Fontan Udenafil Exercise Longitudinal Assessment Trial - 2

NCT05918211 | Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: MZ_Udenafil-05
• Study Type: interventional
• Target: 436
• Locations: 2 countries
• Sites: 40

Brief Summary

This study will evaluate the clinical efficacy and safety of udenafil, an orally administered, potent and selective inhibitor of PDE5, versus placebo for the treatment of adolescent who have had the Fontan procedure.

Conditions

Single Ventricle Heart Disease

Keywords

Fontan; Maximal Oxygen Consumption; Work Rate; VO2 at VAT; Ventilatory Efficiency

Outcome Measures

Primary Outcomes (1)

• Change in Exercise Capacity

  The change in exercise capacity (as measured by maximal VO2 at maximum exercise effort) from baseline to 26 weeks (or study completion)

  Baseline to 26 Weeks

Secondary Outcomes (4)

• Change in VO2 at the ventilatory anaerobic threshold (VAT)

  Baseline to 26 weeks

• Change in Enhanced Liver Fibrosis (ELF) Score

  Baseline to 26 weeks

• Work rate (watts) at ventilatory anaerobic threshold (VAT)

  Baseline to 26 weeks

• Change in ventilatory efficiency (VE/VCO2) at ventilatory anaerobic threshold

  Baseline to 26 weeks

Study Arms (2)

Drug

EXPERIMENTAL

Approximately 218 subjects dosed with udenafil will be enrolled at approximately 30 sites.

Drug: Udenafil

Placebo

EXPERIMENTAL

Approximately 218 subjects dosed with matching placebo will be enrolled at approximately 30 sites.

Drug: Placebo

Interventions

Udenafil DRUG

Active drug

Drug

Placebo DRUG

Matching Placebo

Placebo

Eligibility Criteria

• Age: 12 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Males and females with Fontan physiology who are 12 to less than 19 years of age at enrollment.
• Participant consent or parental/guardian consent and participant assent.
• Participant fluency in primary language of country in which study is being conducted.
• Current antiplatelet or anticoagulant therapy.

You may not qualify if:

• Height \< 132 cm.
• Weight \< 40 kg.
• Hospitalization for acute decompensated heart failure within the last 12 months.
• Current intravenous inotropic drugs.
• Undergoing evaluation for heart transplantation or listed for transplantation.
• Diagnosis of active protein losing enteropathy or plastic bronchitis within the last 3 years, or a history of liver cirrhosis.
• Known Fontan baffle obstruction, branch pulmonary artery stenosis, or pulmonary vein stenosis resulting in a mean gradient of \> 4 mmHg between the regions proximal and distal to the obstruction as measured by either catheterization or echocardiography, obtained prior to screening for the trial.
• Single lung physiology with greater than 80% flow to one lung.
• Failure to achieve maximal exertion (defined as RER \< 1.10) on screening/baseline exercise test.
• Peak minute oxygen consumption (VO2) less than 45% or ≥ 80% of predicated for age and gender at enrollment.
• Severe ventricular dysfunction assessed qualitatively by clinical echocardiography within 6 months prior to enrollment.
• Severe valvar regurgitation, ventricular outflow obstruction, or severe aortic arch obstruction assessed by clinical echocardiography within six months prior to enrollment.
• History of significant renal (serum creatinine \> 2.0), hepatic (serum AST and/or ALT \> 3 times upper limit of normal), gastrointestinal or biliary disorders that could impair absorption, metabolism or excretion of orally administered medications.
• Inability to complete exercise testing at baseline screening.
• Subjects with a pacemaker whose heart rate at peak exercise is controlled by the extrinsic pacemaker as opposed to a native atrial rhythm.

Sponsors & Collaborators

• Mezzion Pharma Co. Ltd: lead

Study Sites (40)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

RECRUITING

Arkansas Children's

Little Rock, Arkansas, 72202, United States

RECRUITING

Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States

RECRUITING

Rady Children's Hospital

San Diego, California, 92123, United States

RECRUITING

UCSF Benioff Children's Hospital

San Francisco, California, 94158, United States

RECRUITING

Children's Hospital of Colorado

Denver, Colorado, 80045, United States

RECRUITING

Yale School of Medicine

New Haven, Connecticut, 06510, United States

RECRUITING

Nemours Children's Hospital

Wilmington, Delaware, 19803, United States

RECRUITING

Childrens National Medical Center

Washington D.C., District of Columbia, 20010, United States

RECRUITING

UF Health Shands Hospital

Gainesville, Florida, 32608, United States

RECRUITING

Joe DiMaggio Children's Hospital

Hollywood, Florida, 33021, United States

RECRUITING

Johns Hopkins All Children's Hospital

St. Petersburg, Florida, 33701, United States

RECRUITING

Children's Healthcare of Atlanta

Atlanta, Georgia, 30329, United States

RECRUITING

Children's Hospital of Georgia

Augusta, Georgia, 30912, United States

RECRUITING

Lurie Children's Hospital

Chicago, Illinois, 60611, United States

RECRUITING

University of Chicago

Chicago, Illinois, 60637, United States

RECRUITING

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

RECRUITING

University of Michigan

Ann Arbor, Michigan, 48109, United States

RECRUITING

Children's Mercy Hospital Kansas City

Kansas City, Missouri, 64108, United States

RECRUITING

Washington University

St Louis, Missouri, 63130, United States

RECRUITING

University of Nebraska Children's Hospital and Medical Center

Omaha, Nebraska, 68114, United States

RECRUITING

Mt. Sinai Children's Hospital

New York, New York, 10029, United States

NOT YET RECRUITING

New York-Presbyterian Children's Hospital

New York, New York, 10032, United States

RECRUITING

Dana.Amaro@atriumhealth.org

Charlotte, North Carolina, 28204, United States

NOT YET RECRUITING

Duke University Medical Center

Durham, North Carolina, 27710, United States

RECRUITING

Cincinnati Childrens Hospital Medical Center

Cincinnati, Ohio, 45206, United States

RECRUITING

Cleveland Clinic

Cleveland, Ohio, 44195, United States

RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, 43215, United States

RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

RECRUITING

MUSC Pediatric Research Group

Charleston, South Carolina, 29425, United States

RECRUITING

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

RECRUITING

UT Southwestern Medical Center

Dallas, Texas, 75235, United States

RECRUITING

Texas Children's Hospital

Houston, Texas, 77030, United States

RECRUITING

Primary Children's Medical Center

Salt Lake City, Utah, 84113, United States

RECRUITING

Seattle Children's Hospital

Seattle, Washington, 98105, United States

RECRUITING

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53226, United States

RECRUITING

Sejong General Hospital

Bucheon-si, Gyeonggi-do, 14754, South Korea

RECRUITING

Seoul National University Children's Hospital

Seoul, 03080, South Korea

RECRUITING

Yonsei Severance Hospital

Seoul, 03722, South Korea

RECRUITING

MeSH Terms

Interventions

udenafil

Study Officials

• WG Kim

  Mezzion Pharma Co. Ltd

  STUDY DIRECTOR

Central Study Contacts

WG Kim

CONTACT

443.699.6746; wgkim@mezzion.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Active and matching placebo tablets will be identical in appearance.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Randomized, double-blind, placebo-controlled safety and efficacy study of udenafil versus placebo of approximately 436 subjects enrolled equally between the two arms (udenafil and placebo)

Study Record Dates

• First Submitted: June 15, 2023
• First Posted: June 26, 2023
• Study Start: October 30, 2023
• Primary Completion: October 28, 2025
• Study Completion: October 28, 2025
• Last Updated: October 28, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (37); KR (3)