Ignite Creation Date:
2025-12-24 @ 10:55 PM
Ignite Modification Date:
2026-01-18 @ 3:10 PM
Study NCT ID:
NCT02581969
Status:
COMPLETED
Last Update Posted:
2020-11-18
First Post:
2015-10-20
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Extension at 10 Years of the: "Observational Study Evaluating Efficacy and Costs of Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia 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'}]}, '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': 'RETROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 43}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2016-04-14', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-11', 'completionDateStruct': {'date': '2018-06-21', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-11-17', 'studyFirstSubmitDate': '2015-10-20', 'studyFirstSubmitQcDate': '2015-10-20', 'lastUpdatePostDateStruct': {'date': '2020-11-18', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2015-10-21', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2017-03-16', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of joint bleeding episodes per year', 'timeFrame': '5 years period'}], 'secondaryOutcomes': [{'measure': 'Total number of bleeding episodes per year', 'timeFrame': '5 years period'}, {'measure': 'Total amount of Recombinant Factor VIII consumption', 'timeFrame': '5 years period'}, {'measure': 'Changes of prophylaxis dose', 'timeFrame': '5 years period'}, {'measure': 'Changes of frequency of infusions', 'timeFrame': '5 years period'}, {'measure': 'Musculoskeletal evaluation by calculating the World Federation of Haemophilia Orthopedic Joint Score (Gilbert score)', 'timeFrame': '5 years period'}, {'measure': 'Proportions of patients who will change the therapeutic regimen during the study', 'timeFrame': '5 years period'}, {'measure': 'Musculoskeletal evaluation by calculating the Radiological score (Pettersson Score)', 'timeFrame': '5 years period'}, {'measure': 'Evaluating the Questionnaires of Health-Related Quality of Life _Haemo Quality of Life (HaemoQol)', 'timeFrame': '5 years period'}, {'measure': 'Evaluating the Questionnaires of Health-Related Quality of Life _Short Form 36 questions (SF36)', 'timeFrame': '5 years period'}, {'measure': 'Evaluating the Questionnaires of Health-Related Quality of Life _ Euro Quality 5 Dimension (EQ5D)', 'timeFrame': '5 years period'}, {'measure': 'Number of days missed from work or school of patients and caregivers because of all the events haemophilia-related', 'timeFrame': '5 years period'}, {'measure': 'Number of patients with poor compliance', 'timeFrame': '5 years period'}, {'measure': 'Number of patients with adverse events', 'timeFrame': '5 years period'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Hemophilia A, Congenital'], 'conditions': ['Hemophilia A']}, 'descriptionModule': {'briefSummary': 'Following the performance of the POTTER observational study, whose primary objective was to collect data on the benefits of secondary prophylaxis versus on demand treatment in terms of prevention of bleeding episodes, the present study aims to extend the observation time up to 10 years, of the same population involved in the previous study.\n\nThe present study has been designed to allow a focus on long term disease-related damage at joint level in subjects with severe haemophilia A, as well as on the pharmacoeconomics impact of the two different treatment regimens.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '60 Years', 'minimumAge': '17 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'A total of approximately 50 patients with a diagnosis of severe haemophilia A (FVIII \\< 1%) , who are already on Kogenate Bayer treatment ongoing for long-term secondary prophylaxis or on-demand therapy and have properly completed the follow-up in the original Potter study are expected to be enrolled.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Completion of follow-up in the original Potter study without having being excluded from the Intention To Treat efficacy analysis of the primary endpoint;\n* Written informed consent specifically issued for the 5-year extension.\n\nExclusion Criteria:\n\n* Switching of treatment to a Factor VIII concentrate different from Kogenate Bayer/Helixate NexGen after the end of the previous follow-up period.'}, 'identificationModule': {'nctId': 'NCT02581969', 'acronym': 'POTTER 10', 'briefTitle': 'Extension at 10 Years of the: "Observational Study Evaluating Efficacy and Costs of Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia A."', 'organization': {'class': 'INDUSTRY', 'fullName': 'Bayer'}, 'officialTitle': 'Extension at 10 Years of the: "Observational Study Evaluating Efficacy and Costs of Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia A." POTTER-10', 'orgStudyIdInfo': {'id': '18251'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Prophylaxis', 'description': 'Prophylaxis group: treatment is based on regularly repeated infusions of clotting factor, 20-30 IU/kg -3 times a week', 'interventionNames': ['Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)']}, {'label': 'On-demand', 'description': 'On-demand group: treatment administered when bleeding episode occur', 'interventionNames': ['Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)']}], 'interventions': [{'name': 'Recombinant Factor VIII (Kogenate FS, BAY14-2222)', 'type': 'DRUG', 'description': 'Administered 20-30 IU/kg 3 times a week', 'armGroupLabels': ['Prophylaxis']}, {'name': 'Recombinant Factor VIII (Kogenate FS, BAY14-2222)', 'type': 'DRUG', 'description': 'Administered only for bleeding episodes', 'armGroupLabels': ['On-demand']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Multiple Locations', 'country': 'Italy'}], 'overallOfficials': [{'name': 'Bayer Study Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Bayer'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Bayer', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}