Ignite Creation Date:
2025-12-24 @ 11:49 PM
Ignite Modification Date:
2025-12-25 @ 9:43 PM
Study NCT ID:
NCT01856751
Status:
UNKNOWN
Last Update Posted:
2015-12-18
First Post:
2013-05-14
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Use of a TGA and TEM in the Assessment of the Efficacy of Treatment With APCC or rFVIIa
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': 'this data is not applicable'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 80}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'RECRUITING', 'startDateStruct': {'date': '2014-04'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2015-12', 'completionDateStruct': {'date': '2016-06', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2015-12-17', 'studyFirstSubmitDate': '2013-05-14', 'studyFirstSubmitQcDate': '2013-05-16', 'lastUpdatePostDateStruct': {'date': '2015-12-18', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2013-05-17', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2016-04', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': "Assessment of patient's individual response to therapy with by-passing agents by simultaneous use of TGA and TEM methods.", 'timeFrame': '48 hours', 'description': "This is non-inverventional study as the protocol will not assign specific treatment to the particular subjects of the study. Patients will be treated with APCC or rFVIIa based on the experience of the study site. Patients are prescribed a treatment according to their physician's judgement or local clinical practice. This is observation of the everyday clinical practise on site."}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Haemophilia', 'TGA', 'TEM'], 'conditions': ['Haemophilia']}, 'referencesModule': {'references': [{'pmid': '19719549', 'type': 'BACKGROUND', 'citation': 'Dargaud Y, Negrier C. Thrombin generation testing in haemophilia comprehensive care centres. Haemophilia. 2010 Mar;16(2):223-30. doi: 10.1111/j.1365-2516.2009.02082.x. Epub 2009 Aug 27.'}, {'pmid': '19016901', 'type': 'BACKGROUND', 'citation': 'Berntorp E. Differential response to bypassing agents complicates treatment in patients with haemophilia and inhibitors. Haemophilia. 2009 Jan;15(1):3-10. doi: 10.1111/j.1365-2516.2008.01931.x. Epub 2008 Nov 10.'}, {'pmid': '18544431', 'type': 'BACKGROUND', 'citation': 'Dargaud Y, Bordet JC, Lienhart A, Negrier C. Use of the thrombin generation test to evaluate response to treatment with recombinant activated factor VII. Semin Hematol. 2008 Apr;45(2 Suppl 1):S72-3. doi: 10.1053/j.seminhematol.2008.03.008. No abstract available.'}, {'pmid': '18371162', 'type': 'BACKGROUND', 'citation': 'Livnat T, Martinowitz U, Zivelin A, Seligsohn U. Effects of factor VIII inhibitor bypassing activity (FEIBA), recombinant factor VIIa or both on thrombin generation in normal and haemophilia A plasma. Haemophilia. 2008 Jul;14(4):782-6. doi: 10.1111/j.1365-2516.2008.01688.x. Epub 2008 Mar 21.'}]}, 'descriptionModule': {'briefSummary': "Occurrence of inhibitors to coagulation factor VIII is diagnosed in \\~30% patients with haemophilia A. Presence of inhibitor with a titre \\>5 BU/ml requires the use of by-passing agents: recombinant activated Factor VIIa concentrate (rFVIIa) and/or activated prothrombin complex concentrate (APCC). Similarly, haemorrhagic complications in patients with acquired haemophilia and inhibitor titre \\>5 BU/ml should be treated with by-passing agents.\n\nResponse to treatment with by-passing agents is patient-specific, and can vary in the same patient during subsequent bleedings. Some patients have good response to both products, however in other patients a better bleeding control is provided by one of the mentioned above agents (APCC or rFVIIa). There are clinical situations when severe bleedings requires an alternate use of both these agents.\n\nTraditional methods of laboratory tests used post-treatment in patients with haemophilia without inhibitors are useless in the presence of inhibitor. Laboratory monitoring of therapy with by-passing agents is possible with the use of global tests for the coagulation process assessment, which are as follows: thrombin generation assay (TGA) and thromboelastometry (TEM).\n\nSeveral studies revealed that TGA allows a monitoring of therapy with by-passing agents in patients with haemophilia A and inhibitor - the choice of the most effective treatment option - agent type and its dose, as well as laboratory assessment of treatment efficacy.\n\nUp to date, laboratory tests assessing the efficacy of by-passing agents in patients with acquired haemophilia were not conducted.\n\nIn Factor VIII or IX deficiency conditions, fibrin's fibres generated by thrombin are morphologically thicker, and blood clots have increased susceptibility to fibrinolytic enzymes. Blood clot stability may be assessed with the use of thromboelastometry (TEM). We can hypothesize that simultaneous use of TGA and TEM methods may allow for an assessment of patient's individual response to therapy with by-passing agents. Clinical significance of the minimal dose of APCC and rFVIIa, needed to TGA and TEM normalization, requires further studies.\n\nTests' purpose: Examination of the hypothesis that simultaneous use of thrombin generation assay (TGA) and thromboelastometry (TEM) may facilitate the choice of optimal therapy with by-passing agents and laboratory monitoring of efficacy of those agents in patients with acquired haemophilia or haemophilia A with inhibitor.", 'detailedDescription': 'This section is Not applicable'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': '60 patients with acquired haemophilia 20 patients with congenital haemophilia A with inhibitor', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* patients with acquired haemophilia\n* patients with congenital haemophilia A with inhibitor\n\nExclusion Criteria:'}, 'identificationModule': {'nctId': 'NCT01856751', 'acronym': 'Thrombus', 'briefTitle': 'Use of a TGA and TEM in the Assessment of the Efficacy of Treatment With APCC or rFVIIa', 'organization': {'class': 'OTHER', 'fullName': 'Stowarzyszenie Pomocy Chorym na Zakrzepicę i Skazy Krwotoczne Thrombus'}, 'officialTitle': 'Use of a TGA and TEM in the Assessment of the Efficacy of Treatment With APCC or rFVIIa Concentrate in Patients With Acquired Haemophilia and in Patients With Haemophilia A With Inhibitors', 'orgStudyIdInfo': {'id': 'TGA-TEM'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'haemophilia', 'description': 'Patients with acquired haemophilia. Patients with haemophilia A with inhibitor.'}]}, 'contactsLocationsModule': {'locations': [{'zip': '80-952', 'city': 'Gdansk', 'status': 'RECRUITING', 'country': 'Poland', 'contacts': [{'name': 'Andrzej Mital, MD, PhD', 'role': 'CONTACT'}], 'facility': 'Katedra i Klinika Hematologii i Transplantologii Gdanski Uniwersytet Medyczny', 'geoPoint': {'lat': 54.35227, 'lon': 18.64912}}, {'zip': '31-501', 'city': 'Krakow', 'status': 'RECRUITING', 'country': 'Poland', 'contacts': [{'name': 'Jacek Musial, MD, PhD', 'role': 'CONTACT'}], 'facility': 'Szpital Uniwersytecki w Krakowie, Oddzial Kliniczny Alergii i Immunologii', 'geoPoint': {'lat': 50.06143, 'lon': 19.93658}}, {'zip': '93-510', 'city': 'Lodz', 'status': 'RECRUITING', 'country': 'Poland', 'contacts': [{'name': 'Krzysztof Chojnowski, MD, PhD', 'role': 'CONTACT'}], 'facility': 'Klinika Hematologii Uniwersytetu Medycznego w Lodzi Wojewodzki Szpital Specjalistyczny im. M. Kopernika', 'geoPoint': {'lat': 51.77058, 'lon': 19.47395}}, {'zip': '61-505', 'city': 'Poznan', 'status': 'RECRUITING', 'country': 'Poland', 'contacts': [{'name': 'Krystyna Zawilska, MD,PhD', 'role': 'CONTACT'}], 'facility': 'Centrum Diagnostyczno - Lecznicze INTERLAB', 'geoPoint': {'lat': 52.40692, 'lon': 16.92993}}, {'zip': '02-776', 'city': 'Warsaw', 'status': 'RECRUITING', 'country': 'Poland', 'contacts': [{'name': 'Jerzy Windyga, MD, PhD', 'role': 'CONTACT'}], 'facility': 'Instytut Hematologii i Transfuzjologii w Warszawie', 'geoPoint': {'lat': 52.22977, 'lon': 21.01178}}, {'zip': '50-367', 'city': 'Wroclaw', 'status': 'RECRUITING', 'country': 'Poland', 'contacts': [{'name': 'Maria Podolak-Dawidziak, MD, PhD', 'role': 'CONTACT'}], 'facility': 'Katedra i Klinika Hematologii, Nowotworow Krwi i Transplantacji Szpiku, Akademia Medyczna', 'geoPoint': {'lat': 51.10286, 'lon': 17.03006}}], 'centralContacts': [{'name': 'Krystyna M Zawilska, MD, PhD', 'role': 'CONTACT', 'email': 'k.zawilska@interia.pl', 'phone': '48618333949'}], 'overallOfficials': [{'name': 'Krystyna Zawilska, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Centrum Diagnostyczno - Lecznicze INTERLAB'}, {'name': 'Maria Podolak-Dawidziak, MD,PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Katedra i Klinika Hematologii, Nowotworow Krwi i Transplantacji Szpiku, Akademia Medyczna'}, {'name': 'Andrzej Mital, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Katedra i Klinika Hematologii i Transplantologii Gdanski Uniwersytet Medyczny'}, {'name': 'Jerzy Windyga, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Instytut Hematologii i Transfuzjologii w Warszawie'}, {'name': 'Krzysztof Chojnowski, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Klinika Hematologii Uniwersytetu Medycznego w Lodzi Wojewodzki Szpital Specjalistyczny im. M. Kopernika'}, {'name': 'Jacek Musial, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Szpital Uniwersytecki w Krakowie, Oddzial Kliniczny Alergii i Immunologii'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Stowarzyszenie Pomocy Chorym na Zakrzepicę i Skazy Krwotoczne Thrombus', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}