Clinical trial NCT03430583: Evaluation of Fontan-Associated Liver Disease
Official title: Imaging and Biomarker Evaluation of Hepatic Stiffness in Children Enrolled in the Fontan Udenafil Exercise Longitudinal Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Single Ventricle Heart Disease
Keywords: Fontan
Ultrasound
Magnetic Resonance Imaging Elastography
Lead sponsor: Mezzion Pharma Co. Ltd
Sponsor class: INDUSTRY
Collaborators: name: National Heart, Lung, and Blood Institute (NHLBI)
First posted: 2018-02-13
Enrollment: 88
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Male and Female subjects 12 - 19 years old with meet the inclusion/exclusion criteria.

Brief summary: A study to evaluate the efficacy of MZ101 therapy in reducing liver stiffness.

Detailed description: This is open-label, long-term prospective study in a cohort of Fontan subjects taking drug daily for one year. The study will determine the scope of hepatic stiffness in the cohort by ultrasound and magnetic resonance imaging elastography and evaluate MZ101 pharmacotherapy as a potentially efficacious treatment to reduce liver stiffness.

Eligibility criteria: Inclusion Criteria:

1. Enrollment in on-going Phase 3 Open-Label Safety Study
2. Informed assent from subject, informed consent from parent/legal guardian as appropriate

Exclusion Criteria:

1. Non-enrollment in the on-going Phase 3 Open-Label Study
2. Subjects with contra-indications for MRI (these subjects will be excluded from the MRI component of this study)
3. Other exclusionary criteria will match those used for the Open-Label Safety Study

Primary outcomes:
measure: Range of Liver Stiffness in Adolescents with Single Ventricle Heart Disease
timeFrame: 12 months
Secondary outcomes:
measure: Effect of Drug Therapy on Liver Stiffness
timeFrame: 12 months
measure: Effect of Drug Therapy on Brain Type Natriuretic Peptide (BNP) Levels
timeFrame: 12 months
measure: Effect of Drug Therapy on n-Terminal BNP (NT-proBNP)
timeFrame: 12 months
measure: Effect of Drug Therapy on MicroRNA Measures.
timeFrame: 12 months
measure: Effect of Drug Therapy on Enhanced Liver Fibrosis (ELF) Score
timeFrame: 12 months
Other outcomes:
Locations:
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Wilmington
country: United States
facility: Nemours Cardiac Center/Alfred I. duPont Hospital for Children
state: Delaware
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: Kansas City
country: United States
facility: Children's Mercy Hospital
state: Kansas
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan Congenital Heart Center
state: Michigan
city: Kansas City
country: United States
facility: Children's Mercy Hospital Kansas City
state: Missouri
city: Omaha
country: United States
facility: University of Nebraska Children's Hospital and Medical Center
state: Nebraska
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Salt Lake City
country: United States
facility: Primary Children's Medical Hospital/Dept. of Pediatric Cardiology
state: Utah
city: Milwaukee
country: United States
facility: Children's Hospital of Wisconsin
state: Wisconsin
city: Toronto
country: Canada
facility: The Hospital for Sick Children
state: Ontario
Study officials:
affiliation: Children's Hospital of Philadelphia
name: David J Goldberg, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Michigan Congenital Heart Center/C.S. Mott Children's Hospital
name: Kurt R Schumacher, MD
role: PRINCIPAL_INVESTIGATOR
References: