An observational study in Hematopoietic Stem Cell Transplant, sponsored by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK). Not yet recruiting at 1 site in United States. Open to participants aged 3 Years to 90 Years. Per ClinicalTrials.gov, last updated 2026-11-10.
Sponsored by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) · Observational
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.
...
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:
Secondary Endpoints:
Liver dysfunction, characterized by development of the following conditions:
Tertiary Endpoints:
824 studies on the registry are indexed under Liver Diseases; 152 are open to participants now.
This study's planned enrollment of 500 is above the median of 200 across 262 observational studies indexed under Liver Diseases.
Browse Liver Diseases studies →National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) is the lead sponsor of 538 studies on the registry; 55 are open to participants now.
Of its 80 completed or terminated interventional studies of FDA-regulated products, 51 (64%) have results posted.
Counted across the registry records on this site, refreshed daily.
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
An individual who meets any of the following criteria will be included in this study:
EXCLUSION CRITERIA:
An individual who meets any of the following criteria will be excluded from participation in this
study:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
undergoing evaluation for hematopoietic stem cell transplant at the NIH Clinical Center
undergoing evaluation for hematopoietic stem cell transplant at the NIH Clinical Center
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.
Time frame: 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.
Time frame: 91 days after transplant
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
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.
Time frame: 91 days after transplant
Plan to share: Undecided
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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