Trial Outcomes & Findings for Chronic Liver Disease in Urea Cycle Disorders (NCT NCT03721367)

Last Updated: 2023-02-10

Results Overview

Shear wave elastography in m/s is a measure of liver stiffness, a surrogate measure for hepatic fibrosis. Normal liverstiffness is \< 1.35 m/s and abnormal liver stiffness is \>1.35 m/s

Recruitment status

COMPLETED

Target enrollment

28 participants

Primary outcome timeframe

One measurement made on the day of study visit

Results posted on

2023-02-10

Participant Flow

Participant milestones

Participant milestones
Measure	Urea Cycle Disorders Diagnostic Ultrasound: All individuals will undergo a blood draw for measurement of biomarkers of liver disease and an ultrasound with shear-wave elastography.
Overall Study STARTED	28
Overall Study COMPLETED	28
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Chronic Liver Disease in Urea Cycle Disorders

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Urea Cycle Disorders n=28 Participants Diagnostic Ultrasound: All individuals will undergo a blood draw for measurement of biomarkers of liver disease and an ultrasound with shear-wave elastography.
Age, Continuous	15 years n=5 Participants
Sex: Female, Male Female	16 Participants n=5 Participants
Sex: Female, Male Male	12 Participants n=5 Participants
Race/Ethnicity, Customized White	13 Participants n=5 Participants
Race/Ethnicity, Customized Native American	2 Participants n=5 Participants
Race/Ethnicity, Customized Hispanic	9 Participants n=5 Participants
Race/Ethnicity, Customized African-American	3 Participants n=5 Participants
Race/Ethnicity, Customized Asian	1 Participants n=5 Participants
Region of Enrollment United States	28 participants n=5 Participants
UCD diagnosis OTC Deficiency (Male)	3 Participants n=5 Participants
UCD diagnosis OTC Deficiency (Female)	7 Participants n=5 Participants
UCD diagnosis ASS1 Deficiency	4 Participants n=5 Participants
UCD diagnosis ASL Deficiency	8 Participants n=5 Participants
UCD diagnosis ARG1 Deficiency	6 Participants n=5 Participants
BMI Underweight	1 Participants n=5 Participants
BMI Healthy	12 Participants n=5 Participants
BMI Overweight	5 Participants n=5 Participants
BMI Obese	10 Participants n=5 Participants
History of Hyperammonemia	16 Participants n=5 Participants

PRIMARY outcome

Timeframe: One measurement made on the day of study visit

Population: The data for this outcome was reliable for only 27 participants.

Shear wave elastography in m/s is a measure of liver stiffness, a surrogate measure for hepatic fibrosis. Normal liverstiffness is \< 1.35 m/s and abnormal liver stiffness is \>1.35 m/s

Outcome measures

Outcome measures
Measure	Urea Cycle Disorders n=27 Participants Diagnostic Ultrasound: All individuals will undergo a blood draw for measurement of biomarkers of liver disease and an ultrasound with shear-wave elastography.
Liver Stiffness as Measured by Shear Wave Elastography Normal Liver Stiffness	13 Participants
Liver Stiffness as Measured by Shear Wave Elastography Abnormal Liver Stiffness	14 Participants

PRIMARY outcome

Timeframe: Baseline, once

Grey scale ultrasound findings

Outcome measures

Outcome measures
Measure	Urea Cycle Disorders n=28 Participants Diagnostic Ultrasound: All individuals will undergo a blood draw for measurement of biomarkers of liver disease and an ultrasound with shear-wave elastography.
Grey Scale Ultrasound Findings Abnormal hepatic parenchyma on grey scale ultrasound	13 Participants
Grey Scale Ultrasound Findings Normal hepatic parenchyma on grey scale ultrasound	15 Participants

SECONDARY outcome

Timeframe: Baseline, once

Fibrotest is a blood test that is a surrogate measure for hepatic fibrosis. F0 is normal. \>F0 predicts at least minimal fibrosis.

Outcome measures

Outcome measures
Measure	Urea Cycle Disorders n=28 Participants Diagnostic Ultrasound: All individuals will undergo a blood draw for measurement of biomarkers of liver disease and an ultrasound with shear-wave elastography.
Fibrotest F0	19 Participants
Fibrotest >F0	9 Participants

Adverse Events

Urea Cycle Disorders

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Lindsay Burrage

Baylor College of Medicine

Phone: 832-822-4280

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place