A Extension Study of Udenafil in Adolescents

NCT ID: NCT03013751

Last Updated: 2025-12-09

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 301 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-02-06
• Study Completion Date: 2022-12-20

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.

Detailed Description

This will be an open-label extension study. All enrolled subjects will be provided with udenafil for the duration of the study. Subjects completing the Phase III FUEL study will be eligible for recruitment. Additional subjects will be recruited as needed to ensure a minimum of 300 total subjects enroll. Safety, pharmacodynamic and quality of life data will be collected and analyzed.

Conditions

Functional Single Ventricle Heart Disease

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Drug

Udenafil administered for 52 weeks

Group Type: EXPERIMENTAL

Udenafil

Intervention Type: DRUG

Active drug

Interventions

Udenafil

Active drug

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. 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.

2. Participant consent or parental/guardian consent and participant assent.

3. Participant fluent in English, Spanish, or Korean.

4. Current anti-platelet or anticoagulant therapy.

Exclusion Criteria

1. Height < 132 cm.

2. Weight < 40 kg.

3. Hospitalization for acute decompensated heart failure within the last 12 months.

4. Current intravenous inotropic drugs.

5. Undergoing evaluation for heart transplantation or listed for transplantation.

6. Diagnosis of active protein losing enteropathy or plastic bronchitis within the last three years, or a history of liver cirrhosis.

7. 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.

8. Single lung physiology.

9. Maximal VO2 less than 50% of predicted for age and gender at enrollment.

10. Severe ventricular dysfunction assessed qualitatively by clinical echocardiography within six months prior to enrollment.

11. Severe valvar regurgitation, ventricular outflow obstruction, or aortic arch obstruction assessed by clinical echocardiography within six months prior to enrollment.

12. Significant renal, hepatic, gastrointestinal or biliary disorders that could impair absorption, metabolism or excretion of orally administered medications.

13. Inability to complete exercise testing at baseline screening.

14. History of PDE-5 inhibitor use (with the exception of FUEL participation) within 3 months before study onset.

15. Use of any other drug to treat pulmonary hypertension within 3 months before study onset.

16. Known intolerance to oral udenafil.

17. Frequent use of medications or other substances that inhibit or induce CYP3A4.

18. Current use of alpha-blockers or nitrates.

19. Ongoing or planned participation in another research protocol that would either prevent successful completion of planned study testing or invalidate its results.

20. Noncardiac medical, psychiatric, and/or social disorder that would prevent successful completion of planned study testing or would invalidate its results.

21. Cardiac care, ongoing or planned, at a non-study center that would impede study completion.

22. For females: Pregnancy at the time of screening, pregnancy planned before study completion, or refusal to use an acceptable method of contraception for study duration.

23. Unable to abstain or limit intake of grapefruit juice during the duration of the trial.

24. Refusal to provide written informed consent/assent.

25. In the opinion of the primary care physician, the subject is likely to be non-compliant with the study protocol.

26. History of clinically significant thromboembolic event, as adjudicated by study Investigators.

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role: collaborator

Mezzion Pharma Co. Ltd

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Steve Paridon, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital of Philadelphia

Locations

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

Phoenix, Arizona, United States

Cedars/Sinai Heart Institute

Los Angeles, California, United States

Rady Children's Hospital

San Diego, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Nemours Cardiac Center/Alfred I. duPont Hospital for Children

Wilmington, Delaware, United States

Children's National Medical Center

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

Johns Hopkins All Children's Heart Institute

St. Petersburg, Florida, United States

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

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

Indianapolis, Indiana, United States

Boston Children's Hospital

Boston, Massachusetts, United States

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

Ann Arbor, Michigan, United States

Children's Mercy Hospital Kansas City

Kansas City, Missouri, United States

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

St Louis, Missouri, United States

Children's Hospital of New York

New York, New York, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Medical University of South Carolina

Charleston, South Carolina, United States

Texas Children's Hospital

Houston, Texas, United States

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

Salt Lake City, Utah, United States

Seattle Children's Hosptial

Seattle, Washington, United States

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, United States

The Hospital for Sick Children

Toronto, Ontario, Canada

Sejong General Hospital

Bucheon-si, Gyeonggi-do, South Korea

Seoul National University Children's Hospital

Seoul, , South Korea

Countries

United States; Canada; South Korea

References

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.

Reference Type: DERIVED

PMID: 29910047 (View on PubMed)

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

U01HL068270

Identifier Type: NIH

Identifier Source: secondary_id

View Link

PHN-Udenafil-03

Identifier Type: -

Identifier Source: org_study_id