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A Extension Study of Udenafil in Adolescents (FUELExten)

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ClinicalTrials.gov Identifier: NCT03013751
Recruitment Status : Recruiting
First Posted : January 6, 2017
Last Update Posted : January 9, 2018
Sponsor:
Collaborator:
Information provided by (Responsible Party):

Study Description
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.

Condition or disease Intervention/treatment Phase
Functional Single Ventricle Heart Disease Drug: Udenafil Phase 3

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.

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 300 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase III Extension Study of Udenafil in Adolescents With Single Ventricle Physiology After Fontan Palliation
Study Start Date : January 2017
Estimated Primary Completion Date : February 2019
Estimated Study Completion Date : February 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Heart Diseases
U.S. FDA Resources

Arms and Interventions

Arm Intervention/treatment
Experimental: Drug
Udenafil administered for 52 weeks
Drug: Udenafil
Active drug


Outcome Measures

Primary Outcome Measures :
  1. Safety (Adverse Events) [ Time Frame: 52 Weeks ]
    Adverse Events


Secondary Outcome Measures :
  1. Exercise (Change in maximal oxygen consumption) [ Time Frame: 52 Weeks ]
    Change in maximal oxygen consumption.

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

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

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

  5. Biomarkers (Change in serum BNP level) [ Time Frame: 52 Weeks ]
    Change in serum BNP level from baseline to end-of-study


Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03013751


Contacts
Contact: Steve Paridon, MD 2155903532 paridon@email.chop.edu
Contact: David J Goldberg, MD 2674268143 goldbergda@email.chop.edu

  Show 24 Study Locations
Sponsors and Collaborators
Mezzion Pharma Co. Ltd
National Heart, Lung, and Blood Institute (NHLBI)
Investigators
Principal Investigator: Steve Paridon, MD Children's Hospital of Philadelphia
More Information

Responsible Party: Mezzion Pharma Co. Ltd
ClinicalTrials.gov Identifier: NCT03013751     History of Changes
Other Study ID Numbers: PHN-Udenafil-03
U01HL068270 ( U.S. NIH Grant/Contract )
First Posted: January 6, 2017    Key Record Dates
Last Update Posted: January 9, 2018
Last Verified: January 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: Yes

Additional relevant MeSH terms:
Heart Diseases
Cardiovascular Diseases
Udenafil
Phosphodiesterase 5 Inhibitors
Phosphodiesterase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action