Ignite Creation Date:
2024-05-06 @ 6:37 PM
Last Modification Date:
2024-10-26 @ 2:51 PM
Study NCT ID:
NCT05722210
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-07-15
First Post:
2023-02-09
Brief Title:
Prevalence and Development of Liver Dysfunction in Hematopoietic Stem Cell Transplant
Sponsor:
National Institute of Diabetes and Digestive and Kidney Diseases NIDDK
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Prevalence and Development of Liver Dysfunction in Hematopoietic Stem Cell Transplant A Prospective Natural History Study
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-02-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 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
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
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
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 mgdL than those without liver disease at transplant
Secondary Objectives
To understand the impact of liver disease in HSCT on morbiditymortality
To understand the development and progression of liver disease in hematopoietic stem cell transplant
Tertiary Objectives
To identify predictiveprotective 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 mgdL at 3 months Visit 7 after the transplant
Mortality rate
Secondary Endpoints
Morbiditycause of death
Development of portal hypertension and sequelae ie ascites variceal bleed thrombocytopenia elevated portal pressure
Development of liver failure ie coagulopathy with an International Normalized Ration INR 15 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 mgdL 20-40 in HSCT recipients or INR 15
Portal hypertension Presenceabsence of any of the following will qualify as portal hypertension- ascites collateral vessels elevated portal pressure
Liver injury ALT4 times the upper limit of normal 22 IUL in women 29 IUL in men or Alkaline phosphatase 25 times the upper limit of normal
Tertiary Endpoints
Imaging laboratory analysis of liver dysfunction liver tissue pathology medicationstreatment course will be reviewed
Tertiary studies include stool microbiome studies metabolomics microbial translocation markers flow cytometry transcriptomics growth factor measurement cytokines and chemokines measurement
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 mgdL than those without liver disease at transplant
Secondary Objectives
To understand the impact of liver disease in HSCT on morbiditymortality
To understand the development and progression of liver disease in hematopoietic stem cell transplant
Tertiary Objectives
To identify predictiveprotective 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 mgdL at 3 months Visit 7 after the transplant
Mortality rate
Secondary Endpoints
Morbiditycause of death
Development of portal hypertension and sequelae ie ascites variceal bleed thrombocytopenia elevated portal pressure
Development of liver failure ie coagulopathy with an International Normalized Ration INR 15 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 mgdL 20-40 in HSCT recipients or INR 15
Portal hypertension Presenceabsence of any of the following will qualify as portal hypertension- ascites collateral vessels elevated portal pressure
Liver injury ALT4 times the upper limit of normal 22 IUL in women 29 IUL in men or Alkaline phosphatase 25 times the upper limit of normal
Tertiary Endpoints
Imaging laboratory analysis of liver dysfunction liver tissue pathology medicationstreatment 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 Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 000545-DK | None | None | None |