N/A N=28

Chronic Liver Disease in Urea Cycle Disorders

Urea Cycle Disorder

Bottom Line

View on ClinicalTrials.gov: NCT03721367 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Feb 2023

Primary outcome: Primary: Liver Stiffness as Measured by Shear Wave Elastography — 13; 14 Participants

Study Design & Population

Study type: Observational
Phase: N/A
Interventions: Diagnostic Ultrasound (Other)
Age: Pediatric, Adult · 5+ yrs
Sex: All
Sponsor: Baylor College of Medicine
Primary completion: Jun 2020

Outcome Measures

Outcome	Result	p-value
PRIMARY Liver Stiffness as Measured by Shear Wave Elastography	13; 14	—
PRIMARY Grey Scale Ultrasound Findings	15; 13	—
SECONDARY Fibrotest	19; 9	—

Summary

This is a pilot, cross-sectional study to assess liver stiffness and markers of hepatic injury, function, and fibrosis in patients with urea cycle disorders. This study will be conducted at 3 UCDC sites: Baylor College of Medicine in Houston, Texas, University of California San Francisco (UCSF), San Francisco, California and Seattle Children's Hospital, Seattle,Washington

Eligibility Criteria

Inclusion Criteria

Age > 5 years and < 60 years
Weight ≥ 11 kg
Males or females with a diagnosis of OTCD based on molecular or enzymatic testing. Males or females with a diagnosis of CPS1D, citrin deficiency, NAGSD, ASS1D, ASLD or ARG1D based on biochemical OR molecular, OR enzymatic testing

Exclusion Criteria

History of hyperammonemia (blood ammonia greater than 100 micromoles/L) documented in the medical record or reported by the patient in the 30 days preceding enrollment visit
History of Liver transplantation
Current pregnancy
Confirmed diagnosis of chronic viral hepatitis, autoimmune liver disease, or alcohol liver disease

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03721367). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.