Ignite Creation Date:
2025-12-25 @ 2:53 AM
Ignite Modification Date:
2026-03-01 @ 1:44 PM
Study NCT ID:
NCT03154333
Status:
TERMINATED
Last Update Posted:
2019-11-05
First Post:
2017-05-10
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Safety and Efficacy of Diacerein 1% Ointment for Subjects With Epidermolysis Bullosa Simplex (EBS)
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D016110', 'term': 'Epidermolysis Bullosa Simplex'}], 'ancestors': [{'id': 'D004820', 'term': 'Epidermolysis Bullosa'}, {'id': 'D012868', 'term': 'Skin Abnormalities'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D012873', 'term': 'Skin Diseases, Genetic'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D012871', 'term': 'Skin Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D012872', 'term': 'Skin Diseases, Vesiculobullous'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C025292', 'term': 'diacerein'}, {'id': 'D009824', 'term': 'Ointments'}], 'ancestors': [{'id': 'D004304', 'term': 'Dosage Forms'}, {'id': 'D004364', 'term': 'Pharmaceutical Preparations'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'mspellman@castlecreekpharma.com', 'phone': '862 286 0400', 'title': 'Dr. Mary Spellman', 'organization': 'Castle Creek Pharmaceuticals, LLC'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}}, 'adverseEventsModule': {'timeFrame': 'Baseline to Week 16', 'eventGroups': [{'id': 'EG000', 'title': 'Diacerein 1% Ointment', 'description': 'diacerein 1% ointment will be used for 8 weeks\n\ndiacerein 1% ointment: diacerein 1% ointment administered topically', 'otherNumAtRisk': 28, 'deathsNumAtRisk': 28, 'otherNumAffected': 16, 'seriousNumAtRisk': 28, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Vehicle Ointment', 'description': 'vehicle ointment will be used for 8 weeks\n\nvehicle ointment administered topically', 'otherNumAtRisk': 26, 'deathsNumAtRisk': 26, 'otherNumAffected': 15, 'seriousNumAtRisk': 26, 'deathsNumAffected': 0, 'seriousNumAffected': 2}], 'otherEvents': [{'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Skin Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 4, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 5, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 4, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Pruritus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Viral Upper Respiratory Tract Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Upper Respiratory Tract Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Blister', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Skin Abrasion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Nasal Congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Application Site Pruritus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Ear Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Dysmenorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'seriousEvents': [{'term': 'Humerus fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': "Meniere's Disease", 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Proportion of Subjects Who Achieved ≥ 60% Reduction in Body Surface Area (BSA) of EBS Lesions Within Assessment Area', 'denoms': [{'units': 'Participants', 'counts': [{'value': '28', 'groupId': 'OG000'}, {'value': '26', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Diacerein 1% Ointment', 'description': 'diacerein 1% ointment will be used for 8 weeks\n\ndiacerein 1% ointment: diacerein 1% ointment administered topically'}, {'id': 'OG001', 'title': 'Vehicle Ointment', 'description': 'vehicle ointment will be used for 8 weeks\n\nvehicle ointment administered topically'}], 'classes': [{'categories': [{'measurements': [{'value': '16', 'groupId': 'OG000'}, {'value': '14', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.9666', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Risk Ratio (RR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '1.01', 'ciLowerLimit': '0.63', 'ciUpperLimit': '1.62', 'statisticalMethod': 'Cochran-Mantel-Haenszel', 'nonInferiorityType': 'OTHER', 'statisticalComment': 'Log transformation normalized the Risk Ratio (RR) estimates; the Standard Error (SE) of the estimate was obtained from confidence limits for the RR.'}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline to Week 8', 'description': 'Analysis of the proportion of subjects who achieved a ≥60% reduction in Body Surface Area (BSA) of EBS lesions within Assessment Area from Baseline to Week 8', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All efficacy analyses were conducted using the Intent to Treat (ITT) Population that consisted of all randomized subjects who were dispensed study drug. Subjects were included in the treatment group to which they were assigned.'}, {'type': 'SECONDARY', 'title': "The Proportion of Subjects Who Achieved Success on the Investigator's Global Assessment (IGA)", 'denoms': [{'units': 'Participants', 'counts': [{'value': '28', 'groupId': 'OG000'}, {'value': '26', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Diacerein 1% Ointment', 'description': 'diacerein 1% ointment will be used for 8 weeks\n\ndiacerein 1% ointment: diacerein 1% ointment administered'}, {'id': 'OG001', 'title': 'Vehicle Ointment', 'description': 'vehicle ointment will be used for 8 weeks\n\nvehicle ointment administered topically'}], 'classes': [{'categories': [{'measurements': [{'value': '12', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.2861', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Risk Ratio (RR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '1.53', 'ciLowerLimit': '0.41', 'ciUpperLimit': '3.28', 'statisticalMethod': 'Cochran-Mantel-Haenszel', 'nonInferiorityType': 'OTHER', 'statisticalComment': 'Log transformation normalized the Risk Ratio (RR) estimates; the Standard Error (SE) of the estimate was obtained from confidence limits for the RR.'}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline to Week 8', 'description': "The investigator's global assessment (IGA) is a five-point scale that is used for overall clinical assessment of severity of disease and classifies EBS-involved skin with a score ranging from 0-4. Success on the IGA was defined as ≥2-point reduction from Baseline to Visit 6 (Week 8).\n\nIGA Scoring:\n\n0 = Clear; 1 = Near Clear; 2 = Mild; 3 = Moderate; 4 = Severe\n\nMinimum score = 0 Maximum score = 4; higher score = worse outcome", 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All efficacy analyses were conducted using the Intent to Treat (ITT) Population that consisted of all randomized subjects who were dispensed study drug. Subjects were included in the treatment group to which they were assigned.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Diacerein 1% Ointment', 'description': 'diacerein 1% ointment will be used for 8 weeks\n\ndiacerein 1% ointment: diacerein 1% ointment administered topically'}, {'id': 'FG001', 'title': 'Vehicle Ointment', 'description': 'vehicle ointment will be used for 8 weeks\n\nvehicle ointment administered topically'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '28'}, {'groupId': 'FG001', 'numSubjects': '26'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '21'}, {'groupId': 'FG001', 'numSubjects': '21'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '7'}, {'groupId': 'FG001', 'numSubjects': '5'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '28', 'groupId': 'BG000'}, {'value': '26', 'groupId': 'BG001'}, {'value': '54', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Diacerein 1% Ointment', 'description': 'diacerein 1% ointment will be used for 8 weeks\n\ndiacerein 1% ointment: diacerein 1% ointment administered topically'}, {'id': 'BG001', 'title': 'Vehicle Ointment', 'description': 'vehicle ointment will be used for 8 weeks\n\nvehicle ointment administered topically'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Customized', 'classes': [{'title': 'Age <8 years old', 'categories': [{'measurements': [{'value': '13', 'groupId': 'BG000'}, {'value': '12', 'groupId': 'BG001'}, {'value': '25', 'groupId': 'BG002'}]}]}, {'title': 'Age greater than or equal to 8 years old', 'categories': [{'measurements': [{'value': '15', 'groupId': 'BG000'}, {'value': '14', 'groupId': 'BG001'}, {'value': '29', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '14', 'groupId': 'BG000'}, {'value': '16', 'groupId': 'BG001'}, {'value': '30', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '14', 'groupId': 'BG000'}, {'value': '10', 'groupId': 'BG001'}, {'value': '24', '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': '2', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '2', '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': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'White', 'measurements': [{'value': '24', 'groupId': 'BG000'}, {'value': '23', 'groupId': 'BG001'}, {'value': '47', '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': '2', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Baseline BSA(Body Surface Area) of EBS(Epidermolysis Bullosa Simplex) Lesions within Assessment Area', 'classes': [{'categories': [{'measurements': [{'value': '5.76', 'spread': '4.674', 'groupId': 'BG000'}, {'value': '7.13', 'spread': '7.606', 'groupId': 'BG001'}, {'value': '6.42', 'spread': '6.237', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'Percent of BSA', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Baseline IGA Score of 3 or 4', 'classes': [{'title': 'IGA Score: 3', 'categories': [{'measurements': [{'value': '26', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '50', 'groupId': 'BG002'}]}]}, {'title': 'IGA Score: 4', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'description': "The investigator's global assessment (IGA) is a five-point scale that is used for overall clinical assessment of severity of disease and classifies EBS-involved skin with a score ranging from 0-4, with higher numbers indicating more severe disease.", 'unitOfMeasure': 'Participants'}], 'populationDescription': 'All efficacy endpoints were based on the Assessment Area, defined in the protocol, unless otherwise stated. The Intent to Treat (ITT) Population was used for these analyses.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2018-08-31', 'size': 399128, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_000.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2019-07-16T13:25', 'hasProtocol': False}, {'date': '2018-01-02', 'size': 795393, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_001.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2019-09-04T07:37', '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': 54}}, 'statusModule': {'whyStopped': 'Futility', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2017-06-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2019-10', 'completionDateStruct': {'date': '2018-10-31', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2019-10-16', 'studyFirstSubmitDate': '2017-05-10', 'resultsFirstSubmitDate': '2019-09-05', 'studyFirstSubmitQcDate': '2017-05-12', 'lastUpdatePostDateStruct': {'date': '2019-11-05', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2019-10-16', 'studyFirstPostDateStruct': {'date': '2017-05-16', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2019-11-05', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2018-10-15', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Proportion of Subjects Who Achieved ≥ 60% Reduction in Body Surface Area (BSA) of EBS Lesions Within Assessment Area', 'timeFrame': 'Baseline to Week 8', 'description': 'Analysis of the proportion of subjects who achieved a ≥60% reduction in Body Surface Area (BSA) of EBS lesions within Assessment Area from Baseline to Week 8'}], 'secondaryOutcomes': [{'measure': "The Proportion of Subjects Who Achieved Success on the Investigator's Global Assessment (IGA)", 'timeFrame': 'Baseline to Week 8', 'description': "The investigator's global assessment (IGA) is a five-point scale that is used for overall clinical assessment of severity of disease and classifies EBS-involved skin with a score ranging from 0-4. Success on the IGA was defined as ≥2-point reduction from Baseline to Visit 6 (Week 8).\n\nIGA Scoring:\n\n0 = Clear; 1 = Near Clear; 2 = Mild; 3 = Moderate; 4 = Severe\n\nMinimum score = 0 Maximum score = 4; higher score = worse outcome"}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Epidermolysis', 'bullosa', 'simplex', 'EBS'], 'conditions': ['Epidermolysis Bullosa Simplex']}, 'descriptionModule': {'briefSummary': 'Epidermolysis bullosa simplex (EBS) is a rare genetic skin disease characterized by fragility of the skin and mucous membranes resulting in painful blisters and erosions after minor trauma. The purpose of this study is to compare the efficacy of diacerein 1% ointment to vehicle ointment when applied once-daily for 8 weeks in subjects with EBS.', 'detailedDescription': 'The study is an international, multicenter, randomized, double-blind, vehicle-controlled, parallel group study to evaluate the safety and efficacy of diacerein 1% ointment for the treatment of subjects with EBS. Participants were assigned to study groups receiving either diacerein 1% ointment or vehicle ointment for 8 weeks, followed by an 8 week follow-up period. Approximately 80 subjects were to be randomized to one of the 2 treatment groups.\n\nThe primary objective of this study is to compare the efficacy of diacerein 1% ointment to vehicle ointment based on reduction in body surface area (BSA) of EBS lesions being treated when applied once-daily for 8 weeks in subjects with EBS. The secondary objectives are to compare the effects of diacerein 1% ointment to vehicle ointment in subjects with EBS in changes in Investigator Global Assessment (IGA) scores, pain, pruritus, mobility, and safety and tolerability.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '4 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Key Inclusion Criteria:\n\n* Subject is at least 4 years of age at Screening\n* Subject has a documented genetic mutation consistent with EBS. Gene mutations acceptable for inclusion are as follows: KRT5, KRT14, PLEC1, TGM5, PKP1, DSP, FERMT1, EXPH5, DST, KLHL24.\n* Subject has an Assessment Area of EBS lesions to be treated, that is ≥2% body surface area (BSA) and the EBS lesions are in one or both of the following body areas:\n\n * Localized: plantar and/or palmar areas\n * Generalized: arms, legs, torso, hands and feet\n* Subject's EBS lesions in the Assessment Area have an Investigator's Global Assessment (IGA) score of ≥3\n* Subject/caregiver agrees to not use any topical therapies other than the study medication that might influence the status of the EBS lesions during the duration of the study\n* Subject is non-pregnant as confirmed by a negative urine pregnancy screen, non-lactating and is not planning for pregnancy during the study period\n* If the subject is a woman of childbearing potential, agrees to use an approved effective method of birth control\n* Subject is in good general health and free of any known disease state or physical condition which might impair evaluation of the EBS lesions or which exposes the subject to an unacceptable risk by study participation\n\nKey Exclusion Criteria:\n\n* Subject has EBS lesions to be treated that are infected\n* Subject has used any diacerein containing product within 6 months prior to Screening\n* Subject has used systemic immunotherapy or cytotoxic chemotherapy within 60 days prior to Screening\n* Subject has used systemic steroidal therapy or has used topical steroidal therapy on the EBS lesions to be treated within 30 days prior to Baseline\n* Subject has evidence of a systemic infection or has used systemic antibiotics within 7 days prior to Screening\n* Subject is currently using systemic analgesics and/or anti-histamine therapy, for treatment of EBS lesions unless on a stable regimen (i.e., the same dosing regimen) for at least 4 weeks prior to Screening\n* Subject has used any systemic diuretics or cardiac glycosides or any systemic product that might put the subject at undue risk\n* Subject has used any topical product containing allantoin on the EBS lesions to be treated within 30 days prior to Screening\n* Subject has a current malignancy, or a history of treatment for a malignancy within 2 years prior to Screening\n* Subject currently has diabetes mellitus (HbA1c ≥6.5%) or controlled diabetes (HbA1c \\< 6.5%)\n* Subject has a history of cardiac, hepatic (ALT and or AST \\>2x ULN, Total bilirubin \\>1.5x ULN at Screening), or renal disease (eGFR\\<30 ml/min/1.73 m\\^2)\n* Subject has a non-EBS skin disease or condition (e.g., sunburn) that might put the subject at undue risk by study participation or interferes with the study medication application or the study assessments"}, 'identificationModule': {'nctId': 'NCT03154333', 'briefTitle': 'Safety and Efficacy of Diacerein 1% Ointment for Subjects With Epidermolysis Bullosa Simplex (EBS)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Castle Creek Pharmaceuticals, LLC'}, 'officialTitle': 'An International, Multicenter, Randomized, Double-Blind, Parallel-Group Phase 2 Study Evaluating the Safety and Efficacy of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex', 'orgStudyIdInfo': {'id': 'CCP-020-301'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'diacerein 1% ointment', 'description': 'diacerein 1% ointment will be used for 8 weeks', 'interventionNames': ['Drug: diacerein 1% ointment']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'vehicle ointment', 'description': 'vehicle ointment will be used for 8 weeks', 'interventionNames': ['Drug: A placebo ointment']}], 'interventions': [{'name': 'diacerein 1% ointment', 'type': 'DRUG', 'otherNames': ['CCP-020'], 'description': 'diacerein 1% ointment administered topically', 'armGroupLabels': ['diacerein 1% ointment']}, {'name': 'A placebo ointment', 'type': 'DRUG', 'description': 'vehicle ointment administered topically', 'armGroupLabels': ['vehicle ointment']}]}, 'contactsLocationsModule': {'locations': [{'zip': '85016', 'city': 'Phoenix', 'state': 'Arizona', 'country': 'United States', 'facility': 'Phoenix Childrens Hospital', 'geoPoint': {'lat': 33.44838, 'lon': -112.07404}}, {'zip': '94304', 'city': 'Palo Alto', 'state': 'California', 'country': 'United States', 'facility': 'Stanford University', 'geoPoint': {'lat': 37.44188, 'lon': -122.14302}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': "Children's Hospital Colorado", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': "Ann and Robert H. Lurie Children's Hospital of Chicago", 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'Northwestern University', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '55455', 'city': 'Minneapolis', 'state': 'Minnesota', 'country': 'United States', 'facility': 'University of Minnesota', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}, {'zip': '65212', 'city': 'Columbia', 'state': 'Missouri', 'country': 'United States', 'facility': 'University of Missouri Healthcare', 'geoPoint': {'lat': 38.95171, 'lon': -92.33407}}, {'zip': '11790', 'city': 'Stony Brook', 'state': 'New York', 'country': 'United States', 'facility': 'Stony Brook University Medical Center', 'geoPoint': {'lat': 40.92565, 'lon': -73.14094}}, {'zip': '27516', 'city': 'Chapel Hill', 'state': 'North Carolina', 'country': 'United States', 'facility': 'University of North Carolina - Chapel Hill', 'geoPoint': {'lat': 35.9132, 'lon': -79.05584}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': "Cincinnati Children's Hospital Medical Center", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '29425-5780', 'city': 'Charleston', 'state': 'South Carolina', 'country': 'United States', 'facility': 'Medical University of South Carolina (MUSC)', 'geoPoint': {'lat': 32.77632, 'lon': -79.93275}}, {'zip': '78218', 'city': 'San Antonio', 'state': 'Texas', 'country': 'United States', 'facility': "Children's Hospital of San Antonio ; Texas Dermatology and Laser Specialists", 'geoPoint': {'lat': 29.42412, 'lon': -98.49363}}, {'zip': '2217', 'city': 'Kogarah', 'state': 'New South Wales', 'country': 'Australia', 'facility': 'Premier Specialists Pty Ltd; The Church', 'geoPoint': {'lat': -33.9681, 'lon': 151.13564}}, {'zip': 'A-5020', 'city': 'Salzburg', 'country': 'Austria', 'facility': 'EB House Austria', 'geoPoint': {'lat': 47.79941, 'lon': 13.04399}}, {'zip': '75010', 'city': 'Paris', 'state': 'Cedex', 'country': 'France', 'facility': 'Hopital Saint Louis', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '75015', 'city': 'Paris', 'state': 'Cedex', 'country': 'France', 'facility': 'Hopital Necker-Enfants Malades', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '06202', 'city': 'Nice', 'country': 'France', 'facility': "CHU de NICE - Hopital de l'Archet II - Service de Dermatologie", 'geoPoint': {'lat': 43.70313, 'lon': 7.26608}}, {'zip': '79104', 'city': 'Freiburg im Breisgau', 'country': 'Germany', 'facility': 'University Medical Center Freiburg', 'geoPoint': {'lat': 47.9959, 'lon': 7.85222}}, {'zip': '6423906', 'city': 'Tel Aviv', 'country': 'Israel', 'facility': 'Tel Aviv Sourasky Medical Center', 'geoPoint': {'lat': 32.08088, 'lon': 34.78057}}, {'zip': 'AB20;30,001', 'city': 'Groningen', 'country': 'Netherlands', 'facility': 'University Medical Center Groningen', 'geoPoint': {'lat': 53.21917, 'lon': 6.56667}}, {'zip': 'WC1N 3JH', 'city': 'London', 'state': 'England', 'country': 'United Kingdom', 'facility': 'Great Ormond Street Hospital', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'SE1 7EH', 'city': 'London', 'country': 'United Kingdom', 'facility': "St. Thomas' Hospital - St Johns Institute of Dermatology", 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'Mary Spellman, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Castle Creek Pharmaceuticals'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Castle Creek Pharmaceuticals, LLC', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}