Clinical Trials Logo

Clinical 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. 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. ...


Clinical Trial 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


Related Conditions & MeSH terms


NCT number NCT05722210
Study type Observational
Source National Institutes of Health Clinical Center (CC)
Contact Shani C Scott, R.N.
Phone (301) 435-6121
Email shani.scott@nih.gov
Status Not yet recruiting
Phase
Start date June 26, 2024
Completion date June 30, 2031

See also
  Status Clinical Trial Phase
Withdrawn NCT02451462 - Pilot Trial of Zoledronic Acid to Prevent Bone Loss in Hematopoietic Cell Transplant Recipients N/A
Terminated NCT01215981 - Influenza Vaccine Post Allogeneic Transplant N/A
Recruiting NCT03613532 - Venetoclax Added to Fludarabine + Busulfan Prior to Transplant and to Maintenance Therapy for AML, MDS, and MDS/MPN Phase 1
Completed NCT01337648 - Radiation Biodosimetry in Children Undergoing Total Body Irradiation N/A
Recruiting NCT03250546 - Haploidentical and Mismatched Unrelated Donors Hematopoietic Stem Cell Transplant Phase 2
Recruiting NCT02982902 - T Cell Therapy of Opportunistic Cytomegalovirus Infection Early Phase 1
Completed NCT02441075 - 70% Ethanol for Decontamination of CVL Exposed to Calcineurine Inhibitors Version 1.0, 1/9/2014 N/A
Completed NCT04549038 - Time Restricted Nutrition in Pediatric Stem Cell Transplant Recipients N/A
Completed NCT03886909 - Impact of Prehabilitation in Oncology Via Exercise- Bone Marrow Transplant N/A
Not yet recruiting NCT06328127 - Positive Psychology Intervention for Hematopoietic Stem Cell Transplantation Survivors N/A
Completed NCT03509051 - Prospective Study on the Vaccine Response to Meningococcal B Vaccine After Allogeneic Stem Cell Transplantation N/A
Terminated NCT02350777 - T-cell Depleted Hematopoietic Stem Cell Boosts Without Conditioning for Poor Marrow Graft Function Following Allogeneic Hematopoietic Stem Cell Transplantation Phase 2
Completed NCT04853277 - Patient Reported Outcomes and Patient Education in Cellular Therapy Patients N/A
Completed NCT03378089 - Music Therapy and Hematopoietic Stem Cell Transplant N/A
Completed NCT03039257 - Vitamin A Replacement in Patients Undergoing HSCT and Its Role on MBI-LCBI Rates N/A
Completed NCT03202849 - A Randomized Trial of Vitamin D Supplementation With or Without Vitamin A in Stem Cell Transplantation N/A
Completed NCT01581164 - Studies of the Immune Response to Vaccination After Hematopoietic Stem Cell Transplantation N/A
Completed NCT01504152 - International Travel Patterns and Health Preparations of Hematopoietic Stem Cell Transplant Recipients at Memorial Sloan-Kettering Cancer Center N/A
Completed NCT03759262 - Ultra-high Dose Vitamin D for HSCT Phase 1
Recruiting NCT04263597 - Oral Supplementation of 2'-Fucosyllactose in Allogeneic Bone Marrow Transplant Recipients Phase 1/Phase 2