Evaluation of Fontan-Associated Liver Disease
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 88
- Enrollment in on-going Phase 3 Open-Label Safety Study
- Informed assent from subject, informed consent from parent/legal guardian as appropriate
- Non-enrollment in the on-going Phase 3 Open-Label Study
- Subjects with contra-indications for MRI (these subjects will be excluded from the MRI component of this study)
- 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 Healthcare of Atlanta
🇺🇸Atlanta, Georgia, United States
Children's Mercy Hospital
🇺🇸Kansas City, Kansas, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
University of Michigan Congenital Heart Center
🇺🇸Ann Arbor, Michigan, United States
Children's Mercy Hospital Kansas City
🇺🇸Kansas City, Missouri, United States
University of Nebraska Children's Hospital and Medical Center
🇺🇸Omaha, Nebraska, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
Scroll for more (7 remaining)Children's Hospital Colorado🇺🇸Aurora, Colorado, United States