Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
62 participants
OBSERVATIONAL
2021-11-04
2025-12-31
Brief Summary
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Detailed Description
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Currently, there are no guidelines for monitoring hepatic complications or extent of liver disease in UCDs. The gold standard for staging of fibrosis or confirming cirrhosis has traditionally been liver biopsy, an invasive procedure with inherent risks, particularly in the setting of a UCD and compromised coagulation. Recently, non-invasive serum and imaging-based biomarkers have been introduced to assess hepatic fibrosis in adults and children who are at increased risk. Utilization of these technique in individuals with UCDs could be invaluable in both the research and clinical arenas.
The purpose of this study is:
1\) To assess risk for increased fibrosis using serum biomarkers and/or VCTE in distal disorders (ASS1D, ASLD and ARG1D) as compared to OTCD 2 ) To assess risk for hepatic fibrosis (liver stiffness as measured by MRE) in individuals with UCDs who have abnormal serum biomarkers and/or VCTE as those who have normal values
Conditions
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Study Design
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OTHER
CROSS_SECTIONAL
Eligibility Criteria
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Inclusion Criteria
* Weight ≥ 11 kg at time of screening
* A molecular or biochemical diagnosis of OTCD, ASS1D, ASLD, or ARG1D.
• Participation in Stage A of this study
Exclusion Criteria
* 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
6 Years
65 Years
ALL
No
Sponsors
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Children's National Research Institute
OTHER
Seattle Children's Hospital
OTHER
Children's Hospital Colorado
OTHER
Children's Hospital of Philadelphia
OTHER
Baylor College of Medicine
OTHER
Responsible Party
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Lindsay Burrage
Principal Investigator
Principal Investigators
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Lindsay Burrage, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
Baylor College of Medicine
Locations
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Children's Hospital Colorado
Aurora, Colorado, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Baylor College of Medicine
Houston, Texas, United States
Seattle Children's Hospital
Seattle, Washington, United States
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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H-50295
Identifier Type: -
Identifier Source: org_study_id
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