Evaluation of Fontan-Associated Liver Disease

Completed

• Conditions: Single Ventricle Heart Disease
• Interventions: Drug: MZ101

• Registration Number: NCT03430583
• Lead Sponsor: Mezzion Pharma Co. Ltd

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.

Study Timeline

• Study First Submitted: 2018-01-22
• Study First Submitted QC: 2018-02-04
• Study First Posted: 2018-02-13
• Study Start: 2018-02-20
• Primary Completion: 2020-09-15
• Study Completion: 2020-09-16
• Status Verified: 2025-04
• Last Update Submitted: 2025-05-01
• Last Update Posted: 2025-05-06

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 88

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
MZ101	MZ101	Dosing per treatment regimen

Primary Outcome Measures

Name	Time	Method
Range of Liver Stiffness in Adolescents with Single Ventricle Heart Disease	12 months	Liver Stiffness as measured by Shear Wave Ultrasound Elastography (meters/second) or Magnetic Resonance Ultrasound (kilopascals).

Secondary Outcome Measures

Name	Time	Method
Effect of Drug Therapy on Liver Stiffness	12 months	Liver Stiffness as measured by Shear Wave Ultrasound Elastography (meters/second) or Magnetic Resonance Ultrasound (kilopascals).
Effect of Drug Therapy on Enhanced Liver Fibrosis (ELF) Score	12 months	Measurement of ELF Score
Effect of Drug Therapy on Brain Type Natriuretic Peptide (BNP) Levels	12 months	Measurement of plasma levels of BNP
Effect of Drug Therapy on n-Terminal BNP (NT-proBNP)	12 months	Measurement of plasma levels of NT-proBNP
Effect of Drug Therapy on MicroRNA Measures.	12 months	Measurement of MicroRNA by miRNA qRT-PCR analysis of total RNA from plasma

Trial Locations

Locations (17)

Children's Hospital Colorado; 🇺🇸 Aurora, Colorado, United States

Nemours Cardiac Center/Alfred I. duPont Hospital for Children; 🇺🇸 Wilmington, Delaware, United States

Children's National Medical Center; 🇺🇸 Washington, District of Columbia, United States

Children's Mercy Hospital; 🇺🇸 Kansas City, Kansas, United States

University of Michigan Congenital Heart Center; 🇺🇸 Ann Arbor, Michigan, United States

Children's Mercy Hospital Kansas City; 🇺🇸 Kansas City, Missouri, United States

Medical University of South Carolina; 🇺🇸 Charleston, South Carolina, United States

Children's Healthcare of Atlanta; 🇺🇸 Atlanta, Georgia, United States

Cincinnati Children's Hospital Medical Center; 🇺🇸 Cincinnati, Ohio, United States

Boston Children's Hospital; 🇺🇸 Boston, Massachusetts, United States

Texas Children's Hospital; 🇺🇸 Houston, Texas, United States

Primary Children's Medical Hospital/Dept. of Pediatric Cardiology; 🇺🇸 Salt Lake City, Utah, United States

Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

Vanderbilt University Medical Center; 🇺🇸 Nashville, Tennessee, United States

The Hospital for Sick Children; 🇨🇦 Toronto, Ontario, Canada

University of Nebraska Children's Hospital and Medical Center; 🇺🇸 Omaha, Nebraska, United States

Children's Hospital of Wisconsin; 🇺🇸 Milwaukee, Wisconsin, United States