Ignite Creation Date:
2025-12-25 @ 2:00 AM
Ignite Modification Date:
2026-01-06 @ 2:51 PM
Study NCT ID:
NCT03199794
Status:
COMPLETED
Last Update Posted:
2022-07-21
First Post:
2017-06-23
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Prospective and Retrospective, Non-interventional Study to Evaluate the Safety and Effectiveness of Obizur in Real-life Practice
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'C536392', 'term': 'Factor 8 deficiency, acquired'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D005169', 'term': 'Factor VIII'}], 'ancestors': [{'id': 'D001779', 'term': 'Blood Coagulation Factors'}, {'id': 'D001798', 'term': 'Blood Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D011498', 'term': 'Protein Precursors'}, {'id': 'D001685', 'term': 'Biological Factors'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 50}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2016-12-14', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-07', 'completionDateStruct': {'date': '2021-07-30', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2022-07-18', 'studyFirstSubmitDate': '2017-06-23', 'studyFirstSubmitQcDate': '2017-06-23', 'lastUpdatePostDateStruct': {'date': '2022-07-21', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2017-06-27', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-07-30', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of AEs and SAEs including seriousness, severity and outcome', 'timeFrame': 'From first administration of Obizur up to 180 days after the last administration of Obizur.', 'description': 'AE - adverse event, SAE - serious adverse event.'}, {'measure': 'Number of AESIs including seriousness, severity, relationship to therapy, outcome, and treatment discontinuation', 'timeFrame': 'From first administration of Obizur up to 180 days after the last administration of Obizur.', 'description': 'Adverse Events of Special Interest (AESI) are as follows: hypersensitivity reactions, thromboembolic events and dose dispensing medication errors.'}, {'measure': 'Number of thromboembolic events', 'timeFrame': 'From first administration of Obizur up to 180 days after the last administration of Obizur.', 'description': 'Thromboembolic events include disseminated intravascular coagulation (DIC), venous thrombosis, pulmonary embolism, myocardial infarction and stroke.'}, {'measure': 'Number of dose dispensing medication errors', 'timeFrame': 'From first administration of Obizur up to 180 days after the last administration of Obizur.', 'description': "Dose dispensing medication erros include miscalculation of dose while prescribing (calculation of correct dose based on the participant's weight) or administration of the incorrect dose."}], 'secondaryOutcomes': [{'measure': 'Immunogenicity; newly recognized anti-pFVIII inhibitor or increase in titre of anti-pFVIII inhibitors and evolution of titre over time', 'timeFrame': 'From first administration of Obizur up to 180 days after the last administration of Obizur.'}, {'measure': 'Obizur treatment regimen, as available', 'timeFrame': 'From first administration of Obizur up to 180 days after the last administration of Obizur.', 'description': 'This may include details of the Obizur treatment regimen utilized, as available'}, {'measure': 'Other medication administered for haemostatic control, as available', 'timeFrame': 'From first administration of Obizur up to 180 days after the last administration of Obizur.', 'description': 'This may include additional medications, treatments and procedures (other than Obizur) undertaken to control a bleeding episode.'}, {'measure': 'Overall effectiveness assessment for resolution of bleeding', 'timeFrame': 'From first administration of Obizur up to 180 days after the last administration of Obizur.', 'description': 'Resolution of bleeding determined as either bleeding stopped or did not stop. If bleeding did not stop, a reason should be provided.'}, {'measure': 'Dose per infusion administered to achieve bleeding control, death or change in haemostatic treatment other than Obizur', 'timeFrame': 'From first administration of Obizur up to 180 days after the last administration of Obizur.', 'description': 'Bleeding control defined as all bleeding stopped.'}, {'measure': 'Number of infusions administered to achieve bleeding control, death or change in haemostatic treatment other than Obizur', 'timeFrame': 'From first administration of Obizur up to 180 days after the last administration of Obizur.', 'description': 'Bleeding control defined as all bleeding stopped.'}, {'measure': 'Time to achieve bleeding control, death or change in haemostatic treatment other than Obizur', 'timeFrame': 'From first administration of Obizur up to 180 days after the last administration of Obizur.', 'description': 'Bleeding control defined as all bleeding stopped.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'conditions': ['Acquired Hemophilia A']}, 'referencesModule': {'references': [{'pmid': '39228858', 'type': 'DERIVED', 'citation': 'Miesbach W, Curry N, Knobl P, Percy C, Santoro R, Schmaier AH, Trautmann-Grill K, Badejo K, Chen J, Nouri M, Oberai P, Klamroth R. Real-world use of recombinant porcine sequence factor VIII in the treatment of acquired hemophilia A: EU PASS. Ther Adv Hematol. 2024 Sep 2;15:20406207241260332. doi: 10.1177/20406207241260332. eCollection 2024.'}], 'seeAlsoLinks': [{'url': 'https://clinicaltrials.takeda.com/study-detail/5f6b5fc64db2bf003ab45d45?idFilter=%5B%22241501', 'label': 'To obtain more information on the study, click here/on this link'}]}, 'descriptionModule': {'briefSummary': 'The study addresses the safety, utilisation and effectiveness of Obizur in the treatment of bleeding episodes in real-life clinical practice in Europe and the United States.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'A participant with acquired hemophilia (AH) must be prescribed Obizur for the treatment of a bleeding episode by a physician, independent of and prior to the decision to enrol the participant in the study.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Adult participant (or legal representative) is willing to provide informed consent\n* Participant is being treated or was treated (treatment initiation within 30 days) with Obizur in routine clinical practice\n\nExclusion Criteria:\n\n* Participant has known anaphylactic reactions to the active substance, hamster protein or to any of the following excipients: Polysorbate 80; sodium chloride; calcium chloride dihydrate; sucrose; Tris Base; Tris HCl; Tri-sodium citrate dihydrate; sterilized water for injections\n* Participant has participated in a clinical study involving a medicinal product or device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving a medicinal product or device at study entry'}, 'identificationModule': {'nctId': 'NCT03199794', 'briefTitle': 'Prospective and Retrospective, Non-interventional Study to Evaluate the Safety and Effectiveness of Obizur in Real-life Practice', 'organization': {'class': 'INDUSTRY', 'fullName': 'Takeda'}, 'officialTitle': 'Prospective and Retrospective, Non-interventional Study to Evaluate the Safety and Effectiveness of Obizur in Real-life Practice', 'orgStudyIdInfo': {'id': '241501'}, 'secondaryIdInfos': [{'id': 'EUPAS16055', 'type': 'REGISTRY', 'domain': 'ENCePP'}]}, 'armsInterventionsModule': {'armGroups': [{'label': 'OBIZUR participants', 'description': 'Participants previously treated with OBIZUR and continue to be treated with OBIZUR during the study.', 'interventionNames': ['Biological: OBIZUR']}], 'interventions': [{'name': 'OBIZUR', 'type': 'BIOLOGICAL', 'otherNames': ['Recombinant pFVIII', 'Porcine Sequence', 'rpFVIII', 'Antihemophilic Factor (Recombinant)'], 'description': 'Treating physician will determine treatment regimen and frequency of laboratory and clinical assessments according to routine clinical practice.', 'armGroupLabels': ['OBIZUR participants']}]}, 'contactsLocationsModule': {'locations': [{'zip': '32608', 'city': 'Gainesville', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Florida - Shands', 'geoPoint': {'lat': 29.65163, 'lon': -82.32483}}, {'zip': '48202', 'city': 'Detroit', 'state': 'Michigan', 'country': 'United States', 'facility': 'Henry Ford Hospital', 'geoPoint': {'lat': 42.33143, 'lon': -83.04575}}, {'zip': '27710', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Duke University Medical Center', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '44103', 'city': 'Cleveland', 'state': 'Ohio', 'country': 'United States', 'facility': 'Cleveland Clinic', 'geoPoint': {'lat': 41.4995, 'lon': -81.69541}}, {'zip': '44106', 'city': 'Cleveland', 'state': 'Ohio', 'country': 'United States', 'facility': 'University Hospitals Cleveland Medical Center', 'geoPoint': {'lat': 41.4995, 'lon': -81.69541}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'The University of Texas Health Science Center at Houston', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '1090', 'city': 'Vienna', 'country': 'Austria', 'facility': 'AKH - Medizinische Universität Wien', 'geoPoint': {'lat': 48.20849, 'lon': 16.37208}}, {'zip': '76000', 'city': 'Rouen', 'country': 'France', 'facility': 'CHU de Rouen - Hôpital Charles Nicolle', 'geoPoint': {'lat': 49.44313, 'lon': 1.09932}}, {'zip': '10249', 'city': 'Berlin', 'country': 'Germany', 'facility': 'Vivantes Klinikum im Friedrichshain', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}, {'zip': '53127', 'city': 'Bonn', 'country': 'Germany', 'facility': 'Universitaetsklinikum Bonn', 'geoPoint': {'lat': 50.73438, 'lon': 7.09549}}, {'zip': '01304', 'city': 'Dresden', 'country': 'Germany', 'facility': 'Universitaetsklinikum Carl Gustav Carus TU Dresden', 'geoPoint': {'lat': 51.05089, 'lon': 13.73832}}, {'zip': '60590', 'city': 'Frankfurt', 'country': 'Germany', 'facility': 'Klinikum der Johann Wolfgang Goethe-Universitaet', 'geoPoint': {'lat': 49.68333, 'lon': 10.53333}}, {'zip': '30625', 'city': 'Hanover', 'country': 'Germany', 'facility': 'Medizinische Hochschule Hannover', 'geoPoint': {'lat': 52.37052, 'lon': 9.73322}}, {'zip': '15100', 'city': 'Alessandria', 'country': 'Italy', 'facility': 'Azienda Ospedaliera Nazionale Santi Antonio e Biagio e Cesare Arrigo', 'geoPoint': {'lat': 44.90924, 'lon': 8.61007}}, {'zip': '88100', 'city': 'Catanzaro', 'country': 'Italy', 'facility': 'Azienda Ospedaliera Pugliese Ciaccio', 'geoPoint': {'lat': 38.88247, 'lon': 16.60086}}, {'zip': '90127', 'city': 'Palermo', 'country': 'Italy', 'facility': 'Azienda Ospedaliero Universitaria Policlinico Paolo Giaccone', 'geoPoint': {'lat': 38.1166, 'lon': 13.3636}}, {'zip': '27100', 'city': 'Pavia', 'country': 'Italy', 'facility': 'Fondazione IRCCS Policlinico San Matteo', 'geoPoint': {'lat': 45.19205, 'lon': 9.15917}}, {'zip': '00168', 'city': 'Roma', 'country': 'Italy', 'facility': 'Policlinico Universitario Agostino Gemelli', 'geoPoint': {'lat': 44.99364, 'lon': 11.10642}}, {'zip': '00161', 'city': 'Rome', 'country': 'Italy', 'facility': 'Umberto I Pol. di Roma-Università di Roma La Sapienza', 'geoPoint': {'lat': 41.89193, 'lon': 12.51133}}, {'zip': '20089', 'city': 'Rozzano', 'country': 'Italy', 'facility': 'Istituto Clinico Humanitas', 'geoPoint': {'lat': 45.38193, 'lon': 9.1559}}, {'zip': '6525 GA', 'city': 'Nijmegen', 'country': 'Netherlands', 'facility': 'Radboud University Medical Centre', 'geoPoint': {'lat': 51.8425, 'lon': 5.85278}}, {'zip': 'B15 2TH', 'city': 'Birmingham', 'country': 'United Kingdom', 'facility': 'University Hospital Birmingham', 'geoPoint': {'lat': 52.48142, 'lon': -1.89983}}, {'zip': 'LS9 7TF', 'city': 'Leeds', 'country': 'United Kingdom', 'facility': "St James's University Hospital", 'geoPoint': {'lat': 53.79648, 'lon': -1.54785}}, {'zip': 'NW3 2QG', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Royal Free Hospital', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'OX3 7LE', 'city': 'Oxford', 'country': 'United Kingdom', 'facility': 'Churchill Hospital', 'geoPoint': {'lat': 51.75222, 'lon': -1.25596}}, {'zip': 'SO16 5YA', 'city': 'Southampton', 'country': 'United Kingdom', 'facility': 'Southampton General Hospital', 'geoPoint': {'lat': 50.90395, 'lon': -1.40428}}], 'overallOfficials': [{'name': 'Study Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Shire'}]}, 'ipdSharingStatementModule': {'url': 'https://vivli.org/ourmember/takeda/', 'infoTypes': ['STUDY_PROTOCOL', 'SAP', 'ICF', 'CSR'], 'ipdSharing': 'YES', 'description': "Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.", 'accessCriteria': 'IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Baxalta now part of Shire', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Baxalta Innovations GmbH, now part of Shire', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}