Hematopoietic Stem Cell TransplantNCT05722210
Prevalence and Development of Liver Dysfunction in Hematopoietic Stem Cell Transplant
An observational study of Hematopoietic Stem Cell Transplant, run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) at 1 site in United States.
Trial summary
Background: Hematopoietic stem cell transplant (HSCT) is a common treatment for many cancers and other illnesses. But many people who have HSCT go on to develop liver dysfunction. Researchers want to know more about how and why this happens. Read more about this study
- Who is it for?
- Ages 3 to 90 years
- Conditions the study is for
- Hematopoietic Stem Cell Transplant
- How many people
- 500 participants
- Where
- 1 siteUnited States
- Study dates
- 10 Nov 2026to 30 Jun 2031
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 3 to 90 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: male and female subjects: adults 18 years of age and older, children 3-17 years of age undergoing hematopoietic stem cell transplant at the National Institutes of Health.
Who may be able to join
- An individual who meets any of the following criteria will be included in this study:
- Male and female adults >=18 years of age and children 3-17 years of age
- Undergoing evaluation for hematopoietic stem cell transplant at the NIH Clinical Center
Who cannot join
- An individual who meets any of the following criteria will be excluded from participation in this
- study:
- Pregnancy or lactation
- Unable to comply with study procedures
- Inability to provide written informed consent
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
NIH Clinical Center Office of Patient Recruitment (OPR), 800-411-1222 ext. TTY dial 711, ccopr@nih.gov
About this study
Official title: Prevalence and Development of Liver Dysfunction in Hematopoietic Stem Cell Transplant: A Prospective Natural History Study
Background:
Hematopoietic stem cell transplant (HSCT) is a common treatment for many cancers and other illnesses. But many people who have HSCT go on to develop liver dysfunction. Researchers want to know more about how and why this happens. In this natural history study, they will try to learn what factors lead to liver dysfunction; how underlying liver disease may affect the results of HSCT; and how HSCT may contribute to liver dysfunction.
Read more
Objective:
To understand the links between HSCT and liver dysfunction.
Eligibility:
Adults aged 18 years or older and children 3 to 17 years who are being evaluated for HSCT.
Design:
This study involves 11 visits in 4 years. Most visits will be in the first year.
Before and after their HSCT, participants will undergo these tests:
Physical exam, including blood tests and a test of heart function. Participants will provide stool samples.
Liver biopsies. Samples of liver tissue will be removed. This may be done either by inserting a needle through the right side of the chest, or with a thin tube threaded to the liver from a vein in the neck. Adult participants will undergo this procedure 2 times: once before the HSCT and once about a year later.
Imaging scans. Participants will lie on a bed that moves into either a cylinder or a donut-shaped machine.
Liver ultrasound. Participants will lie still. A probe that uses sound waves will be slid over their skin to get pictures of the liver.
Fibroscan exam. This is like an ultrasound that uses a special probe to measure the toughness of the liver.
...
Read the detailed description
Study Description:
Liver dysfunction is common in patients that have undergone hematopoietic stem cell transplant (HSCT) and is associated with increased mortality. We aim to study the natural history of liver dysfunction in HSCT, what factors contribute to the development of liver dysfunction, and how underlying liver disease affects complications and outcomes of HSCT. We hypothesize that those patients with underlying liver disease or those who develop liver disease have increased morbidity and mortality compared to those without liver disease.
Objectives:
Primary Objective:
To determine whether, at 3 months (Visit 7) after transplant, patients with liver disease at transplant are more likely to have died or have a total bilirubin >=4 mg/dL than those without liver disease at transplant.
Secondary Objectives:
To understand the impact of liver disease in HSCT on morbidity/mortality.
To understand the development and progression of liver disease in hematopoietic stem cell transplant
Tertiary Objectives:
To identify predictive/protective factors associated with presence or absence of liver disease, and severity of liver disease in patients receiving hematopoietic stem cell transplant.
Endpoints:
Primary Endpoints:
- Death or total bilirubin >=4 mg/dL at 3 months (Visit 7) after the transplant
- Mortality rate
Secondary Endpoints:
- Morbidity/cause of death
- Development of portal hypertension and sequelae (i.e., ascites, variceal bleed, thrombocytopenia, elevated portal pressure)
- Development of liver failure (i.e., coagulopathy with an International Normalized Ration (INR) 1.5, and any degree of mental alteration (encephalopathy in a subject without preexisting cirrhosis and with an illness of < 26 weeks duration, including subjects with Wilson s disease, vertically acquired HBV, or autoimmune hepatitis per AASLD guidelines 2011)
- Rate of infection (type bacterial, viral fungal, location: central line, organ infection, sepsis, etc.)
Liver dysfunction, characterized by development of the following conditions:
- Synthetic dysfunction: Total bilirubin > 4 mg/dL (20-40% in HSCT recipients or INR > 1.5
- Portal hypertension: Presence/absence of any of the following will qualify as portal hypertension- ascites, collateral vessels, elevated portal pressure
- Liver injury: ALT>4 times the upper limit of normal (22 IU/L in women, 29 IU/L in men) or Alkaline phosphatase >2.5 times the upper limit of normal
Tertiary Endpoints:
- Imaging, laboratory analysis of liver dysfunction, liver tissue pathology, medications/treatment course will be reviewed.
- Tertiary studies include stool microbiome studies, metabolomics, microbial translocation markers, flow cytometry, transcriptomics, growth factor measurement, cytokines, and chemokines measurement.
- Conditions
- Hematopoietic Stem Cell Transplant
- Keywords
- Liver Disease, Liver Dysfunction, Natural History
- Medical subject headings
- Liver Diseases
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 |
|---|---|---|
| Adults >= 18 years of age | undergoing evaluation for hematopoietic stem cell transplant at the NIH Clinical Center | |
| Children 3-17 years of age | undergoing evaluation for hematopoietic stem cell transplant at the NIH Clinical Center |
What is being measured
The primary outcome is the main question the study is designed to answer.
Death or total bilirubin >=4 at 91 days after the transplant
To determine whether, at 91 days after transplant, patients with liver disease at transplant are more likely to have died or have a total bilirubin >=4 than those without liver disease at transplant.
Measured: 91 days after transplant
Mortality rate
To determine whether, at 91 days after transplant, patients with liver disease at transplant are more likely to have died or have a total bilirubin >=4 than those without liver disease at transplant.
Measured: 91 days after transplant
Secondary outcomes (4)
Rate of infection (type: bacterial, viral, fungal; location: central line, organ infection, sepsis, etc.)
Measured: 91 days after transplant
Development of portal hypertension and sequelae (i.e., ascites, variceal bleed, thrombocytopenia, elevated portal pressure)
Measured: 91 days after transplant
Morbitity/cause of death
Measured: 91 days after transplant
Development of liver failure
i.e., coagulopathy with an International Normalized Ratio (INR) 1.5, and any degree of mental alteration (encephalopathy) in a subject without preexisting cirrhosis and with an illness of <26 weeks duration, including patients with Wilson s disease, vertically acquired HBV, or autoimmune hepatitis per AASLD guidelines 2011.
Measured: 91 days after transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Feb 2023
- Study start10 Nov 2026 (estimated)
- Primary completion30 Jun 2031 (estimated)
- Study completion30 Jun 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematopoietic Stem Cell TransplantStudies of the same condition, open and under way.
- More trials run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)The sponsor's other registered studies.
- 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
- 500 participants (estimated)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Theo Heller, M.D., Principal investigator, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05722210
- Sponsor's protocol ID
- 10000545
- Other IDs
- 000545-DK
- Status last verified
- 27 Oct 2026
- First submitted
- 9 Feb 2023
- Last updated
- 5 Nov 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prevalence and Development of Liver Dysfunction in Hematopoietic Stem Cell Transplant: A Prospective Natural History Study
- ClinicalTrials.gov ID
- NCT05722210
- Acronym / sponsor ID
- 10000545
- Conditions
- Hematopoietic Stem Cell Transplant
- Keywords
- Liver Disease, Liver Dysfunction, Natural History
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 500 participants (estimated)
- Lead sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)US National Institutes of Health
- Study start
- 10 Nov 2026 (estimated)
- Primary completion
- 30 Jun 2031 (estimated)
- Study completion
- 30 Jun 2031 (estimated)
- First posted
- 10 Feb 2023 (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.
Background:
Hematopoietic stem cell transplant (HSCT) is a common treatment for many cancers and other illnesses. But many people who have HSCT go on to develop liver dysfunction. Researchers want to know more about how and why this happens. In this natural history study, they will try to learn what factors lead to liver dysfunction; how underlying liver disease may affect the results of HSCT; and how HSCT may contribute to liver dysfunction.
Objective:
To understand the links between HSCT and liver dysfunction.
Eligibility:
Adults aged 18 years or older and children 3 to 17 years who are being evaluated for HSCT.
Design:
This study involves 11 visits in 4 years. Most visits will be in the first year.
Before and after their HSCT, participants will undergo these tests:
Physical exam, including blood tests and a test of heart function. Participants will provide stool samples.
Liver biopsies. Samples of liver tissue will be removed. This may be done either by inserting a needle through the right side of the chest, or with a thin tube threaded to the liver from a vein in the neck. Adult participants will undergo this procedure 2 times: once before the HSCT and once about a year later.
Imaging scans. Participants will lie on a bed that moves into either a cylinder or a donut-shaped machine.
Liver ultrasound. Participants will lie still. A probe that uses sound waves will be slid over their skin to get pictures of the liver.
Fibroscan exam. This is like an ultrasound that uses a special probe to measure the toughness of the liver.
...
Detailed description
Study Description:
Liver dysfunction is common in patients that have undergone hematopoietic stem cell transplant (HSCT) and is associated with increased mortality. We aim to study the natural history of liver dysfunction in HSCT, what factors contribute to the development of liver dysfunction, and how underlying liver disease affects complications and outcomes of HSCT. We hypothesize that those patients with underlying liver disease or those who develop liver disease have increased morbidity and mortality compared to those without liver disease.
Objectives:
Primary Objective:
To determine whether, at 3 months (Visit 7) after transplant, patients with liver disease at transplant are more likely to have died or have a total bilirubin >=4 mg/dL than those without liver disease at transplant.
Secondary Objectives:
To understand the impact of liver disease in HSCT on morbidity/mortality.
To understand the development and progression of liver disease in hematopoietic stem cell transplant
Tertiary Objectives:
To identify predictive/protective factors associated with presence or absence of liver disease, and severity of liver disease in patients receiving hematopoietic stem cell transplant.
Endpoints:
Primary Endpoints:
- Death or total bilirubin >=4 mg/dL at 3 months (Visit 7) after the transplant
- Mortality rate
Secondary Endpoints:
- Morbidity/cause of death
- Development of portal hypertension and sequelae (i.e., ascites, variceal bleed, thrombocytopenia, elevated portal pressure)
- Development of liver failure (i.e., coagulopathy with an International Normalized Ration (INR) 1.5, and any degree of mental alteration (encephalopathy in a subject without preexisting cirrhosis and with an illness of < 26 weeks duration, including subjects with Wilson s disease, vertically acquired HBV, or autoimmune hepatitis per AASLD guidelines 2011)
- Rate of infection (type bacterial, viral fungal, location: central line, organ infection, sepsis, etc.)
Liver dysfunction, characterized by development of the following conditions:
- Synthetic dysfunction: Total bilirubin > 4 mg/dL (20-40% in HSCT recipients or INR > 1.5
- Portal hypertension: Presence/absence of any of the following will qualify as portal hypertension- ascites, collateral vessels, elevated portal pressure
- Liver injury: ALT>4 times the upper limit of normal (22 IU/L in women, 29 IU/L in men) or Alkaline phosphatase >2.5 times the upper limit of normal
Tertiary Endpoints:
- Imaging, laboratory analysis of liver dysfunction, liver tissue pathology, medications/treatment course will be reviewed.
- Tertiary studies include stool microbiome studies, metabolomics, microbial translocation markers, flow cytometry, transcriptomics, growth factor measurement, cytokines, and chemokines measurement.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Adults >= 18 years of age | – | –undergoing evaluation for hematopoietic stem cell transplant at the NIH Clinical Center |
| Children 3-17 years of age | – | –undergoing evaluation for hematopoietic stem cell transplant at the NIH Clinical Center |
Who may qualify
- Ages
- 3 to 90 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- male and female subjects: adults 18 years of age and older, children 3-17 years of age undergoing hematopoietic stem cell transplant at the National Institutes of Health.
Key inclusion criteria (3 listed)
- Male and female adults >=18 years of age and children 3-17 years of age
- Undergoing evaluation for hematopoietic stem cell transplant at the NIH Clinical Center
Key exclusion criteria (5 listed)
- An individual who meets any of the following criteria will be excluded from participation in this
- Pregnancy or lactation
- Unable to comply with study procedures
- Inability to provide written informed consent
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
Death or total bilirubin >=4 at 91 days after the transplant
Time frame: 91 days after transplant
To determine whether, at 91 days after transplant, patients with liver disease at transplant are more likely to have died or have a total bilirubin >=4 than those without liver disease at transplant.
Mortality rate
Time frame: 91 days after transplant
To determine whether, at 91 days after transplant, patients with liver disease at transplant are more likely to have died or have a total bilirubin >=4 than those without liver disease at transplant.
4 secondary outcomes
Rate of infection (type: bacterial, viral, fungal; location: central line, organ infection, sepsis, etc.)
Time frame: 91 days after transplant
Development of portal hypertension and sequelae (i.e., ascites, variceal bleed, thrombocytopenia, elevated portal pressure)
Time frame: 91 days after transplant
Morbitity/cause of death
Time frame: 91 days after transplant
Development of liver failure
Time frame: 91 days after transplant
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2026.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: Undecided
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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.
Theo Heller, M.D. principal investigator
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hematopoietic Stem Cell Transplant, run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) at 1 site 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 3 inclusion and 5 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: opening soon, as of the sponsor's last update on 5 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jaha F Norman-Wheeler ((301) 435-6122, jaha.norman-wheeler@nih.gov), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Death or total bilirubin >=4 at 91 days after the transplant (time frame: 91 days after transplant). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 5 Nov 2026.