ATHN 11: Liver Transplantation Outcomes Study (HOT)
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT04055051 |
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Recruitment Status :
Recruiting
First Posted : August 13, 2019
Last Update Posted : February 16, 2022
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| Condition or disease |
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| Hemophilia A and B Liver Transplantation |
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.
ATHN, 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.
The 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.
All study related contact will be timed to coincide with routine, scheduled care whenever possible. The study schedule will include:
Study 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.
Participants will be asked to complete health related (QoL) questionnaires and provide medical history. These questionnaires include:
- 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.
- 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.
- 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.
| Study Type : | Observational |
| Estimated Enrollment : | 112 participants |
| Observational Model: | Cohort |
| Time Perspective: | Prospective |
| Official Title: | An Observational Cohort Study of Long-Term Outcomes of Orthotopic Liver Transplantation in People With Hemophilia |
| Actual Study Start Date : | June 18, 2020 |
| Estimated Primary Completion Date : | December 31, 2022 |
| Estimated Study Completion Date : | June 2023 |
| Group/Cohort |
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Hemophilia A and B Cases
No intervention. Only patients that have undergone a liver transplant per study eligibility are in this cohort.
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Hemophilia A and B Controls
No intervention. Comparable patients to those in Case cohort will be put in this cohort.
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- Quality of life (Haem-A-QoL) [ Time Frame: 1 year ]
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.
For 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.
- Quality of life (PROMIS-29) [ Time Frame: 1 year ]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.
- Quality of Life (HOT) [ Time Frame: 1 year ]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.
- Clotting factor protein [ Time Frame: 1year ]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.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
Inclusion Criteria
Participants who meet the following inclusion criteria are eligible for enrollment into the study:
- Congenital hemophilia A or B of any severity, who have and have not undergone a liver transplant;
- Age > 18; and
- Sex assigned at birth was male
Exclusion Criteria
Participants who fall into any of the following exclusion criteria at the time of screening are not eligible for enrollment into the study:
1. Age <18.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04055051
| Contact: Carol Fedor, ND, RN, CCRC | 800-360-2846 | cfedor@athn.org |
| United States, California | |
| Orthopaedic Institute for Children Hemophilia Program | Recruiting |
| Los Angeles, California, United States, 90007 | |
| Contact: Christopher Chan, FNP 213-742-1402 christopherchan@mednet.ucla.edu | |
| Principal Investigator: Doris Quon, MD | |
| University of California, San Francisco | Recruiting |
| San Francisco, California, United States, 94117 | |
| Contact: Sonam Choden 415-476-4170 sonam.choden@ucsf.edu | |
| Principal Investigator: Andrew Leavitt, MD | |
| United States, Illinois | |
| Bleeding and Clotting Disorders Institute | Recruiting |
| Peoria, Illinois, United States, 61614 | |
| Contact: Elizabeth Taggart 309-692-5337 elizabetht@ilbcdi.org | |
| Principal Investigator: Michael Tarantino, MD | |
| United States, Indiana | |
| Indiana Hemophilia and Thrombosis Center (IHTC) | Recruiting |
| Indianapolis, Indiana, United States, 46260 | |
| Contact: Young Chong 317-871-4511 ychong@ihtc.org | |
| Principal Investigator: Amy Shapiro, MD | |
| United States, Maine | |
| Maine Hemophilia and Thrombosis Center | Recruiting |
| Scarborough, Maine, United States, 04074 | |
| Contact: Celeste Prescott 207-396-7683 CPrescott@mmc.org | |
| Principal Investigator: Eric Larsen, MD | |
| United States, Michigan | |
| Henry Ford Health System | Recruiting |
| Detroit, Michigan, United States, 48202 | |
| Contact: Francesca Picotte 313-916-3790 fpicott1@hfhs.org | |
| Principal Investigator: Philip Kuriakose, MD | |
| United States, Minnesota | |
| Mayo Comprehensive Hemophilia Center | Recruiting |
| Rochester, Minnesota, United States, 55905 | |
| Contact: Tiffany Plowman 507-284-5095 plowman.tiffany@mayo.edu | |
| Principal Investigator: Rajiv Pruthi, MD | |
| United States, New York | |
| Mary M. Gooley Hemophilia Center, Inc. | Recruiting |
| Rochester, New York, United States, 14621 | |
| Contact: Heather Bacchetta 585-922-4807 heather.bacchetta@rochesterregional.org | |
| Principal Investigator: Peter Kouides, MD | |
| United States, North Carolina | |
| Comprehensive Hemophilia Treatment Center, University of North Carolina at Chapel Hill | Recruiting |
| Chapel Hill, North Carolina, United States, 27517 | |
| Contact: Kristi Kirkland 919-491-7137 kristi_kirkland@med.unc.edu | |
| Principal Investigator: Nigel Key, MD | |
| United States, Ohio | |
| Ohio State University Medical Center Hemophilia Treatment Center | Recruiting |
| Columbus, Ohio, United States, 43210 | |
| Contact: Abigail Bartosic 614-685-7243 abigail.bartosic@osumc.edu | |
| Principal Investigator: Eric Kraut, MD | |
| United States, Pennsylvania | |
| University of Pittsburgh | Recruiting |
| Pittsburgh, Pennsylvania, United States, 15213 | |
| Contact: Dana Ivanco des2@pitt.edu | |
| Principal Investigator: Margaret Ragni, MD, MPH | |
| United States, Texas | |
| Gulf States Hemophilia and Thrombophilia Center | Recruiting |
| Houston, Texas, United States, 77030 | |
| Contact: Madeline Cantini 713-500-8377 madeline.cantini@uth.tmc.edu | |
| Principal Investigator: Miguel Escobar, MD | |
| United States, Utah | |
| Utah Center for Bleeding and Clotting Disorders | Recruiting |
| Salt Lake City, Utah, United States, 84113 | |
| Contact: Ming Lim, MD 801-662-4728 ming.lim@hsc.utah.edu | |
| Principal Investigator: Ming Lim, MD | |
| United States, Wisconsin | |
| Hemophilia Outreach Center Green Bay | Recruiting |
| Green Bay, Wisconsin, United States, 54311 | |
| Contact: Sumedha Ghate 920-965-0606 sumedhag@hocgb.org | |
| Principal Investigator: Kenneth Friedman, MD | |
| Comprehensive Center for Bleeding Disorders, Milwaukee | Recruiting |
| Milwaukee, Wisconsin, United States, 53226 | |
| Contact: Karen Stephany 414-937-6581 kstephany@versiti.org | |
| Principal Investigator: Lynn Malec, MD | |
| Principal Investigator: | Margaret Ragni, MD, MPH | University of Pittsburgh Medical Center |
| Responsible Party: | American Thrombosis and Hemostasis Network |
| ClinicalTrials.gov Identifier: | NCT04055051 |
| Other Study ID Numbers: |
ATHN 11: Liver Transplantation |
| First Posted: | August 13, 2019 Key Record Dates |
| Last Update Posted: | February 16, 2022 |
| Last Verified: | February 2022 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |
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Hemophilia A Hemophilia B Liver Transplantation Quality of Life Hemophilia Treatment Centers (HTCs) |
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Hemophilia A Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases |
Coagulation Protein Disorders Hemorrhagic Disorders Genetic Diseases, Inborn |