Hemophilia A and BHOTNCT04055051
ATHN 11: Liver Transplantation Outcomes Study
An observational study of Hemophilia A and B and Liver Transplantation, run by American Thrombosis and Hemostasis Network at 15 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hemophilia A and B, Liver Transplantation
- How many people
- 86 participants
- Where
- 15 sitesUnited States
- Study dates
- 18 Jun 2020to 1 Dec 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: 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.
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
15 sites in 1 country.
15 sites in 1 country
United States15 sites
Comprehensive Hemophilia Treatment Center, University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27517
Ohio State University Medical Center Hemophilia Treatment Center
Columbus, Ohio, 43210
Henry Ford Health System
Detroit, Michigan, 48202
Hemophilia Outreach Center Green Bay
Green Bay, Wisconsin, 54311
Gulf States Hemophilia and Thrombophilia Center
Houston, Texas, 77030
Indiana Hemophilia and Thrombosis Center (IHTC)
Indianapolis, Indiana, 46260
Orthopaedic Institute for Children Hemophilia Program
Los Angeles, California, 90007
Comprehensive Center for Bleeding Disorders, Milwaukee
Milwaukee, Wisconsin, 53226
Bleeding and Clotting Disorders Institute
Peoria, Illinois, 61614
University of Pittsburgh
Pittsburgh, Pennsylvania, 15213
Mayo Comprehensive Hemophilia Center
Rochester, Minnesota, 55905
Mary M. Gooley Hemophilia Center, Inc.
Rochester, New York, 14621
Utah Center for Bleeding and Clotting Disorders
Salt Lake City, Utah, 84113
University of California, San Francisco
San Francisco, California, 94117
Maine Hemophilia and Thrombosis Center
Scarborough, Maine, 04074
About this study
Official title: An Observational Cohort Study of Long-Term Outcomes of Orthotopic Liver Transplantation in People With Hemophilia
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.
Read the detailed description
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.
- Conditions
- Hemophilia A and B, Liver Transplantation
- Keywords
- Hemophilia A, Hemophilia B, Liver Transplantation, Quality of Life, Hemophilia Treatment Centers (HTCs)
- Medical subject headings
- Hemophilia A, Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Hemophilia A and B Cases | No intervention. Only patients that have undergone a liver transplant per study eligibility are in this cohort. | |
| Hemophilia A and B Controls | No intervention. Comparable patients to those in Case cohort will be put in this cohort. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Quality of life (Haem-A-QoL)
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.
Measured: 1 year
Quality of life (PROMIS-29)
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.
Measured: 1 year
Quality of Life (HOT)
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.
Measured: 1 year
Secondary outcomes (1)
Clotting factor protein
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.
Measured: 1year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Aug 2019
- Study start18 Jun 2020
- Primary completion1 Dec 2022
- Study completion1 Dec 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (9)Papers the sponsor has linked to this study.
- Other trials for Hemophilia A and BStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 86 participants (actual)
Sponsor and investigators
- Lead sponsor
- American Thrombosis and Hemostasis Network
- Sponsor type
- Research network
- Collaborators
- BioMarin Pharmaceutical
- Responsible party
- Sponsor
- Investigators
- Margaret Ragni, MD, MPH, Principal investigator, University of Pittsburgh Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04055051
- Sponsor's protocol ID
- ATHN 11: Liver Transplantation
- Acronym
- HOT
- Status last verified
- May 2023
- First submitted
- 8 Aug 2019
- Last updated
- 10 May 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 19470440Subramanian 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.
PubMed 19800334Ragni 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.
PubMed 20722744Ragni 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.
PubMed 22762561Terrault 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.
PubMed 22328294Aguero 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.
PubMed 26415077Mehta 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.
PubMed 27813318Ragni 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.
PubMed 27935212Ragni 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.
PubMed 19347994
Study at a glance
From the registry record, last updated by the sponsor on 10 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Observational Cohort Study of Long-Term Outcomes of Orthotopic Liver Transplantation in People With Hemophilia
- ClinicalTrials.gov ID
- NCT04055051
- Acronym / sponsor ID
- HOT / ATHN 11: Liver Transplantation
- Conditions
- Hemophilia A and B, Liver Transplantation
- Keywords
- Hemophilia A, Hemophilia B, Liver Transplantation, Quality of Life, Hemophilia Treatment Centers (HTCs)
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 86 participants (actual)
- Lead sponsor
- American Thrombosis and Hemostasis NetworkResearch network
- Collaborators
- BioMarin Pharmaceutical
- Study start
- 18 Jun 2020 (actual)
- Primary completion
- 1 Dec 2022 (actual)
- Study completion
- 1 Dec 2022 (actual)
- First posted
- 13 Aug 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Hemophilia A and B Cases | – | –No intervention. Only patients that have undergone a liver transplant per study eligibility are in this cohort. |
| Hemophilia A and B Controls | – | –No intervention. Comparable patients to those in Case cohort will be put in this cohort. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- 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.
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
1 secondary outcome
Clotting factor protein
Time frame: 1year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 May 2023.
- Background: PubMed 19470440Madeira 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.
- Background: PubMed 19800334Subramanian 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.
- Background: PubMed 20722744Ragni 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.
- Background: PubMed 22762561Ragni 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.
- Background: PubMed 22328294Terrault 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.
- Background: PubMed 26415077Aguero 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.
- Background: PubMed 27813318Mehta 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.
- Background: PubMed 27935212Ragni 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.
- Background: PubMed 19347994Ragni 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.
- Individual participant dataSharing planned: No
Related trials for Hemophilia A and B
RecruitingPhase 3
A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
Hemophilia
RecruitingPhase 3
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
Hemophilia A, Hemophilia B
Not yet recruitingObservational study
A Study to Describe Patient REal-world FitusiRan ExpeRience
Hemophilia A or B
Not yet recruitingObservational study
Liver Biopsy Following Gene Therapy For Hemophilia
Hemophilia A, Hemophilia B
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Margaret Ragni, MD, MPH principal investigator
University of Pittsburgh Medical Center
15 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A and B and Liver Transplantation, run by American Thrombosis and Hemostasis Network at 15 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 May 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Quality of life (Haem-A-QoL) (time frame: 1 year). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04055051, which also lists 9 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 May 2023.