Ignite Creation Date:
2025-12-25 @ 4:27 AM
Ignite Modification Date:
2026-02-24 @ 5:03 PM
Study NCT ID:
NCT02670720
Status:
TERMINATED
Last Update Posted:
2016-02-17
First Post:
2016-01-20
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Open-label, Long-term Safety Study of Avoralstat in Subjects With Hereditary Angioedema
Sponsor:
Organization:
Raw JSON
{'hasResults': False, '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': 'C000722673', 'term': 'avoralstat'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'PREVENTION', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 6}}, 'statusModule': {'whyStopped': 'preceding efficacy study of avoralstat in its current formulation was not significant and does not support continued development', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2015-12'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2016-02', 'completionDateStruct': {'date': '2016-02', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2016-02-15', 'studyFirstSubmitDate': '2016-01-20', 'studyFirstSubmitQcDate': '2016-01-28', 'lastUpdatePostDateStruct': {'date': '2016-02-17', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2016-02-02', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2016-02', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Proportion of subjects who discontinue due to a treatment-emergent adverse event, who experience a treatment-emergent serious AE, who experience a Grade 3 or 4 AE and who experience Grade 3 or 4 laboratory abnormalities', 'timeFrame': 'Up to 72 weeks'}], 'secondaryOutcomes': [{'measure': 'Angioedema attack rate (subject-reported HAE attacks normalized for time on study)', 'timeFrame': 'Up to 72 weeks'}, {'measure': 'Durability in response (assessed as subject-reported HAE attacks)', 'timeFrame': 'change over time through 72 weeks'}, {'measure': 'Proportion of subjects who discontinue avoralstat with a reason of lack of efficacy', 'timeFrame': 'Up to 72 weeks'}, {'measure': 'Quality of life as determined by the EQ-5D-5L', 'timeFrame': 'Up to 72 weeks'}, {'measure': 'Quality of life as determined by the Angioedema Quality of Life Questionnaire', 'timeFrame': 'Up to 72 weeks'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['BCX4161', 'avoralstat', 'HAE', 'hereditary angioedema', 'prophylaxis'], 'conditions': ['Hereditary Angioedema', 'HAE']}, 'descriptionModule': {'briefSummary': 'This is an open-label study designed to evaluate the long-term safety of prophylactic avoralstat (500 mg three times daily) when given to approximately 150 patients with hereditary angioedema (HAE) for a duration of up to 72 weeks. The study will also evaluate the long-term efficacy and impact on quality of life of avoralstat prophylactic treatment.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Males and non-pregnant, non-lactating females age ≥ 18 years.\n* Provide written, informed consent.\n* Prior completion of an avoralstat therapeutic study OR a confirmed diagnosis of HAE Type 1 or HAE Type 2.\n* Access to appropriate medication for the treatment of acute HAE attacks.\n* Adequate contraception.\n\nExclusion Criteria:\n\n* Females who are pregnant or breast feeding.\n* Clinically significant medical condition or medical history.\n* Abnormal screening ECG, laboratory or urinalysis finding that is clinically significant.\n* Investigational drug exposure within 30 days (except avoralstat).\n* History of or current alcohol or drug abuse.\n* HIV or active HBV or HCV infection.'}, 'identificationModule': {'nctId': 'NCT02670720', 'acronym': 'OPuS-4', 'briefTitle': 'Open-label, Long-term Safety Study of Avoralstat in Subjects With Hereditary Angioedema', 'organization': {'class': 'INDUSTRY', 'fullName': 'BioCryst Pharmaceuticals'}, 'officialTitle': 'OPuS-4: An Open-label Study to Evaluate the Long-term Safety of Avoralstat in Subjects With Hereditary Angioedema', 'orgStudyIdInfo': {'id': 'BCX4161-303'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'avoralstat', 'description': 'Five avoralstat capsules (100 mg) to be taken three times daily by mouth', 'interventionNames': ['Drug: avoralstat']}], 'interventions': [{'name': 'avoralstat', 'type': 'DRUG', 'otherNames': ['BCX4161'], 'armGroupLabels': ['avoralstat']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Leuven', 'country': 'Belgium', 'geoPoint': {'lat': 50.87959, 'lon': 4.70093}}, {'city': 'Grenoble', 'country': 'France', 'geoPoint': {'lat': 45.17869, 'lon': 5.71479}}, {'city': 'Lille', 'country': 'France', 'geoPoint': {'lat': 50.63391, 'lon': 3.05512}}], 'overallOfficials': [{'name': 'Henriette Farkas, MD, PhD, DSc', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Semmelweis University'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'BioCryst Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}