RECRUITING

Liver Disease in Urea Cycle Disorders

Conditions

Urea Cycle Disorder Ornithine Transcarbamylase Deficiency Citrullinemia 1 ARGI Deficiency ASL Deficiency Argininosuccinic Aciduria ASS Deficiency Hyperargininemia Liver disease Fibroscan Magnetic resonance elastography Fibrotest

Quick Navigation

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

This is a multi-center, cross-sectional study to assess risk for liver fibrosis and hepatic injury in individuals with urea cycle disorders (UCDs) using serum biomarkers, Fibroscan, and MRE. This study will be conducted at 5 sites of the Urea Cycle Disorders Consortium: Baylor College of Medicine in Houston, TX, Seattle Children's Hospital in Seattle, WA, Children's Hospital Colorado in Aurora, CO, Children's Hospital of Philadelphia in Philadelphia, PA, and Children's National Medical Center in Washington D.C.

Official Title

Noninvasive Biomarkers of Hepatic Fibrosis in Urea Cycle Disorders

Quick Facts

Study Start:2021-11-04

Study Completion:2024-12-31

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT04612764

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:6 Years to 65 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
* Age \> 6 years and \< 65 years * Weight ≥ 11 kg at time of screening * A molecular or biochemical diagnosis of OTCD, ASS1D, ASLD, or ARG1D.	* Prior liver transplantation * Episode of acute hyperammonemia (≥100 umol/L) in the 30 days prior to enrollment * Confirmed diagnosis of chronic viral hepatitis, autoimmune liver disease, short gut, small bowel syndrome, alcohol liver disease, TPN requirement, or TPN-related cholestatic disease * Adults with BMI ≥ 45 kg/m2 * Current pregnancy * Open wound near expected Fibroscan® probe application site * Use of implantable active medical device such as cardiac pacemaker or implantable cardioverter-defibrillator * Individuals with claustrophobia or other inability to complete * Known diagnosis of hemochromatosis * Presence of implants or devices incompatible with MRI * Inability to breath-hold for 20 seconds for the elastography sequence * Current pregnancy * Confirmed diagnosis of chronic viral hepatitis, autoimmune liver disease, short gut, small bowel syndrome, alcohol liver disease, TPN requirement, or TPN-related cholestatic disease * Episode of documented acute hyperammonemia (ammonia ≥ 100 umol/L) in the 30 days prior to scheduled visit for Stage B

Inclusion Criteria

Exclusion Criteria

* Age \> 6 years and \< 65 years
* Weight ≥ 11 kg at time of screening
* A molecular or biochemical diagnosis of OTCD, ASS1D, ASLD, or ARG1D.

* Prior liver transplantation
* Episode of acute hyperammonemia (≥100 umol/L) in the 30 days prior to enrollment
* Confirmed diagnosis of chronic viral hepatitis, autoimmune liver disease, short gut, small bowel syndrome, alcohol liver disease, TPN requirement, or TPN-related cholestatic disease
* Adults with BMI ≥ 45 kg/m2
* Current pregnancy
* Open wound near expected Fibroscan® probe application site
* Use of implantable active medical device such as cardiac pacemaker or implantable cardioverter-defibrillator
* Individuals with claustrophobia or other inability to complete
* Known diagnosis of hemochromatosis
* Presence of implants or devices incompatible with MRI
* Inability to breath-hold for 20 seconds for the elastography sequence
* Current pregnancy
* Confirmed diagnosis of chronic viral hepatitis, autoimmune liver disease, short gut, small bowel syndrome, alcohol liver disease, TPN requirement, or TPN-related cholestatic disease
* Episode of documented acute hyperammonemia (ammonia ≥ 100 umol/L) in the 30 days prior to scheduled visit for Stage B

Contacts and Locations

Study Contact

Saima Ali, MSN

CONTACT

832-822-4183

saima.ali@bcm.edu

Principal Investigator

Lindsay Burrage, MD, PhD

PRINCIPAL_INVESTIGATOR

Baylor College of Medicine

Study Locations (Sites)

Children's Hospital Colorado

Aurora, Colorado, 80045

United States

Children's National Medical Center

Washington, District of Columbia, 20010

United States

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104

United States

Baylor College of Medicine

Houston, Texas, 77030

United States

Seattle Children's Hospital

Seattle, Washington, 98105

United States

Collaborators and Investigators

Sponsor: Baylor College of Medicine

Lindsay Burrage, MD, PhD, PRINCIPAL_INVESTIGATOR, Baylor College of Medicine

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-11-04

Study Completion Date2024-12-31

Study Record Updates

Study Start Date2021-11-04

Study Completion Date2024-12-31

Terms related to this study

Keywords Provided by Researchers

Liver disease
Fibroscan
Magnetic resonance elastography
Fibrotest

Additional Relevant MeSH Terms

Urea Cycle Disorder
Ornithine Transcarbamylase Deficiency
Citrullinemia 1
ARGI Deficiency
ASL Deficiency
Argininosuccinic Aciduria
ASS Deficiency
Hyperargininemia