Evaluation of Fontan-Associated Liver Disease

Single Ventricle Heart Disease Clinical Trial

— FALD  

Official title:

Imaging and Biomarker Evaluation of Hepatic Stiffness in Children Enrolled in the Fontan Udenafil Exercise Longitudinal Study

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03430583
• Other study ID #: PHN-Udenafil-04
• Secondary ID: U01HL068270
• Status: Active, not recruiting
• Start date: February 20, 2018
• Est. completion date: August 31, 2021

Study information

• Verified date: November 2020
• Source: Mezzion Pharma Co. Ltd
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 100
• Est. completion date: August 31, 2021
• Est. primary completion date: June 30, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Heart Diseases
• Single Ventricle Heart Disease

Intervention

Drug:
• MZ101
Drug

Locations

Country	Name	City	State
Canada	The Hospital for Sick Children	Toronto	Ontario
United States	University of Michigan Congenital Heart Center	Ann Arbor	Michigan
United States	Children's Healthcare of Atlanta	Atlanta	Georgia
United States	Children's Hospital Colorado	Aurora	Colorado
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Medical University of South Carolina	Charleston	South Carolina
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Texas Children's Hospital	Houston	Texas
United States	Children's Mercy Hospital	Kansas City	Kansas
United States	Children's Mercy Hospital Kansas City	Kansas City	Missouri
United States	Children's Hospital of Wisconsin	Milwaukee	Wisconsin
United States	Vanderbilt University Medical Center	Nashville	Tennessee
United States	University of Nebraska Children's Hospital and Medical Center	Omaha	Nebraska
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Primary Children's Medical Hospital/Dept. of Pediatric Cardiology	Salt Lake City	Utah
United States	Children's National Medical Center	Washington	District of Columbia
United States	Nemours Cardiac Center/Alfred I. duPont Hospital for Children	Wilmington	Delaware

Sponsors (2)

Lead Sponsor	Collaborator
Mezzion Pharma Co. Ltd	National Heart, Lung, and Blood Institute (NHLBI)

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Range of Liver Stiffness in Adolescents with Single Ventricle Heart Disease	Liver Stiffness as measured by Shear Wave Ultrasound Elastography (meters/second) or Magnetic Resonance Ultrasound (kilopascals).	12 months
Secondary	Effect of Drug Therapy on Liver Stiffness	Liver Stiffness as measured by Shear Wave Ultrasound Elastography (meters/second) or Magnetic Resonance Ultrasound (kilopascals).	12 months
Secondary	Effect of Drug Therapy on Brain Type Natriuretic Peptide (BNP) Levels	Measurement of plasma levels of BNP	12 months
Secondary	Effect of Drug Therapy on n-Terminal BNP (NT-proBNP)	Measurement of plasma levels of NT-proBNP	12 months
Secondary	Effect of Drug Therapy on MicroRNA Measures.	Measurement of MicroRNA by miRNA qRT-PCR analysis of total RNA from plasma	12 months
Secondary	Effect of Drug Therapy on Enhanced Liver Fibrosis (ELF) Score	Measurement of ELF Score	12 months