Ignite Creation Date:
2025-12-24 @ 2:59 PM
Ignite Modification Date:
2025-12-28 @ 10:04 PM
Study NCT ID:
NCT04739059
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-08-19
First Post:
2021-02-01
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema Attacks
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'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'PREVENTION', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 171}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2021-04-29', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-08', 'completionDateStruct': {'date': '2025-11', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-08-18', 'studyFirstSubmitDate': '2021-02-01', 'studyFirstSubmitQcDate': '2021-02-01', 'lastUpdatePostDateStruct': {'date': '2025-08-19', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-02-04', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-11', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of subjects with treatment emergent adverse events (TEAEs)', 'timeFrame': 'Up to 45 months'}, {'measure': 'Percentage of subjects with TEAEs', 'timeFrame': 'Up to 45 months'}, {'measure': 'TEAEs rates per injection', 'timeFrame': 'Up to 45 months'}, {'measure': 'TEAEs rates per subject year', 'timeFrame': 'Up to 45 months'}], 'secondaryOutcomes': [{'measure': 'The time-normalized number (per month and year) of Hereditary Angioedema (HAE attacks) for the entire study', 'timeFrame': 'Up to 43 months'}, {'measure': 'The percentage reduction and the number of subjects experiencing at least ≥ 50% ≥ 70%, ≥ 90 or equal to 100% (attack free) reduction in the time-normalized number of HAE attacks on Treatment compared to Run-in Period', 'timeFrame': 'Up to 43 months'}, {'measure': 'The time-normalized number (per month and year) of HAE attacks requiring on-demand treatment in subjects on treatment', 'timeFrame': 'Up to 43 months'}, {'measure': 'The time-normalized number (per month and year) of moderate and/or severe HAE attacks in subjects on treatment', 'timeFrame': 'Up to 43 months'}, {'measure': 'Number and percentage of subjects rating their response to therapy as good or excellent', 'timeFrame': 'Up to 43 months'}, {'measure': 'The number and percentage of subjects experiencing TEAEs', 'timeFrame': 'Up to 45 months'}, {'measure': 'The number and percentage of subjects experiencing adverse events of special interest (AESIs)', 'timeFrame': 'Up to 45 months'}, {'measure': 'The number and percentage of subjects experiencing serious adverse events (SAEs), including deaths', 'timeFrame': 'Up to 45 months'}, {'measure': 'The number and percentage of subjects experiencing CSL312 induced anti-CSL312 antibodies', 'timeFrame': 'Up to 45 months'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Hereditary Angioedema']}, 'descriptionModule': {'briefSummary': 'This phase 3b study will evaluate long-term safety and efficacy of CSL312 (also known as garadacimab) when administered subcutaneously (SC)'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Males and females aged ≥ 12 years\n* Diagnosed with clinically confirmed C1-INH HAE\n* Experienced ≥ 3 HAE attacks during the 3 months before Screening\n* Participated in the Run-in Period for at least 1 month (CSL312-naïve subjects only)\n* Experienced at least an average of 1 HAE attack per month during the Run-in Period\n\nExclusion Criteria:\n\n* Concomitant diagnosis of another form of angioedema, such as idiopathic or acquired angioedema or recurrent angioedema associated with urticaria\n* Use of C1-INH products, androgens, antifibrinolytics or other small molecule medications for routine prophylaxis against HAE attacks at least 2 weeks before the first day of the Run-in Period\n* Use of monoclonal antibodies such as lanadelumab (Takhzyro®) 3 months before the first day of the Run-in Period.\n* Female subjects use estrogen-containing oral contraceptives or hormone replacement therapy within 4 weeks prior to screening\n* Female or male subjects who are fertile and sexually active not using or not willing to use an acceptable method of contraception to avoid pregnancy during the study and for 30 days after receipt of the last dose of CSL312\n* Pregnant, breastfeeding, or not willing to cease breastfeeding'}, 'identificationModule': {'nctId': 'NCT04739059', 'briefTitle': 'Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema Attacks', 'organization': {'class': 'INDUSTRY', 'fullName': 'CSL Behring'}, 'officialTitle': 'An Open-label Study to Evaluate the Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema', 'orgStudyIdInfo': {'id': 'CSL312_3002'}, 'secondaryIdInfos': [{'id': '2020-003918-12', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'CSL312', 'description': 'Fully human immunoglobulin G subclass 4/lambda recombinant inhibitor monoclonal antibody administered subcutaneously', 'interventionNames': ['Biological: CSL312']}], 'interventions': [{'name': 'CSL312', 'type': 'BIOLOGICAL', 'otherNames': ['Factor XIIa inhibitor monoclonal antibody', 'garadacimab'], 'description': 'Fully human immunoglobulin G subclass 4/lambda recombinant inhibitor monoclonal antibody', 'armGroupLabels': ['CSL312']}]}, 'contactsLocationsModule': {'locations': [{'zip': '35209', 'city': 'Birmingham', 'state': 'Alabama', 'country': 'United States', 'facility': 'Clinical Research Center of Alabama', 'geoPoint': {'lat': 33.52066, 'lon': -86.80249}}, {'zip': '85340', 'city': 'Litchfield Park', 'state': 'Arizona', 'country': 'United States', 'facility': 'Research Solutions of Arizona', 'geoPoint': {'lat': 33.49337, 'lon': -112.35794}}, {'zip': '85251', 'city': 'Scottsdale', 'state': 'Arizona', 'country': 'United States', 'facility': 'Medical Research of Arizona', 'geoPoint': {'lat': 33.50921, 'lon': -111.89903}}, {'zip': '72205', 'city': 'Little Rock', 'state': 'Arkansas', 'country': 'United States', 'facility': 'Little Rock Allergy & Asthma Clinic', 'geoPoint': {'lat': 34.74648, 'lon': -92.28959}}, {'zip': '92868', 'city': 'Orange', 'state': 'California', 'country': 'United States', 'facility': 'Donald S. Levy M.D.', 'geoPoint': {'lat': 33.78779, 'lon': -117.85311}}, {'zip': '90404', 'city': 'Santa Monica', 'state': 'California', 'country': 'United States', 'facility': 'Raffi Tachdjian MD, Inc.', 'geoPoint': {'lat': 34.01949, 'lon': -118.49138}}, {'zip': '94598', 'city': 'Walnut Creek', 'state': 'California', 'country': 'United States', 'facility': 'Allergy & Asthma Clinical Research', 'geoPoint': {'lat': 37.90631, 'lon': -122.06496}}, {'zip': '20815', 'city': 'Chevy Chase', 'state': 'Maryland', 'country': 'United States', 'facility': 'Institute for Asthma and Allergy PC', 'geoPoint': {'lat': 39.00287, 'lon': -77.07115}}, {'zip': '45236', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': 'Bernstein Clinical Research Center, LLC', 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '17033', 'city': 'Hershey', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'PennState Health Milton S. 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For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.\n\nApplicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD.\n\nIf the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.', 'accessCriteria': "Requests may only be made by systematic review groups or bona-fide researchers whose proposed use of the IPD is non-commercial in nature and has been approved by an internal review committee.\n\nAn IPD request will not be considered by CSL unless the proposed research question seeks to answer a significant and unknown medical science or patient care question as determined by CSL's internal review committee.\n\nThe requesting party must execute an appropriate data sharing agreement before IPD will be made available."}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'CSL Behring', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}