 'minimumAge': '1 Week', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'SMA patients are diagnosed with SMN1 gene deletion or mutation.', 'healthyVolunteers': True, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Patients with SMA types I, II and III\n* Asymptomatic SMA carriers\n* Relatives of SMA patients or carriers\n* Unrelated healthy controls\n* Participants or Parent(s)/legal guardian(s) willing and able to complete the informed consent process\n\nExclusion Criteria:\n\n\\* Participants are unable to comply with trial procedures and visit schedule'}, 'identificationModule': {'nctId': 'NCT04010604', 'briefTitle': 'A Registered Cohort Study on SMA', 'organization': {'class': 'OTHER', 'fullName': 'First Affiliated Hospital of Fujian Medical University'}, 'officialTitle': 'A Registered Cohort Study on Spinal Muscular Atrophy', 'orgStudyIdInfo': {'id': 'MRCTA,ECFAHOFFMU[2019]196'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'SMA type I'}, {'label': 'SMA type II'}, {'label': 'SMA type III'}, {'label': 'Asymptomatic carriers of SMA'}, {'label': 'Relatives of SMA patients and carriers'}, {'label': 'Unrelated healthy controls'}]}, 'contactsLocationsModule': {'locations': [{'zip': '350005', 'city': 'Fuzhou', 'state': 'Fujian', 'status': 'RECRUITING', 'country': 'China', 'contacts': [{'name': 'Wan-Jin Chen, PhD', 'role': 'CONTACT', 'email': 'wanjinchen75@fjmu.edu.cn', 'phone': '86-0591-87982772', 'phoneExt': '+1386061359'}, {'name': 'Wan-Jin Chen, MD,PhD', 'role': 'PRINCIPAL_INVESTIGATOR'}, {'name': 'Ning Wang, MD, PhD', 'role': 'SUB_INVESTIGATOR'}], 'facility': 'Department of Neurology, First Affiliated Hospital Fujian Medical University', 'geoPoint': {'lat': 26.06139, 'lon': 119.30611}}], 'centralContacts': [{'name': 'Yi Lin, PhD', 'role': 'CONTACT', 'email': 'linyi7811@163.com', 'phone': '86-0591-87982772'}, {'name': 'Wan-Jian Chen, PhD', 'role': 'CONTACT', 'email': 'wanjinchen75@fjmu.edu.cn', 'phone': '86-0591-87982772'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Wan-Jin Chen', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR_INVESTIGATOR', 'investigatorTitle': 'The Vice-Director for the Department of Neurology', 'investigatorFullName': 'Wan-Jin Chen', 'investigatorAffiliation': 'First Affiliated Hospital of Fujian Medical University'}}}}