Ignite Creation Date:
2025-12-24 @ 3:02 PM
Ignite Modification Date:
2026-02-11 @ 12:46 PM
Study NCT ID:
NCT02584959
Status:
COMPLETED
Last Update Posted:
2021-06-08
First Post:
2015-10-20
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Study to Evaluate the Clinical Efficacy and Safety of Subcutaneously Administered C1 Esterase Inhibitor for the Prevention of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema
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'}, {'id': 'D005440', 'term': 'Fluid Therapy'}], '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'}, {'id': 'D004358', 'term': 'Drug Therapy'}, {'id': 'D013812', 'term': 'Therapeutics'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'ClinicalTransparency@shire.com', 'phone': '+1 866 842 5335', 'title': 'Study Director', 'organization': 'Shire'}, 'certainAgreement': {'otherDetails': 'If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Adverse events were reported from the time the informed consent was signed through 7 days afer the last dose of investigational product was received (week 1 to 15 for treatment period 1 and 2).', 'eventGroups': [{'id': 'EG000', 'title': 'Treatment C1 INH', 'description': 'Participants who received C1 Esterase Inhibitor (C1 INH) treatment (in period 1 of the Experimental/Placebo arm, in period 2 of the Placebo/Experimental arm and in period 1 and 2 of the Experimental/Experimental arm).', 'otherNumAtRisk': 71, 'deathsNumAtRisk': 71, 'otherNumAffected': 23, 'seriousNumAtRisk': 71, 'deathsNumAffected': 0, 'seriousNumAffected': 2}, {'id': 'EG001', 'title': 'Treatment Placebo', 'description': 'Participants who received Placebo treatment (in period 2 of the Experimental/Placebo arm and in period 1 of the Placebo/Experimental arm).', 'otherNumAtRisk': 57, 'deathsNumAtRisk': 57, 'otherNumAffected': 14, 'seriousNumAtRisk': 57, 'deathsNumAffected': 0, 'seriousNumAffected': 3}], 'otherEvents': [{'term': 'Viral upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numEvents': 11, 'numAffected': 11}, {'groupId': 'EG001', 'numAtRisk': 57, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numEvents': 7, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 57, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 57, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numEvents': 10, 'numAffected': 6}, {'groupId': 'EG001', 'numAtRisk': 57, 'numEvents': 7, 'numAffected': 6}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}], 'seriousEvents': [{'term': 'Cardiac arrest', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 57, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Hereditary angioedema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 57, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Congenital, familial and genetic disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Cholelithiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 57, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Appendicitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 57, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Staphylococcal infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 57, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Time-Normalized Number of Attacks (NNA) for Participants During a Treatment Period', 'denoms': [{'units': 'Participants', 'counts': [{'value': '56', 'groupId': 'OG000'}, {'value': '57', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment C1 INH', 'description': 'Participants who received C1 Esterase Inhibitor (C1 INH) treatment (in period 1 of the Experimental/Placebo arm and in period 2 of the Placebo/Experimental arm).'}, {'id': 'OG001', 'title': 'Treatment Placebo', 'description': 'Participants who received Placebo treatment (in period 2 of the Experimental/Placebo arm and in period 1 of the Placebo/Experimental arm).'}], 'classes': [{'categories': [{'measurements': [{'value': '1.611', 'groupId': 'OG000', 'lowerLimit': '1.067', 'upperLimit': '2.156'}, {'value': '3.931', 'groupId': 'OG001', 'lowerLimit': '3.391', 'upperLimit': '4.471'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference in LS means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-2.320', 'ciLowerLimit': '-2.895', 'ciUpperLimit': '-1.744', 'groupDescription': 'The LS means, 95% CIs, and p-values were based on a mixed effect linear model with period, sequence ,use of prophylactic therapy with C1 INH at randomization, and treatment as fixed effects and subject nested within sequence as a random effect.', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Weeks 1 to 14 for treatment period 1 and 2', 'description': 'The angioedema attacks were recorded in the electronic patient diary. The investigator completed a separate angioedema eCRF for each attack based on the review of the patients diary. Time-normalized number of angioedema attacks was expressed as the number of attacks per month (ie, 30.4 days) of exposure. NNA=30.4 \\* (number of attacks during treatment period) / (days of treatment period).', 'unitOfMeasure': 'Number of attacks', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Data were not collected for the Experimental/Experimental Arm for this outcome measure and this represents the full analysis set.'}, {'type': 'SECONDARY', 'title': 'Proportion of Participants Meeting at Least a 50% Reduction in NNA (Normalized Number of Angioedema Attacks) During the Experimental Injection Treatment Period Relative to the Placebo Period.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '49', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Full Analysis Set', 'description': 'Including all participants in the cross-over sequences who had data in both treatment periods and who received at least one dose of C1 Esterase Inhibitor and had at least 1 post-baseline primary efficacy assessment.'}], 'classes': [{'categories': [{'measurements': [{'value': '38', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Weeks 1 to 14 for treatment period 1 and 2', 'description': 'The angioedema attacks were recorded in the electronic patient diary. The investigator completed a separate angioedema eCRF for each attack based on the review of the patients diary. Time-Normalized Number of Attacks was expressed as the number of attacks per month (ie, 30.4 days) of exposure. NNA = 30.4 x (number of attacks during treatment period) / (days of treatment period).', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Analysis done on participants from the crossover sequences (Experimental/Placebo, Placebo/Experimental) who were dosed in both treatment periods. As the measure is implicitly a within subject comparison of the two arms the full analysis set is reported here. Participants with 0 attacks in the placebo period were excluded as %-reduct. not calculated'}, {'type': 'SECONDARY', 'title': 'Time-Normalized Number of Attacks (NNA) for Participants During Each Treatment Period Excluding the First 2 Weeks.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '55', 'groupId': 'OG000'}, {'value': '55', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment C1 INH', 'description': 'Participants who received C1 Esterase Inhibitor (C1 INH) treatment (in period 1 of the Experimental/Placebo arm and in period 2 of the Placebo/Experimental arm).'}, {'id': 'OG001', 'title': 'Treatment Placebo', 'description': 'Participants who received Placebo treatment (in period 2 of the Experimental/Placebo arm and in period 1 of the Placebo/Experimental arm).'}], 'classes': [{'categories': [{'measurements': [{'value': '1.524', 'groupId': 'OG000', 'lowerLimit': '0.912', 'upperLimit': '2.136'}, {'value': '3.847', 'groupId': 'OG001', 'lowerLimit': '3.237', 'upperLimit': '4.457'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference in LS means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-2.323', 'ciLowerLimit': '-2.969', 'ciUpperLimit': '-1.677', 'groupDescription': 'The LS means, 95% CIs, and p-values were based on a mixed effect linear model with period, sequence ,use of prophylactic therapy with C1 INH at randomization, and treatment as fixed effects and subject nested within sequence as a random effect.', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Weeks 3 to 14 for treatment period 1 and 2', 'description': 'The angioedema attacks were recorded in the electronic patient diary. The investigator completed a separate angioedema eCRF for each attack based on the review of the patients diary. Time-normalized number of angioedema attacks was expressed as the number of attacks per month (ie, 30.4 days) of exposure. NNA=30.4 \\* (number of attacks during treatment period) / (days of treatment period).', 'unitOfMeasure': 'Number of Attacks', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Data were not collected for the Experimental/Experimental Arm for this outcome measure and this represents the full analysis set.'}, {'type': 'SECONDARY', 'title': 'Proportion of Participants Meeting at Least a 50% Reduction in NNA (Normalized Number of Angioedema Attacks) During the Experimental Injection Treatment Period Relative to the Placebo Period Excluding the First 2 Weeks of Each Treatment Period.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '47', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Full Analysis Data Set', 'description': 'Including all participants in the cross-over sequences who had data in both treatment periods and who received at least one dose of C1 Esterase Inhibitor and had at least 1 post-baseline primary efficacy assessment.'}], 'classes': [{'categories': [{'measurements': [{'value': '36', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Weeks 3 to 14 for treatment period 1 and 2', 'description': 'The angioedema attacks were recorded in the electronic patient diary. The investigator completed a separate angioedema eCRF for each attack based on the review of the patients diary. Time-Normalized Number of Attacks was expressed as the number of attacks per month (ie, 30.4 days) of exposure. NNA = 30.4 x (number of attacks during treatment period) / (days of treatment period).', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Analysis done on participants from the crossover sequences (Experimental/Placebo, Placebo/Experimental) who were dosed in both treatment periods. As the measure is implicitly a within subject comparison of the two arms the full analysis set is reported here. Participants with 0 attacks in the placebo period were excluded as %-reduct. not calculated'}, {'type': 'SECONDARY', 'title': 'Proportion of Participants Meeting at Least a 50% Reduction in NNA (Normalized Number of Angioedema Attacks) During the Experimental Injection Treatment Period Relative to the Pre-treatment Assessment.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '53', 'groupId': 'OG000'}, {'value': '55', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment C1 INH', 'description': 'Participants who received C1 Esterase Inhibitor (C1 INH) treatment (in period 1 of the Experimental/Placebo arm and in period 2 of the Placebo/Experimental arm).'}, {'id': 'OG001', 'title': 'Treatment Placebo', 'description': 'Participants who received Placebo treatment (in period 2 of the Experimental/Placebo arm and in period 1 of the Placebo/Experimental arm).'}], 'classes': [{'categories': [{'measurements': [{'value': '41', 'groupId': 'OG000'}, {'value': '13', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Weeks 1 to 14 for treatment period 1 and 2', 'description': 'The angioedema attacks were recorded in the electronic patient diary. The investigator completed a separate angioedema eCRF for each attack based on the review of the patients diary. Time-Normalized Number of Attacks was expressed as the number of attacks per month (ie, 30.4 days) of exposure. NNA = 30.4 x (number of attacks during treatment period) / (days of treatment period).', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Full analysis set from the crossover sequences (Experimental/Placebo arm and Placebo/Experimental arm) was used for analysis of this outcome measure.Participants with zero attacks in the placebo period were excluded because a percent reduction could not be calculated. Analysis was done on participants with pretreatment and post-treatment NNA values'}, {'type': 'SECONDARY', 'title': 'Cumulative Attack Severity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '56', 'groupId': 'OG000'}, {'value': '57', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment C1 INH', 'description': 'Participants who received C1 Esterase Inhibitor (C1 INH) treatment (in period 1 of the Experimental/Placebo arm and in period 2 of the Placebo/Experimental arm).'}, {'id': 'OG001', 'title': 'Treatment Placebo', 'description': 'Participants who received Placebo treatment (in period 2 of the Experimental/Placebo arm and in period 1 of the Placebo/Experimental arm).'}], 'classes': [{'categories': [{'measurements': [{'value': '3.159', 'groupId': 'OG000', 'lowerLimit': '1.856', 'upperLimit': '4.463'}, {'value': '8.041', 'groupId': 'OG001', 'lowerLimit': '6.746', 'upperLimit': '9.336'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference in LS means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-4.881', 'ciLowerLimit': '-6.113', 'ciUpperLimit': '-3.649', 'groupDescription': 'The LS means, 95% CIs, and p-values were based on a mixed effect linear model with period, sequence ,use of prophylactic therapy with C1 INH at randomization, and treatment as fixed effects and subject nested within sequence as a random effect.', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Weeks 1 to 14 for treatment period 1 and 2', 'description': "Severity of the angioedema attack sign/symptom was characterized as None: no symptom; Mild: noticeable symptom but easily tolerated by the participant and did not interfere with routine activities; Moderate: symptom interfered with the participant's ability to attend school or participate in family life and social/recreational activities; Severe: symptom significantly limited the participant's ability to attend school or participate in family life and social/recreational activities. Symptom severity score was assigned as Mild = 1, Moderate = 2 and Severe = 3. Cumulative attack severity score was the sum of the maximum symptom severity scores recorded for each angioedema attack in a treatment period. Cumulative attack-severity score normalized per month \\[(raw score/number of days of participation in that treatment period)\\*30.4\\] was reported here. Cumulative attack-severity score normalized per month ranged from 0 to 19.83 and higher scores represent worse symptoms.", 'unitOfMeasure': 'Score on a scale', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Data were not collected for the Experimental/Experimental Arm for this outcome measure and this represents the full analysis set.'}, {'type': 'SECONDARY', 'title': 'Number of Attack-free Days', 'denoms': [{'units': 'Participants', 'counts': [{'value': '56', 'groupId': 'OG000'}, {'value': '57', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment C1 INH', 'description': 'Participants who received C1 Esterase Inhibitor (C1 INH) treatment (in period 1 of the Experimental/Placebo arm and in period 2 of the Placebo/Experimental arm).'}, {'id': 'OG001', 'title': 'Treatment Placebo', 'description': 'Participants who received Placebo treatment (in period 2 of the Experimental/Placebo arm and in period 1 of the Placebo/Experimental arm).'}], 'classes': [{'categories': [{'measurements': [{'value': '26.788', 'groupId': 'OG000', 'lowerLimit': '25.106', 'upperLimit': '28.470'}, {'value': '21.353', 'groupId': 'OG001', 'lowerLimit': '19.681', 'upperLimit': '23.025'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference in LS means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '5.435', 'ciLowerLimit': '3.981', 'ciUpperLimit': '6.889', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Weeks 1 to 14 for treatment period 1 and 2', 'description': 'Attack free days were normalized per month.', 'unitOfMeasure': 'Days', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Data were not collected for the Experimental/Experimental Arm for this outcome measure and this represents the full analysis set.'}, {'type': 'SECONDARY', 'title': 'Number of Angioedema Attacks Requiring Acute Treatment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '56', 'groupId': 'OG000'}, {'value': '57', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment C1 INH', 'description': 'Participants who received C1 Esterase Inhibitor (C1 INH) treatment (in period 1 of the Experimental/Placebo arm and in period 2 of the Placebo/Experimental arm).'}, {'id': 'OG001', 'title': 'Treatment Placebo', 'description': 'Participants who received Placebo treatment (in period 2 of the Experimental/Placebo arm and in period 1 of the Placebo/Experimental arm).'}], 'classes': [{'categories': [{'measurements': [{'value': '1.454', 'groupId': 'OG000', 'lowerLimit': '0.906', 'upperLimit': '2.002'}, {'value': '3.628', 'groupId': 'OG001', 'lowerLimit': '3.085', 'upperLimit': '4.172'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference in LS means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-2.175', 'ciLowerLimit': '-2.750', 'ciUpperLimit': '-1.599', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Weeks 1 to 14 for treatment period 1 and 2', 'description': 'Angioedema attacks were normalized per month.', 'unitOfMeasure': 'Number of attacks', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Data were not collected for the Experimental/Experimental Arm for this outcome measure and this represents the full analysis set.'}, {'type': 'SECONDARY', 'title': 'Response to Icatibant When Administered for an Acute Attack', 'denoms': [{'units': 'Participants', 'counts': [{'value': '71', 'groupId': 'OG000'}, {'value': '57', 'groupId': 'OG001'}]}, {'units': 'Attacks requiring Icatibant', 'counts': [{'value': '181', 'groupId': 'OG000'}, {'value': '453', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment C1 INH', 'description': 'Participants who received C1 Esterase Inhibitor (C1 INH) treatment (in period 1 of the Experimental/Placebo arm, in period 2 of the Placebo/Experimental arm and in period 1 and 2 of the Experimental/Experimental arm).'}, {'id': 'OG001', 'title': 'Treatment Placebo', 'description': 'Participants who received Placebo treatment (in period 2 of the Experimental/Placebo arm and in period 1 of the Placebo/Experimental arm).'}], 'classes': [{'categories': [{'title': 'requiring 1 injection', 'measurements': [{'value': '129', 'groupId': 'OG000'}, {'value': '306', 'groupId': 'OG001'}]}, {'title': 'requiring 2 injections', 'measurements': [{'value': '38', 'groupId': 'OG000'}, {'value': '89', 'groupId': 'OG001'}]}, {'title': 'requiring 3 injections', 'measurements': [{'value': '13', 'groupId': 'OG000'}, {'value': '30', 'groupId': 'OG001'}]}, {'title': 'requiring >= 4 injections', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '28', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_UNITS', 'timeFrame': 'Weeks 1 to 14 for treatment period 1 and 2', 'description': 'The number of Acute Hereditary Angioedema Attacks that required Icatibant as acute therapy is presented by the number of Icatibant injections.', 'unitOfMeasure': 'Attacks requiring Icatibant', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Attacks requiring Icatibant', 'denomUnitsSelected': 'Attacks requiring Icatibant', 'populationDescription': 'For treatment with C1 INH (Overall treatment with C1 INH group) all participants who received C1 INH in each of the randomized arms (experimental/placebo, placebo/experimental and experimental/experimental) are included.'}, {'type': 'SECONDARY', 'title': 'Number of Patients With Adverse Events (AEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '71', 'groupId': 'OG000'}, {'value': '57', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment C1 INH', 'description': 'Participants who received C1 Esterase Inhibitor (C1 INH) treatment (in period 1 of the Experimental/Placebo arm, in period 2 of the Placebo/Experimental arm and in period 1 and 2 of the Experimental/Experimental arm).'}, {'id': 'OG001', 'title': 'Treatment Placebo', 'description': 'Participants who received Placebo treatment (in period 2 of the Experimental/Placebo arm and in period 1 of the Placebo/Experimental arm).'}], 'classes': [{'title': 'Any TEAE', 'categories': [{'measurements': [{'value': '42', 'groupId': 'OG000'}, {'value': '32', 'groupId': 'OG001'}]}]}, {'title': 'Serious TEAE', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}]}, {'title': 'Severe TEAE', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}]}, {'title': 'TEAE within 24 hours of IP administration', 'categories': [{'measurements': [{'value': '10', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}]}]}, {'title': 'Serious TEAE within 24 hours of IP administration', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Treatment-related TEAE within 24 hrs of IP admin.', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}]}, {'title': 'Treatment-related SAE within 24 hrs of IP admin.', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'TEAE within 24 hrs IP admin. leading to withdrawal', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Deaths due to TEAE', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Hospitalizations due to TEAE', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}]}, {'title': 'TEAE leading to withdrawal', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}]}, {'title': 'Treatment-related TEAE', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}]}, {'title': 'Treatment-related SAE', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Treatment-related severe TEAE', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Treatment-related TEAE leading to withdrawal', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Weeks 1 to 14 for treatment period 1 and 2', 'description': 'Treatment-emergent adverse events (TEAE) were counted by the treatment most recently taken when the event occurred. Participants were counted once per category per treatment.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Injection Site Reactions', 'denoms': [{'units': 'Participants', 'counts': [{'value': '71', 'groupId': 'OG000'}, {'value': '57', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment C1 INH', 'description': 'Participants who received C1 Esterase Inhibitor (C1 INH) treatment (in period 1 of the Experimental/Placebo arm, in period 2 of the Placebo/Experimental arm and in period 1 and 2 of the Experimental/Experimental arm).'}, {'id': 'OG001', 'title': 'Treatment Placebo', 'description': 'Participants who received Placebo treatment (in period 2 of the Experimental/Placebo arm and in period 1 of the Placebo/Experimental arm).'}], 'classes': [{'title': 'Any injection site reaction', 'categories': [{'measurements': [{'value': '42', 'groupId': 'OG000'}, {'value': '15', 'groupId': 'OG001'}]}]}, {'title': 'Any severe injection site reaction', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Any mild injection site reaction', 'categories': [{'measurements': [{'value': '42', 'groupId': 'OG000'}, {'value': '15', 'groupId': 'OG001'}]}]}, {'title': 'Any moderate injection site reaction', 'categories': [{'measurements': [{'value': '14', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Weeks 1 to 14 for treatment period 1 and 2', 'description': 'Injection site reactions (Erythema, Swelling, Cutaneous pain, Burning sensation, Itching/Pruritus, Warm sensation) were recorded on a designated eCRF page by the site personnel who monitored the local reaction for 1 hour after IP administration 5 times during each treatment period.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of Patients With Positive Anti-C1 INH Antibodies', 'denoms': [{'units': 'Participants', 'counts': [{'value': '71', 'groupId': 'OG000'}, {'value': '57', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment C1 INH', 'description': 'Participants who received C1 Esterase Inhibitor (C1 INH) treatment (in period 1 of the Experimental/Placebo arm, in period 2 of the Placebo/Experimental arm and in period 1 and 2 of the Experimental/Experimental arm).'}, {'id': 'OG001', 'title': 'Treatment Placebo', 'description': 'Participants who received Placebo treatment (in period 2 of the Experimental/Placebo arm and in period 1 of the Placebo/Experimental arm).'}], 'classes': [{'title': 'Positive anti-C1 INH antibodies prior treatment', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Positive anti-C1 INH antibodies developed', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Weeks 1 to 14 for treatment period 1 and 2', 'description': 'Anti-C1 INH antibodies were measured during study time.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'PK Parameters: AUC (0-96) and AUC (0-t) for Functional C1 INH Binding Activity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment C1 INH', 'description': 'Participants who received C1 Esterase Inhibitor (C1 INH) treatment (in period 1 of the Experimental/Placebo arm, in period 2 of the Placebo/Experimental arm and in period 1 and 2 of the Experimental/Experimental arm).'}, {'id': 'OG001', 'title': 'Treatment Placebo', 'description': 'Participants who received Placebo treatment (in period 2 of the Experimental/Placebo arm and in period 1 of the Placebo/Experimental arm).'}], 'classes': [{'title': 'AUC (0-96)', 'categories': [{'measurements': [{'value': '31070', 'spread': '17396', 'groupId': 'OG000'}, {'value': '13860', 'spread': '7269.0', 'groupId': 'OG001'}]}]}, {'title': 'AUC (0-t)', 'categories': [{'measurements': [{'value': '31190', 'spread': '17389', 'groupId': 'OG000'}, {'value': '13860', 'spread': '7268.3', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Within 15 min prior dosing at week 1, week 2, week 8, week 16, week 24, week 27/28 and 48 (± 3) hours after dose in week 27/28 in period 1 and 2. In addition 24 (±3) hours, 72 (±6) hours and 96 (±6) hours post dose in week 28 for period 2.', 'description': 'AUC(0-96)=area under the plasma concentration-time curve from time zero to last measurable concentration; AUC(0-t)=area under the plasma concentration-time curve from time zero extrapolated to the end of the dosing interval tau, where tau is approximately 84 hours (ie, average of every 3 or 4 days) AUC(0-96) = AUC(0-tau).', 'unitOfMeasure': 'mU*h/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants included who have taken at least one dose of investigational product and for whom the primary PK data are considered sufficient and interpretable.'}, {'type': 'SECONDARY', 'title': 'PK Parameters: AUC (0-96) and AUC (0-t) for C1 INH Antigen Concentrations', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment C1 INH', 'description': 'Participants who received C1 Esterase Inhibitor (C1 INH) treatment (in period 1 of the Experimental/Placebo arm, in period 2 of the Placebo/Experimental arm and in period 1 and 2 of the Experimental/Experimental arm).'}, {'id': 'OG001', 'title': 'Treatment Placebo', 'description': 'Participants who received Placebo treatment (in period 2 of the Experimental/Placebo arm and in period 1 of the Placebo/Experimental arm).'}], 'classes': [{'title': 'AUC (0-96)', 'categories': [{'measurements': [{'value': '6882', 'spread': '4586.0', 'groupId': 'OG000'}, {'value': '1849', 'spread': '426.36', 'groupId': 'OG001'}]}]}, {'title': 'AUC (0-t)', 'categories': [{'measurements': [{'value': '6902', 'spread': '4574.0', 'groupId': 'OG000'}, {'value': '1849', 'spread': '426.09', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Within 15 min prior dosing at week 1, week 2, week 8, week 16, week 24, week 27/28 and 48 (± 3) hours after dose in week 27/28 in period 1 and 2. In addition 24 (±3) hours, 72 (±6) hours and 96 (±6) hours post dose in week 28 for period 2.', 'description': 'AUC(0-96)=area under the plasma concentration-time curve from time zero to last measurable concentration; AUC(0-t)=area under the plasma concentration-time curve from time zero extrapolated to the end of the dosing interval tau, where tau is approximately 84 hours (ie, average of every 3 or 4 days) AUC(0-96) = AUC(0-tau).', 'unitOfMeasure': 'mcg*h/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants included who have taken at least one dose of investigational product and for whom the primary PK data are considered sufficient and interpretable.'}, {'type': 'SECONDARY', 'title': 'PK Parameters: AUC (0-96) and AUC (0-t) for Complement C4 Concentrations (Treamtment C1 INH)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment C1 INH', 'description': 'Participants who received C1 Esterase Inhibitor (C1 INH) treatment (in period 1 of the Experimental/Placebo arm, in period 2 of the Placebo/Experimental arm and in period 1 and 2 of the Experimental/Experimental arm).'}], 'classes': [{'title': 'AUC (0-96)', 'categories': [{'measurements': [{'value': '16690', 'spread': '803.60', 'groupId': 'OG000'}]}]}, {'title': 'AUC (0-t)', 'categories': [{'measurements': [{'value': '16780', 'spread': '895.63', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Within 15 min prior dosing at week 1, week 2, week 8, week 16, week 24, week 27/28 and 48 (± 3) hours after dose in week 27/28 in period 1 and 2. In addition 24 (±3) hours, 72 (±6) hours and 96 (±6) hours post dose in week 28 for period 2.', 'description': 'AUC(0-96)=area under the plasma concentration-time curve from time zero to last measurable concentration; AUC(0-t)=area under the plasma concentration-time curve from time zero extrapolated to the end of the dosing interval tau, where tau is approximately 84 hours (ie, average of every 3 or 4 days) AUC(0-96) = AUC(0-tau).', 'unitOfMeasure': 'mg*h/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants included who have taken at least one dose of investigational product and for whom the primary PK data are considered sufficient and interpretable.'}, {'type': 'SECONDARY', 'title': 'PK Parameters: AUC (0-96) and AUC (0-t) for Complement C4 Concentrations (Treatment Placebo)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment Placebo', 'description': 'Participants who received Placebo treatment (in period 2 of the Experimental/Placebo arm and in period 1 of the Placebo/Experimental arm).'}], 'classes': [{'title': 'AUC (0-96) - Participant 1', 'categories': [{'measurements': [{'value': '8840', 'groupId': 'OG000'}]}]}, {'title': 'AUC (0-96) - Participant 2', 'categories': [{'measurements': [{'value': '2150', 'groupId': 'OG000'}]}]}, {'title': 'AUC (0-t) - Participant 1', 'categories': [{'measurements': [{'value': '8840', 'groupId': 'OG000'}]}]}, {'title': 'AUC (0-t) - Participant 2', 'categories': [{'measurements': [{'value': '2150', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Within 15 min prior dosing at week 1, week 2, week 8, week 16, week 24, week 27/28 and 48 (± 3) hours after dose in week 27/28 in period 1 and 2. In addition 24 (±3) hours, 72 (±6) hours and 96 (±6) hours post dose in week 28 for period 2.', 'description': 'AUC(0-96)=area under the plasma concentration-time curve from time zero to last measurable concentration; AUC(0-t)=area under the plasma concentration-time curve from time zero extrapolated to the end of the dosing interval tau, where tau is approximately 84 hours (ie, average of every 3 or 4 days) AUC(0-96) = AUC(0-tau). Participant wise data was reported for this outcome.', 'unitOfMeasure': 'mg*h/L', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants included who have taken at least one dose of investigational product and for whom the primary PK data are considered sufficient and interpretable. No summary analysis was done and participant wise data are reported.'}, {'type': 'SECONDARY', 'title': 'PK Parameters: Cmax and Cmin for Functional C1 INH Binding Activity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment C1 INH', 'description': 'Participants who received C1 Esterase Inhibitor (C1 INH) treatment (in period 1 of the Experimental/Placebo arm, in period 2 of the Placebo/Experimental arm and in period 1 and 2 of the Experimental/Experimental arm).'}, {'id': 'OG001', 'title': 'Treatment Placebo', 'description': 'Participants who received Placebo treatment (in period 2 of the Experimental/Placebo arm and in period 1 of the Placebo/Experimental arm).'}], 'classes': [{'title': 'Cmax', 'categories': [{'measurements': [{'value': '396.20', 'spread': '273.013', 'groupId': 'OG000'}, {'value': '159.50', 'spread': '82.982', 'groupId': 'OG001'}]}]}, {'title': 'Cmin', 'categories': [{'measurements': [{'value': '258.15', 'spread': '138.232', 'groupId': 'OG000'}, {'value': '125.90', 'spread': '62.329', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Within 15 min prior dosing at week 1, week 2, week 8, week 16, week 24, week 27/28 and 48 (± 3) hours after dose in week 27/28 in period 1 and 2 and in addition 24 (±3) hours, 72 (±6) hours and 96 (±6) hours post dose in week 28 for period 2.', 'description': 'Cmax=maximum observed plasma concentration and Cmin=minimum observed plasma concentration', 'unitOfMeasure': 'mU/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants included who have taken at least one dose of investigational product and for whom the primary PK data are considered sufficient and interpretable.'}, {'type': 'SECONDARY', 'title': 'PK Parameters: Cmax and Cmin for C1 INH Antigen Concentrations', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment C1 INH', 'description': 'Participants who received C1 Esterase Inhibitor (C1 INH) treatment (in period 1 of the Experimental/Placebo arm, in period 2 of the Placebo/Experimental arm and in period 1 and 2 of the Experimental/Experimental arm).'}, {'id': 'OG001', 'title': 'Treatment Placebo', 'description': 'Participants who received Placebo treatment (in period 2 of the Experimental/Placebo arm and in period 1 of the Placebo/Experimental arm)..'}], 'classes': [{'title': 'Cmax', 'categories': [{'measurements': [{'value': '77.680', 'spread': '52.4375', 'groupId': 'OG000'}, {'value': '21.257', 'spread': '5.1647', 'groupId': 'OG001'}]}]}, {'title': 'Cmin', 'categories': [{'measurements': [{'value': '65.562', 'spread': '45.9331', 'groupId': 'OG000'}, {'value': '17.913', 'spread': '4.4316', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Within 15 min prior dosing at week 1, week 2, week 8, week 16, week 24, week 27/28 and 48 (± 3) hours after dose in week 27/28 in period 1 and 2 and in addition 24 (±3) hours, 72 (±6) hours and 96 (±6) hours post dose in week 28 for period 2', 'description': 'Cmax=maximum observed plasma concentration and Cmin=minimum observed plasma concentration', 'unitOfMeasure': 'µg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants included who have taken at least one dose of investigational product and for whom the primary PK data are considered sufficient and interpretable.'}, {'type': 'SECONDARY', 'title': 'PK Parameters: Cmax and Cmin for Complement C4 Concentrations (Treatment C1 INH)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment C1 INH', 'description': 'Participants who received C1 Esterase Inhibitor (C1 INH) treatment (in period 1 of the Experimental/Placebo arm, in period 2 of the Placebo/Experimental arm and in period 1 and 2 of the Experimental/Experimental arm).'}], 'classes': [{'title': 'Cmax', 'categories': [{'measurements': [{'value': '200', 'spread': '30.82', 'groupId': 'OG000'}]}]}, {'title': 'Cmin', 'categories': [{'measurements': [{'value': '158', 'spread': '13.04', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Within 15 min prior dosing at week 1, week 2, week 8, week 16, week 24, week 27/28 and 48 (± 3) hours after dose in week 27/28 in period 1 and 2 and in addition 24 (±3) hours, 72 (±6) hours and 96 (±6) hours post dose in week 28 for period 2.', 'description': 'Cmax=maximum observed plasma concentration and Cmin=minimum observed plasma concentration', 'unitOfMeasure': 'mg/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants included who have taken at least one dose of investigational product and for whom the primary PK data are considered sufficient and interpretable.'}, {'type': 'SECONDARY', 'title': 'PK Parameters: Cmax and Cmin for Complement C4 Concentrations (Treatment Placebo)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment Placebo', 'description': 'Participants who received Placebo treatment (in period 2 of the Experimental/Placebo arm and in period 1 of the Placebo/Experimental arm).'}], 'classes': [{'title': 'Cmax - Participant 1', 'categories': [{'measurements': [{'value': '120', 'groupId': 'OG000'}]}]}, {'title': 'Cmax - Participant 2', 'categories': [{'measurements': [{'value': '27', 'groupId': 'OG000'}]}]}, {'title': 'Cmin - Participant 1', 'categories': [{'measurements': [{'value': '82', 'groupId': 'OG000'}]}]}, {'title': 'Cmin - Participant 2', 'categories': [{'measurements': [{'value': '18', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Within 15 min prior dosing at week 1, week 2, week 8, week 16, week 24, week 27/28 and 48 (± 3) hours after dose in week 27/28 in period 1 and 2 and in addition 24 (±3) hours, 72 (±6) hours and 96 (±6) hours post dose in week 28 for period 2.', 'description': 'Cmax=maximum observed plasma concentration and Cmin=minimum observed plasma concentration. Participant wise data was reported for this outcome.', 'unitOfMeasure': 'mg/L', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants included who have taken at least one dose of investigational product and for whom the primary PK data are considered sufficient and interpretable. No summary analysis was done and participant wise data are reported.'}, {'type': 'SECONDARY', 'title': 'PK Parameters: Tmax', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment C1 INH', 'description': 'Participants who received C1 Esterase Inhibitor (C1 INH) treatment (in period 1 of the Experimental/Placebo arm, in period 2 of the Placebo/Experimental arm and in period 1 and 2 of the Experimental/Experimental arm).'}, {'id': 'OG001', 'title': 'Treatment Placebo', 'description': 'Participants who received Placebo treatment (in period 2 of the Experimental/Placebo arm and in period 1 of the Placebo/Experimental arm).'}], 'classes': [{'title': 'Functional C1 INH Binding Activity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '31.597', 'spread': '12.7038', 'groupId': 'OG000'}, {'value': '55.689', 'spread': '49.4851', 'groupId': 'OG001'}]}]}, {'title': 'C1 INH Antigen Concentration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '31.656', 'spread': '24.6912', 'groupId': 'OG000'}, {'value': '47.578', 'spread': '47.3106', 'groupId': 'OG001'}]}]}, {'title': 'Complement C4 Concentration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '33.417', 'spread': '36.5183', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Within 15 min prior dosing at week 1, week 2, week 8, week 16, week 24, week 27/28 and 48 (± 3) hours after dose in week 27/28 in period 1 and 2 and in addition 24 (±3) hours, 72 (±6) hours and 96 (±6) hours post dose in week 28 for period 2.', 'description': 'tmax=time of maximum observed plasma concentration', 'unitOfMeasure': 'hours', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants included who have taken at least one dose of investigational product and for whom the primary PK data are considered sufficient and interpretable. For Complement C4 Concentration no summary analysis was done for participants in the placebo group and participant wise data for 2 participants are reported in Outcome measure #19.'}, {'type': 'SECONDARY', 'title': 'PK Parameters: Tmax for Complement C4 Concentrations (Placebo Group)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment Placebo', 'description': 'Participants who received Placebo treatment (in period 2 of the Experimental/Placebo arm and in period 1 of the Placebo/Experimental arm).'}], 'classes': [{'title': 'tmax - Participant 1', 'categories': [{'measurements': [{'value': '94.62', 'groupId': 'OG000'}]}]}, {'title': 'tmax - Participant 2', 'categories': [{'measurements': [{'value': '48.12', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Within 15 min prior dosing at week 1, week 2, week 8, week 16, week 24, week 27/28 and 48 (± 3) hours after dose in week 27/28 in period 1 and 2 and in addition 24 (±3) hours, 72 (±6) hours and 96 (±6) hours post dose in week 28 for period 2.', 'description': 'tmax=time of maximum observed plasma concentration. Participant wise data was reported for this outcome.', 'unitOfMeasure': 'mg/L', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants included who have taken at least one dose of investigational product and for whom the primary PK data are considered sufficient and interpretable. No summary analysis was done and participant wise data are reported.'}, {'type': 'SECONDARY', 'title': 'Assess Disease Activity as Measured by the Angioedema Activity Score (AAS) Normalized Per Month', 'denoms': [{'units': 'Participants', 'counts': [{'value': '56', 'groupId': 'OG000'}, {'value': '57', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment C1 INH', 'description': 'Participants who received C1 Esterase Inhibitor (C1 INH) treatment (in period 1 of the Experimental/Placebo arm and in period 2 of the Placebo/Experimental arm).'}, {'id': 'OG001', 'title': 'Treatment Placebo', 'description': 'Participants who received Placebo treatment (in period 2 of the Experimental/Placebo arm and in period 1 of the Placebo/Experimental arm).'}], 'classes': [{'categories': [{'measurements': [{'value': '25.433', 'groupId': 'OG000', 'lowerLimit': '11.204', 'upperLimit': '39.662'}, {'value': '57.168', 'groupId': 'OG001', 'lowerLimit': '43.010', 'upperLimit': '71.326'}]}]}], 'analyses': [{'pValue': '0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference in LS means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-31.735', 'ciLowerLimit': '-42.696', 'ciUpperLimit': '-20.773', 'groupDescription': 'The Least Square means, 95% confidence intervals and p-values are based on mixed effect linear model with period, sequence, use of prophylactic therapy with C1 INH at randomization and treatment as fixed effects and subject nested within sequence as a random effect.', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Weeks 1 to 14 for treatment period 1 and 2', 'description': 'Disease activity was measured using a 98-day Angioedema Activity Score (AAS). The AAS collects information of disease activity in the last 24 hours. The following items are assessed: experience of swelling, severity of the swelling, timing of the swelling, extent of discomfort due to the swelling, extent that the swelling caused limitations in daily life, and feelings of being disfigured by the swelling. The instrument uses a binary response option for the first item and a three-point response scale for the 5 items thereafter. The daily AAS was the sum of the AAS items per day. Total daily ASS scores range between 0 and 15 points. Higher values stand for higher disease activity. The normalized 98-day AAS per month for a participant is calculated by (the sum of daily AAS within a treatment period/the number of days that a subject has AAS records within the treatment period)\\*30.4.', 'unitOfMeasure': 'Score on a scale', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Data were not collected for the Experimental/Experimental Arm for this outcome measure and this represents the full analysis set.'}, {'type': 'SECONDARY', 'title': 'Participant Experience With Self-administration: Overall Experience With the Syringe', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}, {'value': '40', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment C1 INH', 'description': 'Participants who received C1 Esterase Inhibitor (C1 INH) treatment (in period 1 of the Experimental/Placebo arm, in period 2 of the Placebo/Experimental arm and in period 1 and 2 of the Experimental/Experimental arm).'}, {'id': 'OG001', 'title': 'Treatment Placebo', 'description': 'Participants who received Placebo treatment (in period 2 of the Experimental/Placebo arm and in period 1 of the Placebo/Experimental arm).'}], 'classes': [{'title': 'Visit 28: Easy to use', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}, {'value': '40', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '48', 'groupId': 'OG000'}, {'value': '36', 'groupId': 'OG001'}]}]}, {'title': 'Visit 28: Somewhat difficult to use', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}, {'value': '40', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '11', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}]}, {'title': 'Visiti 28: Difficult to use', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}, {'value': '40', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Visit 28b: Easy to use', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '11', 'groupId': 'OG000'}]}]}, {'title': 'Visit 28b: Somewhat difficult to use', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Visiti 28b: Difficult to use', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Week 14 for treatment period 1 and 2', 'description': 'Self-administration survey with questions about the overall experience with the syringe was assessed in week 14 (visit 28 and 28b). Visit 28 summarizes treatment period 1 of the experimental/experimental arm and treatment periods 1 and 2 of the experimental/placebo arm and the placebo/experimental arm. Visit 28b summarizes treatment period 2 of the experimental/experimental arm.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Visit 28b summarizes only treatment period 2 of the Experimental/Experimental arm. Therefore no participants were analyzed in the placebo group.'}, {'type': 'SECONDARY', 'title': 'Participant Experience With Self-administration: How Many Visits for Confidence With Self-administration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}, {'value': '40', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment C1 INH', 'description': 'Participants who received C1 Esterase Inhibitor (C1 INH) treatment (in period 1 of the Experimental/Placebo arm, in period 2 of the Placebo/Experimental arm and in period 1 and 2 of the Experimental/Experimental arm).'}, {'id': 'OG001', 'title': 'Treatment Placebo', 'description': 'Participants who received Placebo treatment (in period 2 of the Experimental/Placebo arm and in period 1 of the Placebo/Experimental arm).'}], 'classes': [{'title': 'Visit 28', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}, {'value': '40', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '1.8', 'spread': '1.70', 'groupId': 'OG000'}, {'value': '2.0', 'spread': '2.49', 'groupId': 'OG001'}]}]}, {'title': 'Visit 28b', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '1.8', 'spread': '1.22', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Week 14 for treatment period 1 and 2', 'description': 'The self-administration survey includes the number of visits needed for participants to be able to self-administer investigational product with confidence and all participants could self-administer without supervision. Visit 28 summarizes treatment period 1 of the experimental/experimental arm and treatment periods 1 and 2 of the experimental/placebo arm and the placebo/experimental arm. Visit 28b summarizes treatment period 2 of the experimental/experimental arm.', 'unitOfMeasure': 'Number of visits', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Visit 28b summarizes only treatment period 2 of the Experimental/Experimental arm. Therefore no participants were analyzed in the placebo group.'}, {'type': 'SECONDARY', 'title': 'Participant Experience With Self-administration: Better Long-term Option and Preferred Administration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}, {'value': '40', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment C1 INH', 'description': 'Participants who received C1 Esterase Inhibitor (C1 INH) treatment (in period 1 of the Experimental/Placebo arm, in period 2 of the Placebo/Experimental arm and in period 1 and 2 of the Experimental/Experimental arm).'}, {'id': 'OG001', 'title': 'Treatment Placebo', 'description': 'Participants who received Placebo treatment (in period 2 of the Experimental/Placebo arm and in period 1 of the Placebo/Experimental arm).'}], 'classes': [{'title': 'Visit 28: SC better long term option', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}, {'value': '40', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '57', 'groupId': 'OG000'}, {'value': '39', 'groupId': 'OG001'}]}]}, {'title': 'Visit 28: IV better long term option', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}, {'value': '40', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Visit 28: SC preferred administration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}, {'value': '40', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '56', 'groupId': 'OG000'}, {'value': '40', 'groupId': 'OG001'}]}]}, {'title': 'Visit 28: IV preferred administration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}, {'value': '40', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Visit 28: Preference for admin - very strong', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}, {'value': '40', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '47', 'groupId': 'OG000'}, {'value': '33', 'groupId': 'OG001'}]}]}, {'title': 'Visit 28: Preference for admin - fairly strong', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}, {'value': '40', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '12', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}]}]}, {'title': 'Visit 28: Preference for admin - not very strong', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}, {'value': '40', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Visit 28b: SC better long term option', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '12', 'groupId': 'OG000'}]}]}, {'title': 'Visit 28b: IV better long term option', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Visit 28b: SC preferred administration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '11', 'groupId': 'OG000'}]}]}, {'title': 'Visit 28b: IV preferred administration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Visit 28b: Preference for admin - very strong', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '11', 'groupId': 'OG000'}]}]}, {'title': 'Visit 28b: Preference for admin - fairly strong', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Visit 28b: Preference for admin - not very strong', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Week 14 for treatment period 1 and 2', 'description': 'The self-administration survey includes the number of visits needed for participants to be able to self-administer investigational product with confidence and all participants could self-administer without supervision. Visit 28 summarizes treatment period 1 of the experimental/experimental arm and treatment periods 1 and 2 of the experimental/placebo arm and the placebo/experimental arm. Visit 28b summarizes treatment period 2 of the experimental/experimental arm.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Visit 28b summarizes only treatment period 2 of the Experimental/Experimental arm. Therefore no participants were analyzed in the placebo group.'}, {'type': 'SECONDARY', 'title': 'Mean Change in Angioedema Quality of Life Questionnaire Scores From Baseline to Week 13', 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}, {'value': '28', 'groupId': 'OG001'}, {'value': '29', 'groupId': 'OG002'}, {'value': '25', 'groupId': 'OG003'}, {'value': '15', 'groupId': 'OG004'}, {'value': '15', 'groupId': 'OG005'}]}], 'groups': [{'id': 'OG000', 'title': 'Experimental/Placebo Arm - Treatment C1 INH', 'description': 'Participants randomized to the Experimental/Placebo arm received C1 Esterase Inhibitor (C1 INH) in treatment period 1.'}, {'id': 'OG001', 'title': 'Experimental/Placebo Arm - Treatment Placebo', 'description': 'Participants randomized to the Experimental/Placebo arm received Placebo in treatment period 2.'}, {'id': 'OG002', 'title': 'Placebo/Experimental Arm - Treatment Placebo', 'description': 'Participants randomized to the Placebo/Experimental arm received Placebo in treatment period 1.'}, {'id': 'OG003', 'title': 'Placebo/Experimental Arm - Treatment C1 INH', 'description': 'Participants randomized to the Placebo/Experimental arm received C1 Esterase Inhibitor (C1 INH) in treatment period 2.'}, {'id': 'OG004', 'title': 'Experimental/Experimental Arm - Period 1 Treatment C1 INH', 'description': 'Participants randomized to the Experimental/Experimental arm received C1 Esterase Inhibitor (C1 INH) in treatment period 1.'}, {'id': 'OG005', 'title': 'Experimental/Experimental Arm - Period 2 Treatment C1 INH', 'description': 'Participants randomized to the Experimental/Experimental arm received C1 Esterase Inhibitor (C1 INH) in treatment period 2.'}], 'classes': [{'title': 'AE-QoL Total', 'categories': [{'measurements': [{'value': '-10.35', 'spread': '17.75', 'groupId': 'OG000'}, {'value': '4.77', 'spread': '12.14', 'groupId': 'OG001'}, {'value': '-6.86', 'spread': '10.72', 'groupId': 'OG002'}, {'value': '-12.10', 'spread': '9.83', 'groupId': 'OG003'}, {'value': '-11.65', 'spread': '9.87', 'groupId': 'OG004'}, {'value': '-0.52', 'spread': '6.58', 'groupId': 'OG005'}]}]}, {'title': 'Functioning', 'categories': [{'measurements': [{'value': '-9.25', 'spread': '21.29', 'groupId': 'OG000'}, {'value': '6.11', 'spread': '20.62', 'groupId': 'OG001'}, {'value': '-10.56', 'spread': '17.23', 'groupId': 'OG002'}, {'value': '-23.33', 'spread': '19.45', 'groupId': 'OG003'}, {'value': '-20.50', 'spread': '14.62', 'groupId': 'OG004'}, {'value': '-2.78', 'spread': '10.34', 'groupId': 'OG005'}]}]}, {'title': 'Fatigue/Mood', 'categories': [{'measurements': [{'value': '-8.40', 'spread': '21.94', 'groupId': 'OG000'}, {'value': '2.44', 'spread': '14.20', 'groupId': 'OG001'}, {'value': '-9.11', 'spread': '10.16', 'groupId': 'OG002'}, {'value': '-10.10', 'spread': '13.42', 'groupId': 'OG003'}, {'value': '-8.40', 'spread': '12.29', 'groupId': 'OG004'}, {'value': '3.11', 'spread': '9.33', 'groupId': 'OG005'}]}]}, {'title': 'Fear/Shame', 'categories': [{'measurements': [{'value': '-12.50', 'spread': '21.46', 'groupId': 'OG000'}, {'value': '5.19', 'spread': '12.06', 'groupId': 'OG001'}, {'value': '-1.67', 'spread': '10.62', 'groupId': 'OG002'}, {'value': '-5.40', 'spread': '10.67', 'groupId': 'OG003'}, {'value': '-9.67', 'spread': '11.91', 'groupId': 'OG004'}, {'value': '-1.85', 'spread': '11.19', 'groupId': 'OG005'}]}]}, {'title': 'Nutrition', 'categories': [{'measurements': [{'value': '-11.00', 'spread': '22.45', 'groupId': 'OG000'}, {'value': '6.67', 'spread': '19.70', 'groupId': 'OG001'}, {'value': '-9.44', 'spread': '25.55', 'groupId': 'OG002'}, {'value': '-14.76', 'spread': '17.50', 'groupId': 'OG003'}, {'value': '-8.00', 'spread': '16.19', 'groupId': 'OG004'}, {'value': '-1.11', 'spread': '7.82', 'groupId': 'OG005'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to week 13 for treatment period 1 and 2', 'description': 'The AE-QoL is a questionnaire on the quality of life of patients suffering from recurrent angioedema. It consists of 17 specific questions that are associated with work, physical activity, free time, social relations, and food. Each of the 17 questions has a five-point response scale ranging from 1 (Never) to 5 (Very Often). The AE-QoL consists of 4 dimensions (functioning=4 questions(qns) fatigue/mood=5 qns, fears/shame=6 qns, nutrition=2 qns) and a total score (all 17 questions).All scores were calculated by using the following formula: (Σ items - min Σ items / max Σ items - min Σ items) x 100. Σ items=sum of response by participant, min Σ items=minimum response possible, max Σ items=maximum response possible. Scores range from 0 to 100 , with higher scores indicating greater impairment. Absolute change calculated as visit score at week 13 minus score at baseline per period.', 'unitOfMeasure': 'Mean change in AE-QoL scores', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Experimental/Placebo', 'description': 'Participants were randomized to receive C1 Esterase Inhibitor in the 1st treatment period and then switched to Placebo in the 2nd treatment period.'}, {'id': 'FG001', 'title': 'Placebo/Experimental', 'description': 'Participants were randomized to receive placebo treatment in the 1st treatment period and then switched to receive C1 Esterase Inhibitor in the 2nd treatment period.'}, {'id': 'FG002', 'title': 'Experimental/ Experimental', 'description': 'Participants were randomized to receive C1 Esterase Inhibitor in both 1st as well as the 2nd treatment period.'}], 'periods': [{'title': 'Period 1', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '31'}, {'groupId': 'FG001', 'numSubjects': '29'}, {'groupId': 'FG002', 'numSubjects': '15'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '28'}, {'groupId': 'FG001', 'numSubjects': '25'}, {'groupId': 'FG002', 'numSubjects': '15'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '4'}, {'groupId': 'FG002', 'numSubjects': '0'}]}], 'dropWithdraws': [{'type': 'Withdrawn from study', 'reasons': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '4'}, {'groupId': 'FG002', 'numSubjects': '0'}]}]}, {'title': 'Period 2', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '28'}, {'groupId': 'FG001', 'numSubjects': '25'}, {'groupId': 'FG002', 'numSubjects': '15'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '22'}, {'groupId': 'FG001', 'numSubjects': '24'}, {'groupId': 'FG002', 'numSubjects': '13'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '6'}, {'groupId': 'FG001', 'numSubjects': '1'}, {'groupId': 'FG002', 'numSubjects': '2'}]}], 'dropWithdraws': [{'type': 'Withdrawn from study', 'reasons': [{'groupId': 'FG000', 'numSubjects': '6'}, {'groupId': 'FG001', 'numSubjects': '1'}, {'groupId': 'FG002', 'numSubjects': '2'}]}]}], 'recruitmentDetails': 'This was a multicenter study conducted at 33 sites/centers in 7 countries: United States, Canada , Germany, Hungary, Israel, Spain, and Romania .', 'preAssignmentDetails': 'Of the 81 participants screened, 6 subjects failed to meet the randomization criteria and were not randomly assigned to a treatment sequence . All 75 randomly assigned participants received at least 1 dose of the IP.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'BG000'}, {'value': '29', 'groupId': 'BG001'}, {'value': '15', 'groupId': 'BG002'}, {'value': '75', 'groupId': 'BG003'}]}], 'groups': [{'id': 'BG000', 'title': 'Experimental/Placebo', 'description': 'Subjects will be randomized to receive C1 Esterase Inhibitor in the 1st Treatment period and then switch to Placebo in the 2nd treatment period.'}, {'id': 'BG001', 'title': 'Placebo/Experimental', 'description': 'Subjects will be randomized to receive a placebo treatment in the 1st Treatment period and then switch to receive C1 Esterase Inhibitor in the 2nd treatment period.'}, {'id': 'BG002', 'title': 'Experimental/ Experimental', 'description': 'Subjects will be randomized and receive C1 Esterase Inhibitor in both 1st as well as the 2nd treatment period'}, {'id': 'BG003', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}, {'value': '3', 'groupId': 'BG003'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '30', 'groupId': 'BG000'}, {'value': '25', 'groupId': 'BG001'}, {'value': '13', 'groupId': 'BG002'}, {'value': '68', 'groupId': 'BG003'}]}, {'title': '>=65 years', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}, {'value': '4', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '40.5', 'spread': '13.16', 'groupId': 'BG000'}, {'value': '40.7', 'spread': '15.34', 'groupId': 'BG001'}, {'value': '44.4', 'spread': '16.40', 'groupId': 'BG002'}, {'value': '41.3', 'spread': '14.58', 'groupId': 'BG003'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '23', 'groupId': 'BG000'}, {'value': '21', 'groupId': 'BG001'}, {'value': '8', 'groupId': 'BG002'}, {'value': '52', 'groupId': 'BG003'}]}, {'title': 'Male', 'measurements': [{'value': '8', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '7', 'groupId': 'BG002'}, {'value': '23', 'groupId': 'BG003'}]}]}], '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'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Black or African American', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '2', 'groupId': 'BG003'}]}, {'title': 'White', 'measurements': [{'value': '30', 'groupId': 'BG000'}, {'value': '27', 'groupId': 'BG001'}, {'value': '15', 'groupId': 'BG002'}, {'value': '72', 'groupId': 'BG003'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2015-05-28', 'size': 1094178, 'label': 'Study Protocol: Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2018-07-17T07:17', 'hasProtocol': True}, {'date': '2015-07-22', 'size': 1223145, 'label': 'Study Protocol: Amendment1', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_001.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2018-07-17T07:18', 'hasProtocol': True}, {'date': '2015-09-03', 'size': 1210855, 'label': 'Study Protocol: Amendment2', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_002.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2018-07-17T07:20', 'hasProtocol': True}, {'date': '2016-01-11', 'size': 1562709, 'label': 'Study Protocol: Amendment3', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_003.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2018-07-17T07:20', 'hasProtocol': True}, {'date': '2017-02-08', 'size': 1226704, 'label': 'Study Protocol: Amendment4', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_004.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2018-07-17T07:21', 'hasProtocol': True}, {'date': '2017-08-17', 'size': 1245418, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_005.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2018-07-17T07:49', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'DOUBLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR']}, 'primaryPurpose': 'PREVENTION', 'interventionModel': 'CROSSOVER'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 75}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2015-11-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-05', 'completionDateStruct': {'date': '2017-07-24', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-05-14', 'studyFirstSubmitDate': '2015-10-20', 'resultsFirstSubmitDate': '2018-07-17', 'studyFirstSubmitQcDate': '2015-10-21', 'lastUpdatePostDateStruct': {'date': '2021-06-08', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2018-11-02', 'studyFirstPostDateStruct': {'date': '2015-10-23', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2018-11-28', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2017-07-24', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Time-Normalized Number of Attacks (NNA) for Participants During a Treatment Period', 'timeFrame': 'Weeks 1 to 14 for treatment period 1 and 2', 'description': 'The angioedema attacks were recorded in the electronic patient diary. The investigator completed a separate angioedema eCRF for each attack based on the review of the patients diary. Time-normalized number of angioedema attacks was expressed as the number of attacks per month (ie, 30.4 days) of exposure. NNA=30.4 \\* (number of attacks during treatment period) / (days of treatment period).'}], 'secondaryOutcomes': [{'measure': 'Proportion of Participants Meeting at Least a 50% Reduction in NNA (Normalized Number of Angioedema Attacks) During the Experimental Injection Treatment Period Relative to the Placebo Period.', 'timeFrame': 'Weeks 1 to 14 for treatment period 1 and 2', 'description': 'The angioedema attacks were recorded in the electronic patient diary. The investigator completed a separate angioedema eCRF for each attack based on the review of the patients diary. Time-Normalized Number of Attacks was expressed as the number of attacks per month (ie, 30.4 days) of exposure. NNA = 30.4 x (number of attacks during treatment period) / (days of treatment period).'}, {'measure': 'Time-Normalized Number of Attacks (NNA) for Participants During Each Treatment Period Excluding the First 2 Weeks.', 'timeFrame': 'Weeks 3 to 14 for treatment period 1 and 2', 'description': 'The angioedema attacks were recorded in the electronic patient diary. The investigator completed a separate angioedema eCRF for each attack based on the review of the patients diary. Time-normalized number of angioedema attacks was expressed as the number of attacks per month (ie, 30.4 days) of exposure. NNA=30.4 \\* (number of attacks during treatment period) / (days of treatment period).'}, {'measure': 'Proportion of Participants Meeting at Least a 50% Reduction in NNA (Normalized Number of Angioedema Attacks) During the Experimental Injection Treatment Period Relative to the Placebo Period Excluding the First 2 Weeks of Each Treatment Period.', 'timeFrame': 'Weeks 3 to 14 for treatment period 1 and 2', 'description': 'The angioedema attacks were recorded in the electronic patient diary. The investigator completed a separate angioedema eCRF for each attack based on the review of the patients diary. Time-Normalized Number of Attacks was expressed as the number of attacks per month (ie, 30.4 days) of exposure. NNA = 30.4 x (number of attacks during treatment period) / (days of treatment period).'}, {'measure': 'Proportion of Participants Meeting at Least a 50% Reduction in NNA (Normalized Number of Angioedema Attacks) During the Experimental Injection Treatment Period Relative to the Pre-treatment Assessment.', 'timeFrame': 'Weeks 1 to 14 for treatment period 1 and 2', 'description': 'The angioedema attacks were recorded in the electronic patient diary. The investigator completed a separate angioedema eCRF for each attack based on the review of the patients diary. Time-Normalized Number of Attacks was expressed as the number of attacks per month (ie, 30.4 days) of exposure. NNA = 30.4 x (number of attacks during treatment period) / (days of treatment period).'}, {'measure': 'Cumulative Attack Severity', 'timeFrame': 'Weeks 1 to 14 for treatment period 1 and 2', 'description': "Severity of the angioedema attack sign/symptom was characterized as None: no symptom; Mild: noticeable symptom but easily tolerated by the participant and did not interfere with routine activities; Moderate: symptom interfered with the participant's ability to attend school or participate in family life and social/recreational activities; Severe: symptom significantly limited the participant's ability to attend school or participate in family life and social/recreational activities. Symptom severity score was assigned as Mild = 1, Moderate = 2 and Severe = 3. Cumulative attack severity score was the sum of the maximum symptom severity scores recorded for each angioedema attack in a treatment period. Cumulative attack-severity score normalized per month \\[(raw score/number of days of participation in that treatment period)\\*30.4\\] was reported here. Cumulative attack-severity score normalized per month ranged from 0 to 19.83 and higher scores represent worse symptoms."}, {'measure': 'Number of Attack-free Days', 'timeFrame': 'Weeks 1 to 14 for treatment period 1 and 2', 'description': 'Attack free days were normalized per month.'}, {'measure': 'Number of Angioedema Attacks Requiring Acute Treatment', 'timeFrame': 'Weeks 1 to 14 for treatment period 1 and 2', 'description': 'Angioedema attacks were normalized per month.'}, {'measure': 'Response to Icatibant When Administered for an Acute Attack', 'timeFrame': 'Weeks 1 to 14 for treatment period 1 and 2', 'description': 'The number of Acute Hereditary Angioedema Attacks that required Icatibant as acute therapy is presented by the number of Icatibant injections.'}, {'measure': 'Number of Patients With Adverse Events (AEs)', 'timeFrame': 'Weeks 1 to 14 for treatment period 1 and 2', 'description': 'Treatment-emergent adverse events (TEAE) were counted by the treatment most recently taken when the event occurred. Participants were counted once per category per treatment.'}, {'measure': 'Number of Participants With Injection Site Reactions', 'timeFrame': 'Weeks 1 to 14 for treatment period 1 and 2', 'description': 'Injection site reactions (Erythema, Swelling, Cutaneous pain, Burning sensation, Itching/Pruritus, Warm sensation) were recorded on a designated eCRF page by the site personnel who monitored the local reaction for 1 hour after IP administration 5 times during each treatment period.'}, {'measure': 'Number of Patients With Positive Anti-C1 INH Antibodies', 'timeFrame': 'Weeks 1 to 14 for treatment period 1 and 2', 'description': 'Anti-C1 INH antibodies were measured during study time.'}, {'measure': 'PK Parameters: AUC (0-96) and AUC (0-t) for Functional C1 INH Binding Activity', 'timeFrame': 'Within 15 min prior dosing at week 1, week 2, week 8, week 16, week 24, week 27/28 and 48 (± 3) hours after dose in week 27/28 in period 1 and 2. In addition 24 (±3) hours, 72 (±6) hours and 96 (±6) hours post dose in week 28 for period 2.', 'description': 'AUC(0-96)=area under the plasma concentration-time curve from time zero to last measurable concentration; AUC(0-t)=area under the plasma concentration-time curve from time zero extrapolated to the end of the dosing interval tau, where tau is approximately 84 hours (ie, average of every 3 or 4 days) AUC(0-96) = AUC(0-tau).'}, {'measure': 'PK Parameters: AUC (0-96) and AUC (0-t) for C1 INH Antigen Concentrations', 'timeFrame': 'Within 15 min prior dosing at week 1, week 2, week 8, week 16, week 24, week 27/28 and 48 (± 3) hours after dose in week 27/28 in period 1 and 2. In addition 24 (±3) hours, 72 (±6) hours and 96 (±6) hours post dose in week 28 for period 2.', 'description': 'AUC(0-96)=area under the plasma concentration-time curve from time zero to last measurable concentration; AUC(0-t)=area under the plasma concentration-time curve from time zero extrapolated to the end of the dosing interval tau, where tau is approximately 84 hours (ie, average of every 3 or 4 days) AUC(0-96) = AUC(0-tau).'}, {'measure': 'PK Parameters: AUC (0-96) and AUC (0-t) for Complement C4 Concentrations (Treamtment C1 INH)', 'timeFrame': 'Within 15 min prior dosing at week 1, week 2, week 8, week 16, week 24, week 27/28 and 48 (± 3) hours after dose in week 27/28 in period 1 and 2. In addition 24 (±3) hours, 72 (±6) hours and 96 (±6) hours post dose in week 28 for period 2.', 'description': 'AUC(0-96)=area under the plasma concentration-time curve from time zero to last measurable concentration; AUC(0-t)=area under the plasma concentration-time curve from time zero extrapolated to the end of the dosing interval tau, where tau is approximately 84 hours (ie, average of every 3 or 4 days) AUC(0-96) = AUC(0-tau).'}, {'measure': 'PK Parameters: AUC (0-96) and AUC (0-t) for Complement C4 Concentrations (Treatment Placebo)', 'timeFrame': 'Within 15 min prior dosing at week 1, week 2, week 8, week 16, week 24, week 27/28 and 48 (± 3) hours after dose in week 27/28 in period 1 and 2. In addition 24 (±3) hours, 72 (±6) hours and 96 (±6) hours post dose in week 28 for period 2.', 'description': 'AUC(0-96)=area under the plasma concentration-time curve from time zero to last measurable concentration; AUC(0-t)=area under the plasma concentration-time curve from time zero extrapolated to the end of the dosing interval tau, where tau is approximately 84 hours (ie, average of every 3 or 4 days) AUC(0-96) = AUC(0-tau). Participant wise data was reported for this outcome.'}, {'measure': 'PK Parameters: Cmax and Cmin for Functional C1 INH Binding Activity', 'timeFrame': 'Within 15 min prior dosing at week 1, week 2, week 8, week 16, week 24, week 27/28 and 48 (± 3) hours after dose in week 27/28 in period 1 and 2 and in addition 24 (±3) hours, 72 (±6) hours and 96 (±6) hours post dose in week 28 for period 2.', 'description': 'Cmax=maximum observed plasma concentration and Cmin=minimum observed plasma concentration'}, {'measure': 'PK Parameters: Cmax and Cmin for C1 INH Antigen Concentrations', 'timeFrame': 'Within 15 min prior dosing at week 1, week 2, week 8, week 16, week 24, week 27/28 and 48 (± 3) hours after dose in week 27/28 in period 1 and 2 and in addition 24 (±3) hours, 72 (±6) hours and 96 (±6) hours post dose in week 28 for period 2', 'description': 'Cmax=maximum observed plasma concentration and Cmin=minimum observed plasma concentration'}, {'measure': 'PK Parameters: Cmax and Cmin for Complement C4 Concentrations (Treatment C1 INH)', 'timeFrame': 'Within 15 min prior dosing at week 1, week 2, week 8, week 16, week 24, week 27/28 and 48 (± 3) hours after dose in week 27/28 in period 1 and 2 and in addition 24 (±3) hours, 72 (±6) hours and 96 (±6) hours post dose in week 28 for period 2.', 'description': 'Cmax=maximum observed plasma concentration and Cmin=minimum observed plasma concentration'}, {'measure': 'PK Parameters: Cmax and Cmin for Complement C4 Concentrations (Treatment Placebo)', 'timeFrame': 'Within 15 min prior dosing at week 1, week 2, week 8, week 16, week 24, week 27/28 and 48 (± 3) hours after dose in week 27/28 in period 1 and 2 and in addition 24 (±3) hours, 72 (±6) hours and 96 (±6) hours post dose in week 28 for period 2.', 'description': 'Cmax=maximum observed plasma concentration and Cmin=minimum observed plasma concentration. Participant wise data was reported for this outcome.'}, {'measure': 'PK Parameters: Tmax', 'timeFrame': 'Within 15 min prior dosing at week 1, week 2, week 8, week 16, week 24, week 27/28 and 48 (± 3) hours after dose in week 27/28 in period 1 and 2 and in addition 24 (±3) hours, 72 (±6) hours and 96 (±6) hours post dose in week 28 for period 2.', 'description': 'tmax=time of maximum observed plasma concentration'}, {'measure': 'PK Parameters: Tmax for Complement C4 Concentrations (Placebo Group)', 'timeFrame': 'Within 15 min prior dosing at week 1, week 2, week 8, week 16, week 24, week 27/28 and 48 (± 3) hours after dose in week 27/28 in period 1 and 2 and in addition 24 (±3) hours, 72 (±6) hours and 96 (±6) hours post dose in week 28 for period 2.', 'description': 'tmax=time of maximum observed plasma concentration. Participant wise data was reported for this outcome.'}, {'measure': 'Assess Disease Activity as Measured by the Angioedema Activity Score (AAS) Normalized Per Month', 'timeFrame': 'Weeks 1 to 14 for treatment period 1 and 2', 'description': 'Disease activity was measured using a 98-day Angioedema Activity Score (AAS). The AAS collects information of disease activity in the last 24 hours. The following items are assessed: experience of swelling, severity of the swelling, timing of the swelling, extent of discomfort due to the swelling, extent that the swelling caused limitations in daily life, and feelings of being disfigured by the swelling. The instrument uses a binary response option for the first item and a three-point response scale for the 5 items thereafter. The daily AAS was the sum of the AAS items per day. Total daily ASS scores range between 0 and 15 points. Higher values stand for higher disease activity. The normalized 98-day AAS per month for a participant is calculated by (the sum of daily AAS within a treatment period/the number of days that a subject has AAS records within the treatment period)\\*30.4.'}, {'measure': 'Participant Experience With Self-administration: Overall Experience With the Syringe', 'timeFrame': 'Week 14 for treatment period 1 and 2', 'description': 'Self-administration survey with questions about the overall experience with the syringe was assessed in week 14 (visit 28 and 28b). Visit 28 summarizes treatment period 1 of the experimental/experimental arm and treatment periods 1 and 2 of the experimental/placebo arm and the placebo/experimental arm. Visit 28b summarizes treatment period 2 of the experimental/experimental arm.'}, {'measure': 'Participant Experience With Self-administration: How Many Visits for Confidence With Self-administration', 'timeFrame': 'Week 14 for treatment period 1 and 2', 'description': 'The self-administration survey includes the number of visits needed for participants to be able to self-administer investigational product with confidence and all participants could self-administer without supervision. Visit 28 summarizes treatment period 1 of the experimental/experimental arm and treatment periods 1 and 2 of the experimental/placebo arm and the placebo/experimental arm. Visit 28b summarizes treatment period 2 of the experimental/experimental arm.'}, {'measure': 'Participant Experience With Self-administration: Better Long-term Option and Preferred Administration', 'timeFrame': 'Week 14 for treatment period 1 and 2', 'description': 'The self-administration survey includes the number of visits needed for participants to be able to self-administer investigational product with confidence and all participants could self-administer without supervision. Visit 28 summarizes treatment period 1 of the experimental/experimental arm and treatment periods 1 and 2 of the experimental/placebo arm and the placebo/experimental arm. Visit 28b summarizes treatment period 2 of the experimental/experimental arm.'}, {'measure': 'Mean Change in Angioedema Quality of Life Questionnaire Scores From Baseline to Week 13', 'timeFrame': 'Baseline to week 13 for treatment period 1 and 2', 'description': 'The AE-QoL is a questionnaire on the quality of life of patients suffering from recurrent angioedema. It consists of 17 specific questions that are associated with work, physical activity, free time, social relations, and food. Each of the 17 questions has a five-point response scale ranging from 1 (Never) to 5 (Very Often). The AE-QoL consists of 4 dimensions (functioning=4 questions(qns) fatigue/mood=5 qns, fears/shame=6 qns, nutrition=2 qns) and a total score (all 17 questions).All scores were calculated by using the following formula: (Σ items - min Σ items / max Σ items - min Σ items) x 100. Σ items=sum of response by participant, min Σ items=minimum response possible, max Σ items=maximum response possible. Scores range from 0 to 100 , with higher scores indicating greater impairment. Absolute change calculated as visit score at week 13 minus score at baseline per period.'}]}, 'conditionsModule': {'conditions': ['Hereditary Angioedema (HAE)']}, 'referencesModule': {'references': [{'pmid': '36326435', 'type': 'DERIVED', 'citation': 'Beard N, Frese M, Smertina E, Mere P, Katelaris C, Mills K. Interventions for the long-term prevention of hereditary angioedema attacks. Cochrane Database Syst Rev. 2022 Nov 3;11(11):CD013403. doi: 10.1002/14651858.CD013403.pub2.'}, {'pmid': '30682573', 'type': 'DERIVED', 'citation': 'Lumry WR, Martinez-Saguer I, Yang WH, Bernstein JA, Jacobs J, Moldovan D, Riedl MA, Johnston DT, Li HH, Tang Y, Schranz J, Lu P, Vardi M, Farkas H; SAHARA study group. Fixed-Dose Subcutaneous C1-Inhibitor Liquid for Prophylactic Treatment of C1-INH-HAE: SAHARA Randomized Study. J Allergy Clin Immunol Pract. 2019 May-Jun;7(5):1610-1618.e4. doi: 10.1016/j.jaip.2019.01.021. Epub 2019 Jan 23.'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to assess the efficacy and safety of subcutaneous administration of a liquid formulation of C1 esterase inhibitor for the prevention of angioedema attacks in adolescent and adult subjects with hereditary angioedema.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'The maximum duration of participation is approximately 9 months. Patients will complete a screening period of up to 21 days. Following screening, eligible patients will be randomly assigned to 1 of 3 treatment sequences. Each patient will undergo 2 14-week treatment periods for a total of 28 weeks (Treatment Period 1 and Treatment Period 2). After completing the 2 treatment periods, patients will enter a 1-month follow-up period.'}, 'identificationModule': {'nctId': 'NCT02584959', 'briefTitle': 'Study to Evaluate the Clinical Efficacy and Safety of Subcutaneously Administered C1 Esterase Inhibitor for the Prevention of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema', 'organization': {'class': 'INDUSTRY', 'fullName': 'Takeda'}, 'officialTitle': 'A Phase 3, Randomized, Double-blind, Placebo-controlled, Two-period, Three-sequence, Partial Crossover Study to Evaluate the Efficacy and Safety of Subcutaneous Administration of 2000 IU of C1 Esterase Inhibitor [Human] Liquid for Injection for the Prevention of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema', 'orgStudyIdInfo': {'id': 'SHP616-300'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Experimental/Placebo', 'description': 'Subjects will be randomized to receive C1 Esterase Inhibitor in the 1st Treatment period and then switch to Placebo in the 2nd treatment period.', 'interventionNames': ['Drug: C1 esterase inhibitor [human] liquid', 'Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'Placebo/Experimental', 'description': 'Subjects will be randomized to receive a placebo treatment in the 1st Treatment period and then switch to receive C1 Esterase Inhibitor in the 2nd treatment period.', 'interventionNames': ['Drug: C1 esterase inhibitor [human] liquid', 'Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'Experimental/ Experimental', 'description': 'Subjects will be randomized and receive C1 Esterase Inhibitor in both 1st as well as the 2nd treatment period', 'interventionNames': ['Drug: C1 esterase inhibitor [human] liquid']}], 'interventions': [{'name': 'C1 esterase inhibitor [human] liquid', 'type': 'DRUG', 'description': 'C1 Esterase Inhibitor \\[Human\\] Liquid administered Subcutaneously as specified on specified days', 'armGroupLabels': ['Experimental/ Experimental', 'Experimental/Placebo', 'Placebo/Experimental']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Placebo', 'armGroupLabels': ['Experimental/Placebo', 'Placebo/Experimental']}]}, 'contactsLocationsModule': {'locations': [{'zip': '85251', 'city': 'Scottsdale', 'state': 'Arizona', 'country': 'United States', 'facility': 'Medical Research of Arizona', 'geoPoint': {'lat': 33.50921, 'lon': -111.89903}}, {'zip': '90404', 'city': 'Santa Monica', 'state': 'California', 'country': 'United States', 'facility': 'AIRE Medical of Los Angeles', 'geoPoint': {'lat': 34.01949, 'lon': -118.49138}}, {'zip': '94598', 'city': 'Walnut Creek', 'state': 'California', 'country': 'United States', 'facility': 'Bay Area Allergy', 'geoPoint': {'lat': 37.90631, 'lon': -122.06496}}, {'zip': '80112', 'city': 'Centennial', 'state': 'Colorado', 'country': 'United States', 'facility': 'IMMUNOe Research Centers', 'geoPoint': {'lat': 39.57916, 'lon': -104.87692}}, {'zip': '80907', 'city': 'Colorado Springs', 'state': 'Colorado', 'country': 'United States', 'facility': 'Asthma and Allergy Associates PC', 'geoPoint': {'lat': 38.83388, 'lon': -104.82136}}, {'zip': '30060', 'city': 'Marietta', 'state': 'Georgia', 'country': 'United States', 'facility': 'Atlanta Allergy and Asthma Clinic', 'geoPoint': {'lat': 33.9526, 'lon': -84.54993}}, {'zip': '20815', 'city': 'Chevy Chase', 'state': 'Maryland', 'country': 'United States', 'facility': 'Institute For Asthma and Allergy', 'geoPoint': {'lat': 39.00287, 'lon': -77.07115}}, {'zip': '63141', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Washington University', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '07410', 'city': 'Fair Lawn', 'state': 'New Jersey', 'country': 'United States', 'facility': 'Allergy Asthma and Immunology', 'geoPoint': {'lat': 40.94038, 'lon': -74.13181}}, {'zip': '08830', 'city': 'Iselin', 'state': 'New Jersey', 'country': 'United States', 'facility': 'Allergy Treatment Center of New Jersey', 'geoPoint': {'lat': 40.57538, 'lon': -74.32237}}, {'zip': '28277', 'city': 'Charlotte', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Clinical Research of Charlotte', 'geoPoint': {'lat': 35.22709, 'lon': -80.84313}}, {'zip': '45231', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': 'Bernstein Clinical Research Center Inc', 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '44130', 'city': 'Middleburg Heights', 'state': 'Ohio', 'country': 'United States', 'facility': 'Clinical Research Solutions', 'geoPoint': {'lat': 41.36144, 'lon': -81.81291}}, {'zip': '74133', 'city': 'Tulsa', 'state': 'Oklahoma', 'country': 'United States', 'facility': 'Allergy Clinic of Tulsa', 'geoPoint': {'lat': 36.15398, 'lon': -95.99277}}, {'zip': '75231', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': 'AARA Research Center', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '99202', 'city': 'Spokane', 'state': 'Washington', 'country': 'United States', 'facility': 'Premier Clinical Research', 'geoPoint': {'lat': 47.65966, 'lon': -117.42908}}, {'zip': 'L8N 325', 'city': 'Hamilton', 'state': 'Ontario', 'country': 'Canada', 'facility': 'McMaster University Medical Center', 'geoPoint': {'lat': 43.25011, 'lon': -79.84963}}, {'zip': 'K1Y 4G2', 'city': 'Ottawa', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Ottawa Allergy Research Corporation', 'geoPoint': {'lat': 45.41117, 'lon': -75.69812}}, {'zip': '64546', 'city': 'Mörfelden-Walldorf', 'country': 'Germany', 'facility': 'Hämophilie Zentrum Rhein Main GmbH', 'geoPoint': {'lat': 49.99472, 'lon': 8.58361}}, {'zip': '1125', 'city': 'Budapest', 'country': 'Hungary', 'facility': 'Semmelweis Egyetem', 'geoPoint': {'lat': 47.49835, 'lon': 19.04045}}, {'zip': '52621', 'city': 'Ramat Gan', 'country': 'Israel', 'facility': 'Chaim Sheba Medical Center', 'geoPoint': {'lat': 32.08227, 'lon': 34.81065}}, {'zip': '64239', 'city': 'Tel Aviv', 'country': 'Israel', 'facility': 'Tel Aviv Sourasky Medical Center', 'geoPoint': {'lat': 32.08088, 'lon': 34.78057}}, {'zip': '540072', 'city': 'Târgu Mureş', 'country': 'Romania', 'facility': 'MediQuest Clinical Research Center', 'geoPoint': {'lat': 46.54245, 'lon': 24.55747}}, {'zip': '8035', 'city': 'Barcelona', 'country': 'Spain', 'facility': "Hospital Universitario Vall d'Hebron", 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'zip': '28046', 'city': 'Madrid', 'country': 'Spain', 'facility': 'Hospital Universitario La Paz', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}, {'zip': '41013', 'city': 'Seville', 'country': 'Spain', 'facility': 'Hospital Universitario Virgen del Rocio', 'geoPoint': {'lat': 37.38283, 'lon': -5.97317}}, {'zip': '46026', 'city': 'Valencia', 'country': 'Spain', 'facility': 'Hospital Universitari i Politecnic La Fe de Valencia', 'geoPoint': {'lat': 39.47391, 'lon': -0.37966}}], 'overallOfficials': [{'name': 'Study Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Takeda'}]}, 'ipdSharingStatementModule': {'url': 'https://vivli.org/ourmember/takeda/', 'infoTypes': ['STUDY_PROTOCOL', 'SAP', 'ICF', 'CSR'], 'ipdSharing': 'YES', 'description': "Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.", 'accessCriteria': 'IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Shire', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}