Ignite Creation Date:
2025-12-25 @ 4:56 AM
Ignite Modification Date:
2026-03-11 @ 3:41 PM
Study NCT ID:
NCT04139018
Status:
COMPLETED
Last Update Posted:
2021-08-24
First Post:
2019-10-23
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Timolol Gel for Epistaxis in Hereditary Hemorrhagic Telangiectasia
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2021-07-30', 'type': 'ACTUAL'}}}}, 'conditionBrowseModule': {'meshes': [{'id': 'D013683', 'term': 'Telangiectasia, Hereditary Hemorrhagic'}, {'id': 'D004844', 'term': 'Epistaxis'}], 'ancestors': [{'id': 'D020141', 'term': 'Hemostatic Disorders'}, {'id': 'D014652', 'term': 'Vascular Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D013684', 'term': 'Telangiectasis'}, {'id': 'D006474', 'term': 'Hemorrhagic Disorders'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D054079', 'term': 'Vascular Malformations'}, {'id': 'D018376', 'term': 'Cardiovascular Abnormalities'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D009668', 'term': 'Nose Diseases'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}, {'id': 'D010038', 'term': 'Otorhinolaryngologic Diseases'}, {'id': 'D006470', 'term': 'Hemorrhage'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D012818', 'term': 'Signs and Symptoms, Respiratory'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'piccirij@wustl.edu', 'phone': '314-362-8641', 'title': 'Jay Piccirillo, M.D., Vice Chair for Research', 'organization': 'Washington University School of Medicine Department of Otolaryngology - Head and Neck Surgery'}, 'certainAgreement': {'piSponsorEmployee': True}}, 'adverseEventsModule': {'timeFrame': "Adverse events collected throughout the whole duration of the study and assessed multiple times throughout a participant's 8 weeks of intervention.", 'eventGroups': [{'id': 'EG000', 'title': 'Timolol Gel Arm', 'description': 'Participants in the timolol gel arm (active medication arm) will receive timolol nasal gel 0.1% with 0.5 mL applied to each nostril twice daily via a syringe that will amount to a 2 mg total daily dose.\n\nTimolol Gel: Timolol nasal gel 0.1% will be prepared with a poloxamer gel (combination of poloxamer 188 and 407; pH adjusted to 4.5-6.5) and 0.5 ml applied to each nostril twice daily. The total daily dose would amount to 2 mg.', 'otherNumAtRisk': 14, 'deathsNumAtRisk': 14, 'otherNumAffected': 7, 'seriousNumAtRisk': 14, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Placebo Gel Arm', 'description': 'Participants in the placebo gel arm will receive the gel itself with no active medication.\n\nPlacebo Gel: Placebo gel is prepared with poloxamers and no active ingredients.', 'otherNumAtRisk': 13, 'deathsNumAtRisk': 13, 'otherNumAffected': 3, 'seriousNumAtRisk': 13, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Shortness of breath', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 13, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Rhinorrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Bad taste of medication', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Sore throat', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Irritation upon applying gel', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change in Assisted Epistaxis Severity Scale (aESS) Score From Baseline at 8 Week Follow-up', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}, {'value': '13', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Timolol Gel Arm', 'description': 'Participants in the timolol gel arm (active medication arm) will receive timolol nasal gel 0.1% with 0.5 mL applied to each nostril twice daily via a syringe that will amount to a 2 mg total daily dose.\n\nTimolol Gel: Timolol nasal gel 0.1% will be prepared with a poloxamer gel (combination of poloxamer 188 and 407; pH adjusted to 4.5-6.5) and 0.5 ml applied to each nostril twice daily. The total daily dose would amount to 2 mg.'}, {'id': 'OG001', 'title': 'Placebo Gel Arm', 'description': 'Participants in the placebo gel arm will receive the gel itself with no active medication.\n\nPlacebo Gel: Placebo gel is prepared with poloxamers and no active ingredients.'}], 'classes': [{'categories': [{'measurements': [{'value': '2.32', 'groupId': 'OG000', 'lowerLimit': '0.22', 'upperLimit': '5.97'}, {'value': '1.96', 'groupId': 'OG001', 'lowerLimit': '-0.91', 'upperLimit': '5.98'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Baseline to 8-week follow-up', 'description': "Assessment of epistaxis severity will be obtained by the validated instrument, the Epistaxis Severity Score (ESS). To complete the ESS, patients are asked to consider typical symptoms over the previous 3 months. The ESS contains 6 items - frequency, duration, and intensity of nosebleeds, whether patient has sought medication attention, whether patient is anemic, and whether patient has received a blood transfusion. The overall score ranges from 0 to 10, with severity of nosebleed based on score graded as None composite score of 0-1, Mild 1-4, Moderate 4-7, and Severe as 7-10.The minimal important difference noticeable by both patients and clinicians in the ESS scoring system is estimated as a change of 0.71. The scoring and MCID of the aESS is the same as the ESS.\n\nThe aESS references a participant's epistaxis over the past 1 month, and the change in aESS was calculated as the aESS score at 8 weeks minus the aESS score at baseline.", 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'Analysis was used with intention-to-treat principles, so all patients enrolled with baseline data were included. Note: the screen fail participant within the timolol gel arm had no baseline data say they were excluded from the analysis.'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Improved Response on Clinical Global Impression - Improvement (CGI-I) Scale', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Timolol Gel Arm', 'description': 'Participants in the timolol gel arm (active medication arm) will receive timolol nasal gel 0.1% with 0.5 mL applied to each nostril twice daily via a syringe that will amount to a 2 mg total daily dose.\n\nTimolol Gel: Timolol nasal gel 0.1% will be prepared with a poloxamer gel (combination of poloxamer 188 and 407; pH adjusted to 4.5-6.5) and 0.5 ml applied to each nostril twice daily. The total daily dose would amount to 2 mg.'}, {'id': 'OG001', 'title': 'Placebo Gel Arm', 'description': 'Participants in the placebo gel arm will receive the gel itself with no active medication.\n\nPlacebo Gel: Placebo gel is prepared with poloxamers and no active ingredients.'}], 'classes': [{'categories': [{'title': 'No Change', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Slightly Improved', 'measurements': [{'value': '4', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}, {'title': 'Much Improved', 'measurements': [{'value': '7', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Scores at 8-week follow-up only', 'description': 'CGI-I is a global rating of improvement scale, which requires subjects to rate their degree of improvement on a seven-point scale: "Compared to your condition at admission to the project \\[prior to medication initiation\\], how would you rate your overall response: 1=very much improved since the initiation of treatment; 2=much improved; 3=minimally improved; 4=no change from baseline (the initiation of treatment); 5=minimally worse; 6= much worse; 7=very much worse since the initiation of treatment."', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Timolol Gel Arm', 'description': 'Participants in the timolol gel arm (active medication arm) will receive timolol nasal gel 0.1% with 0.5 mL applied to each nostril twice daily via a syringe that will amount to a 2 mg total daily dose.\n\nTimolol Gel: Timolol nasal gel 0.1% will be prepared with a poloxamer gel (combination of poloxamer 188 and 407; pH adjusted to 4.5-6.5) and 0.5 ml applied to each nostril twice daily. The total daily dose would amount to 2 mg.'}, {'id': 'FG001', 'title': 'Placebo Gel Arm', 'description': 'Participants in the placebo gel arm will receive the gel itself with no active medication.\n\nPlacebo Gel: Placebo gel is prepared with poloxamers and no active ingredients.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '14'}, {'groupId': 'FG001', 'numSubjects': '13'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '11'}, {'groupId': 'FG001', 'numSubjects': '12'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Screen fail', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'Recruitment occurred from October 2019 through March 2020 at Washington University HHT Center of Excellence', 'preAssignmentDetails': '272 participants total were assessed for eligibility; 245 met exclusion criteria'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'BG000'}, {'value': '13', 'groupId': 'BG001'}, {'value': '27', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Timolol Gel Arm', 'description': 'Participants in the timolol gel arm (active medication arm) will receive timolol nasal gel 0.1% with 0.5 mL applied to each nostril twice daily via a syringe that will amount to a 2 mg total daily dose.\n\nTimolol Gel: Timolol nasal gel 0.1% will be prepared with a poloxamer gel (combination of poloxamer 188 and 407; pH adjusted to 4.5-6.5) and 0.5 ml applied to each nostril twice daily. The total daily dose would amount to 2 mg.'}, {'id': 'BG001', 'title': 'Placebo Gel Arm', 'description': 'Participants in the placebo gel arm will receive the gel itself with no active medication.\n\nPlacebo Gel: Placebo gel is prepared with poloxamers and no active ingredients.'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '58', 'groupId': 'BG000', 'lowerLimit': '20', 'upperLimit': '76'}, {'value': '52', 'groupId': 'BG001', 'lowerLimit': '30', 'upperLimit': '65'}, {'value': '55', 'groupId': 'BG002', 'lowerLimit': '20', 'upperLimit': '76'}]}]}], 'paramType': 'MEDIAN', 'unitOfMeasure': 'years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '10', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '14', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '13', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}, {'title': 'White', 'measurements': [{'value': '14', 'groupId': 'BG000'}, {'value': '11', 'groupId': 'BG001'}, {'value': '25', 'groupId': 'BG002'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '14', 'groupId': 'BG000'}, {'value': '13', 'groupId': 'BG001'}, {'value': '27', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Hypertension', 'classes': [{'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Seasonal Allergies', 'classes': [{'categories': [{'measurements': [{'value': '9', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '18', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Comorbidity Status', 'classes': [{'categories': [{'title': 'None', 'measurements': [{'value': '9', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '17', 'groupId': 'BG002'}]}, {'title': 'Mild', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '7', 'groupId': 'BG002'}]}, {'title': 'Moderate', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'Severe', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Baseline Clinical Global Impression - Severity', 'classes': [{'categories': [{'title': 'No Problem', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Mild Problem', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '7', 'groupId': 'BG002'}]}, {'title': 'Moderate/Severe Problem', 'measurements': [{'value': '10', 'groupId': 'BG000'}, {'value': '5', 'groupId': 'BG001'}, {'value': '15', 'groupId': 'BG002'}]}, {'title': 'Problem as bad as it could be', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Unknown', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'description': 'Measures severity of epistaxis on a 5-point Likert scale from "No problem" to "Problem as bad as it could be"', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2019-11-01', 'size': 955712, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_001.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2021-07-29T12:52', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 27}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2019-10-20', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-07', 'completionDateStruct': {'date': '2020-05-20', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-07-29', 'studyFirstSubmitDate': '2019-10-23', 'resultsFirstSubmitDate': '2021-07-08', 'studyFirstSubmitQcDate': '2019-10-23', 'lastUpdatePostDateStruct': {'date': '2021-08-24', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2021-07-29', 'studyFirstPostDateStruct': {'date': '2019-10-25', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2021-08-24', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2020-05-20', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change in Assisted Epistaxis Severity Scale (aESS) Score From Baseline at 8 Week Follow-up', 'timeFrame': 'Baseline to 8-week follow-up', 'description': "Assessment of epistaxis severity will be obtained by the validated instrument, the Epistaxis Severity Score (ESS). To complete the ESS, patients are asked to consider typical symptoms over the previous 3 months. The ESS contains 6 items - frequency, duration, and intensity of nosebleeds, whether patient has sought medication attention, whether patient is anemic, and whether patient has received a blood transfusion. The overall score ranges from 0 to 10, with severity of nosebleed based on score graded as None composite score of 0-1, Mild 1-4, Moderate 4-7, and Severe as 7-10.The minimal important difference noticeable by both patients and clinicians in the ESS scoring system is estimated as a change of 0.71. The scoring and MCID of the aESS is the same as the ESS.\n\nThe aESS references a participant's epistaxis over the past 1 month, and the change in aESS was calculated as the aESS score at 8 weeks minus the aESS score at baseline."}], 'secondaryOutcomes': [{'measure': 'Number of Participants With Improved Response on Clinical Global Impression - Improvement (CGI-I) Scale', 'timeFrame': 'Scores at 8-week follow-up only', 'description': 'CGI-I is a global rating of improvement scale, which requires subjects to rate their degree of improvement on a seven-point scale: "Compared to your condition at admission to the project \\[prior to medication initiation\\], how would you rate your overall response: 1=very much improved since the initiation of treatment; 2=much improved; 3=minimally improved; 4=no change from baseline (the initiation of treatment); 5=minimally worse; 6= much worse; 7=very much worse since the initiation of treatment."'}]}, 'oversightModule': {'isUsExport': True, 'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Epistaxis'], 'conditions': ['Hereditary Hemorrhagic Telangiectasia']}, 'referencesModule': {'references': [{'pmid': '32940653', 'type': 'DERIVED', 'citation': 'Peterson AM, Lee JJ, Kallogjeri D, Schneider JS, Chakinala MM, Piccirillo JF. Efficacy of Timolol in a Novel Intranasal Thermosensitive Gel for Hereditary Hemorrhagic Telangiectasia-Associated Epistaxis: A Randomized Clinical Trial. JAMA Otolaryngol Head Neck Surg. 2020 Nov 1;146(11):1006-1014. doi: 10.1001/jamaoto.2020.3025.'}]}, 'descriptionModule': {'briefSummary': 'This study is a double-blinded, randomized controlled trial to evaluate the efficacy of an intranasal topical timolol gel in the care for epistaxis in adults with hereditary hemorrhagic telangiectasia.', 'detailedDescription': 'This study is a double-blinded, placebo-controlled, 8-week randomized clinical trial investigating the efficacy of timolol gel in the management of epistaxis in adults with HHT.\n\nThe Specific Aims are to determine in adults with HHT-associated epistaxis:\n\n1. If topical timolol gel is more effective than placebo in reducing the frequency and severity of epistaxis.\n2. If topical timolol gel is more effective than placebo in improving hemoglobin levels.\n3. The frequency of adverse events, side effects, and safety profile of topical timolol gel delivered to the nasal mucosa.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '20 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Adults ages 20 and older\n2. Confirmed clinical (meeting at least 3 of the 4 Curaçao Criteria) or genetic diagnosis of HHT\n3. Epistaxis Severity Score (ESS) ≥ 4 and 2 or more nosebleeds per week with a cumulative nosebleed duration of at least 5 minutes per week\n4. Stable nasal hygiene and medical regimen for preceding 1 month\n5. Stable epistaxis pattern over the preceding 3 months\n\nExclusion Criteria:\n\n1. Contraindications for systemic β adrenergic blocker administration\n\n 1. Hypersensitivity to β adrenergic blockers\n 2. Asthma or bronchospasm\n 3. Congestive heart failure with LVEF \\<40%\n 4. Hereditary pulmonary arterial hypertension\n 5. Baseline bradycardia (HR \\<55 beats per minute)\n 6. Sick Sinus Syndrome\n 7. 2nd or 3rd degree heart block, left or right bundle branch block, or bifasicular block\n 8. Uncontrolled diabetes mellitus (most recent HbA1c \\>9%) or diabetic ketoacidosis within last 6 months\n 9. Hypotension (systolic blood pressure \\< 90)\n2. Known hypersensitivity to timolol\n3. Severe peripheral circulatory disturbances (Raynaud phenomenon)\n4. Known intermediate or poor metabolizer variant of the liver enzyme CYP2D6\n5. Current use of any of the following known strong CYP2D6 inhibitors: fluoxetine (Prozac), paroxetine (Paxil), bupropion (Welbutrin), quinidine, quinine, ritonavir (Norvir), and terbinafine (Lamisil)\n6. Current use of the following other drugs known to pharmacodynamically interact with timolol: diltiazem, verapamil, digoxin, digitalis, propafenone, disopyramide, clonidine, flecainide, or lidocaine\n7. Patients currently treated or who plan to initiate treatment with β-blockers\n8. Use of any anti-angiogenic medication in the last month prior to recruitment, including bevacizumab, pazopanib, thalidomide, or lenalidomide\n9. Illicit drug use, except marijuana\n10. Known pheochromocytoma\n11. Use of anticoagulants, antiplatelet, or fibrinolytic therapies within the last month prior to recruitment, except for low-dose (81 mg or less) of aspirin\n12. Pregnancy or planned pregnancy in the next 6 months or currently breastfeeding\n13. Inability to read or understand English\n14. Inability to complete 8 weeks of therapy for any reason'}, 'identificationModule': {'nctId': 'NCT04139018', 'acronym': 'ETIC-HHT', 'briefTitle': 'Timolol Gel for Epistaxis in Hereditary Hemorrhagic Telangiectasia', 'organization': {'class': 'OTHER', 'fullName': 'Washington University School of Medicine'}, 'officialTitle': 'Efficacy of a Timolol Gel in the Care for Epistaxis in Patients With Hereditary Hemorrhagic Telangiectasia: A Double-Blinded, Randomized Controlled Trial', 'orgStudyIdInfo': {'id': '201908160'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Timolol Gel Arm', 'description': 'Participants in the timolol gel arm (active medication arm) will receive timolol nasal gel 0.1% with 0.5 mL applied to each nostril twice daily via a syringe that will amount to a 2 mg total daily dose.', 'interventionNames': ['Drug: Timolol Gel']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo Gel Arm', 'description': 'Participants in the placebo gel arm will receive the gel itself with no active medication.', 'interventionNames': ['Drug: Placebo Gel']}], 'interventions': [{'name': 'Timolol Gel', 'type': 'DRUG', 'description': 'Timolol nasal gel 0.1% will be prepared with a poloxamer gel (combination of poloxamer 188 and 407; pH adjusted to 4.5-6.5) and 0.5 ml applied to each nostril twice daily. The total daily dose would amount to 2 mg.', 'armGroupLabels': ['Timolol Gel Arm']}, {'name': 'Placebo Gel', 'type': 'DRUG', 'description': 'Placebo gel is prepared with poloxamers and no active ingredients.', 'armGroupLabels': ['Placebo Gel Arm']}]}, 'contactsLocationsModule': {'locations': [{'zip': '63110', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Washington University', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}], 'overallOfficials': [{'name': 'Jay F Piccirillo, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Washington University School of Medicine'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Washington University School of Medicine', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Professor and Vice Chair for Research, Department of Otolaryngology - Head and Neck Surgery', 'investigatorFullName': 'Jay F. Piccirillo, MD', 'investigatorAffiliation': 'Washington University School of Medicine'}}}}