A Extension Study of Udenafil in Adolescents

NCT03013751 | Completed Phase 3 Results DMC FDA Drug

• 2 other identifiers: PHN-Udenafil-03U01HL068270
• Study Type: interventional
• Target: 301
• Locations: 3 countries
• Sites: 24

Brief Summary

This study is a 12-month (52 week) safety extension study to supplement the FUEL Phase III clinical trial to provide safety information regarding the long-term use of udenafil in adolescents with single ventricle congenital heart disease.

Conditions

Functional Single Ventricle Heart Disease

Outcome Measures

Primary Outcomes (1)

• Treatment-Emergent Adverse Events

  Summary of Treatment Emergent Adverse Events

  52 Weeks

Secondary Outcomes (10)

• Change in Maximal VO2

  52 Weeks

• Change in Log-transformed Reactive Hyperemia Index (lnRHI)

  52 Weeks

• Change in Serum BNP

  52 Weeks

• Change in Myocardial Performance Index (MPI)

  52 Weeks

• Change in PedsQL Physical Functioning (Child Reported)

  52 Weeks

Study Arms (1)

Drug

EXPERIMENTAL

Udenafil administered for 52 weeks

Drug: Udenafil

Interventions

Udenafil DRUG

Active drug

Drug

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 participated in the FUEL trial or, if they did not participate in FUEL, those who are 12 to less than 19 years of age at enrollment.
• Participant consent or parental/guardian consent and participant assent.
• Participant fluent in English, Spanish, or Korean.
• Current anti-platelet 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 three 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 mm Hg between the regions proximal and distal to the obstruction as measured by either catheterization or echocardiography.
• Single lung physiology.
• Maximal VO2 less than 50% of predicted for age and gender at enrollment.
• Severe ventricular dysfunction assessed qualitatively by clinical echocardiography within six months prior to enrollment.
• Severe valvar regurgitation, ventricular outflow obstruction, or aortic arch obstruction assessed by clinical echocardiography within six months prior to enrollment.
• Significant renal, hepatic, gastrointestinal or biliary disorders that could impair absorption, metabolism or excretion of orally administered medications.
• Inability to complete exercise testing at baseline screening.
• History of PDE-5 inhibitor use (with the exception of FUEL participation) within 3 months before study onset.
• Use of any other drug to treat pulmonary hypertension within 3 months before study onset.

Sponsors & Collaborators

• Mezzion Pharma Co. Ltd: lead
• National Heart, Lung, and Blood Institute (NHLBI): collaborator

Study Sites (24)

Phoenix Children's Hospital/Children's Heart Center at Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

Cedars/Sinai Heart Institute

Los Angeles, California, 90048, United States

Location

Rady Children's Hospital

San Diego, California, 92123, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Nemours Cardiac Center/Alfred I. duPont Hospital for Children

Wilmington, Delaware, 19803, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20008, United States

Location

Johns Hopkins All Children's Heart Institute

St. Petersburg, Florida, 33701, United States

Location

Children's Healthcare of Atlanta

Atlanta, Georgia, 30322, United States

Location

Riley Hospital for Children/Herman B. Wells Center for Pediatric Research

Indianapolis, Indiana, 46201, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

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

Ann Arbor, Michigan, 48109-4204, United States

Location

Children's Mercy Hospital Kansas City

Kansas City, Missouri, 64108, United States

Location

Washington University St. Louis/St.Louis Children's Hospital

St Louis, Missouri, 63110, United States

Location

Children's Hospital of New York

New York, New York, 10032, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

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

Salt Lake City, Utah, 84113, United States

Location

Seattle Children's Hosptial

Seattle, Washington, 98105, United States

Location

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

The Hospital for Sick Children

Toronto, Ontario, ON M5G 1X8, Canada

Location

Sejong General Hospital

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

Location

Seoul National University Children's Hospital

Seoul, 110-744, South Korea

Location

Related Publications (1)

• Goldberg DJ, Zak V, Goldstein BH, McCrindle BW, Menon SC, Schumacher KR, Payne RM, Rhodes J, McHugh KE, Penny DJ, Trachtenberg F, Hamstra MS, Richmond ME, Frommelt PC, Files MD, Yeager JL, Pemberton VL, Stylianou MP, Pearson GD, Paridon SM; Pediatric Heart Network Investigators. Design and rationale of the Fontan Udenafil Exercise Longitudinal (FUEL) trial. Am Heart J. 2018 Jul;201:1-8. doi: 10.1016/j.ahj.2018.03.015. Epub 2018 Apr 3.

  PMID: 29910047 DERIVED

MeSH Terms

Interventions

udenafil

Results Point of Contact

• Title: Director of Clinical Operatons
• Organization: Mezzion Pharmaceuticals, Inc

wg.kim@mezzion.com

3128980292

Study Officials

• Steve Paridon, MD

  Children's Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 5, 2017
• First Posted: January 6, 2017
• Study Start: February 6, 2017
• Primary Completion: December 30, 2019
• Study Completion: December 20, 2022
• Last Updated: December 9, 2025
• Results First Posted: December 9, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (21); CA (1); KR (2)