Ignite Creation Date:
2025-12-24 @ 7:21 PM
Ignite Modification Date:
2026-01-07 @ 2:55 AM
Study NCT ID:
NCT00168103
Status:
COMPLETED
Last Update Posted:
2015-03-31
First Post:
2005-09-12
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Human C1 Esterase Inhibitor (C1-INH) in Subjects With Acute Abdominal or Facial Hereditary Angioedema (HAE) Attacks
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D054179', 'term': 'Angioedemas, Hereditary'}], 'ancestors': [{'id': 'D000799', 'term': 'Angioedema'}, {'id': 'D014652', 'term': 'Vascular Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D000081208', 'term': 'Hereditary Complement Deficiency Diseases'}, {'id': 'D000081207', 'term': 'Primary Immunodeficiency Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D014581', 'term': 'Urticaria'}, {'id': 'D017445', 'term': 'Skin Diseases, Vascular'}, {'id': 'D012871', 'term': 'Skin Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D006969', 'term': 'Hypersensitivity, Immediate'}, {'id': 'D006967', 'term': 'Hypersensitivity'}, {'id': 'D007154', 'term': 'Immune System Diseases'}, {'id': 'D007153', 'term': 'Immunologic Deficiency Syndromes'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D050718', 'term': 'Complement C1 Inhibitor Protein'}], 'ancestors': [{'id': 'D006023', 'term': 'Glycoproteins'}, {'id': 'D006001', 'term': 'Glycoconjugates'}, {'id': 'D002241', 'term': 'Carbohydrates'}, {'id': 'D003174', 'term': 'Complement C1 Inactivator Proteins'}, {'id': 'D015843', 'term': 'Serpins'}, {'id': 'D010455', 'term': 'Peptides'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D003169', 'term': 'Complement Inactivator Proteins'}, {'id': 'D003165', 'term': 'Complement System Proteins'}, {'id': 'D007162', 'term': 'Immunoproteins'}, {'id': 'D001798', 'term': 'Blood Proteins'}, {'id': 'D011506', 'term': 'Proteins'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'clinicaltrials@cslbehring.com', 'phone': 'Use email contact', 'title': 'Clinical Program Director', 'organization': 'CSL Behring'}, 'certainAgreement': {'otherDetails': 'The investigator must provide a copy of any results communication to the sponsor for review at least 30 days prior to public release. The sponsor may request any changes necessary to prevent forfeiture of patent rights to data not in the public domain. For a multi-center study, the investigator must wait (i) at least 1 year after the study is completed at all sites or (ii) until notified by the sponsor that no multi-center publication is planned before seeking publication review.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}, 'limitationsAndCaveats': {'description': 'A test for futility of the C1-INH 10 U/kg bw group conducted during a planned interim analysis led to ceasing recruitment for the C1-INH 10 U/kg bw group.'}}, 'adverseEventsModule': {'timeFrame': 'AEs for each study arm are reported for the first 4 hours after start of study treatment.', 'description': 'Safety data were analyzed according to the actual treatment received. The analysis of AEs within 4 hours of the start of initial treatment permits an unbiased comparison of treatment groups without the confounding effect of any rescue medication.', 'eventGroups': [{'id': 'EG000', 'title': 'C1-INH 10 U/kg bw', 'description': 'Includes subjects receiving 10 U/kg bw C1-INH and no rescue study medication within 4 hours after the start of the initial treatment.', 'otherNumAtRisk': 39, 'otherNumAffected': 10, 'seriousNumAtRisk': 39, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'C1-INH 20 U/kg bw', 'description': 'Includes subjects receiving 20 U/kg bw C1-INH and no rescue study medication within 4 hours after the start of the initial treatment (n=43). An additional 3 subjects not randomized to but treated with 20 U/kg bw C1-INH in this time period were also included in this group for the safety analysis.', 'otherNumAtRisk': 46, 'otherNumAffected': 10, 'seriousNumAtRisk': 46, 'seriousNumAffected': 0}, {'id': 'EG002', 'title': 'Placebo', 'description': 'Includes subjects receiving Placebo and no rescue study medication within 4 hours after the start of the initial treatment.', 'otherNumAtRisk': 41, 'otherNumAffected': 18, 'seriousNumAtRisk': 41, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 46, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 41, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 10.0'}, {'term': 'Diarrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 46, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 41, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 10.0'}, {'term': 'Dysgeusia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 46, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 41, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 10.0'}, {'term': 'Edema peripheral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 46, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 41, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 10.0'}, {'term': 'Face edema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 46, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 10.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 46, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 41, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 10.0'}, {'term': 'Lip swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 46, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 10.0'}, {'term': 'Muscle spasms', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 46, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 41, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 10.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 46, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 41, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 10.0'}, {'term': 'Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 46, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 10.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 46, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 41, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 10.0'}], 'frequencyThreshold': '1'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Time to Start of Relief of Symptoms From HAE Attack', 'denoms': [{'units': 'Participants', 'counts': [{'value': '39', 'groupId': 'OG000'}, {'value': '43', 'groupId': 'OG001'}, {'value': '42', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'C1-INH 10 U/kg bw', 'description': 'Includes all subjects enrolled and randomized to the C1-INH 10 U/kg bw arm.'}, {'id': 'OG001', 'title': 'C1-INH 20 U/kg bw', 'description': 'Includes all subjects enrolled and randomized to the C1-INH 20 U/kg bw arm.'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Includes all subjects enrolled and randomized to the Placebo arm.'}], 'classes': [{'categories': [{'measurements': [{'value': '1.17', 'spread': '10.513', 'groupId': 'OG000', 'lowerLimit': '0.17', 'upperLimit': '24.00'}, {'value': '0.5', 'spread': '8.202', 'groupId': 'OG001', 'lowerLimit': '0.17', 'upperLimit': '24.00'}, {'value': '1.5', 'spread': '11.481', 'groupId': 'OG002', 'lowerLimit': '0.20', 'upperLimit': '24.00'}]}]}], 'analyses': [{'pValue': '0.0025', 'groupIds': ['OG001', 'OG002'], 'paramType': 'Median Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.525', 'ciLowerLimit': '-2.217', 'ciUpperLimit': '-0.033', 'pValueComment': '1-sided P-value. The a priori threshold was 0.024 (overall Type 1 error 0.025 adjusted for alpha spending for an interim analysis).', 'estimateComment': 'The median difference was estimated by the Hodges-Lehmann estimate.', 'statisticalMethod': 'Wilcoxon (Mann-Whitney)', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'statisticalComment': '1-sided', 'testedNonInferiority': False}], 'paramType': 'MEDIAN', 'timeFrame': 'Up to 24 h after start of study treatment', 'description': 'The start of symptom relief was determined by subject self-assessment. Time to start of symptom relief was set to 24 hours if the subject received rescue medication (blinded study medication, narcotic analgesics, antiemetics, open-label C1-INH, or fresh frozen plasma) at any time point after the start of study treatment but before start of relief.', 'unitOfMeasure': 'Hours', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'Analysis was based on the ITT population which included all subjects receiving any portion of the randomized study medication.'}, {'type': 'SECONDARY', 'title': 'Number of Subjects With Worsened Intensity of Clinical HAE Symptoms', 'denoms': [{'units': 'Participants', 'counts': [{'value': '39', 'groupId': 'OG000'}, {'value': '43', 'groupId': 'OG001'}, {'value': '42', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'C1-INH 10 U/kg bw', 'description': 'Includes all subjects enrolled and randomized to the C1-INH 10 U/kg bw arm.'}, {'id': 'OG001', 'title': 'C1-INH 20 U/kg bw', 'description': 'Includes all subjects enrolled and randomized to the C1-INH 20 U/kg bw arm.'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Includes all subjects enrolled and randomized to the Placebo arm.'}], 'classes': [{'categories': [{'measurements': [{'value': '8', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '13', 'groupId': 'OG002'}]}]}], 'analyses': [{'pValue': '0.0014', 'groupIds': ['OG001', 'OG002'], 'paramType': 'Odds Ratio (OR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '80', 'paramValue': '0.1088', 'ciLowerLimit': '0.0392', 'ciUpperLimit': '0.3023', 'pValueComment': '1-sided P-value. The a priori threshold for significance was 0.1 (trend).', 'estimateComment': 'The Odds Ratio was calculated as C1-INH 20 U/kg bw (numerator) versus Placebo (denominator).', 'groupDescription': 'Worsened intensity was evaluated between 2 and 4 hours after start of study treatment relative to baseline for at least 1 of the HAE symptoms present at baseline.', 'statisticalMethod': 'Fisher Exact', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'statisticalComment': '1-sided', 'testedNonInferiority': False}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline and between 2 and 4 h after start of study treatment', 'description': 'Includes any worsening of intensity of at least 1 of the HAE symptoms present at baseline. Routinely checked symptoms included pain, nausea, vomiting, cramps, and diarrhea.', 'unitOfMeasure': 'Subjects', 'reportingStatus': 'POSTED', 'populationDescription': 'Analysis was based on the ITT population which included all subjects receiving any portion of the randomized study medication.'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Time to Complete Resolution of All HAE Symptoms, Including Pain', 'denoms': [{'units': 'Participants', 'counts': [{'value': '39', 'groupId': 'OG000'}, {'value': '43', 'groupId': 'OG001'}, {'value': '42', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'C1-INH 10 U/kg bw', 'description': 'Includes all subjects enrolled and randomized to the C1-INH 10 U/kg bw arm.'}, {'id': 'OG001', 'title': 'C1-INH 20 U/kg bw', 'description': 'Includes all subjects enrolled and randomized to the C1-INH 20 U/kg bw arm.'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Includes all subjects enrolled and randomized to the Placebo arm.'}], 'classes': [{'categories': [{'measurements': [{'value': '20.00', 'spread': '494.230', 'groupId': 'OG000', 'lowerLimit': '0.47', 'upperLimit': '1486.17'}, {'value': '4.92', 'spread': '314.347', 'groupId': 'OG001', 'lowerLimit': '0.47', 'upperLimit': '1486.17'}, {'value': '7.79', 'spread': '382.815', 'groupId': 'OG002', 'lowerLimit': '0.33', 'upperLimit': '1486.17'}]}]}], 'analyses': [{'pValue': '0.0237', 'groupIds': ['OG001', 'OG002'], 'paramType': 'Median Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '80', 'paramValue': '-3.292', 'ciLowerLimit': '-5.150', 'ciUpperLimit': '-1.050', 'pValueComment': '1-sided, exploratory test.', 'estimateComment': 'The median difference was estimated by the Hodge-Lehmann estimate.', 'groupDescription': 'This was an exploratory analysis.', 'statisticalMethod': 'Wilcoxon (Mann-Whitney)', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'statisticalComment': '1-sided', 'testedNonInferiority': False}], 'paramType': 'MEDIAN', 'timeFrame': 'Up to 24 h after start of study treatment', 'description': 'Complete resolution of symptoms was determined by subject self-assessment.', 'unitOfMeasure': 'Hours', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'Analysis was based on the ITT population which included all subjects receiving any portion of the randomized study medication.'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Number of Subjects Receiving Rescue Study Medication', 'denoms': [{'units': 'Participants', 'counts': [{'value': '39', 'groupId': 'OG000'}, {'value': '43', 'groupId': 'OG001'}, {'value': '42', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'C1-INH 10 U/kg bw', 'description': 'Includes all subjects enrolled and randomized to the C1-INH 10 U/kg bw arm.'}, {'id': 'OG001', 'title': 'C1-INH 20 U/kg bw', 'description': 'Includes all subjects enrolled and randomized to the C1-INH 20 U/kg bw arm.'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Includes all subjects enrolled and randomized to the Placebo arm.'}], 'classes': [{'categories': [{'measurements': [{'value': '13', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '24', 'groupId': 'OG002'}]}]}], 'analyses': [{'groupIds': ['OG001', 'OG002'], 'paramType': 'Odds Ratio (OR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.1714', 'ciLowerLimit': '0.0642', 'ciUpperLimit': '0.4575', 'estimateComment': 'The Odds Ratio was calculated as C1-INH 20 U/kg bw (numerator) versus Placebo (denominator).', 'groupDescription': 'This was an exploratory analysis.', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}], 'paramType': 'NUMBER', 'timeFrame': 'Within 4 h after start of study treatment', 'unitOfMeasure': 'Subjects', 'reportingStatus': 'POSTED', 'populationDescription': 'Analysis was based on the ITT population which included all subjects receiving any portion of the randomized study medication.'}, {'type': 'SECONDARY', 'title': 'Number of Vomiting Episodes', 'denoms': [{'units': 'Participants', 'counts': [{'value': '39', 'groupId': 'OG000'}, {'value': '43', 'groupId': 'OG001'}, {'value': '42', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'C1-INH 10 U/kg bw', 'description': 'Includes all subjects enrolled and randomized to the C1-INH 10 U/kg bw arm.'}, {'id': 'OG001', 'title': 'C1-INH 20 U/kg bw', 'description': 'Includes all subjects enrolled and randomized to the C1-INH 20 U/kg bw arm.'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Includes all subjects enrolled and randomized to the Placebo arm.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'spread': '0.77', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '4'}, {'value': '0', 'spread': '0.41', 'groupId': 'OG001', 'lowerLimit': '0', 'upperLimit': '2'}, {'value': '0', 'spread': '2.59', 'groupId': 'OG002', 'lowerLimit': '0', 'upperLimit': '16'}]}]}], 'analyses': [{'pValue': '0.0329', 'groupIds': ['OG001', 'OG002'], 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '80', 'pValueComment': '1-sided P-value. The a priori threshold for significance was 0.1 (trend).', 'statisticalMethod': 'Wilcoxon (Mann-Whitney)', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'statisticalComment': '1-sided', 'testedNonInferiority': False}], 'paramType': 'MEDIAN', 'timeFrame': 'Within 4 h after start of study treatment', 'unitOfMeasure': 'Episodes per subject', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'Analysis was based on the ITT population which included all subjects receiving any portion of the randomized study medication.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'C1-INH 10 U/kg bw', 'description': 'Includes all subjects enrolled and randomized to the C1 Esterase Inhibitor (C1-INH) 10 Units (U)/kg body weight (bw) arm.'}, {'id': 'FG001', 'title': 'C1-INH 20 U/kg bw', 'description': 'Includes all subjects enrolled and randomized to the C1-INH 20 U/kg bw arm.'}, {'id': 'FG002', 'title': 'Placebo', 'description': 'Includes all subjects enrolled and randomized to the Placebo arm.'}, {'id': 'FG003', 'title': 'Not Randomized', 'description': 'Includes one subject enrolled who was not randomized but received treatment with 20 U/kg bw C1-INH.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '40'}, {'groupId': 'FG001', 'numSubjects': '43'}, {'groupId': 'FG002', 'numSubjects': '42'}, {'groupId': 'FG003', 'numSubjects': '1'}]}, {'type': 'COMPLETED', 'achievements': [{'comment': '1 randomized subject received open-label rescue medication before receiving randomized treatment.', 'groupId': 'FG000', 'numSubjects': '38'}, {'groupId': 'FG001', 'numSubjects': '38'}, {'groupId': 'FG002', 'numSubjects': '41'}, {'groupId': 'FG003', 'numSubjects': '1'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '5'}, {'groupId': 'FG002', 'numSubjects': '1'}, {'groupId': 'FG003', 'numSubjects': '0'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '2'}, {'groupId': 'FG002', 'numSubjects': '1'}, {'groupId': 'FG003', 'numSubjects': '0'}]}, {'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '3'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'This was a multinational study enrolling subjects at 36 study centers in 15 countries.', 'preAssignmentDetails': 'A screening visit was performed before the subject presented with an hereditary angioedema (HAE) attack at the study center. Study entry was defined to occur with administration of study treatment.\n\nOne subject enrolled received study treatment without being randomized and is listed separately in the participant flow.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '39', 'groupId': 'BG000'}, {'value': '43', 'groupId': 'BG001'}, {'value': '42', 'groupId': 'BG002'}, {'value': '124', 'groupId': 'BG003'}]}], 'groups': [{'id': 'BG000', 'title': 'C1-INH 10 U/kg bw', 'description': 'Baseline characteristics were calculated only for the intention to treat (ITT) and per protocol (PP) analysis populations, not for all enrolled subjects. Baseline data presented here are for subjects included in the ITT population. One (1) subject enrolled and randomized to the C1-INH 10 U/kg bw group was excluded from the ITT analysis population.'}, {'id': 'BG001', 'title': 'C1-INH 20 U/kg bw', 'description': 'Baseline data presented here are for subjects included in the ITT population. All subjects enrolled and randomized to the C1-INH 20 U/kg bw arm were included in the ITT analysis population.'}, {'id': 'BG002', 'title': 'Placebo', 'description': 'Baseline data presented here are for subjects included in the ITT population. All subjects enrolled and randomized to the Placebo arm were included in the ITT analysis population.'}, {'id': 'BG003', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Customized', 'classes': [{'title': '3 to < 12 years', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '3', 'groupId': 'BG003'}]}]}, {'title': '12 to < 17 years', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '10', 'groupId': 'BG003'}]}]}, {'title': '17 to < 65 years', 'categories': [{'measurements': [{'value': '35', 'groupId': 'BG000'}, {'value': '35', 'groupId': 'BG001'}, {'value': '37', 'groupId': 'BG002'}, {'value': '107', 'groupId': 'BG003'}]}]}, {'title': '>= 65 years', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '4', 'groupId': 'BG003'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '33.1', 'spread': '12.77', 'groupId': 'BG000'}, {'value': '34.6', 'spread': '14.91', 'groupId': 'BG001'}, {'value': '31.5', 'spread': '13.57', 'groupId': 'BG002'}, {'value': '33.1', 'spread': '13.76', 'groupId': 'BG003'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '26', 'groupId': 'BG000'}, {'value': '30', 'groupId': 'BG001'}, {'value': '28', 'groupId': 'BG002'}, {'value': '84', 'groupId': 'BG003'}]}, {'title': 'Male', 'measurements': [{'value': '13', 'groupId': 'BG000'}, {'value': '13', 'groupId': 'BG001'}, {'value': '14', 'groupId': 'BG002'}, {'value': '40', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'White', 'categories': [{'measurements': [{'value': '36', 'groupId': 'BG000'}, {'value': '38', 'groupId': 'BG001'}, {'value': '37', 'groupId': 'BG002'}, {'value': '111', 'groupId': 'BG003'}]}]}, {'title': 'Black', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}, {'value': '4', 'groupId': 'BG003'}]}]}, {'title': 'Hispanic', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}, {'value': '5', 'groupId': 'BG003'}]}]}, {'title': 'Asian', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '3', 'groupId': 'BG003'}]}]}, {'title': 'American Indian or Alaskan Native', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'Participants'}, {'title': 'Intensity of Baseline HAE Attack', 'classes': [{'title': 'Moderate', 'categories': [{'measurements': [{'value': '32', 'groupId': 'BG000'}, {'value': '27', 'groupId': 'BG001'}, {'value': '26', 'groupId': 'BG002'}, {'value': '85', 'groupId': 'BG003'}]}]}, {'title': 'Severe', 'categories': [{'measurements': [{'value': '7', 'groupId': 'BG000'}, {'value': '16', 'groupId': 'BG001'}, {'value': '16', 'groupId': 'BG002'}, {'value': '39', 'groupId': 'BG003'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'Participants'}, {'title': 'Primary Disease Characteristic', 'classes': [{'title': 'Type I HAE', 'categories': [{'measurements': [{'value': '35', 'groupId': 'BG000'}, {'value': '35', 'groupId': 'BG001'}, {'value': '38', 'groupId': 'BG002'}, {'value': '108', 'groupId': 'BG003'}]}]}, {'title': 'Type II HAE', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}, {'value': '15', 'groupId': 'BG003'}]}]}, {'title': 'Missing', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}]}]}], 'paramType': 'NUMBER', 'description': 'Type I HAE (common form genotype): An impaired synthesis and an elevated turnover of a normal and functionally active C1-INH molecule occurs, causing a reduction in the availability of functionally active C1-INH to levels of 5% to 30% of normal.\n\nType II HAE (variant form genotype): Normal levels of a functionally impaired C1-INH molecule are synthesized while the normal form of C1-INH is considerably reduced in the circulation.', 'unitOfMeasure': 'Participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2', 'PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'DOUBLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 126}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2005-06'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2011-02', 'completionDateStruct': {'date': '2007-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2015-03-11', 'studyFirstSubmitDate': '2005-09-12', 'resultsFirstSubmitDate': '2010-04-21', 'studyFirstSubmitQcDate': '2005-09-12', 'lastUpdatePostDateStruct': {'date': '2015-03-31', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2010-07-26', 'studyFirstPostDateStruct': {'date': '2005-09-14', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2010-08-24', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2007-10', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Time to Complete Resolution of All HAE Symptoms, Including Pain', 'timeFrame': 'Up to 24 h after start of study treatment', 'description': 'Complete resolution of symptoms was determined by subject self-assessment.'}, {'measure': 'Number of Subjects Receiving Rescue Study Medication', 'timeFrame': 'Within 4 h after start of study treatment'}], 'primaryOutcomes': [{'measure': 'Time to Start of Relief of Symptoms From HAE Attack', 'timeFrame': 'Up to 24 h after start of study treatment', 'description': 'The start of symptom relief was determined by subject self-assessment. Time to start of symptom relief was set to 24 hours if the subject received rescue medication (blinded study medication, narcotic analgesics, antiemetics, open-label C1-INH, or fresh frozen plasma) at any time point after the start of study treatment but before start of relief.'}], 'secondaryOutcomes': [{'measure': 'Number of Subjects With Worsened Intensity of Clinical HAE Symptoms', 'timeFrame': 'Baseline and between 2 and 4 h after start of study treatment', 'description': 'Includes any worsening of intensity of at least 1 of the HAE symptoms present at baseline. Routinely checked symptoms included pain, nausea, vomiting, cramps, and diarrhea.'}, {'measure': 'Number of Vomiting Episodes', 'timeFrame': 'Within 4 h after start of study treatment'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['C1 Inhibitor', 'Hereditary angioedema', 'Acute HAE attack'], 'conditions': ['Hereditary Angioedema']}, 'referencesModule': {'references': [{'pmid': '21262096', 'type': 'BACKGROUND', 'citation': 'Bernstein JA, Ritchie B, Levy RJ, Wasserman RL, Bewtra AK, Hurewitz DS, Obtulowicz K, Reshef A, Moldovan D, Shirov T, Grivcheva-Panovska V, Kiessling PC, Keinecke HO, Craig TJ. Hereditary angioedema: Validation of the end point time to onset of relief by correlation with symptom intensity. Allergy Asthma Proc. 2011 Jan-Feb;32(1):36-42. doi: 10.2500/aap.2011.32.3404.'}, {'pmid': '19767078', 'type': 'RESULT', 'citation': 'Craig TJ, Levy RJ, Wasserman RL, Bewtra AK, Hurewitz D, Obtulowicz K, Reshef A, Ritchie B, Moldovan D, Shirov T, Grivcheva-Panovska V, Kiessling PC, Keinecke HO, Bernstein JA. Efficacy of human C1 esterase inhibitor concentrate compared with placebo in acute hereditary angioedema attacks. J Allergy Clin Immunol. 2009 Oct;124(4):801-8. doi: 10.1016/j.jaci.2009.07.017. Epub 2009 Sep 19.'}, {'pmid': '23987198', 'type': 'DERIVED', 'citation': 'Craig TJ, Rojavin MA, Machnig T, Keinecke HO, Bernstein JA. Effect of time to treatment on response to C1 esterase inhibitor concentrate for hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2013 Sep;111(3):211-5. doi: 10.1016/j.anai.2013.06.021. Epub 2013 Jul 16.'}, {'pmid': '20674826', 'type': 'DERIVED', 'citation': 'Bernstein JA, Ritchie B, Levy RJ, Wasserman RL, Bewtra AK, Hurewitz DS, Obtulowicz K, Reshef A, Moldovan D, Shirov T, Grivcheva-Panovska V, Kiessling PC, Schindel F, Craig TJ. Population pharmacokinetics of plasma-derived C1 esterase inhibitor concentrate used to treat acute hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2010 Aug;105(2):149-54. doi: 10.1016/j.anai.2010.06.005.'}]}, 'descriptionModule': {'briefSummary': 'HAE is a rare disorder characterized by functional C1 esterase inhibitor deficiency. If not treated adequately, the acute attacks of HAE can be life-threatening and may even result in fatalities, especially in case of swelling of the larynx. This clinical Phase 2/Phase 3 study was designed to provide clinically relevant data on dosing, efficacy and safety in subjects with HAE.', 'detailedDescription': 'For each subject, only a single abdominal or facial attack was treated and evaluated. After receiving treatment, subjects were observed for a minimum of 4 hours, after which they could be discharged from the study center if they reported onset of symptom relief. Starting from 4 hours after treatment, subjects who reported insufficient or no symptom relief could receive a second dose of double-blind treatment (called "rescue medication") as follows: C1-INH 20 U/kg bw for subjects initially receiving placebo, C1-INH 10 U/kg bw for subjects initially receiving C1-INH 10 U/kg bw, and placebo for subjects initially receiving C1-INH 20 U/kg bw.\n\nThe study was defined to be successful if the primary outcome measure and at least one of the secondary outcome measures were met in the comparison between the C1-INH 20 U/kg bw group and the Placebo group.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n* Documented congenital C1-INH deficiency\n* Acute facial or abdominal HAE attack\n\nKey Exclusion Criteria:\n\n* Acquired angioedema\n* Treatment with any other investigational drug within the last 30 days before study entry\n* Treatment with any C1-INH concentrate within the previous 7 days'}, 'identificationModule': {'nctId': 'NCT00168103', 'briefTitle': 'Human C1 Esterase Inhibitor (C1-INH) in Subjects With Acute Abdominal or Facial Hereditary Angioedema (HAE) Attacks', 'organization': {'class': 'INDUSTRY', 'fullName': 'CSL Behring'}, 'officialTitle': 'Human Pasteurized C1 Esterase Inhibitor Concentrate (CE1145) in Subjects With Congenital C1-INH Deficiency and Acute Abdominal or Facial HAE Attacks', 'orgStudyIdInfo': {'id': 'CE1145_3001'}, 'secondaryIdInfos': [{'id': '2004-001186-17', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'C1-INH 10 U/kg bw', 'description': '10 Units (U)/kg body weight (bw) dose', 'interventionNames': ['Biological: C1 Esterase Inhibitor']}, {'type': 'EXPERIMENTAL', 'label': 'C1-INH 20 U/kg bw', 'description': '20 U/kg bw dose', 'interventionNames': ['Biological: C1 Esterase Inhibitor']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'interventionNames': ['Biological: Placebo']}], 'interventions': [{'name': 'C1 Esterase Inhibitor', 'type': 'BIOLOGICAL', 'otherNames': ['Berinert', 'Berinert P', 'CE1145'], 'description': 'Single application of C1-INH administered intravenously by slow injection or infusion at a recommended rate of 4mL/min.', 'armGroupLabels': ['C1-INH 10 U/kg bw', 'C1-INH 20 U/kg bw']}, {'name': 'Placebo', 'type': 'BIOLOGICAL', 'otherNames': ['Physiological saline solution'], 'description': 'Single application of physiological saline solution equivalent to the volume calculated for subjects in the C1-INH 20 U/kg bw arm.', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '91344', 'city': 'Granada Hills', 'state': 'California', 'country': 'United States', 'facility': 'Study Site', 'geoPoint': {'lat': 34.26472, 'lon': -118.52314}}, {'zip': '33331', 'city': 'Weston', 'state': 'Florida', 'country': 'United States', 'facility': 'Study Site', 'geoPoint': {'lat': 26.10037, 'lon': -80.39977}}, {'zip': '30342', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': 'Study Site', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '83404', 'city': 'Idaho Falls', 'state': 'Idaho', 'country': 'United States', 'facility': 'Study Site', 'geoPoint': {'lat': 43.46658, 'lon': -112.03414}}, {'zip': '60612', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'Study Site', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '71130', 'city': 'Shreveport', 'state': 'Louisiana', 'country': 'United States', 'facility': 'Study Site', 'geoPoint': {'lat': 32.52515, 'lon': -93.75018}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Study Site', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '55411', 'city': 'Plymouth', 'state': 'Minnesota', 'country': 'United States', 'facility': 'Study Site', 'geoPoint': 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'responsibleParty': {'oldNameTitle': 'Global Head Clinical Research & Development', 'oldOrganization': 'CSL Behring'}}}}