Ignite Creation Date:
2025-12-24 @ 10:57 PM
Ignite Modification Date:
2026-04-13 @ 6:35 PM
Study NCT ID:
NCT03932669
Status:
COMPLETED
Last Update Posted:
2021-09-29
First Post:
2019-04-25
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Effect of Nilotinib in Cerebellar Ataxia Patients
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'submissionInfos': [{'resetDate': '2024-03-14', 'releaseDate': '2023-09-05'}], 'estimatedResultsFirstSubmitDate': '2023-09-05'}}, 'conditionBrowseModule': {'meshes': [{'id': 'D002524', 'term': 'Cerebellar Ataxia'}, {'id': 'D001259', 'term': 'Ataxia'}, {'id': 'D020754', 'term': 'Spinocerebellar Ataxias'}], 'ancestors': [{'id': 'D002526', 'term': 'Cerebellar Diseases'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D020820', 'term': 'Dyskinesias'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D013132', 'term': 'Spinocerebellar Degenerations'}, {'id': 'D013118', 'term': 'Spinal Cord Diseases'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C498826', 'term': 'nilotinib'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 70}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2018-11-19', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-09', 'completionDateStruct': {'date': '2020-08-13', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-09-23', 'studyFirstSubmitDate': '2019-04-25', 'studyFirstSubmitQcDate': '2019-04-26', 'lastUpdatePostDateStruct': {'date': '2021-09-29', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2019-05-01', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2020-08-13', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Occurrence of adverse events', 'timeFrame': 'Baseline, 1 month, 3 month, 6 month, 12 month', 'description': 'CTCAE version 4.0'}], 'primaryOutcomes': [{'measure': 'Activity of daily living', 'timeFrame': '12 month', 'description': 'Barthel index: score range 0-6'}], 'secondaryOutcomes': [{'measure': 'Cerebellar function', 'timeFrame': '12 month', 'description': 'Scale for the assessment and rating of ataxia (SARA): score range 0-36'}, {'measure': 'General function', 'timeFrame': '12 month', 'description': "Rating scale for Friedreich's ataxia I: score range 0-6"}, {'measure': 'Activity of daily living', 'timeFrame': '12 month', 'description': "Rating scale for Friedreich's ataxia II: score range 0-36"}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['spinocerebellar ataxia', 'treatment', 'nilotinib', 'bcr-abl', 'receptor-tyrosine-kinase-inhibitor'], 'conditions': ['Ataxia, Cerebellar', 'Ataxia, Progressive']}, 'referencesModule': {'references': [{'pmid': '28601294', 'type': 'RESULT', 'citation': 'Lee WJ, Moon J, Kim TJ, Jun JS, Lee HS, Ryu YJ, Lee ST, Jung KH, Park KI, Jung KY, Kim M, Lee SK, Chu K. The c-Abl inhibitor, nilotinib, as a potential therapeutic agent for chronic cerebellar ataxia. J Neuroimmunol. 2017 Aug 15;309:82-87. doi: 10.1016/j.jneuroim.2017.05.015. Epub 2017 May 24.'}, {'pmid': '39009709', 'type': 'DERIVED', 'citation': 'Lee WJ, Moon J, Jang Y, Shin YW, Son H, Shin S, Jeon D, Han D, Lee ST, Park KI, Jung KH, Lee SK, Chu K. Nilotinib treatment outcomes in autosomal dominant spinocerebellar ataxia over one year. Sci Rep. 2024 Jul 15;14(1):16303. doi: 10.1038/s41598-024-67072-z.'}]}, 'descriptionModule': {'briefSummary': "This is an institutional cohort study. Patients confirmed with spinocerebellar ataxia (SCA) and taking or planning to take Nilotinib (Tasigna®) are enrolled in this study. The daily dose of Nilotinib is 150mg-300mg and the patients will be followed up at 1, 3, 6, and 12 months. Rating scale for Friedreich's ataxia I and II and Barthel index are used as general function and daily living performance index. Scale for assessment and rating of ataxia (SARA) are used as an objective measure of cerebellar function. Adverse drug reactions are evaluated based on CTCAE version 4.0.", 'detailedDescription': "1. Patients taking Nilotinib 1-1.Baseline information: demographics, genetic type of SCA, brain MRI, electrocardiography, routine blood evaluations 1-2. Time point of evaluation: At initiation of taking Nilotinib, 1, 3, 6, and 12 months 1-3. Evaluation items (at each time point) Routine check-up: electrocardiography, routine blood evaluations Adverse events: CTCAE version 4.0 Daily living performance: Barthel index General function: Friedreich's ataxia I and II 1-4. Change of drug dose Based on the attending physician's decision\n2. Patients planning to take Nilotinib 2-1.Baseline information: demographics, genetic type of SCA, brain MRI, electrocardiography, routine blood evaluations 2-2. Time point of evaluation: Baseline, 1, 3, 6, and 12 months 2-3. Evaluation items (at each time point) Routine check-up: electrocardiography, routine blood evaluations Adverse events: CTCAE version 4.0 Cerebellar function: Scale for assessment and rating of ataxia (SARA) Daily living performance: Barthel index General function: Friedreich's ataxia I and II 2-4. Change of drug dose Based on the attending physician's decision"}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '85 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Diagnosed as chronic cerebellar ataxia\n* Confirmed as spinocerebellar ataxia by gene test\n\nExclusion Criteria:\n\n* Laboratory abnormalities that could interfere with the proper use of Nilotinib QTc interval \\>450ms on initial electrocardiograph, Hb \\<8.0, WBC\\<2000, ANC \\<1600, PLT \\<140,000, AST \\>200, ALT\\>200, ALP\\>575, Positive HIV serology, active hepatitis B\n* Unstable mental or physical status that could interfere with precise evaluation and proper management of SCA Heart failure (NYHA Grade III or IV), history of major heart disease Pregnancy, on breast feeding'}, 'identificationModule': {'nctId': 'NCT03932669', 'briefTitle': 'Effect of Nilotinib in Cerebellar Ataxia Patients', 'organization': {'class': 'OTHER', 'fullName': 'Seoul National University Hospital'}, 'officialTitle': 'Effect of Nilotinib in Cerebellar Ataxia Patients', 'orgStudyIdInfo': {'id': '1811-074-985'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Nilotinib group', 'description': 'Patients with SCA and taking nilotinib treatment', 'interventionNames': ['Drug: Nilotinib']}], 'interventions': [{'name': 'Nilotinib', 'type': 'DRUG', 'description': '150-300mg daily dose of nilotinib', 'armGroupLabels': ['Nilotinib group']}]}, 'contactsLocationsModule': {'locations': [{'zip': '03080', 'city': 'Seoul', 'country': 'South Korea', 'facility': 'Seoul National University Hospital', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}], 'overallOfficials': [{'name': 'Kon Chu, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Seoul National University Hospital'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO', 'description': 'The IPD will be provided upon request from any qualified investigator.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Seoul National University Hospital', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Professor', 'investigatorFullName': 'Kon Chu', 'investigatorAffiliation': 'Seoul National University Hospital'}}}, 'annotationSection': {'annotationModule': {'unpostedAnnotation': {'unpostedEvents': [{'date': '2023-09-05', 'type': 'RELEASE'}, {'date': '2024-03-14', 'type': 'RESET'}], 'unpostedResponsibleParty': 'Kon Chu, Professor, Seoul National University Hospital'}}}}