Prevalence of Liver Disease in Patients Dependent on Parenteral Nutrition

NCT05011370 | Completed

• 1 other identifier: TARA-001-502
• Study Type: observational
• Target: 80
• Locations: 4 countries
• Sites: 15

Brief Summary

This is a multi-center prospective cross-sectional observational study that will assess the prevalence of liver disease in patients dependent on parenteral nutrition (PN) for 4 or more days per week. Liver disease will be determined by the presence of choline deficiency, cholestasis (confirmed by elevated serum alkaline phosphatase (ALP) liver isoenzyme level), and steatosis (confirmed by magnetic resonance imaging-derived proton density fat fraction (MRI-PDFF). The objective of this study is to investigate the presence/prevalence of liver disease in patients dependent on PN (≥4 days a week).

Conditions

Intestinal Failure-associated Liver Disease

Keywords

Intestinal failure-associated liver disease (IFALD); IFALD; Liver disease; Parenteral nutrition; Choline deficiency; Hepatic steatosis; Cholestasis; parenteral nutrition-associated liver disease

Outcome Measures

Primary Outcomes (2)

• Percentage of patients with choline deficiency

  Choline deficiency is defined as plasma-free choline concentration less than 7 nmol/mL.

  Day 1

• Percentage of patients with choline deficiency, elevated serum ALP liver isoenzyme level and MRI-PDFF ≥8%

  Choline deficiency is defined as plasma-free choline concentration less than 7 nmol/mL. Elevated serum ALP liver isoenzyme level is defined as \>1.5x upper limit of normal. MRI-PDFF ≥8 %

  Day 1

Secondary Outcomes (3)

• Percentage of patients with MRI-PDFF ≥8%

  Day 1

• Percentage of patients with elevated serum ALP liver isoenzyme level (defined as ≥ 1.5x upper limit of normal)

  Day 1

• Percentage of patients with abnormal liver tests including serum direct bilirubin, AST, ALT and GGT

  Day 1

Eligibility Criteria

• Age: 12 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

This study will enroll male and female adults and adolescent participants 12 years and older who are dependent on PN (≥ 4 days a week) in the primary care clinic setting.

You may qualify if:

• The participant and/or their parent/Legally Authorized Representative is willing and able to provide signed informed consent or assent as appropriate
• Male or female adults 18 to 80 years of age, or adolescents 12 to 17 years of age
• Patients dependent on parenteral nutrition (PN) that receive PN for an average ≥ 4 days a week for 10 weeks or longer prior to screening to meet nutritional, caloric, fluid, and/or electrolyte needs
• The Investigator expects no changes in the lipid, dextrose, amino acid, or vitamin regimen to be medically necessary during the participant's participation in the study
• Willingness of participant to maintain his/her current habitual oral diet and fluids regimen for the study duration

You may not qualify if:

• Participants taking steatogenic medications for ≥12 weeks in the past 12 months (e.g., amiodarone, tamoxifen, methotrexate, tetracycline, glucocorticoids, anabolic steroids, over the usual dose of estrogen for hormone replacement therapy, and valproate); those taking any medicine (e.g., metformin, thiazolidinediones, ursodeoxycholic acid, pentoxifylline, S-adenosyl-L-Methionine, and betaine) that could affect the measurement of IFALD within 12 weeks prior to study entry
• Participants taking potential hepatotoxic medications that in the judgement of the Investigator is causing hepatic abnormalities
• Participants with a cardiac pacemaker, intravascular stents, other metallic devices, and claustrophobia which are contraindicated to magnetic resonance imaging
• Participants who took choline supplements or choline-containing multivitamins within 14 days of screening
• History of major organ transplant (e.g., heart, kidney, liver, etc.)
• For more information on eligibility criteria, please contact the sponsor.

Sponsors & Collaborators

• Protara Therapeutics: lead

Study Sites (15)

Ronald Reagan Medical Center of UCLA

Los Angeles, California, 90095, United States

Location

IHS Health

Kissimmee, Florida, 34741, United States

Location

The University of Chicago

Chicago, Illinois, 60637, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Cincinnati Children's Hospital

Cincinnati, Ohio, 45229, United States

Location

Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

Location

Seattle Children's

Seattle, Washington, 98105, United States

Location

Rigshospitalet

Copenhagen, Denmark

Location

Center for Chronic Intestinal Failure, St. Orsola Hospital Dept of Medical and Surgical Sciences, University of Bologna

Bologna, Italy

Location

St. Mark's Hospital

Harrow, England, HA1 3UJ, United Kingdom

Location

University College London Hospitals

London, England, NW1 2BU, United Kingdom

Location

Northern Care Alliance NHS Foundation

Salford, England, United Kingdom

Location

MeSH Terms

Conditions

Liver Diseases; Hyperphagia; Choline Deficiency; Fatty Liver; Cholestasis

Condition Hierarchy (Ancestors)

Digestive System Diseases; Signs and Symptoms, Digestive; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Vitamin B Deficiency; Avitaminosis; Deficiency Diseases; Malnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases; Bile Duct Diseases; Biliary Tract Diseases

Study Officials

• Chief Scientific Operations Officer

  Protara Therapeutics

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 11, 2021
• First Posted: August 18, 2021
• Study Start: August 23, 2021
• Primary Completion: June 29, 2023
• Study Completion: June 29, 2023
• Last Updated: October 3, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will not share

Locations

IT (1); DK (1); US (10); GB (3)