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

Mezzion Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Single Ventricle Heart Disease

Treatments: Drug: Udenafil

Study type: Interventional

Funder types: Industry; NIH

Identifiers

NCT03430583

U01HL068270 (U.S. NIH Grant/Contract)

PHN-Udenafil-04

Details and patient eligibility

About

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

Full 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.

Enrollment: 88 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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88 participants in 2 patient groups

Udenafil Continuation (U+)

Experimental group

Description:

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

Treatment:

Drug: Udenafil

Udenafil Naive (U-)

Experimental group

Description:

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

Treatment:

Drug: Udenafil