Ignite Creation Date:
2025-12-26 @ 2:38 PM
Ignite Modification Date:
2025-12-29 @ 11:41 AM
Study NCT ID:
NCT05360706
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-08-26
First Post:
2022-04-26
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study of AAV5-hFIX in Severe or Moderately Severe Haemophilia B
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D002836', 'term': 'Hemophilia B'}], 'ancestors': [{'id': 'D025861', 'term': 'Blood Coagulation Disorders, Inherited'}, {'id': 'D001778', 'term': 'Blood Coagulation Disorders'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D020147', 'term': 'Coagulation Protein Disorders'}, {'id': 'D006474', 'term': 'Hemorrhagic Disorders'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITHOUT_DNA', 'description': 'After biological samples have been analyzed, they will remain stored for potential re-analysis at any time during the study to a maximum of up to 1 year after the study has been completed, before being destroyed.'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 9}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2021-03-18', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-08', 'completionDateStruct': {'date': '2026-05', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-08-25', 'studyFirstSubmitDate': '2022-04-26', 'studyFirstSubmitQcDate': '2022-04-29', 'lastUpdatePostDateStruct': {'date': '2025-08-26', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2022-05-04', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-05', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Adverse events Possibly or probably related to previous AAV5-hFIX administration', 'timeFrame': '6-10 years after dosing in CT-AMT-060-01 study'}, {'measure': 'Neutralizing FIX antibodies (FIX inhibitors)', 'timeFrame': '6-10 years after dosing in CT-AMT-060-01 study'}, {'measure': 'ALT/AST levels', 'timeFrame': '6-10 years after dosing in CT-AMT-060-01 study'}, {'measure': 'Liver pathology score Assessed by ultrasound every 6 months', 'timeFrame': '6-10 years after dosing in CT-AMT-060-01 study'}, {'measure': 'Alfa fetoprotein levels', 'timeFrame': '6-10 years after dosing in CT-AMT-060-01 study'}], 'secondaryOutcomes': [{'measure': 'Endogenous Percent FIX activity', 'timeFrame': '6-10 years after dosing in CT-AMT-060-01 study'}, {'measure': 'Total Consumption of FIX Replacement Therapy on-demand and prophylactic', 'timeFrame': '6-10 years after dosing in CT-AMT-060-01 study'}, {'measure': 'Annualized bleeding rate Including all bleeds (treated and untreated), spontaneous bleeds, traumatic bleeds and joint bleeds', 'timeFrame': '6-10 years after dosing in CT-AMT-060-01 study'}, {'measure': 'Number of Procedures (including major and minor surgeries)', 'timeFrame': '6-10 years after dosing in CT-AMT-060-01 study'}, {'measure': 'Quality of Life questionnaire SF-36 score', 'timeFrame': '6-10 years after dosing in CT-AMT-060-01 study'}, {'measure': 'Quality of Life questionnaire EQ-5D-5L score', 'timeFrame': '6-10 years after dosing in CT-AMT-060-01 study'}, {'measure': 'Hemophilia Joint Health Score', 'timeFrame': '6-10 years after dosing in CT-AMT-060-01 study'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Hemophilia B']}, 'descriptionModule': {'briefSummary': 'This is an open-label, extension study enrolling patients who have successfully completed all assessments in Study CT-AMT-060-01 (Years 1-5). Assessment phase will begin at Visit 36 (the first clinical visit in this extension study, approximately 5.5 years after the initial dosing visit Study CT-AMT-060-01) and go to Visit 45 (10-years post-dosing in Study CT-AMT-060-01).'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Male subjects with severe or moderately severe haemophilia B, who previously received an infusion of AMT-060 and completed all assessments in Study CTAMT-060-01', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Subjects with congenital hemophilia B who completed Study CTAMT-060-01\n* Able to provide informed consent following receipt of verbal and written information about the trial.\n\nExclusion Criteria:\n\n* Enrolled subjects will have already been assessed based on the exclusion criteria for Study CT-AMT-060-01.'}, 'identificationModule': {'nctId': 'NCT05360706', 'briefTitle': 'Study of AAV5-hFIX in Severe or Moderately Severe Haemophilia B', 'organization': {'class': 'INDUSTRY', 'fullName': 'CSL Behring'}, 'officialTitle': 'A Phase I/IIb Extension Study Assessing the Long-term Safety and Efficacy of an Adeno-associated Viral Vector Containing a Codon-optimized Human Factor IX Gene (AAV5-hFIX) Previously Administered to Adult Patients With Severe or Moderately Severe Haemophilia B During the CT-AMT-060-01 Phase I/II Study', 'orgStudyIdInfo': {'id': 'CSL220_1002 (CT-AMT-060-04)'}, 'secondaryIdInfos': [{'id': '2020-000739-28', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'label': 'CSL220', 'description': 'AAV5 containing a codon-optimized human factor IX gene', 'interventionNames': ['Genetic: AAV5-hFIX']}], 'interventions': [{'name': 'AAV5-hFIX', 'type': 'GENETIC', 'otherNames': ['CSL220'], 'description': 'AAV5 containing a codon-optimized human factor IX gene', 'armGroupLabels': ['CSL220']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Berlin', 'country': 'Germany', 'facility': 'Vivantes Klinikum im Friedrichshain', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}, {'city': 'Frankfurt', 'country': 'Germany', 'facility': 'Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet', 'geoPoint': {'lat': 49.68333, 'lon': 10.53333}}, {'city': 'Amsterdam', 'country': 'Netherlands', 'facility': 'Amsterdam UMC - Locatie AMC', 'geoPoint': {'lat': 52.37403, 'lon': 4.88969}}, {'city': 'Groningen', 'country': 'Netherlands', 'facility': 'Groningen UMC', 'geoPoint': {'lat': 53.21917, 'lon': 6.56667}}, {'city': 'Rotterdam', 'country': 'Netherlands', 'facility': 'Erasmus MC', 'geoPoint': {'lat': 51.9225, 'lon': 4.47917}}, {'city': 'Utrecht', 'country': 'Netherlands', 'facility': 'Universitair Medisch Centrum Utrecht', 'geoPoint': {'lat': 52.09083, 'lon': 5.12222}}], 'overallOfficials': [{'name': 'Study Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'CSL Behring'}]}, 'ipdSharingStatementModule': {'infoTypes': ['STUDY_PROTOCOL'], 'timeFrame': 'Requests for IPD will generally be considered once review by major regulatory authorities (ie FDA, EMA) is complete and the primary publication is available.', 'ipdSharing': 'YES', 'description': 'CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.', 'accessCriteria': 'Proposed research should seek to answer a previously unanswered important medical or scientific question.\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.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'CSL Behring', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}