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

NCT ID: NCT03430583

Last Updated: 2025-10-14

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

88 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-02-20

Study Completion Date

2020-09-16

Brief Summary

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

Detailed Description

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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.

Conditions

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Single Ventricle Heart Disease

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Udenafil Continuation (U+)

Subjects who were receiving Udenafil 87.5 mg BID in FUEL study prior to enrolling to this study (FALD).

Group Type EXPERIMENTAL

Udenafil

Intervention Type DRUG

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

Udenafil Naive (U-)

Subjects who were receiving Placebo BID in FUEL study prior to enrolling to this study (FALD).

Group Type EXPERIMENTAL

Udenafil

Intervention Type DRUG

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

Interventions

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Udenafil

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

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role collaborator

Mezzion Pharma Co. Ltd

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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David J Goldberg, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital of Philadelphia

Kurt R Schumacher, MD

Role: PRINCIPAL_INVESTIGATOR

University of Michigan Congenital Heart Center/C.S. Mott Children's Hospital

Locations

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Children's Hospital Colorado

Aurora, Colorado, United States

Site Status

Nemours Cardiac Center/Alfred I. duPont Hospital for Children

Wilmington, Delaware, United States

Site Status

Children's National Medical Center

Washington D.C., District of Columbia, United States

Site Status

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Site Status

Children's Mercy Hospital

Kansas City, Kansas, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

University of Michigan Congenital Heart Center

Ann Arbor, Michigan, United States

Site Status

Children's Mercy Hospital Kansas City

Kansas City, Missouri, United States

Site Status

University of Nebraska Children's Hospital and Medical Center

Omaha, Nebraska, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

Texas Children's Hospital

Houston, Texas, United States

Site Status

Primary Children's Medical Hospital/Dept. of Pediatric Cardiology

Salt Lake City, Utah, United States

Site Status

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

The Hospital for Sick Children

Toronto, Ontario, Canada

Site Status

Countries

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United States Canada

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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U01HL068270

Identifier Type: NIH

Identifier Source: secondary_id

View Link

PHN-Udenafil-04

Identifier Type: -

Identifier Source: org_study_id

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