Fontan Udenafil Exercise Longitudinal Assessment Trial - 2

NCT ID: NCT05918211

Last Updated: 2025-10-28

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

436 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-10-30

Study Completion Date

2025-10-28

Brief Summary

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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.

Detailed Description

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This study is a 26 week, prospective, multicenter, randomized, double-blinded, placebo-controlled safety and efficacy study of udenafil vs. placebo in adolescent subjects who have had the Fontan procedure. The primary efficacy endpoint will be change from baseline at 26 weeks in peak minute oxygen consumption (VO2 mL/kg/min) as measured by maximal cardiopulmonary exercise test (CPET) reading laboratory who will be blinded to treatment allocation.

Conditions

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Single Ventricle Heart Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

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)
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
Active and matching placebo tablets will be identical in appearance.

Study Groups

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Drug

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

Group Type EXPERIMENTAL

Udenafil

Intervention Type DRUG

Active drug

Placebo

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

Group Type EXPERIMENTAL

Placebo

Intervention Type DRUG

Matching Placebo

Interventions

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Udenafil

Active drug

Intervention Type DRUG

Placebo

Matching Placebo

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Males and females with Fontan physiology who are 12 to less than 19 years of age at enrollment.
2. Participant consent or parental/guardian consent and participant assent.
3. Participant fluency in primary language of country in which study is being conducted.
4. Current antiplatelet 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 3 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 mmHg between the regions proximal and distal to the obstruction as measured by either catheterization or echocardiography, obtained prior to screening for the trial.
8. Single lung physiology with greater than 80% flow to one lung.
9. Failure to achieve maximal exertion (defined as RER \< 1.10) on screening/baseline exercise test.
10. Peak minute oxygen consumption (VO2) less than 45% or ≥ 80% of predicated for age and gender at enrollment.
11. Severe ventricular dysfunction assessed qualitatively by clinical echocardiography within 6 months prior to enrollment.
12. Severe valvar regurgitation, ventricular outflow obstruction, or severe aortic arch obstruction assessed by clinical echocardiography within six months prior to enrollment.
13. 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.
14. Inability to complete exercise testing at baseline screening.
15. Subjects with a pacemaker whose heart rate at peak exercise is controlled by the extrinsic pacemaker as opposed to a native atrial rhythm.
16. History of PDE-5 inhibitor use within 12 months prior to enrollment. (Treatment is defined as chronic therapy as opposed to a single dose.)
17. History of any other medication for treatment of pulmonary hypertension within 3 months before study onset.
18. Known intolerance to oral udenafil.
19. Frequent use of medications or other substances that inhibit or induce CYP3A4.
20. Current use of alpha-blockers or nitrates.
21. Ongoing or planned participation in another research protocol that would either prevent successful completion of planned study testing or invalidate its results.
22. Noncardiac medical, psychiatric, and/or social disorder that would prevent successful completion of planned study testing or would invalidate its results.
23. Cardiac care, ongoing or planned, at a non-study center that would impede study completion.
24. 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 if sexually active.
25. Unable to abstain or limit intake of grapefruit juice and grapefruit containing drinks during the duration of the trial.
26. Refusal to provide written informed consent/assent.
27. In the opinion of the investigator, the subject is likely to be non-compliant with the study protocol.
28. History of clinically significant thromboembolic event, in the option of the site Principal Investigator, that may put the subject at increased risk of a subsequent event while participating in the study.
29. Coronavirus disease 2019 (COVID-19) vaccination or symptoms of COVID-19 infection within 7 days of Visit 1.
30. Not taking antiplatelet or anticoagulant therapy.
Minimum Eligible Age

12 Years

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Mezzion Pharma Co. Ltd

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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WG Kim

Role: STUDY_DIRECTOR

Mezzion Pharma Co. Ltd

Locations

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Phoenix Children's Hospital

Phoenix, Arizona, United States

Site Status RECRUITING

Arkansas Children's

Little Rock, Arkansas, United States

Site Status RECRUITING

Children's Hospital of Los Angeles

Los Angeles, California, United States

Site Status RECRUITING

Rady Children's Hospital

San Diego, California, United States

Site Status RECRUITING

UCSF Benioff Children's Hospital

San Francisco, California, United States

Site Status RECRUITING

Children's Hospital of Colorado

Denver, Colorado, United States

Site Status RECRUITING

Yale School of Medicine

New Haven, Connecticut, United States

Site Status RECRUITING

Nemours Children's Hospital

Wilmington, Delaware, United States

Site Status RECRUITING

Childrens National Medical Center

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

Site Status RECRUITING

UF Health Shands Hospital

Gainesville, Florida, United States

Site Status RECRUITING

Joe DiMaggio Children's Hospital

Hollywood, Florida, United States

Site Status RECRUITING

Johns Hopkins All Children's Hospital

St. Petersburg, Florida, United States

Site Status RECRUITING

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Site Status RECRUITING

Children's Hospital of Georgia

Augusta, Georgia, United States

Site Status RECRUITING

Lurie Children's Hospital

Chicago, Illinois, United States

Site Status RECRUITING

University of Chicago

Chicago, Illinois, United States

Site Status RECRUITING

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status RECRUITING

University of Michigan

Ann Arbor, Michigan, United States

Site Status RECRUITING

Children's Mercy Hospital Kansas City

Kansas City, Missouri, United States

Site Status RECRUITING

Washington University

St Louis, Missouri, United States

Site Status RECRUITING

University of Nebraska Children's Hospital and Medical Center

Omaha, Nebraska, United States

Site Status RECRUITING

Mt. Sinai Children's Hospital

New York, New York, United States

Site Status NOT_YET_RECRUITING

New York-Presbyterian Children's Hospital

New York, New York, United States

Site Status RECRUITING

[email protected]

Charlotte, North Carolina, United States

Site Status NOT_YET_RECRUITING

Duke University Medical Center

Durham, North Carolina, United States

Site Status RECRUITING

Cincinnati Childrens Hospital Medical Center

Cincinnati, Ohio, United States

Site Status RECRUITING

Cleveland Clinic

Cleveland, Ohio, United States

Site Status RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status RECRUITING

MUSC Pediatric Research Group

Charleston, South Carolina, United States

Site Status RECRUITING

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status RECRUITING

UT Southwestern Medical Center

Dallas, Texas, United States

Site Status RECRUITING

Texas Children's Hospital

Houston, Texas, United States

Site Status RECRUITING

Primary Children's Medical Center

Salt Lake City, Utah, United States

Site Status RECRUITING

Seattle Children's Hospital

Seattle, Washington, United States

Site Status RECRUITING

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, United States

Site Status RECRUITING

Sejong General Hospital

Bucheon-si, Gyeonggi-do, South Korea

Site Status RECRUITING

Seoul National University Children's Hospital

Seoul, , South Korea

Site Status RECRUITING

Yonsei Severance Hospital

Seoul, , South Korea

Site Status RECRUITING

Countries

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United States South Korea

Central Contacts

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WG Kim

Role: CONTACT

443.699.6746

Facility Contacts

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Jade Porche

Role: primary

602-933-6835

Wendy Haley

Role: primary

Matthew Dam

Role: primary

323-361-7881

Mark Abcede

Role: primary

619-987-0650

Aisling Conneally

Role: primary

510-600-2965

Andrea DeMarsh

Role: primary

216-906-7931

Amanda Catucci

Role: primary

631-897-6722

Carol Prospero

Role: primary

302-651-6686

Stacy Davenport

Role: primary

240-547-9804

Dalia Lopez-Colon

Role: primary

352-281-6723

Norma Barton

Role: primary

954-265-4466

Lexie Dallas

Role: primary

727-767-2465

Jessica Braun

Role: primary

404-785-3828

Michelle Collins

Role: primary

706-721-9680

Jessica Bhavsar

Role: primary

312-227-4432

Becky Puplava

Role: primary

773-702-2879

Tom Giorgio

Role: primary

617-355-4201

Cynthia Smith

Role: primary

734-615-0590

Dara Watkins

Role: primary

816-302-3633

Mason Basler

Role: primary

618-579-5674

Morgan Loeffelbein

Role: primary

402-559-0681

Xueru Mu

Role: primary

347-335-7148

Katrina Golub

Role: primary

212-342-1562

Dana Amaro

Role: primary

704-355-4692

Alexi Pifer

Role: primary

Melissa Harward

Role: backup

Mariah Palmer

Role: primary

513-636-9516

Ewing Paige

Role: primary

216-445-8798

Victoria Shay

Role: primary

614-355-5819

Faith Alunni

Role: primary

570-430-5058

Niklas Gerhart

Role: primary

412-692-6742

Susannah C Wakefield

Role: primary

843-792-8317

Sherry Bowman

Role: primary

615-936-0061

Aruna Ayalasomayajula

Role: primary

702-340-7830

Sara Stephens

Role: primary

832-826-5613

Andrea Dunn

Role: primary

801-213-7609

Ranjini Prakash

Role: primary

206-987-2000

Jennifer Yauck

Role: primary

414-266-2325

Jiyeun Jeong

Role: primary

010-9221-5114

Yubin Min

Role: backup

032-340-1782

Gowoon Choi

Role: primary

Juwon Heo

Role: primary

Other Identifiers

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MZ_Udenafil-05

Identifier Type: -

Identifier Source: org_study_id

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