Ignite Creation Date:
2025-12-24 @ 11:54 PM
Ignite Modification Date:
2025-12-25 @ 9:50 PM
Study NCT ID:
NCT04055051
Status:
COMPLETED
Last Update Posted:
2023-05-10
First Post:
2019-08-08
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
ATHN 11: Liver Transplantation Outcomes Study
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006467', 'term': 'Hemophilia A'}, {'id': 'D002836', 'term': 'Hemophilia B'}], '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'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 86}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2020-06-18', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-05', 'completionDateStruct': {'date': '2022-12-01', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-05-09', 'studyFirstSubmitDate': '2019-08-08', 'studyFirstSubmitQcDate': '2019-08-12', 'lastUpdatePostDateStruct': {'date': '2023-05-10', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2019-08-13', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-12-01', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Quality of life (Haem-A-QoL)', 'timeFrame': '1 year', 'description': 'Health related quality of life questionnaire Hemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL): Domains include physical health, feelings, view of self, sports and leisure, work and school, dealing with hemophilia, treatment, future, family planning, and partnership and sexuality.\n\nFor comparing questionnaire scores between two cohorts, we could use a combined score of PROMIS-29, Haem-A-QoL, and HOT questionnaires rescaled in 0-100 or use each separate survey.'}, {'measure': 'Quality of life (PROMIS-29)', 'timeFrame': '1 year', 'description': 'Health related Quality of Life measured by the Patient Reported Outcome Measurement Information System (PROMIS-29) Profile measure physical function, anxiety, depression, fatigue, sleep disturbance, ability to participate in social roles and activities, pain interference, and pain intensity.'}, {'measure': 'Quality of Life (HOT)', 'timeFrame': '1 year', 'description': 'Hemophilia Orthotopic Liver Transplantation QoL Questionnaire (HOT) Domains include physical well-being, psychological well-being, stress, anxiety, fear, work, family, relationships and spiritual well-being.'}], 'secondaryOutcomes': [{'measure': 'Clotting factor protein', 'timeFrame': '1year', 'description': 'impact of sustained increase in clotting factor proteins (FVIII and FIX) levels after liver transplantation. The medical record will be accessed to record baseline hemophilia history including primary factor diagnosis and baseline factor level used to access disease severity, post-transplant factor levels and genotype.'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Hemophilia A', 'Hemophilia B', 'Liver Transplantation', 'Quality of Life', 'Hemophilia Treatment Centers (HTCs)'], 'conditions': ['Hemophilia A and B', 'Liver Transplantation']}, 'referencesModule': {'references': [{'pmid': '19470440', 'type': 'BACKGROUND', 'citation': 'Madeira CL, Layman ME, de Vera RE, Fontes PA, Ragni MV. Extrahepatic factor VIII production in transplant recipient of hemophilia donor liver. Blood. 2009 May 21;113(21):5364-5. doi: 10.1182/blood-2009-02-206979. No abstract available.'}, {'pmid': '19800334', 'type': 'BACKGROUND', 'citation': 'Subramanian A, Sulkowski M, Barin B, Stablein D, Curry M, Nissen N, Dove L, Roland M, Florman S, Blumberg E, Stosor V, Jayaweera DT, Huprikar S, Fung J, Pruett T, Stock P, Ragni M. MELD score is an important predictor of pretransplantation mortality in HIV-infected liver transplant candidates. Gastroenterology. 2010 Jan;138(1):159-64. doi: 10.1053/j.gastro.2009.09.053. Epub 2009 Sep 30.'}, {'pmid': '20722744', 'type': 'BACKGROUND', 'citation': 'Ragni MV, Moore CG, Soadwa K, Nalesnik MA, Zajko AB, Cortese-Hassett A, Whiteside TL, Hart S, Zeevi A, Li J, Shaikh OS; HHH Study Group. Impact of HIV on liver fibrosis in men with hepatitis C infection and haemophilia. Haemophilia. 2011 Jan;17(1):103-11. doi: 10.1111/j.1365-2516.2010.02366.x. Epub 2010 Aug 16.'}, {'pmid': '22762561', 'type': 'BACKGROUND', 'citation': 'Ragni MV, Devera ME, Roland ME, Wong M, Stosor V, Sherman KE, Hardy D, Blumberg E, Fung J, Barin B, Stablein D, Stock PG. Liver transplant outcomes in HIV+ haemophilic men. Haemophilia. 2013 Jan;19(1):134-40. doi: 10.1111/j.1365-2516.2012.02905.x. Epub 2012 Jul 5.'}, {'pmid': '22328294', 'type': 'BACKGROUND', 'citation': 'Terrault NA, Roland ME, Schiano T, Dove L, Wong MT, Poordad F, Ragni MV, Barin B, Simon D, Olthoff KM, Johnson L, Stosor V, Jayaweera D, Fung J, Sherman KE, Subramanian A, Millis JM, Slakey D, Berg CL, Carlson L, Ferrell L, Stablein DM, Odim J, Fox L, Stock PG; Solid Organ Transplantation in HIV: Multi-Site Study Investigators. Outcomes of liver transplant recipients with hepatitis C and human immunodeficiency virus coinfection. Liver Transpl. 2012 Jun;18(6):716-26. doi: 10.1002/lt.23411.'}, {'pmid': '26415077', 'type': 'BACKGROUND', 'citation': 'Aguero F, Rimola A, Stock P, Grossi P, Rockstroh JK, Agarwal K, Garzoni C, Barcan LA, Maltez F, Manzardo C, Mari M, Ragni MV, Anadol E, Di Benedetto F, Nishida S, Gastaca M, Miro JM; FIPSE/NIH HIVTR/NEAT023 Investigators. Liver Retransplantation in Patients With HIV-1 Infection: An International Multicenter Cohort Study. Am J Transplant. 2016 Feb;16(2):679-87. doi: 10.1111/ajt.13461. Epub 2015 Sep 28.'}, {'pmid': '27813318', 'type': 'BACKGROUND', 'citation': 'Mehta KD, Ragni MV. Bleeding and liver transplantation outcomes in haemophilia. Haemophilia. 2017 Mar;23(2):230-237. doi: 10.1111/hae.13104. Epub 2016 Nov 4.'}, {'pmid': '27935212', 'type': 'BACKGROUND', 'citation': 'Ragni MV, Humar A, Stock PG, Blumberg EA, Eghtesad B, Fung JJ, Stosor V, Nissen N, Wong MT, Sherman KE, Stablein DM, Barin B. Hemophilia Liver Transplantation Observational Study. Liver Transpl. 2017 Jun;23(6):762-768. doi: 10.1002/lt.24688.'}, {'pmid': '19347994', 'type': 'BACKGROUND', 'citation': 'Ragni MV, Nalesnik MA, Schillo R, Dang Q. Highly active antiretroviral therapy improves ESLD-free survival in HIV-HCV co-infection. Haemophilia. 2009 Mar;15(2):552-8. doi: 10.1111/j.1365-2516.2008.01935.x.'}]}, 'descriptionModule': {'briefSummary': 'This study is designed as a multi-center, observational cohort study of participants with hemophilia A and B who have and have not undergone liver transplantation. Participants will be asked to complete health related quality of life questionnaires and provide medical history.', 'detailedDescription': 'At a time when gene therapy is becoming a reality for individuals with hemophilia A and B, little is known about long-term cures. Yet, there are few data to provide real life testing of the impact of long-term cures. Specifically, little is known about the relationship between factor level achieved with gene therapy and clinical outcomes or quality of life. The analogy to liver transplant is clear. There has been no systematic method to collect and analyze the long-term outcomes of liver transplantation in individuals with hemophilia. Previous efforts to access data through the United Network for Organ Sharing national database have been complicated by lack of identification of those with hemophilia. As most of the individuals with hemophilia who undergo liver transplantation receive care at a hemophilia treatment center (HTC), ATHN proposes to leverage its existing relationship with the HTCs to support HTC execution of this study.\n\nATHN, in collaboration with US Hemophilia Treatment Centers (HTCs), can provide the infrastructure and organization to support a longitudinal cohort study of subjects with congenital hemophilia who have and have not undergone liver transplantation. This cohort study will compare quality of life outcomes between cases that have undergone liver transplantation and controls that have not undergone liver transplantation. The study will determine if factor levels attained post-transplantation correlate with or predict improvement in quality of life measures.\n\nThe existence of ATHN and the ATHN System for clinical care and research provides an opportunity to observe a cohort of subjects after liver transplantation with facility. During the study period, it is predicted that sustained normalization in factor VIII or IX levels following liver transplantation improves clinical functioning and quality of life in adults with hemophilia A and B.\n\nAll study related contact will be timed to coincide with routine, scheduled care whenever possible. The study schedule will include:\n\nStudy enrollment - Study activities will begin after participant consent. Activities include the documentation of relevant medical history and the administration of quality of life (QoL) questionnaires.\n\nParticipants will be asked to complete health related (QoL) questionnaires and provide medical history. These questionnaires include:\n\n1. Hemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL): A quality of life questionnaire designed to asses health-related quality of life in adult patients with hemophilia. Domains include physical health, feelings, view of self, sports and leisure, work and school, dealing with hemophilia, treatment, future, family planning, and partnership and sexuality.\n2. Patient Reported Outcomes Measurement Information System (PROMIS-29): A quality of life questionnaire designed to assess physical function, anxiety, depression, fatigue, sleep disturbance, ability to participate in social roles and activities, pain interference, and pain intensity.\n3. Hemophilia Orthotopic Liver Transplantation QoL Questionnaire (HOT): A quality of life questionnaire, adapted from a cancer survivorship tool, designed to assess adults with hemophilia who have received a liver transplant. Domains include physical well-being, psychological well-being, stress, anxiety, fear, work, family, relationships, and spiritual well-being.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'The study will enroll a cohort of approximately 112 participants born with hemophilia who meet the eligibility criteria and are receiving care from one of approximately 12 participating HTCs. The participants will be approached for enrollment and matched 1:3 with age-, race-, ethnicity-matched controls (N=84) with hemophilia who have not undergone transplantation, for a total of 112 individuals with hemophilia who will compete quality of life questionnaires.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria\n\nParticipants who meet the following inclusion criteria are eligible for enrollment into the study:\n\n1. Congenital hemophilia A or B of any severity, who have and have not undergone a liver transplant;\n2. Age \\> 18; and\n3. Sex assigned at birth was male\n\nExclusion Criteria\n\nParticipants who fall into any of the following exclusion criteria at the time of screening are not eligible for enrollment into the study:\n\n1\\. Age \\<18.'}, 'identificationModule': {'nctId': 'NCT04055051', 'acronym': 'HOT', 'briefTitle': 'ATHN 11: Liver Transplantation Outcomes Study', 'organization': {'class': 'NETWORK', 'fullName': 'American Thrombosis and Hemostasis Network'}, 'officialTitle': 'An Observational Cohort Study of Long-Term Outcomes of Orthotopic Liver Transplantation in People With Hemophilia', 'orgStudyIdInfo': {'id': 'ATHN 11: Liver Transplantation'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Hemophilia A and B Cases', 'description': 'No intervention. Only patients that have undergone a liver transplant per study eligibility are in this cohort.'}, {'label': 'Hemophilia A and B Controls', 'description': 'No intervention. Comparable patients to those in Case cohort will be put in this cohort.'}]}, 'contactsLocationsModule': {'locations': [{'zip': '90007', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'Orthopaedic Institute for Children Hemophilia Program', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '94117', 'city': 'San Francisco', 'state': 'California', 'country': 'United States', 'facility': 'University of California, San Francisco', 'geoPoint': {'lat': 37.77493, 'lon': -122.41942}}, {'zip': '61614', 'city': 'Peoria', 'state': 'Illinois', 'country': 'United States', 'facility': 'Bleeding and Clotting Disorders Institute', 'geoPoint': {'lat': 40.69365, 'lon': -89.58899}}, {'zip': '46260', 'city': 'Indianapolis', 'state': 'Indiana', 'country': 'United States', 'facility': 'Indiana Hemophilia and Thrombosis Center (IHTC)', 'geoPoint': {'lat': 39.76838, 'lon': -86.15804}}, {'zip': '04074', 'city': 'Scarborough', 'state': 'Maine', 'country': 'United States', 'facility': 'Maine Hemophilia and Thrombosis Center', 'geoPoint': {'lat': 43.57814, 'lon': -70.32172}}, {'zip': '48202', 'city': 'Detroit', 'state': 'Michigan', 'country': 'United States', 'facility': 'Henry Ford Health System', 'geoPoint': {'lat': 42.33143, 'lon': -83.04575}}, {'zip': '55905', 'city': 'Rochester', 'state': 'Minnesota', 'country': 'United States', 'facility': 'Mayo Comprehensive Hemophilia Center', 'geoPoint': {'lat': 44.02163, 'lon': -92.4699}}, {'zip': '14621', 'city': 'Rochester', 'state': 'New York', 'country': 'United States', 'facility': 'Mary M. Gooley Hemophilia Center, Inc.', 'geoPoint': {'lat': 43.15478, 'lon': -77.61556}}, {'zip': '27517', 'city': 'Chapel Hill', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Comprehensive Hemophilia Treatment Center, University of North Carolina at Chapel Hill', 'geoPoint': {'lat': 35.9132, 'lon': -79.05584}}, {'zip': '43210', 'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'facility': 'Ohio State University Medical Center Hemophilia Treatment Center', 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '15213', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'University of Pittsburgh', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'Gulf States Hemophilia and Thrombophilia Center', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '84113', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': 'Utah Center for Bleeding and Clotting Disorders', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '54311', 'city': 'Green Bay', 'state': 'Wisconsin', 'country': 'United States', 'facility': 'Hemophilia Outreach Center Green Bay', 'geoPoint': {'lat': 44.51916, 'lon': -88.01983}}, {'zip': '53226', 'city': 'Milwaukee', 'state': 'Wisconsin', 'country': 'United States', 'facility': 'Comprehensive Center for Bleeding Disorders, Milwaukee', 'geoPoint': {'lat': 43.0389, 'lon': -87.90647}}], 'overallOfficials': [{'name': 'Margaret Ragni, MD, MPH', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of Pittsburgh Medical Center'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'American Thrombosis and Hemostasis Network', 'class': 'NETWORK'}, 'collaborators': [{'name': 'BioMarin Pharmaceutical', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}