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Completed

NCT Number: NCT03430583

Evaluation of Fontan-associated Hepatic Stiffness and the Efficacy of Udenafil in Reducing Liver Stiffness

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

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Key information

Conditions

Single Ventricle Heart Disease

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

The Hospital for Sick Children, Toronto, Ontario, Canada

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

  • 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

Treatment and study plan

Udenafil

Drug

Open-label extension with Udenafil 87.5 mg BID treatment for 52 weeks.

Primary outcomes

  1. Effect of Udenafil 87.5 mg BID Treatment on Liver Stiffness in Udenafil Continuation and Naive Groups After 52 Weeks, as Determined by Ultrasound Shear Wave Elastography

    Time frame: 52 weeks

    Liver stiffness was measured at baseline and 52 weeks using Ultrasound Shear Wave Elastography (USWE) separately in Udenafil Continuation (U+) and Udenafil Naive (U-) groups. A decrease in value indicates a decrease in liver stiffness.

  2. Effect of Udenafil 87.5 mg BID Treatment on Liver Stiffness in Udenafil Continuation and Naive Groups After 52 Weeks, as Determined by Magnetic Resonance Elastography

    Time frame: 52 weeks

    Liver stiffness was measured at baseline and 52 weeks using Magnetic Resonance Elastography (MRE) separately in Udenafil Continuation (U+) and Udenafil Naive (U-) groups. A decrease in value indicates a decrease in liver stiffness.

Secondary outcomes

  1. Effect of Udenafil 87.5 mg BID on Enhanced Liver Fibrosis (ELF) Score After 52 Weeks of Treatment

    Time frame: 52 weeks

    Overall ELF score. ELF score is based on the serum levels of hyaluronic acid (HA), amino-terminal propeptide of type III collagen (PIIINP), and tissue inhibitor of metalloproteinase-1 (TIMP-1), and calculated using the following formula: ELF score= 2.494+0.846 In(CHA)+0.391 In(CPIIINP)+0.391 In(CTIMP-1). The levels of each component were as assessed individually and as part of the calculated ELF score as continuous variables. The normal range in healthy subjects ≤20 years of age is 6.9 - 8.8. Correlations between ELF and the degree of liver fibrosis have not been established in patients with Fontan circulation, however, a decrease in ELF score is associated with improved liver health in other disease states.

  2. Effect of Udenafil 87.5 mg BID on Brain Natriuretic Peptide After 52 Weeks of Treatment

    Time frame: 52 weeks

    Brain Natriuretic Peptide (BNP) was measured at baseline and 52 weeks of treatment. BNP is a biomarker of overall cardiac function across a variety of disease states. A decrease in value is associated with improved cardiac function.

Sponsors and collaborators

Lead sponsor

Mezzion Pharma Co. Ltd

Industry

Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

Registry information

Official study title

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

Acronym: FALD

Important dates

Study start
2018
Primary completion
2020
Study completion
2020
First posted
Feb 13, 2018
Registry last updated
Oct 14, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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