Ignite Creation Date:
2025-12-24 @ 2:13 PM
Ignite Modification Date:
2025-12-26 @ 2:39 AM
Study NCT ID:
NCT04431895
Status:
COMPLETED
Last Update Posted:
2025-09-16
First Post:
2020-06-12
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Safety And Efficacy Of Tofacitinib In Patients With Refractory Myasthenia Gravis: A Pilot Study
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009157', 'term': 'Myasthenia Gravis'}], 'ancestors': [{'id': 'D020361', 'term': 'Paraneoplastic Syndromes, Nervous System'}, {'id': 'D009423', 'term': 'Nervous System Neoplasms'}, {'id': 'D009371', 'term': 'Neoplasms by Site'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D010257', 'term': 'Paraneoplastic Syndromes'}, {'id': 'D020274', 'term': 'Autoimmune Diseases of the Nervous System'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D020511', 'term': 'Neuromuscular Junction Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D001327', 'term': 'Autoimmune Diseases'}, {'id': 'D007154', 'term': 'Immune System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C479163', 'term': 'tofacitinib'}]}}, 'protocolSection': {'designModule': {'phases': ['EARLY_PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 19}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2020-06-11', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-09', 'completionDateStruct': {'date': '2024-03-31', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-09-10', 'studyFirstSubmitDate': '2020-06-12', 'studyFirstSubmitQcDate': '2020-06-12', 'lastUpdatePostDateStruct': {'date': '2025-09-16', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2020-06-16', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2024-03-21', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Change From Baseline in Serum Titers of anti-acetylcholine receptor antibodies at Week 16', 'timeFrame': 'Baseline, Week 16', 'description': 'Anti-acetylcholine receptor antibodies are the major pathological autoantibodies of MG. For anti-AChR antibodies-positive candidates, we followed the antibodies titers at Week 16.'}, {'measure': 'Change From Baseline in Inflammatory Cytokines at Week 12', 'timeFrame': 'Baseline, Week 12', 'description': 'MG is an autoimmune disease manifested by impairment at the neuromuscular junction featured by weakness and fatigability. We compared the inflammatory cytokines (IL-6, IL-10, IL-17A, and IL-23) at Baseline and Week 12'}], 'primaryOutcomes': [{'measure': 'Change From Baseline in Quantitative Myasthenia Gravis (QMG) Scores at Month 6', 'timeFrame': 'Baseline, Month 6', 'description': 'A quantitative MG scoring system (QMG Score) is essential in the objective evaluation of therapy for MG.This scoring system is based on quantitative testing of sentinel muscle groups. Total QMG score range from 0 (extreme disease severity) to 39 (none), higher score indicated less disease severity.'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline in Myasthenia Gravis-related Activities of Daily Living (MG-ADL) Scores at Month 6', 'timeFrame': 'Baseline, Month 6', 'description': 'The myasthenia gravis activities of daily living (MG-ADL) profile is an eight-item patient-reported scale developed to assess MG symptoms and their effects on daily activities.Total MG-ADL score range from 0 (none) to 24 (extreme disease conditions), higher score indicated more disease severity.'}, {'measure': 'Change From Baseline in Myasthenia Gravis Composite (MGC) Scores at Month 6', 'timeFrame': 'Baseline, Month 6', 'description': 'The MGC score consists of test items that measure symptoms and signs of MG, with weighted response options. Total MGC score range from 0 (none) to 50 (extreme muscle weakness), higher score indicated more disease severity.'}, {'measure': 'Change From Baseline in Myasthenia Gravis-Quality of Life Questionnaire-15 item (MG-QOL15) Scores at Month 6', 'timeFrame': 'Baseline, Month 6', 'description': "The MG-QOL15 is helpful in informing the clinician about the patient's perception of the extent of and dissatisfaction with MG-related dysfunction. Total MG-QOL15 score range from 0 (none) to 60 (extreme disease severity), higher score indicated more disease severity."}, {'measure': 'Change From Baseline in Doses of Steroids at Month 6', 'timeFrame': 'Baseline, Month 6', 'description': 'Steroids is recommended as the first-line treatment for myasthenia gravis. Here we followed the combined doseage of steroids while administrating tofacitinib for enrolled participants.'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['myasthenia gravis', 'tofacitinib'], 'conditions': ['Myasthenia Gravis, Generalized']}, 'descriptionModule': {'briefSummary': 'This is a 6 month study that will evaluate the efficacy of 5mg tofacitinib tablet taken twice a day in patients with refractory myasthenia gravis. Twenty adult participants will receive a four month treatment of tofacitinib followed by elution for one month.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '70 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* fluctuating muscle weakness and fatigability classified as the Myasthenia Gravis Foundation of America (MGFA) clinical classification Type IIa-IVa;\n* more than 10% amplitude decrement in low frequency repetitive nerve stimulation, and less than 10% amplitude increment in high frequency repetitive nerve stimulation;\n* understanding and assigning the informed consent form, and having a good compliance with treatment.\n\nmeet all of the criteria in above as well as at least one of the below:\n\n* refractory: insufficient response to full dose- and course- steroids treatment (0.75-1mg per kg per day for at least 3-6 months), failure to respond adequately to tacrolimus (no less than 3mg per day) for 3 months;\n* severe or intolerable adverse effects from conventional immunosuppressive therapy.\n\nExclusion Criteria:\n\n* positive for muscle-specific kinase (MuSK) antibodies;\n* a tumor history except for thymoma;\n* a history of Type B and Type C hepatitis;\n* a history of tuberculosis or T-SPOT.TB tset positivity;\n* hepatic, renal and cardial insufficiency (baseline: ALT/AST\\>50U/L;BNP\\>200pg/ml）;\n* severe allergy or infection, or chronic or recurrent infection;\n* pregnancy;\n* hyperlipidemia;\n* participating other clinical trials'}, 'identificationModule': {'nctId': 'NCT04431895', 'briefTitle': 'Safety And Efficacy Of Tofacitinib In Patients With Refractory Myasthenia Gravis: A Pilot Study', 'organization': {'class': 'OTHER', 'fullName': 'Huashan Hospital'}, 'officialTitle': 'A Prospective,Single-center,Single-Arm,Single-Blind Pilot Study To Investigate The Efficacy Of Tofacitinib In Patients With Refractory Myasthenia Gravis', 'orgStudyIdInfo': {'id': 'KY2020-019'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'tofacitinib 5mg twice a day', 'interventionNames': ['Drug: Tofacitinib 5 MG']}], 'interventions': [{'name': 'Tofacitinib 5 MG', 'type': 'DRUG', 'description': 'tofacitinib 5 mg tablet administered twice a day for 6 months', 'armGroupLabels': ['tofacitinib 5mg twice a day']}]}, 'contactsLocationsModule': {'locations': [{'zip': '200040', 'city': 'Shanghai', 'country': 'China', 'facility': 'Huashan Hospital', 'geoPoint': {'lat': 31.22222, 'lon': 121.45806}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO', 'description': 'Individual participant data (IPD) are not available to other researchers.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Huashan Hospital', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Professor', 'investigatorFullName': 'Chongbo Zhao', 'investigatorAffiliation': 'Huashan Hospital'}}}}