Ignite Creation Date:
2025-12-24 @ 5:35 PM
Ignite Modification Date:
2025-12-28 @ 7:38 PM
Study NCT ID:
NCT01817868
Status:
COMPLETED
Last Update Posted:
2020-05-27
First Post:
2013-03-12
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Comparison of Efficacy, Safety and Costs of Recombinant FVIII Products Between On-demand and Secondary Prophylaxis Groups in Haemophilia A Patients
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'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D005169', 'term': 'Factor VIII'}, {'id': 'C078147', 'term': 'F8 protein, human'}, {'id': 'C414350', 'term': 'BAY 14-2222'}], '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': 73}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2013-01-04', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-05', 'completionDateStruct': {'date': '2019-05-23', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-05-22', 'studyFirstSubmitDate': '2013-03-12', 'studyFirstSubmitQcDate': '2013-03-21', 'lastUpdatePostDateStruct': {'date': '2020-05-27', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2013-03-26', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2018-07-16', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Median ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group', 'timeFrame': 'After 4 years'}, {'measure': 'Mean ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group', 'timeFrame': 'After 4 years'}], 'secondaryOutcomes': [{'measure': 'Number of overall bleeding episodes', 'timeFrame': 'After 4 years'}, {'measure': 'Musculoskeletal evaluation recommended by World Federation of Hemophilia: Orthopedic Joint Score (Gilbert Score)', 'timeFrame': 'After 4 years'}, {'measure': 'Musculoskeletal evaluation recommended by World Federation of Hemophilia: Radiological evaluation (Pettersson Score)', 'timeFrame': 'After 4 years'}, {'measure': 'Cost-effectiveness (cost of additional joint bleed)', 'timeFrame': 'After 4 years'}, {'measure': 'Cost-utility', 'timeFrame': 'After 4 years', 'description': 'The costs of care of subjects with haemophilia will be evaluated adopting the perspective of the payer, i.e. the Social Security Institution \\[SSI\\].'}, {'measure': 'Comparison of patient compliance between prophylaxis and on-demand therapy groups', 'timeFrame': 'After 4 years'}, {'measure': 'Number of spontaneous bleeds', 'timeFrame': 'After 4 years'}, {'measure': 'Quality of life as measured with the SF-36', 'timeFrame': 'Baseline and after 4 years'}, {'measure': 'Quality of life as measured with Hemo-QoL', 'timeFrame': 'Baseline and after 4 years'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Hemophilia', 'Turkey', 'Observational', 'Adult'], 'conditions': ['Hemophilia']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'https://clinicaltrials.bayer.com/', 'label': 'Click here to find results for studies related to Bayer products.'}]}, 'descriptionModule': {'briefSummary': 'The project is an observational, multi-central, prospective, non-interventional and open-label data collection study on secondary prophylaxis with recombinant FVIII products in adolescents and adults with severe hemophilia A (FVIII \\< 1%).\n\nIt will be a controlled observation of patients on secondary prophylaxis versus on-demand treatment regimen. Patients will be enrolled preferably on a 1:1 basis with regards to prophylaxis and on-demand treatment.\n\nThe patient enrollment period will be 2 years with a follow-up (observation period) of 2 years for each patient. Based on the primary effectiveness parameters (joint bleeds and overall bleeds per year) an observation period of 2 years is considered sufficient although it has to be admitted that it is rather short to assess the progression of orthopedic status. Previously treated prophylaxis patients with at least 50 exposure days and patients with continuing prophylaxis treatment will be included.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Adult haemophilia patients already on treatment with commercially available recombinant FVIII products.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Age ≥ 18 years\n* Severe hemophilia A (FVIII\\<1%) diagnosis\n* Prior treatment or ongoing treatment with on-demand or prophylaxis regimens according to Turkish guidelines and requirements\n* Previously treated patients with at least \\>50 exposure days\n* Written informed consent signed by patient/legal representative\n\nExclusion Criteria:\n\n* Currently on immune tolerance treatment\n* Platelet count \\< 75,000/mm3\n* Participation in another study\n* Existence of inhibitors in the past and in the last currently available blood sample prior to study start (Bethesda titer \\< 0.6 BU/ml)\n* Existence of inhibitor history in family members who also are diagnosed with hemophilia A\n* Having been on primary prophylaxis as defined in the introduction\n* Participation in another study'}, 'identificationModule': {'nctId': 'NCT01817868', 'briefTitle': 'Comparison of Efficacy, Safety and Costs of Recombinant FVIII Products Between On-demand and Secondary Prophylaxis Groups in Haemophilia A Patients', 'organization': {'class': 'INDUSTRY', 'fullName': 'Bayer'}, 'officialTitle': 'Evaluation of Efficacy, Safety and Costs of Recombinant FVIII Products Applied to Severe Hemophilia A Patients: Observational Data Collection Study Evaluating On-demand Treatment and Secondary Prophylaxis', 'orgStudyIdInfo': {'id': '16368'}, 'secondaryIdInfos': [{'id': 'KG1210TR', 'type': 'OTHER', 'domain': 'Company Internal'}]}, 'armsInterventionsModule': {'armGroups': [{'label': 'Group 1', 'interventionNames': ['Other: Recombinant Factor VIII (Kogenate, BAY14-2222)']}], 'interventions': [{'name': 'Recombinant Factor VIII (Kogenate, BAY14-2222)', 'type': 'OTHER', 'description': 'All dosage, frequency and duration for drugs will be under the decision of the treating physician.', 'armGroupLabels': ['Group 1']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Multiple Locations', 'country': 'Turkey (Türkiye)'}], 'overallOfficials': [{'name': 'Bayer Study Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Bayer'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED', 'description': 'Availability of this study\'s data will be determined according to Bayer\'s commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.clinicalstudydatarequest.com to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the Study sponsors section of the portal.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Bayer', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}