A Extension Study of Udenafil in Adolescents
- Registration Number
- NCT03013751
- Lead Sponsor
- Mezzion Pharma Co. Ltd
- 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.
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 300
- 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.
- 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.
- Known intolerance to oral udenafil.
- Frequent use of medications or other substances that inhibit or induce CYP3A4.
- Current use of alpha-blockers or nitrates.
- Ongoing or planned participation in another research protocol that would either prevent successful completion of planned study testing or invalidate its results.
- Noncardiac medical, psychiatric, and/or social disorder that would prevent successful completion of planned study testing or would invalidate its results.
- Cardiac care, ongoing or planned, at a non-study center that would impede study completion.
- 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.
- Unable to abstain or limit intake of grapefruit juice during the duration of the trial.
- Refusal to provide written informed consent/assent.
- In the opinion of the primary care physician, the subject is likely to be non-compliant with the study protocol.
- History of clinically significant thromboembolic event, as adjudicated by study Investigators.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Drug Udenafil Udenafil administered for 52 weeks
- Primary Outcome Measures
Name Time Method Safety (Adverse Events) 52 Weeks Adverse Events
- Secondary Outcome Measures
Name Time Method Biomarkers (Change in serum BNP level) 52 Weeks Change in serum BNP level from baseline to end-of-study
Endothelial function (Change in log-transformed Reactive Hyperemia Index) 52 Weeks Change in log-transformed Reactive Hyperemia Index
Function Health Status (Change in full scale Peds QL) 52 Weeks Change in full scale Peds QL
Exercise (Change in maximal oxygen consumption) 52 Weeks Change in maximal oxygen consumption.
Echo (Change in myocardial performance Index) 52 Weeks Change in myocardial performance Index
Trial Locations
- Locations (24)
Phoenix Children's Hospital/Children's Heart Center at Phoenix Children's Hospital
🇺🇸Phoenix, Arizona, United States
Medical University of South Carolina
🇺🇸Charleston, South Carolina, United States
The Hospital for Sick Children
🇨🇦Toronto, Ontario, Canada
Rady Children's Hospital
🇺🇸San Diego, California, United States
Cedars/Sinai Heart Institute
🇺🇸Los Angeles, 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, District of Columbia, United States
Johns Hopkins All Children's Heart Institute
🇺🇸Saint 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
🇺🇸Saint Louis, Missouri, United States
Children's Hospital of New York
🇺🇸New York, New York, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
Sejong General Hospital
🇰🇷Bucheon-si, Gyeonggi-do, Korea, Republic of
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
Seoul National University Children's Hospital
🇰🇷Seoul, Korea, Republic of