A Extension Study of Udenafil in Adolescents

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03013751
• Date of registration: 05/01/2017
• Prospective Registration: No
• Primary sponsor: Mezzion Pharma Co. Ltd
• Public title: A Extension Study of Udenafil in Adolescents FUELExten
• Scientific title: A Phase III Extension Study of Udenafil in Adolescents With Single Ventricle Physiology After Fontan Palliation
• Date of first enrolment: January 2017
• Target sample size: 300
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT03013751
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Canada, Korea, Republic of, United States

Contacts

• Name: Steve Paridon, MD
• Affiliation: Children's Hospital of Philadelphia

Key inclusion & exclusion 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.



Age minimum: 12 Years
Age maximum: 18 Years
Gender: All

Health Condition(s) or Problem(s) studied

Functional Single Ventricle Heart Disease

Intervention(s)

Drug: Udenafil

Primary Outcome(s)

Safety (Adverse Events) [Time Frame: 52 Weeks]

Secondary Outcome(s)

Echo (Change in myocardial performance Index) [Time Frame: 52 Weeks]

Exercise (Change in maximal oxygen consumption) [Time Frame: 52 Weeks]

Endothelial function (Change in log-transformed Reactive Hyperemia Index) [Time Frame: 52 Weeks]

Biomarkers (Change in serum BNP level) [Time Frame: 52 Weeks]

Function Health Status (Change in full scale Peds QL) [Time Frame: 52 Weeks]

Secondary ID(s)

PHN-Udenafil-03

U01HL068270

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

National Heart, Lung, and Blood Institute (NHLBI)