Ignite Creation Date:
2025-12-25 @ 2:54 AM
Ignite Modification Date:
2026-01-09 @ 10:39 AM
Study NCT ID:
NCT03240133
Status:
COMPLETED
Last Update Posted:
2021-04-01
First Post:
2017-08-02
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Study of BCX7353 as a Treatment for Attacks of 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': 'C000706836', 'term': 'berotralstat'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'clinicaltrials@biocryst.com', 'phone': '+1 919-859-1302', 'title': 'Study Director', 'organization': 'BioCryst Pharmaceuticals Inc'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}, 'limitationsAndCaveats': {'description': 'In this interim clinical study report (CSR), the VAS change from pre-dose at 4 hours and the proportion of attacks requiring SOC-Rx through the 24 hour post dose were discussed. Assessment of other efficacy endpoints were to be discussed in a final CSR, but this is no longer planned due to a decision to not proceed further with trials of acute treatment of HAE attacks with berotralstat.'}}, 'adverseEventsModule': {'timeFrame': 'Adverse events (AEs) were collected from informed consent signature until 16 to 19 days after the 3rd or final HAE attack treated with IMP. As the HAE attack frequency varied between subjects, subject duration of study participation and therefore the duration over which AEs were collected was dependent on attack frequency and therefore variable. The maximum period over which AEs were collected was 281 days.', 'description': 'AEs were assigned to an attack treated with placebo or berotralstat depending on which IMP was used most recently prior to AE onset. HAE attacks and their associated symptoms were not defined as AEs, unless they met the criteria for an SAE.', 'eventGroups': [{'id': 'EG000', 'title': 'Part1: Berotralstat 750 mg', 'description': 'Berotralstat : oral liquid formulation', 'otherNumAtRisk': 33, 'deathsNumAtRisk': 33, 'otherNumAffected': 12, 'seriousNumAtRisk': 33, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Part 2: Berotralstat 500 mg', 'description': 'Berotralstat: oral liquid formulation', 'otherNumAtRisk': 14, 'deathsNumAtRisk': 14, 'otherNumAffected': 8, 'seriousNumAtRisk': 14, 'deathsNumAffected': 0, 'seriousNumAffected': 1}, {'id': 'EG002', 'title': 'Part 3: Berotralstat 250 mg', 'description': 'Berotralstat: oral liquid formulation', 'otherNumAtRisk': 11, 'deathsNumAtRisk': 11, 'otherNumAffected': 7, 'seriousNumAtRisk': 11, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG003', 'title': 'Parts 1, 2 and 3: Placebo', 'description': 'Placebo: oral liquid formulation to match Berotralstat', 'otherNumAtRisk': 53, 'deathsNumAtRisk': 53, 'otherNumAffected': 17, 'seriousNumAtRisk': 53, 'deathsNumAffected': 0, 'seriousNumAffected': 1}], 'otherEvents': [{'term': 'Arthropod bite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Limb injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Dysplastic naevus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Dysgeusia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Vessel puncture site reaction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Abdominal discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Faeces discoloured', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Epigastric discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Gastrooesophageal reflux disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Muscle contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Blood creatine phosphokinase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Cholelithiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}], 'seriousEvents': [{'term': 'kidney contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Ankle fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}, {'term': 'Ligament sprain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 53, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.0)'}], 'frequencyThreshold': '2'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Proportion of Subjects With Improved or Stable Composite Visual Analog Scale (VAS) Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '28', 'groupId': 'OG001'}, {'value': '33', 'groupId': 'OG002'}, {'value': '28', 'groupId': 'OG003'}, {'value': '14', 'groupId': 'OG004'}, {'value': '9', 'groupId': 'OG005'}, {'value': '14', 'groupId': 'OG006'}, {'value': '9', 'groupId': 'OG007'}, {'value': '11', 'groupId': 'OG008'}, {'value': '11', 'groupId': 'OG009'}, {'value': '11', 'groupId': 'OG010'}, {'value': '11', 'groupId': 'OG011'}]}, {'units': 'HAE Attacks', 'counts': [{'value': '59', 'groupId': 'OG000'}, {'value': '28', 'groupId': 'OG001'}, {'value': '59', 'groupId': 'OG002'}, {'value': '28', 'groupId': 'OG003'}, {'value': '24', 'groupId': 'OG004'}, {'value': '9', 'groupId': 'OG005'}, {'value': '24', 'groupId': 'OG006'}, {'value': '9', 'groupId': 'OG007'}, {'value': '21', 'groupId': 'OG008'}, {'value': '11', 'groupId': 'OG009'}, {'value': '21', 'groupId': 'OG010'}, {'value': '11', 'groupId': 'OG011'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 1: Berotralstat 750 mg - Predose', 'description': 'HAE attack prior to dosing with 750mg berotralstat'}, {'id': 'OG001', 'title': 'Part 1: Placebo - Pre-dose', 'description': 'HAE attack prior to dosing with placebo'}, {'id': 'OG002', 'title': 'Part 1: Berotralstat 750 mg - 4hr Post-dose', 'description': 'HAE attack 4 hr post dosing with 750mg berotralstat'}, {'id': 'OG003', 'title': 'Part 1: Placebo - 4hr Post-dose', 'description': 'HAE attack 4 hr post dosing with placebo'}, {'id': 'OG004', 'title': 'Part 2: Berotralstat 500 mg - Predose', 'description': 'HAE attack prior to dosing with 500mg berotralstat'}, {'id': 'OG005', 'title': 'Part 2: Placebo - Pre-dose', 'description': 'HAE attack prior to dosing with placebo'}, {'id': 'OG006', 'title': 'Part 2: Berotralstat 500 mg - 4hr Post-dose', 'description': 'HAE attack 4 hr post dosing with 50mg berotralstat'}, {'id': 'OG007', 'title': 'Part 2: Placebo - 4hr Post-dose', 'description': 'HAE attack 4 hr post dosing with placebo'}, {'id': 'OG008', 'title': 'Part 3: Berotralstat 250 mg - Predose', 'description': 'HAE attack prior to dosing with 250mg berotralstat'}, {'id': 'OG009', 'title': 'Part 3: Placebo - Pre-dose', 'description': 'HAE attack prior to dosing with placebo'}, {'id': 'OG010', 'title': 'Part 3: Berotralstat 250 mg - 4hr Post-dose', 'description': 'HAE attack 4 hr post dosing with 250mg berotralstat'}, {'id': 'OG011', 'title': 'Part 3: Placebo - 4hr Post-dose', 'description': 'HAE attack 4 hr post dosing with placebo'}], 'classes': [{'categories': [{'measurements': [{'value': '13.96', 'spread': '9.84', 'groupId': 'OG000'}, {'value': '15.04', 'spread': '11.90', 'groupId': 'OG001'}, {'value': '10.54', 'spread': '11.39', 'groupId': 'OG002'}, {'value': '18.42', 'spread': '14.19', 'groupId': 'OG003'}, {'value': '17.69', 'spread': '15.25', 'groupId': 'OG004'}, {'value': '13.48', 'spread': '16.11', 'groupId': 'OG005'}, {'value': '11.31', 'spread': '15.75', 'groupId': 'OG006'}, {'value': '9.26', 'spread': '11.53', 'groupId': 'OG007'}, {'value': '14.57', 'spread': '11.78', 'groupId': 'OG008'}, {'value': '11.33', 'spread': '10.17', 'groupId': 'OG009'}, {'value': '10.92', 'spread': '10.31', 'groupId': 'OG010'}, {'value': '9.21', 'spread': '9.67', 'groupId': 'OG011'}]}]}], 'analyses': [{'pValue': '0.0024', 'groupIds': ['OG000', 'OG001', 'OG002', 'OG003'], 'paramType': 'Difference in Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-6.98', 'ciLowerLimit': '-11.37', 'ciUpperLimit': '-2.6', 'groupDescription': 'Change in HAE attack symptoms from pre-dose to 4 hours post-dose was assessed following treatment with either 750mg berotralstat or placebo.', 'statisticalMethod': 'Mixed effect linear model', 'nonInferiorityType': 'SUPERIORITY', 'nonInferiorityComment': 'Comparisons were performed separately at each time point using a mixed effect linear model including treatment, period and sequence as fixed effects, subject within sequence as a random effect, and predose 3-symptom composite VAS score as a covariate.'}, {'pValue': '0.6424', 'groupIds': ['OG004', 'OG005', 'OG006', 'OG007'], 'paramType': 'Difference in Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-2.1', 'ciLowerLimit': '-11.49', 'ciUpperLimit': '7.29', 'groupDescription': 'Change in HAE attack symptoms from pre-dose to 4 hours post-dose was assessed following treatment with either 500mg berotralstat or placebo.', 'statisticalMethod': 'Mixed effect linear model', 'nonInferiorityType': 'SUPERIORITY', 'nonInferiorityComment': 'Comparisons were performed separately at each time point using a mixed effect linear model including treatment, period and sequence as fixed effects, subject within sequence as a random effect, and predose 3-symptom composite VAS score as a covariate.'}, {'pValue': '0.8283', 'groupIds': ['OG008', 'OG009', 'OG010', 'OG011'], 'paramType': 'Difference in Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.57', 'ciLowerLimit': '-4.9', 'ciUpperLimit': '6.03', 'groupDescription': 'Change in HAE attack symptoms from pre-dose to 4 hours post-dose was assessed following treatment with either 250mg berotralstat or placebo.', 'statisticalMethod': 'Mixed effect linear model', 'nonInferiorityType': 'SUPERIORITY', 'nonInferiorityComment': 'Comparisons were performed separately at each time point using a mixed effect linear model including treatment, period and sequence as fixed effects, subject within sequence as a random effect, and predose 3-symptom composite VAS score as a covariate.'}], 'paramType': 'MEAN', 'timeFrame': 'Mean composite VAS for HAE attack symptoms severity prior to IMP treatment and 4 hours post-dose', 'description': 'Subjects completed a 3-component VAS on a 100 mm scale for severity of abdominal pain, skin pain and skin swelling associated with the HAE attack, where zero indicated no pain or swelling and 100 mm indicated worst possible pain or swelling. Subjects completed the VAS immediately prior to study drug administration, then at 1, 2, 3, 4, approximately 8 \\& at 24 hours post-dose. The primary endpoint was the proportion of subject attacks with an improved or stable 3-symptom composite VAS score at 4 hours post dose. The 3-symptom composite was calculated as the average of the VAS scores for abdominal pain, skin pain, and skin swelling. A subject was considered improved or stable if the change from baseline (CFB; time of drug administration) in VAS was ≤ 0.', 'unitOfMeasure': 'VAS score - millimeters', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'HAE Attacks', 'denomUnitsSelected': 'HAE Attacks', 'populationDescription': 'The number of participants analyzed in each analysis set corresponds to number of HAE attacks treated with placebo or berotralstat.'}, {'type': 'PRIMARY', 'title': 'Percentage of Attacks Treated With Standard of Care Acute Attack Medication (SOC-Rx) Through 24 Hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '28', 'groupId': 'OG001'}, {'value': '14', 'groupId': 'OG002'}, {'value': '9', 'groupId': 'OG003'}, {'value': '11', 'groupId': 'OG004'}, {'value': '11', 'groupId': 'OG005'}]}, {'units': 'HAE Attacks', 'counts': [{'value': '59', 'groupId': 'OG000'}, {'value': '28', 'groupId': 'OG001'}, {'value': '24', 'groupId': 'OG002'}, {'value': '9', 'groupId': 'OG003'}, {'value': '21', 'groupId': 'OG004'}, {'value': '11', 'groupId': 'OG005'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 1: Berotralstat 750 mg', 'description': 'HAE attack treated with 750mg berotralstat'}, {'id': 'OG001', 'title': 'Part 1: Placebo', 'description': 'HAE attack treated with placebo'}, {'id': 'OG002', 'title': 'Part 2: Berotralstat 500 mg', 'description': 'HAE attack treated with 500mg berotralstat'}, {'id': 'OG003', 'title': 'Part 2: Placebo', 'description': 'HAE attack treated with placebo'}, {'id': 'OG004', 'title': 'Part 3: Berotralstat 250 mg', 'description': 'HAE attack treated with 250mg berotralstat'}, {'id': 'OG005', 'title': 'Part 3: Placebo', 'description': 'HAE attack treated with placebo'}], 'classes': [{'categories': [{'measurements': [{'value': '29.7', 'groupId': 'OG000'}, {'value': '61.3', 'groupId': 'OG001'}, {'value': '32.0', 'groupId': 'OG002'}, {'value': '45.5', 'groupId': 'OG003'}, {'value': '38.1', 'groupId': 'OG004'}, {'value': '45.5', 'groupId': 'OG005'}]}]}], 'analyses': [{'pValue': '0.0029', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Odds Ratio (OR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.196', 'ciLowerLimit': '0.069', 'ciUpperLimit': '0.559', 'groupDescription': 'Analyses were performed using a generalized logistic model including treatment, period and sequence as fixed effects, and subject within sequence as a random effect. The odds of a berotralstat 750 mg-treated-attack requiring SOC-Rx was 0.196 that of a placebo attack.', 'statisticalMethod': 'logistic mixed effect model', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.4048', 'groupIds': ['OG002', 'OG003'], 'paramType': 'Odds Ratio (OR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.472', 'ciLowerLimit': '0.074', 'ciUpperLimit': '2.988', 'groupDescription': 'Analyses were performed using a generalized logistic model including treatment, period and sequence as fixed effects, and subject within sequence as a random effect. The odds of a berotralstat 500 mg-treated-attack requiring SOC-Rx was 0.472 that of a placebo attack.', 'statisticalMethod': 'logistic mixed effect model', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.5984', 'groupIds': ['OG004', 'OG005'], 'paramType': 'Odds Ratio (OR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.587', 'ciLowerLimit': '0.073', 'ciUpperLimit': '4.733', 'groupDescription': 'Analyses were performed using a generalized logistic model including treatment, period and sequence as fixed effects, and subject within sequence as a random effect. The odds of a berotralstat 250 mg-treated-attack requiring SOC-Rx was 0.587 that of a placebo attack.', 'statisticalMethod': 'logistic mixed effect model', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'NUMBER', 'timeFrame': '24 hours', 'description': 'The proportion of attacks for which subjects took SOC-Rx in the 24 hours following treatment with study drug. HAE Rescue Medications included C1-INH (Berinert, Cinryze, Ruconest) and Firazyr/Icatibant.', 'unitOfMeasure': 'Percentage attacks treated with SOC-Rx', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'HAE Attacks', 'denomUnitsSelected': 'HAE Attacks'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Part 1: Berotralstat (750 mg) & Placebo Treated HAE Attacks', 'description': 'Each subject treated 3 HAE attacks, 1 with placebo and 2 with 750 mg berotralstat. Placebo vs berotralstat treatment of an HAE attack was assigned by randomization.'}, {'id': 'FG001', 'title': 'Part 2: Berotralstat (500 mg) & Placebo Treated HAE Attacks', 'description': 'Each subject treated 3 HAE attacks, 1 with placebo and 2 with 500 mg berotralstat. Placebo vs berotralstat treatment of an HAE attack was assigned by randomization.'}, {'id': 'FG002', 'title': 'Part 3: Berotralstat (250 mg) & Placebo Treated HAE Attacks', 'description': 'Each subject treated 3 HAE attacks, 1 with placebo and 2 with 250 mg berotralstat. Placebo vs berotralstat treatment of an HAE attack was assigned by randomization.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '33'}, {'groupId': 'FG001', 'numSubjects': '14'}, {'groupId': 'FG002', 'numSubjects': '11'}]}, {'type': 'Completed Period 1', 'achievements': [{'groupId': 'FG000', 'numSubjects': '33'}, {'groupId': 'FG001', 'numSubjects': '14'}, {'groupId': 'FG002', 'numSubjects': '11'}]}, {'type': 'Completed Period 2', 'achievements': [{'groupId': 'FG000', 'numSubjects': '32'}, {'groupId': 'FG001', 'numSubjects': '11'}, {'groupId': 'FG002', 'numSubjects': '11'}]}, {'type': 'Completed Period 3', 'achievements': [{'groupId': 'FG000', 'numSubjects': '30'}, {'groupId': 'FG001', 'numSubjects': '11'}, {'groupId': 'FG002', 'numSubjects': '11'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '30'}, {'groupId': 'FG001', 'numSubjects': '11'}, {'groupId': 'FG002', 'numSubjects': '10'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '3'}, {'groupId': 'FG002', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '1'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'Rash', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'sponsor closure/completion of Part 1 of the study', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'Intercurrent illness.', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'Sponsor/competent authority/IRB/IEC discontinuation', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '1'}]}]}], 'preAssignmentDetails': 'HAE subjects attended a Screening Visit up to 35 days before the baseline visit, for assessment of eligibility to participate in the study.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'BG000'}, {'value': '14', 'groupId': 'BG001'}, {'value': '11', 'groupId': 'BG002'}, {'value': '58', 'groupId': 'BG003'}]}], 'groups': [{'id': 'BG000', 'title': 'Part 1: Berotralstat (750 mg) & Placebo Treated HAE Attacks', 'description': 'Each subject treated 3 HAE attacks, 1 with placebo and 2 with 750 mg berotralstat. Placebo vs berotralstat treatment of an HAE attack was assigned by randomization.'}, {'id': 'BG001', 'title': 'Part 2: Berotralstat (500 mg) & Placebo Treated HAE Attacks', 'description': 'Each subject treated 3 HAE attacks, 1 with placebo and 2 with 500 mg berotralstat. Placebo vs berotralstat treatment of an HAE attack was assigned by randomization.'}, {'id': 'BG002', 'title': 'Part 3: Berotralstat (250 mg) & Placebo Treated HAE Attacks', 'description': 'Each subject treated 3 HAE attacks, 1 with placebo and 2 with 250 mg berotralstat. Placebo vs berotralstat treatment of an HAE attack was assigned by randomization.'}, {'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': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '32', 'groupId': 'BG000'}, {'value': '14', 'groupId': 'BG001'}, {'value': '11', 'groupId': 'BG002'}, {'value': '57', 'groupId': 'BG003'}]}, {'title': '>=65 years', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '43.7', 'spread': '12.5', 'groupId': 'BG000'}, {'value': '42.1', 'spread': '10.5', 'groupId': 'BG001'}, {'value': '34.9', 'spread': '10.9', 'groupId': 'BG002'}, {'value': '41.6', 'spread': '12.0', 'groupId': 'BG003'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '17', 'groupId': 'BG000'}, {'value': '11', 'groupId': 'BG001'}, {'value': '7', 'groupId': 'BG002'}, {'value': '35', 'groupId': 'BG003'}]}, {'title': 'Male', 'measurements': [{'value': '16', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '4', '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': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', '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': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'White', 'measurements': [{'value': '33', 'groupId': 'BG000'}, {'value': '14', 'groupId': 'BG001'}, {'value': '11', 'groupId': 'BG002'}, {'value': '58', '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'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'Austria', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}]}]}, {'title': 'Romania', 'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '4', 'groupId': 'BG003'}]}]}, {'title': 'Hungary', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '2', 'groupId': 'BG003'}]}]}, {'title': 'Denmark', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '3', 'groupId': 'BG003'}]}]}, {'title': 'North Macedonia', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '2', 'groupId': 'BG003'}]}]}, {'title': 'Poland', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '6', 'groupId': 'BG003'}]}]}, {'title': 'Italy', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '9', 'groupId': 'BG003'}]}]}, {'title': 'United Kingdom', 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '5', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '13', 'groupId': 'BG003'}]}]}, {'title': 'Israel', 'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '6', 'groupId': 'BG003'}]}]}, {'title': 'France', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}]}]}, {'title': 'Switzerland', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '2', 'groupId': 'BG003'}]}]}, {'title': 'Germany', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}, {'value': '9', 'groupId': 'BG003'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Adjusted qualifying HAE attack rate', 'classes': [{'categories': [{'measurements': [{'value': '3.08', 'spread': '1.40', 'groupId': 'BG000'}, {'value': '3.03', 'spread': '1.23', 'groupId': 'BG001'}, {'value': '3.23', 'spread': '1.82', 'groupId': 'BG002'}, {'value': '3.09', 'spread': '1.42', 'groupId': 'BG003'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'HAE attacks/month', 'dispersionType': 'STANDARD_DEVIATION'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2019-01-29', 'size': 2240450, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_000.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2020-10-23T07:16', 'hasProtocol': False}, {'date': '2018-03-16', 'size': 976825, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_001.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2021-02-10T07:35', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'DOUBLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'CROSSOVER'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 58}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2017-07-31', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-03', 'completionDateStruct': {'date': '2019-01-29', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-03-08', 'studyFirstSubmitDate': '2017-08-02', 'resultsFirstSubmitDate': '2020-11-17', 'studyFirstSubmitQcDate': '2017-08-03', 'lastUpdatePostDateStruct': {'date': '2021-04-01', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2021-03-08', 'studyFirstPostDateStruct': {'date': '2017-08-04', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2021-04-01', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-01-29', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Proportion of Subjects With Improved or Stable Composite Visual Analog Scale (VAS) Score', 'timeFrame': 'Mean composite VAS for HAE attack symptoms severity prior to IMP treatment and 4 hours post-dose', 'description': 'Subjects completed a 3-component VAS on a 100 mm scale for severity of abdominal pain, skin pain and skin swelling associated with the HAE attack, where zero indicated no pain or swelling and 100 mm indicated worst possible pain or swelling. Subjects completed the VAS immediately prior to study drug administration, then at 1, 2, 3, 4, approximately 8 \\& at 24 hours post-dose. The primary endpoint was the proportion of subject attacks with an improved or stable 3-symptom composite VAS score at 4 hours post dose. The 3-symptom composite was calculated as the average of the VAS scores for abdominal pain, skin pain, and skin swelling. A subject was considered improved or stable if the change from baseline (CFB; time of drug administration) in VAS was ≤ 0.'}, {'measure': 'Percentage of Attacks Treated With Standard of Care Acute Attack Medication (SOC-Rx) Through 24 Hours', 'timeFrame': '24 hours', 'description': 'The proportion of attacks for which subjects took SOC-Rx in the 24 hours following treatment with study drug. HAE Rescue Medications included C1-INH (Berinert, Cinryze, Ruconest) and Firazyr/Icatibant.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['HAE', 'hereditary angioedema', 'kallikrein inhibitor', 'plasma kallikrein'], 'conditions': ['Hereditary Angioedema (HAE)']}, 'referencesModule': {'references': [{'pmid': '36408587', 'type': 'DERIVED', 'citation': 'Farkas H, Balla Z. A review of berotralstat for the treatment of hereditary angioedema. Expert Rev Clin Immunol. 2023 Feb;19(2):145-153. doi: 10.1080/1744666X.2023.2150611. Epub 2022 Nov 29.'}]}, 'descriptionModule': {'briefSummary': 'This 3-part study will evaluate the efficacy and safety of an oral kallikrein inhibitor, BCX7353, in the treatment angioedema attacks in subjects with Type I or II hereditary angioedema (HAE). In each study part, subjects will treat 3 attacks with BCX7353 (2 attacks) or placebo (1 attack), in a randomly allocated order. In Part 1, the dose of 750mg will be assessed relative to placebo in up to 36 patients. If this is shown to be effective, then a further 12 patients will be enrolled at a 500mg dose (Part 1), followed by a further 12 (if efficacy still shown) at a dose of 250mg (Part 3) to determine the minimum effective dose of BCX7353 compared to placebo for treating HAE attacks. Efficacy will be determined by subject diary entries completed at pre-defined times post-dose.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '70 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n1. Able to provide written, informed consent.\n2. A clinical diagnosis of hereditary angioedema Type 1 or Type 2 as documented at any time in the medical records or at the screening visit.\n3. Access to and ability to use standard of care acute attack treatment for attacks of HAE.\n4. Sexually active women of child-bearing potential and sexually active men must utilize effective contraception.\n\nExclusion Criteria:\n\n1. Women who are pregnant or breast-feeding.\n2. Any clinical condition or medical history that would interfere with the subject's safety or ability to participate in the study.\n3. Use of C1INH, androgens or tranexamic acid for prophylaxis of HAE attacks.\n4. History of or current alcohol or drug abuse.\n5. Infection with hepatitis B, hepatitis C or HIV.\n6. Participation in any other investigational drug study currently or within the last 30 days.\n7. Positive drugs of abuse screen (unless as used as medical treatment, e.g., with a prescription).\n8. An immediate family relationship to either Sponsor employees, the Investigator or employees of the study site."}, 'identificationModule': {'nctId': 'NCT03240133', 'briefTitle': 'Study of BCX7353 as a Treatment for Attacks of Hereditary Angioedema', 'organization': {'class': 'INDUSTRY', 'fullName': 'BioCryst Pharmaceuticals'}, 'officialTitle': 'A Randomized, Double-blind, Placebo-controlled, Dose-ranging, Study to Evaluate the Efficacy, Safety and Tolerability of Single Doses of BCX7353 as an Acute Attack Treatment in Subjects With Hereditary Angioedema', 'orgStudyIdInfo': {'id': 'BCX7353-202'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Part1: BCX7353 750 mg', 'interventionNames': ['Drug: BCX7353']}, {'type': 'EXPERIMENTAL', 'label': 'Part 2: BCX7353 500 mg', 'interventionNames': ['Drug: BCX7353']}, {'type': 'EXPERIMENTAL', 'label': 'Part 3: BCX7353 250 mg', 'interventionNames': ['Drug: BCX7353']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Parts 1, 2 and 3: placebo', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'BCX7353', 'type': 'DRUG', 'description': 'oral liquid formulation', 'armGroupLabels': ['Part 2: BCX7353 500 mg', 'Part 3: BCX7353 250 mg', 'Part1: BCX7353 750 mg']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'oral liquid formulation to match BCX7353', 'armGroupLabels': ['Parts 1, 2 and 3: placebo']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Graz', 'country': 'Austria', 'facility': 'Study Center', 'geoPoint': {'lat': 47.06733, 'lon': 15.44197}}, {'city': 'Odense', 'country': 'Denmark', 'facility': 'Study Center', 'geoPoint': {'lat': 55.39594, 'lon': 10.38831}}, {'city': 'Grenoble', 'country': 'France', 'facility': 'Study Center', 'geoPoint': {'lat': 45.17869, 'lon': 5.71479}}, {'city': 'Lille', 'country': 'France', 'facility': 'Study Center', 'geoPoint': {'lat': 50.63391, 'lon': 3.05512}}, {'city': 'Berlin', 'country': 'Germany', 'facility': 'Study Center', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}, {'city': 'Frankfurt', 'country': 'Germany', 'facility': 'Study Center', 'geoPoint': {'lat': 49.68333, 'lon': 10.53333}}, {'city': 'Budapest', 'country': 'Hungary', 'facility': 'Study Center', 'geoPoint': {'lat': 47.49835, 'lon': 19.04045}}, {'city': 'Ashkelon', 'country': 'Israel', 'facility': 'Study Center', 'geoPoint': {'lat': 31.66926, 'lon': 34.57149}}, {'city': 'Tel Aviv', 'country': 'Israel', 'facility': 'Study Center', 'geoPoint': {'lat': 32.08088, 'lon': 34.78057}}, {'city': 'Tel Litwinsky', 'country': 'Israel', 'facility': 'Study Center', 'geoPoint': {'lat': 32.05096, 'lon': 34.84588}}, {'city': 'Milan', 'country': 'Italy', 'facility': 'Study Center', 'geoPoint': {'lat': 42.78235, 'lon': 12.59836}}, {'city': 'Padua', 'country': 'Italy', 'facility': 'Study center', 'geoPoint': {'lat': 45.40797, 'lon': 11.88586}}, {'city': 'Salerno', 'country': 'Italy', 'facility': 'Study Center', 'geoPoint': {'lat': 40.67545, 'lon': 14.79328}}, {'city': 'Skopje', 'country': 'North Macedonia', 'facility': 'Study Center', 'geoPoint': {'lat': 41.99646, 'lon': 21.43141}}, {'city': 'Krakow', 'country': 'Poland', 'facility': 'Study Center', 'geoPoint': {'lat': 50.06143, 'lon': 19.93658}}, {'city': 'Târgu Mureş', 'country': 'Romania', 'facility': 'Study Center', 'geoPoint': {'lat': 46.54245, 'lon': 24.55747}}, {'city': 'Zurich', 'country': 'Switzerland', 'facility': 'Study Center', 'geoPoint': {'lat': 47.36667, 'lon': 8.55}}, {'city': 'Birmingham', 'country': 'United Kingdom', 'facility': 'Study Center', 'geoPoint': {'lat': 52.48142, 'lon': -1.89983}}, {'city': 'Bristol', 'country': 'United Kingdom', 'facility': 'Study Center', 'geoPoint': {'lat': 51.45523, 'lon': -2.59665}}, {'city': 'Cambridge', 'country': 'United Kingdom', 'facility': 'Study Center', 'geoPoint': {'lat': 52.2, 'lon': 0.11667}}, {'city': 'London', 'country': 'United Kingdom', 'facility': 'Study Center', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'city': 'Manchester', 'country': 'United Kingdom', 'facility': 'Study Center', 'geoPoint': {'lat': 53.48095, 'lon': -2.23743}}, {'city': 'Plymouth', 'country': 'United Kingdom', 'facility': 'Study Center', 'geoPoint': {'lat': 50.37153, 'lon': -4.14305}}, {'city': 'Southampton', 'country': 'United Kingdom', 'facility': 'Study Center', 'geoPoint': {'lat': 50.90395, 'lon': -1.40428}}], 'overallOfficials': [{'name': 'Hilary Longhurst, MBBS, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Barts & The London NHS Trust'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'BioCryst Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}