Ignite Creation Date:
2025-12-25 @ 2:47 AM
Ignite Modification Date:
2025-12-31 @ 12:30 PM
Study NCT ID:
NCT04560933
Status:
COMPLETED
Last Update Posted:
2024-04-24
First Post:
2020-09-15
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006467', 'term': 'Hemophilia A'}], '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'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITHOUT_DNA', 'description': 'Blood sample collection for AAVx serotypes'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'ECOLOGIC_OR_COMMUNITY'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 186}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2020-08-25', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-04', 'completionDateStruct': {'date': '2023-10-20', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-04-22', 'studyFirstSubmitDate': '2020-09-15', 'studyFirstSubmitQcDate': '2020-09-22', 'lastUpdatePostDateStruct': {'date': '2024-04-24', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2020-09-23', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2023-09-20', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Assess the Seroprevalence of antibodies to AAV vectors in patients with hemophilia A', 'timeFrame': 'Baseline', 'description': 'To assess the Seroprevalence of antibodies to AAV vectors in patients with hemophilia A at Baseline'}, {'measure': 'Assess the rate of Seroconversion of antibodies to AAV vectors in patients with hemophilia A', 'timeFrame': 'Change from baseline through Week 12'}], 'secondaryOutcomes': [{'measure': 'To describe and characterize AAV vector titer values in subjects with Hemophilia A', 'timeFrame': 'Change from baseline through Week 12', 'description': 'To describe and characterize AAV vector titer values in subjects with Hemophilia A'}, {'measure': 'To describe and characterize AAV vector titer values in subjects with Hemophilia A', 'timeFrame': 'Change from baseline through Week 24', 'description': 'To describe and characterize AAV vector titer values in subjects with Hemophilia A'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Hemophilia A']}, 'descriptionModule': {'briefSummary': 'To quantify the seroprevalence of antibodies to AAV5, AAV6, and AAV8 and the seroconversion rate over varying follow-up intervals in subjects with hemophilia A', 'detailedDescription': 'This is a single-center, decentralized, patient-centered, prospective, observational study utilizing biospecimen samples collected from hemophilia A subjects across the United States to evaluate and characterize seroprevalence and the rate of seroconversion of antibodies against AAV serotypes and exploratory vectors, and to investigate the associated factors that may influence the vector titers.\n\nRelevant medical findings will also be collected from the subject, as well as symptoms related to hemophilia A. The collection of medical history may include major illnesses, diagnoses, and surgeries.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Subjects diagnosed with Hemophilia A', 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Subjects diagnosed with Hemophilia A\n* Subjects aged 18years or over at time of entry\n\nExclusion Criteria:\n\n* Currently participating in an interventional study of any investigational product, device or procedure.\n* Subjects who have been previously treated with AAV vector gene therapy'}, 'identificationModule': {'nctId': 'NCT04560933', 'acronym': 'SAAVY', 'briefTitle': 'A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A', 'organization': {'class': 'INDUSTRY', 'fullName': 'BioMarin Pharmaceutical'}, 'officialTitle': 'A Prospective, Observational Study Evaluating Seroprevalence and Rate of Seroconversion of Antibodies Against Adeno-associated Virus (AAV) Serotypes and Exploratory Vectors in Subjects With Hemophilia A in the United States', 'orgStudyIdInfo': {'id': '270-701'}}, 'armsInterventionsModule': {'interventions': [{'name': 'Blood sample collection', 'type': 'PROCEDURE', 'description': 'Blood sample collection'}]}, 'contactsLocationsModule': {'locations': [{'zip': '53704', 'city': 'Madison', 'state': 'Wisconsin', 'country': 'United States', 'facility': 'Covance Inc', 'geoPoint': {'lat': 43.07305, 'lon': -89.40123}}], 'overallOfficials': [{'name': 'Stephen Flach, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Covance'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'BioMarin Pharmaceutical', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}