Prevalence of Liver Disease in Patients Dependent on Parenteral Nutrition

NCT ID: NCT05011370

Last Updated: 2023-10-03

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 80 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2021-08-23
• Study Completion Date: 2023-06-29

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).

Detailed Description

Eligible participants will provide blood samples at Visit 1 to determine choline deficiency, elevated serum ALP liver isoenzyme level and liver dysfunction, and complete one imaging study (MRI-PDFF) at Visit 1 to assess steatosis.

The purpose of this study is to understand and document critical epidemiological data related to choline deficiency and the incidence of liver disease in patients dependent on PN (≥ 4 days a week) and to better understand this patient population to help determine who might benefit from innovative treatments including IV Choline Chloride treatment.

Conditions

Intestinal Failure-associated Liver Disease

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Eligibility Criteria

Inclusion Criteria

• 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

Exclusion Criteria

• 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.

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Protara Therapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Chief Scientific Operations Officer

Role: STUDY_DIRECTOR

Protara Therapeutics

Locations

Ronald Reagan Medical Center of UCLA

Los Angeles, California, United States

IHS Health

Kissimmee, Florida, United States

The University of Chicago

Chicago, Illinois, United States

Mayo Clinic

Rochester, Minnesota, United States

Duke University Medical Center

Durham, North Carolina, United States

Cincinnati Children's Hospital

Cincinnati, Ohio, United States

Cleveland Clinic Foundation

Cleveland, Ohio, United States

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Virginia Commonwealth University

Richmond, Virginia, United States

Seattle Children's

Seattle, Washington, United States

Rigshospitalet

Copenhagen, , Denmark

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

Bologna, , Italy

St. Mark's Hospital

Harrow, England, United Kingdom

University College London Hospitals

London, England, United Kingdom

Northern Care Alliance NHS Foundation

Salford, England, United Kingdom

Countries

United States; Denmark; Italy; United Kingdom

Other Identifiers

TARA-001-502

Identifier Type: -

Identifier Source: org_study_id