Ignite Creation Date:
2025-12-24 @ 9:32 PM
Ignite Modification Date:
2025-12-26 @ 7:28 PM
Study NCT ID:
NCT04428632
Status:
APPROVED_FOR_MARKETING
Last Update Posted:
2020-12-11
First Post:
2020-06-10
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Oral Berotralstat Expanded Access Program
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': 'C000706836', 'term': 'berotralstat'}]}}, 'protocolSection': {'designModule': {'studyType': 'EXPANDED_ACCESS', 'expandedAccessTypes': {'intermediate': True}}, 'statusModule': {'overallStatus': 'APPROVED_FOR_MARKETING', 'statusVerifiedDate': '2020-12', 'lastUpdateSubmitDate': '2020-12-09', 'studyFirstSubmitDate': '2020-06-10', 'studyFirstSubmitQcDate': '2020-06-10', 'lastUpdatePostDateStruct': {'date': '2020-12-11', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2020-06-11', 'type': 'ACTUAL'}}, 'conditionsModule': {'keywords': ['Berotralstat', 'BCX7353', 'Hereditary Angioedema', 'HAE'], 'conditions': ['Hereditary Angioedema', 'HAE', 'Prophylaxis']}, 'descriptionModule': {'briefSummary': 'This expanded access program will provide access to berotralstat for eligible participants with hereditary angioedema in the U.S.', 'detailedDescription': 'Physicians may request access to berotralstat for eligible participants with unmet medical need\n\nPhysicians can send email inquiries to access.us@inceptua.com or call 1-888-225-8677\n\nPatients who may be interested should contact their physician about participation\n\nBerotralstat (BCX7353) will be available through this expanded access program until it is commercially available'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '12 Years', 'eligibilityCriteria': "Inclusion Criteria\n\nPatients must meet all of the following inclusion criteria to be eligible for participation in this program:\n\n1. At least 12 years of age\n2. Able to provide written, informed consent or assent\n3. Patients with a clinical diagnosis of HAE Type I or II who, in the opinion of their treating physician, are expected to benefit from an oral treatment for the prevention of angioedema attacks, and who are not eligible or able to access a berotralstat clinical trial\n4. Females must use acceptable effective contraception\n\nExclusion Criteria\n\nPatients must meet none of the below exclusion criteria to be eligible for participation in this program:\n\n1. Pregnancy or breast-feeding\n2. Any clinically significant medical condition or medical history that, in the opinion of the treating physician, would interfere with the patient's safety\n3. Current infection with hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV)"}, 'identificationModule': {'nctId': 'NCT04428632', 'briefTitle': 'Oral Berotralstat Expanded Access Program', 'organization': {'class': 'INDUSTRY', 'fullName': 'BioCryst Pharmaceuticals'}, 'officialTitle': 'Expanded Access Program With Oral Berotralstat for the Prevention of Attacks in Patients With Hereditary Angioedema', 'orgStudyIdInfo': {'id': 'BCX7353-308'}}, 'armsInterventionsModule': {'interventions': [{'name': 'Berotralstat', 'type': 'DRUG', 'otherNames': ['BCX7353'], 'description': 'One 150mg capsule administered orally once daily'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'BioCryst Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}