Ignite Creation Date:
2025-12-25 @ 1:28 AM
Ignite Modification Date:
2025-12-27 @ 11:28 PM
Study NCT ID:
NCT01267994
Status:
COMPLETED
Last Update Posted:
2018-01-10
First Post:
2010-12-28
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Clinical Trial of Anakinra for Steroid-Resistant Autoimmune Inner Ear Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006319', 'term': 'Hearing Loss, Sensorineural'}, {'id': 'D003639', 'term': 'Hearing Loss, Sudden'}, {'id': 'D008575', 'term': 'Meniere Disease'}], 'ancestors': [{'id': 'D034381', 'term': 'Hearing Loss'}, {'id': 'D006311', 'term': 'Hearing Disorders'}, {'id': 'D004427', 'term': 'Ear Diseases'}, {'id': 'D010038', 'term': 'Otorhinolaryngologic Diseases'}, {'id': 'D012678', 'term': 'Sensation Disorders'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D018159', 'term': 'Endolymphatic Hydrops'}, {'id': 'D007759', 'term': 'Labyrinth Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D053590', 'term': 'Interleukin 1 Receptor Antagonist Protein'}], 'ancestors': [{'id': 'D016207', 'term': 'Cytokines'}, {'id': 'D036341', 'term': 'Intercellular Signaling Peptides and Proteins'}, {'id': 'D010455', 'term': 'Peptides'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D001685', 'term': 'Biological Factors'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'vambutas@nshs.edu', 'phone': '718-470-7748', 'title': 'Andrea Vambutas, MD, FACS', 'organization': 'North Shore-LIJ Health System'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}}, 'adverseEventsModule': {'eventGroups': [{'id': 'EG000', 'title': 'Single Arm', 'description': 'Anakinra administered for 84 consecutive days', 'otherNumAtRisk': 12, 'otherNumAffected': 10, 'seriousNumAtRisk': 12, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Injection site reactions', 'notes': 'Injection site reactions', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numAffected': 10}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numAffected': 4}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Vertigo', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numAffected': 3}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Tinnitus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numAffected': 2}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Influenza like illness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Cellulitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Wheezing', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Psoriasis Flare', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Visual Impairment', 'notes': 'floaters', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'To Assess the Potential Efficacy of Anakinra in Improving Hearing Thresholds in Corticosteroid-resistant Patients With Autoimmune Inner Ear Disease', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Arm', 'description': 'Patients with Autoimmune Inner Ear Disease that had \\<5dB hearing improvement in response to corticosteorids following 28 days of treatment'}], 'classes': [{'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '180 days', 'description': 'The primary endpoint is to determine whether those treated with anakinra for 84 days demonstrate an improved hearing threshold compared with their pre-anakinra-treatment threshold. Audiometric thresholds will be compared to those treated with a prolonged corticosteroid taper and those that elect for no further treatment. The durability of the response will be measured over a total of 180 days.', 'unitOfMeasure': 'responders', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of Serious Adverse Events Reported', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Arm', 'description': 'Subject who received at least one injection of anakinra'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '84 days', 'description': 'To assess the number of Serious Adverse Events reported in any subject that received at least one injection dose of anakinra', 'unitOfMeasure': 'reported SAEs', 'reportingStatus': 'POSTED', 'populationDescription': 'Any subject enrolled in this study who received at least 1 injection dose of anakinra'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Open Label Trial', 'description': 'Open label, single arm trial of anakinra for corticosteroid-resistant Autoimmune Inner Ear Disease'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'comment': 'Declined to participate. n=1 Did not receive intervention due to infeciton, n=1', 'groupId': 'FG000', 'numSubjects': '12'}]}, {'type': 'COMPLETED', 'achievements': [{'comment': 'Excluded from analysis, could not reach first milestone (day 28) for efficacy assessment, n=2', 'groupId': 'FG000', 'numSubjects': '10'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Single Arm', 'description': 'Patients with Autoimmune Inner Ear Disease that had \\<5dB hearing improvement in response to corticosteorids following 28 days of treatment'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '12', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '7', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '13', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'description': '14 Non-responders, 1 elected not to participate', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '13', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '13', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}], 'populationDescription': 'Corticosteroid-resistant AIED patients.'}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 13}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2011-06'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-12', 'completionDateStruct': {'date': '2014-09', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2017-12-12', 'studyFirstSubmitDate': '2010-12-28', 'resultsFirstSubmitDate': '2014-09-16', 'studyFirstSubmitQcDate': '2010-12-28', 'lastUpdatePostDateStruct': {'date': '2018-01-10', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2015-03-23', 'studyFirstPostDateStruct': {'date': '2010-12-29', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2015-04-06', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2014-09', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'To Assess the Potential Efficacy of Anakinra in Improving Hearing Thresholds in Corticosteroid-resistant Patients With Autoimmune Inner Ear Disease', 'timeFrame': '180 days', 'description': 'The primary endpoint is to determine whether those treated with anakinra for 84 days demonstrate an improved hearing threshold compared with their pre-anakinra-treatment threshold. Audiometric thresholds will be compared to those treated with a prolonged corticosteroid taper and those that elect for no further treatment. The durability of the response will be measured over a total of 180 days.'}], 'secondaryOutcomes': [{'measure': 'Number of Serious Adverse Events Reported', 'timeFrame': '84 days', 'description': 'To assess the number of Serious Adverse Events reported in any subject that received at least one injection dose of anakinra'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Steroid', 'Steroid-Resistant', 'Autoimmune Inner Ear Disease', 'Immune Mediated Hearing Loss', 'Sudden Sensorineural Hearing Loss', "Meniere's Disease", 'Autoimmune', 'Hearing'], 'conditions': ['Sensorineural Hearing Loss', 'Autoimmune Inner Ear Disease']}, 'referencesModule': {'references': [{'pmid': '25133431', 'type': 'RESULT', 'citation': 'Vambutas A, Lesser M, Mullooly V, Pathak S, Zahtz G, Rosen L, Goldofsky E. Early efficacy trial of anakinra in corticosteroid-resistant autoimmune inner ear disease. J Clin Invest. 2014 Sep;124(9):4115-22. doi: 10.1172/JCI76503. Epub 2014 Aug 18.'}], 'seeAlsoLinks': [{'url': 'http://www.feinsteininstitute.org/Feinstein/Clinical+Trials', 'label': 'Clinical trials at the Feinstein Institute for Medical Research'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to determine if Anakinra (an interleukin-1 receptor antagonist) can improve hearing thresholds in those patients with Autoimmune Inner Ear Disease (AIED) that did not respond to oral steroid therapy for a sudden decline in hearing. The patients to be enrolled will have recently completed a course of oral steroids and demonstrated no change in their audiometric thresholds following corticosteroid therapy.', 'detailedDescription': 'Patients with immune mediated hearing loss (also known as autoimmune hearing loss) are typically treated with corticosteroids. Of those treated, approximately 60% respond, however, that response may be lost over time. Other therapies use to date have proven largely ineffectual in improving hearing. This study proposes a phase I open label clinical trial of Anakinra for corticosteroid-resistant patients to determine if this therapy is efficacious in hearing restoration.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '75 Years', 'minimumAge': '13 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Bilateral sensorineural hearing loss with an active decline in hearing in one ear\n* No audiometric improvement with 28-30 days of oral prednisone or other corticosteroid, including an initial dose of 60mg per day for 14 days\n* Enrollment within 14 days of completion of corticosteroid therapy\n* Age 13 years and older\n* No evidence of neutropenia (low white blood cell count)\n* No evidence of retrocochlear pathology (ie. acoustic neuroma/vestibular schwannoma)\n* May have concurrent, systemic autoimmune disease\n\nExclusion Criteria:\n\n* Age over 75, or less than 13\n* Neutropenia\n* Renal insufficiency\n* Pregnant females\n* Unilateral hearing loss\n* Patients with any immunodeficiency syndrome\n* Patients receiving methotrexate or any TNF (tumor necrosis factor) antagonist therapy\n* Patients with chronic infections\n* Patients treated for a malignancy within the past 3 years\n* Patients with a latex allergy\n* Patients with an inner ear anomaly\n* Patients with retrocochlear pathology'}, 'identificationModule': {'nctId': 'NCT01267994', 'briefTitle': 'A Clinical Trial of Anakinra for Steroid-Resistant Autoimmune Inner Ear Disease', 'organization': {'class': 'OTHER', 'fullName': 'Northwell Health'}, 'officialTitle': 'A Phase I/II Open-label Study of the Effects of Anakinra in Corticosteroid-resistant Subjects With Autoimmune Inner Ear Disease', 'orgStudyIdInfo': {'id': '1R21DC011827-01', 'link': 'https://reporter.nih.gov/quickSearch/1R21DC011827-01', 'type': 'NIH'}, 'secondaryIdInfos': [{'id': 'R33DC011827', 'link': 'https://reporter.nih.gov/quickSearch/R33DC011827', 'type': 'NIH'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Single Arm-Open Label', 'description': 'Single Arm-Open Label use of Anakinra', 'interventionNames': ['Drug: Anakinra']}], 'interventions': [{'name': 'Anakinra', 'type': 'DRUG', 'otherNames': ['Kineret'], 'description': '100mg of anakinra administered by a subcutaneous injection for 84 consecutive days.', 'armGroupLabels': ['Single Arm-Open Label']}]}, 'contactsLocationsModule': {'locations': [{'zip': '11040', 'city': 'New Hyde Park', 'state': 'New York', 'country': 'United States', 'facility': 'North Shore-LIJ Hearing and Speech Center', 'geoPoint': {'lat': 40.7351, 'lon': -73.68791}}], 'overallOfficials': [{'name': 'Andrea Vambutas, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Northwell Health'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Andrea Vambutas', 'class': 'OTHER'}, 'collaborators': [{'name': 'National Institute on Deafness and Other Communication Disorders (NIDCD)', 'class': 'NIH'}], 'responsibleParty': {'type': 'SPONSOR_INVESTIGATOR', 'investigatorTitle': 'Professor of Otolaryngology & Molecular Medicine', 'investigatorFullName': 'Andrea Vambutas', 'investigatorAffiliation': 'Northwell Health'}}}}