Ignite Creation Date:
2025-12-25 @ 2:07 AM
Ignite Modification Date:
2025-12-26 @ 2:40 AM
Study NCT ID:
NCT05187260
Status:
UNKNOWN
Last Update Posted:
2022-01-26
First Post:
2021-12-25
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Antisense Oligonucleotide for Spinal Muscular Atrophy
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009134', 'term': 'Muscular Atrophy, Spinal'}, {'id': 'C537987', 'term': 'Charcot-Marie-Tooth disease, Type 1F'}], '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': {'bioSpec': {'retention': 'SAMPLES_WITH_DNA', 'description': '5ml of CSF is collected and typically discarded as part of the typical clinical encounter when SPINRAZA® (nusinersen) is administered. This 5ml of CSF will be collected for research purposes at each dosing visit. Blood samples, including whole blood, serum, and plasma (less than 10 ml total) will be collected before intrathecal injection of SPINRAZA® (nusinersen) at each dosing visit.'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 1000}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'RECRUITING', 'startDateStruct': {'date': '2022-01-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-01', 'completionDateStruct': {'date': '2024-01-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2022-01-11', 'studyFirstSubmitDate': '2021-12-25', 'studyFirstSubmitQcDate': '2021-12-25', 'lastUpdatePostDateStruct': {'date': '2022-01-26', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2022-01-11', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2024-01-31', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change in CSF Neurofilament Light Chain levels since baseline', 'timeFrame': 'up to 24months', 'description': 'Measured by Single-molecule Array'}], 'secondaryOutcomes': [{'measure': 'Change in serum Neurofilament Light Chain levels since baseline', 'timeFrame': 'up to 24months', 'description': 'Measured by Single-molecule Array'}]}, 'oversightModule': {'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Spinal Muscular Atrophy', 'Antisense oligonucleotide', 'Neurofilament light chain'], 'conditions': ['Spinal Muscular Atrophy']}, 'descriptionModule': {'briefSummary': 'This is a longitudinal, multiple-center, observational study of patients genetically confirmed chromosome 5q SMA to monitor the efficacy, safety, tolerability of SPINRAZA® (nusinersen) for up to 24 months.', 'detailedDescription': 'SPINRAZA® (nusinersen) is an antisense oligonucleotide (ASO) designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. Nusinersen, approved by the FDA for treatment of SMA in 2016, was approved by the Chinese National Medical Products Administration in 2019.\n\nThis is a prospective, longitudinal, multi-center, observational study designed to evaluate the efficacy, safety, tolerability and of nusinersen in patients genetically confirmed chromosome 5q SMA in China. Subjects with SMA I/II/III who are planning to initiate treatment with nusinersen will be enrolled in this study. All patients will be treated by their physicians according to standard clinical practice. SPINRAZA® (nusinersen) is administered as an intrathecal injection. A total of 5ml of cerebrospinal fluid (CSF) will be removed prior to administration of SPINRAZA® (nusinersen), which will be collected by the study. Neurofilament light chain (NfL) in CSF and blood will be assessed for the efficacy of nusinersen, as well as motor and pulmonary function.\n\nThere will be a total of nine visits. All the patients with 5q SMA receiving nusinersen will be visited face to face at baseline, day 14, day 28 and day 63 after treatment initiation, and then 4-month intervals through month 24/22.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '50 Years', 'minimumAge': '1 Week', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Patients genetically confirmed 5q SMA including types I, II and III', 'healthyVolunteers': True, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Patients genetically confirmed 5q SMA including types I, II and III, who are planning to initiate treatment with SPINRAZA® (nusinersen) as part of their clinical care plan.\n* Non-5q SMA patients undergoing clinical standard lumbar puncture\n* Non-SMA subjects including Asymptomatic carriers of SMA, relatives of SMA patients and carriers, and patients undergoing clinical standard lumbar puncture\n* Participants or Parent(s)/legal guardian(s) willing and able to complete the informed consent process\n\nExclusion Criteria:\n\n* Contraindication for lumbar puncture\n* Inability to access intrathecal space for nusinersen injection'}, 'identificationModule': {'nctId': 'NCT05187260', 'briefTitle': 'Antisense Oligonucleotide for Spinal Muscular Atrophy', 'organization': {'class': 'OTHER', 'fullName': 'First Affiliated Hospital of Fujian Medical University'}, 'officialTitle': 'Antisense Oligonucleotide Therapy in Spinal Muscular Atrophy: An Observational Study in China', 'orgStudyIdInfo': {'id': 'MRCTA,ECFAH of FMU [2021]489'}}, 'armsInterventionsModule': {'armGroups': [{'label': '5q SMA type I', 'interventionNames': ['Drug: Nusinersen']}, {'label': '5q SMA type II', 'interventionNames': ['Drug: Nusinersen']}, {'label': '5q SMA type III', 'interventionNames': ['Drug: Nusinersen']}, {'label': 'Non-5q SMA'}, {'label': 'Non-SMA subjects', 'description': 'Including asymptomatic carriers of SMA, relatives of SMA patients and carriers, and patients undergoing clinical standard lumbar puncture'}], 'interventions': [{'name': 'Nusinersen', 'type': 'DRUG', 'description': 'SPINRAZA® (nusinersen) prescribed as part of standard of care', 'armGroupLabels': ['5q SMA type I', '5q SMA type II', '5q SMA type III']}]}, 'contactsLocationsModule': {'locations': [{'zip': '100010', 'city': 'Beijing', 'state': 'Beijing Municipality', 'status': 'RECRUITING', 'country': 'China', 'contacts': [{'name': 'Yi Dai, MD,PhD', 'role': 'CONTACT', 'email': 'pumchdy@pumch.cn', 'phone': '13811177531'}, {'name': 'Yi Dai, MD, PhD', 'role': 'SUB_INVESTIGATOR'}], 'facility': 'Department of Neurology, Peking Union Medical College Hospital', 'geoPoint': {'lat': 39.9075, 'lon': 116.39723}}, {'zip': '350001', 'city': 'Fuzhou', 'state': 'Fujian', 'status': 'RECRUITING', 'country': 'China', 'contacts': [{'name': 'Jun Hu, MD, PhD', 'role': 'CONTACT', 'email': 'hujun2252@126.com', 'phone': '18950472106'}, {'name': 'Jun Hu, MD, PhD', 'role': 'SUB_INVESTIGATOR'}], 'facility': 'Department of Pediatrics, Fujian Medical University Union Hospital', 'geoPoint': {'lat': 26.06139, 'lon': 119.30611}}, {'zip': '350005', 'city': 'Fuzhou', 'state': 'Fujian', 'status': 'NOT_YET_RECRUITING', 'country': 'China', 'contacts': [{'name': 'Yi Lin, MD, PhD', 'role': 'CONTACT', 'email': 'linyi7811@163.com', 'phone': '13615039153'}, {'name': 'Wan-Jin Chen, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Department of Neurology, First Affiliated Hospital Fujian Medical University', 'geoPoint': {'lat': 26.06139, 'lon': 119.30611}}], 'centralContacts': [{'name': 'Yi Lin, MD, PhD', 'role': 'CONTACT', 'email': 'linyi7811@163.com', 'phone': '13615039153'}], 'overallOfficials': [{'name': 'Wan-Jin Chen', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'First Affiliated Hospital Fujian Medical University'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'First Affiliated Hospital of Fujian Medical University', 'class': 'OTHER'}, 'collaborators': [{'name': 'Fujian Medical University Union Hospital', 'class': 'OTHER'}, {'name': 'Peking Union Medical College Hospital', 'class': 'OTHER'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'The Director for the Department of Neurology', 'investigatorFullName': 'Wan-Jin Chen', 'investigatorAffiliation': 'First Affiliated Hospital of Fujian Medical University'}}}}