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CompletedNCT02741115FUELUpdated Jun 17, 2025Results posted

Fontan Udenafil Exercise Longitudinal Assessment Trial

A Phase 3 interventional study of Udenafil and Placebo in Single Ventricle Heart Disease, sponsored by Mezzion Pharma Co. Ltd. Completed at 30 sites in 3 countries. Open to participants aged 12 Years to 18 Years. Per ClinicalTrials.gov, last updated 2025-06-17.

Sponsored by Mezzion Pharma Co. Ltd · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
400
Allocation
Randomized
Ages
12 Years to 18 Years
Sex
All
01

Study summary

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 subjects who have undergone the Fontan procedure.

Read the detailed description

This study is a randomized, double-blinded, efficacy trial of the effects of udenafil vs. placebo on the background of standard therapy on maximal VO2 (ml/kg/min) from baseline to six months in adolescent survivors of the Fontan procedure. . The target sample size is 400 subjects (200 per group).

02

Conditions studied

  • Single Ventricle Heart Disease

Keywords

  • Fontan
  • Maximal Oxygen Consumption
  • Vascular Function
  • EndoPAT
  • Myocardial Performance
03

In context

Heart Diseases

3,639 studies on the registry are indexed under Heart Diseases; 461 are open to participants now.

This study's enrollment of 400 is above the median of 100 across 1,778 interventional studies indexed under Heart Diseases.

Browse Heart Diseases studies →

Lead sponsor

Mezzion Pharma Co. Ltd is the lead sponsor of 5 studies on the registry; 1 is open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
12 Years to 18 Years
Sexes eligible
All
Accepts healthy volunteers
No

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

Exclusion criteria

Exclusion Criteria:

  1. Weight \< 40 kg
  2. Height \< 132 cm.
  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. VO2 less than 50%
  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 (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, based on laboratory assessment six weeks prior to screening for the trial.
  13. Inability to complete exercise testing at baseline screening.
  14. History of PDE-5 inhibitor use within 3 months before study onset.
  15. History of any other medication for treatment of 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 if sexually active.
  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.
05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
400 participants (actual)

Study arms

  • Experimental
    Drug

    Udenafil. One tablet twice daily for 26 weeks

    Drug: Udenafil

  • Experimental
    Placebo

    Placebo. One tablet twice daily for 26 weeks

    Drug: Placebo

Interventions

  • DrugUdenafil

    Active drug

  • DrugPlacebo

    Matching Placebo

06

What researchers measure

Primary outcomes

  1. Change in Maximal VO2 From Baseline to Week 26 Using Last Observation Carried Forward (LOCF)

    The change in exercise capacity (as measured by peak VO2 at maximum exercise effort) from baseline to 26 weeks

    Time frame: Baseline to 26 Weeks

Secondary outcomes

  1. Change in Myocardial Performance Index (MPI) From Baseline to Week 26

    Change in the myocardial performance index (MPI) from baseline to 26 weeks is determined by velocities from blood pool Doppler of the inflow and outflow tract of the dominant ventricle. The measure is the ratio of the sum of isovolumetric contraction time and isovolumetric relaxation time, divided by ventricular ejection time. A lower value is consistent with a more efficient ventricle (better function). A value of zero indicates that there is no isovolumetric contraction or relaxation and, while physiologically implausible, would be consistent with a perfectly efficient ventricle. The mean left ventricular MPI in healthy people without heart disease is approximately 0.35 while the mean right ventricular MPI is approximately 0.1 due to the reduced ventricular afterload of the pulmonary circulation. In general, a decrease in the MPI corresponds to more efficient (better) ventricular function, while an increase in MPI corresponds to less efficient (worse) ventricular function.

    Time frame: Week 26

  2. Change in Log-transformed Reactive Hyperemia Index (InRH) From Baseline to Week 26

    Endothelial pulse amplitude tonometry (Endo-PAT) is a technique for the non-invasive assessment of peripheral vascular function. In adults, Endo-PAT has been demonstrated to identify those with coronary artery dysfunction and to correlate with brachial artery reactivity testing. Endo-PAT use in children has been more limited, but has shown excellent reproducibility. Reactive hyperemia index, a measure of the hyperemic response adjusted for baseline blood flow, is a measure of vascular function. A higher value denotes better, or more healthy, vascular (endothelial) function.

    Time frame: Week 26

  3. Change in Serum Brain-type Natriuretic Peptide (BNP) From Baseline to Week 26.

    BNP is a hormone produced when the heart is enlarged or has to work harder to pump blood to the body. Higher levels of BNP can be a sign of heart failure.

    Time frame: Week 26

  4. Change in VO2 at VAT

    This outcome measures the change in milliliters of oxygen consumption (VO2) per minute at the ventilatory anaerobic threshold (VAT) between Week 26 and Baseline Visits using last observation carried forward (ITT Population). The anaerobic threshold is the period in exercise when metabolism switches from aerobic to anaerobic. This is an useful measure of submaximal exercise capacity particularly in the single ventricle population.

    Time frame: 26 Weeks

  5. Change in Work Rate at VAT From Baseline to Week 26 With LOCF

    This outcome measures the change in work (measured in Watts) at the ventilatory anaerobic threshold (VAT) between Week 26 and Baseline Visits using last observation carried forward (ITT Population). The anaerobic threshold is the period in exercise when metabolism switches from aerobic to anaerobic. This is an useful measure of submaximal exercise capacity particularly in the single ventricle population.

    Time frame: 26 Weeks

  6. Change in VE/VCO2 at VAT From Baseline to Week 26

    This measure is a ratio of the amount of inspired air required to clear a given volume of CO2 from the circulation at the ventilatory anaerobic threshold (VAT). This measurement is made during exercise stress testing. Decreases in this ratio may reflect an improvement in ventilatory efficiency or an improvement in cardiac function.

    Time frame: 26 Weeks

  7. Change in Respiratory Rate at Maximal Exercise Effort From Baseline to Week 26 Using LOCF

    Time frame: Week 26

  8. Change in Minute Oxygen Consumption at Maximal Exercise Effort From Baseline to Week 26

    The amount of air that enters the lungs per minute at maximal exercise effort.

    Time frame: Week 26

  9. Change in Work Rate at Maximal Exercise Effort Between Baseline and Week 26 Using LOCF

    Change in power at maximal exercise effort.

    Time frame: Week 26

  10. Change in Pediatric Quality of Life (PedsQL) Generic Core Scales - Physical Functioning Subscale (Child Reported)

    The PedsQL is a short questionnaire that has been used in multiple studies, including a previous study of Fontan patients. A 5-point Likert response scale (0-4) is used. This score was linearly transformed on a scale of 0-100 (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0). For an individual, a higher score is associated with a higher reported healthcare-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 weeks. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

    Time frame: 26 weeks

  11. Change in Pediatric Quality of Life (PedsQL) Generic Core Scales - Physical Functioning Subscale (Parent Reported)

    The PedsQL is a short questionnaire that has been used in multiple studies, including a previous study of Fontan patients. A 5-point Likert response scale (0-4) is used. This score was linearly transformed on a scale of 0-100 (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0). For an individual, a higher score is associated with a higher reported healthcare-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 weeks. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

    Time frame: 26 weeks

  12. Change in Pediatric Quality of Life (PedsQL) Generic Core Scales - Psychosocial Health Subscale (Child Reported)

    The PedsQL is a short questionnaire that has been used in multiple studies, including a previous study of Fontan patients. A 5-point Likert response scale (0-4) is used. This score was linearly transformed on a scale of 0-100 (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0). For an individual, a higher score is associated with a higher reported healthcare-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 weeks. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

    Time frame: 26 weeks

  13. Change in Pediatric Quality of Life (PedsQL) Generic Core Scales - Psychosocial Health Subscale (Parent Reported)

    The PedsQL is a short questionnaire that has been used in multiple studies, including a previous study of Fontan patients. A 5-point Likert response scale (0-4) is used. This score was linearly transformed on a scale of 0-100 (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0). For an individual, a higher score is associated with a higher reported healthcare-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 weeks. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

    Time frame: 26 weeks

  14. Change in Pediatric Quality of Life (PedsQL) Inventory - Cardiac Module Scale - Treatment II Subscale (Child-reported)

    The PedsQL is a short questionnaire that has been used in multiple studies, including a previous study of Fontan patients. A 5-point Likert response scale (0-4) is used. This score was linearly transformed on a scale of 0-100 (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0). For an individual, a higher score is associated with a higher reported healthcare-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 weeks. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

    Time frame: 26 weeks

  15. Change in Pediatric Quality of Life (PedsQL) Inventory - Cardiac Module Scale - Perceived Physical Appearance Subscale

    The PedsQL is a short questionnaire that has been used in multiple studies, including a previous study of Fontan patients. A 5-point Likert response scale (0-4) is used. This score was linearly transformed on a scale of 0-100 (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0). For an individual, a higher score is associated with a higher reported healthcare-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 weeks. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

    Time frame: 26 weeks

  16. Change in Pediatric Quality of Life (PedsQL) Inventory - Cardiac Module Scale - Treatment Anxiety Subscale

    The PedsQL is a short questionnaire that has been used in multiple studies, including a previous study of Fontan patients. A 5-point Likert response scale (0-4) is used. This score was linearly transformed on a scale of 0-100 (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0). For an individual, a higher score is associated with a higher reported healthcare-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 weeks. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

    Time frame: 26 weeks

  17. Change in Pediatric Quality of Life (PedsQL) Inventory - Cardiac Module Scale - Cognitive Problems Subscale

    The PedsQL is a short questionnaire that has been used in multiple studies, including a previous study of Fontan patients. A 5-point Likert response scale (0-4) is used. This score was linearly transformed on a scale of 0-100 (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0). For an individual, a higher score is associated with a higher reported healthcare-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 weeks. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

    Time frame: 26 weeks

  18. Change in Pediatric Quality of Life (PedsQL) Inventory - Cardiac Module Scale - Communication Problems Subscale

    The PedsQL is a short questionnaire that has been used in multiple studies, including a previous study of Fontan patients. A 5-point Likert response scale (0-4) is used. This score was linearly transformed on a scale of 0-100 (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0). For an individual, a higher score is associated with a higher reported healthcare-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 week. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

    Time frame: 26 weeks

  19. Change in Pediatric Cardiac Quality of Life Inventory (PCQLI) Total Score-Ages 8 to 12 (Child Reported)

    The PCLQI is a disease-specific measure of health-related quality of life for children and adolescents with congenital or acquired heart disease. The PCQLI score is calculated by adding together the scores from the two subscales: Disease Impact and Psychosocial Impact. Each subscale can score a maximum of 50 points, resulting in a total score with a maximum of 100. Higher scores indicate better perceived health-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 weeks. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

    Time frame: 26 weeks

  20. Change in Pediatric Cardiac Quality of Life Inventory (PCQLI) Total Score-Ages 8 to 12 (Parent Reported)

    The PCLQI is a disease-specific measure of health-related quality of life for children and adolescents with congenital or acquired heart disease. The PCQLI score is calculated by adding together the scores from the two subscales: Disease Impact and Psychosocial Impact. Each subscale can score a maximum of 50 points, resulting in a total score with a maximum of 100. Higher scores indicate better perceived health-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 weeks. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

    Time frame: 26 weeks

  21. Change in Pediatric Cardiac Quality of Life Inventory (PCQLI) Total Score-Ages 13 to 18 (Child Reported)

    The PCLQI is a disease-specific measure of health-related quality of life for children and adolescents with congenital or acquired heart disease. The PCQLI score is calculated by adding together the scores from the two subscales: Disease Impact and Psychosocial Impact. Each subscale can score a maximum of 50 points, resulting in a total score with a maximum of 100. Higher scores indicate better perceived health-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 weeks. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

    Time frame: 26 weeks

  22. Change in Pediatric Cardiac Quality of Life Inventory (PCQLI) Total Score-Ages 13 to 18 (Parent Reported)

    The PCLQI is a disease-specific measure of health-related quality of life for children and adolescents with congenital or acquired heart disease. The PCQLI score is calculated by adding together the scores from the two subscales: Disease Impact and Psychosocial Impact. Each subscale can score a maximum of 50 points, resulting in a total score with a maximum of 100. Higher scores indicate better perceived health-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 weeks. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

    Time frame: 26 weeks

07

Results

Posted May 21, 2025

Participant flow

Participant flow — Overall Study
MilestoneDrugPlacebo
Started200200
Completed188194
Not completed126
Withdrew: Adverse event10
Withdrew: Lost to follow-up74
Withdrew: Withdrawal by subject42

Outcome measures

PrimaryChange in Maximal VO2 From Baseline to Week 26 Using Last Observation Carried Forward (LOCF)

The change in exercise capacity (as measured by peak VO2 at maximum exercise effort) from baseline to 26 weeks

Time frame:
Baseline to 26 Weeks
Reported as:
Mean · mL/kg/min
Change in Maximal VO2 From Baseline to Week 26 Using Last Observation Carried Forward (LOCF)
mL/kg/minUdenafilPlacebo
Change in Maximal VO2 From Baseline to Week 26 Using Last Observation Carried Forward (LOCF)-0.23 ± 4.056-0.89 ± 3.672
Statistical analysis
  • Udenafil vs Placebo · ANCOVA · p = 0.092 (LS mean was the estimated treatment difference from the analysis model.) · Mean difference (final values): 0.64ANCOVA fixed factors: ventricular morphology and treatment group with a continuous covariate of Baseline maximal VO2. LOCF imputation used.
SecondaryChange in Myocardial Performance Index (MPI) From Baseline to Week 26

Change in the myocardial performance index (MPI) from baseline to 26 weeks is determined by velocities from blood pool Doppler of the inflow and outflow tract of the dominant ventricle. The measure is the ratio of the sum of isovolumetric contraction time and isovolumetric relaxation time, divided by ventricular ejection time. A lower value is consistent with a more efficient ventricle (better function). A value of zero indicates that there is no isovolumetric contraction or relaxation and, while physiologically implausible, would be consistent with a perfectly efficient ventricle. The mean left ventricular MPI in healthy people without heart disease is approximately 0.35 while the mean right ventricular MPI is approximately 0.1 due to the reduced ventricular afterload of the pulmonary circulation. In general, a decrease in the MPI corresponds to more efficient (better) ventricular function, while an increase in MPI corresponds to less efficient (worse) ventricular function.

Time frame:
Week 26
Reported as:
Mean · ratio
Change in Myocardial Performance Index (MPI) From Baseline to Week 26
ratioDrugPlacebo
Change in Myocardial Performance Index (MPI) From Baseline to Week 26-0.02 ± 0.1120.01 ± 0.132
Statistical analysis
  • Drug vs Placebo · ANCOVA · p = 0.024 (LS mean was the estimated treatment difference from the analytical model.) · Mean difference (final values): -0.03Fixed factors: ventricular morphology and treatment group with a continuous covariate of Baseline maximal VO2.
SecondaryChange in Log-transformed Reactive Hyperemia Index (InRH) From Baseline to Week 26

Endothelial pulse amplitude tonometry (Endo-PAT) is a technique for the non-invasive assessment of peripheral vascular function. In adults, Endo-PAT has been demonstrated to identify those with coronary artery dysfunction and to correlate with brachial artery reactivity testing. Endo-PAT use in children has been more limited, but has shown excellent reproducibility. Reactive hyperemia index, a measure of the hyperemic response adjusted for baseline blood flow, is a measure of vascular function. A higher value denotes better, or more healthy, vascular (endothelial) function.

Time frame:
Week 26
Reported as:
Mean · Natural log-transformed RHI (lnRHI)
Change in Log-transformed Reactive Hyperemia Index (InRH) From Baseline to Week 26
Natural log-transformed RHI (lnRHI)DrugPlacebo
Change in Log-transformed Reactive Hyperemia Index (InRH) From Baseline to Week 260.06 ± 0.3010.04 ± 0.364
Statistical analysis
  • Drug vs Placebo · ANCOVA · p = 0.591 · Mean difference (final values): 0.02
SecondaryChange in Serum Brain-type Natriuretic Peptide (BNP) From Baseline to Week 26.

BNP is a hormone produced when the heart is enlarged or has to work harder to pump blood to the body. Higher levels of BNP can be a sign of heart failure.

Time frame:
Week 26
Reported as:
Mean · pg/mL
Change in Serum Brain-type Natriuretic Peptide (BNP) From Baseline to Week 26.
pg/mLDrugPlacebo
Change in Serum Brain-type Natriuretic Peptide (BNP) From Baseline to Week 26.0.08 ± 0.9050.03 ± 1.137
Statistical analysis
  • Drug vs Placebo · ANCOVA · p = 0.169 · Mean difference (final values): 0.13
SecondaryChange in VO2 at VAT

This outcome measures the change in milliliters of oxygen consumption (VO2) per minute at the ventilatory anaerobic threshold (VAT) between Week 26 and Baseline Visits using last observation carried forward (ITT Population). The anaerobic threshold is the period in exercise when metabolism switches from aerobic to anaerobic. This is an useful measure of submaximal exercise capacity particularly in the single ventricle population.

Time frame:
26 Weeks
Reported as:
Mean · mL/kg/min
Change in VO2 at VAT
mL/kg/minDrugPlacebo
Change in VO2 at VAT-0.07 ± 2.998-0.68 ± 3.216
Statistical analysis
  • Drug vs Placebo · ANCOVA · p = 0.012 (LS mean was the estimated treatment mean difference from the analysis model.) · Median difference (final values): 0.78Fixed factors: ventricular morphology and treatment group, with a continuous covariate of Baseline maximal VO2. LOCF imputation.
SecondaryChange in Work Rate at VAT From Baseline to Week 26 With LOCF

This outcome measures the change in work (measured in Watts) at the ventilatory anaerobic threshold (VAT) between Week 26 and Baseline Visits using last observation carried forward (ITT Population). The anaerobic threshold is the period in exercise when metabolism switches from aerobic to anaerobic. This is an useful measure of submaximal exercise capacity particularly in the single ventricle population.

Time frame:
26 Weeks
Reported as:
Mean · Watts
Change in Work Rate at VAT From Baseline to Week 26 With LOCF
WattsDrugPlacebo
Change in Work Rate at VAT From Baseline to Week 26 With LOCF3.46 ± 15.0760.31 ± 13.246
Statistical analysis
  • Drug vs Placebo · ANCOVA · p = 0.029 (LS mean was the estimated treatment difference in the analysis model.) · Mean difference (final values): 3.20Fixed factors: ventricular morphology and treatment group, with a continuous covariate of Baseline maximal VO2. LOCF imputation.
SecondaryChange in VE/VCO2 at VAT From Baseline to Week 26

This measure is a ratio of the amount of inspired air required to clear a given volume of CO2 from the circulation at the ventilatory anaerobic threshold (VAT). This measurement is made during exercise stress testing. Decreases in this ratio may reflect an improvement in ventilatory efficiency or an improvement in cardiac function.

Time frame:
26 Weeks
Reported as:
Mean · Ratio
Change in VE/VCO2 at VAT From Baseline to Week 26
RatioDrugPlacebo
Change in VE/VCO2 at VAT From Baseline to Week 26-0.76 ± 3.564-0.05 ± 2.967
Statistical analysis
  • Drug vs Placebo · ANCOVA · p = 0.011 (LS mean was the estimated treatment difference from the analysis model.) · Mean difference (final values): -0.82Fixed factors: ventricular morphology and treatment group with a continuous covariate of Baseline maximal VO2. LOCF imputation.
SecondaryChange in Respiratory Rate at Maximal Exercise Effort From Baseline to Week 26 Using LOCF
Time frame:
Week 26
Reported as:
Mean · Breaths/min
Change in Respiratory Rate at Maximal Exercise Effort From Baseline to Week 26 Using LOCF
Breaths/minUdenafilPlacebo
Change in Respiratory Rate at Maximal Exercise Effort From Baseline to Week 26 Using LOCF-1.01 ± 10.030-1.44 ± 9.928
Statistical analysis
  • Udenafil vs Placebo · ANCOVA · p = 0.696 · Mean difference (final values): 0.35Fixed factors: ventricular morphology and treatment group with a continuous covariate of Baseline VO2. LOCF imputation.
SecondaryChange in Minute Oxygen Consumption at Maximal Exercise Effort From Baseline to Week 26

The amount of air that enters the lungs per minute at maximal exercise effort.

Time frame:
Week 26
Reported as:
Mean · L/min
Change in Minute Oxygen Consumption at Maximal Exercise Effort From Baseline to Week 26
L/minUdenafilPlacebo
Change in Minute Oxygen Consumption at Maximal Exercise Effort From Baseline to Week 261.15 ± 13.934-0.10 ± 13.748
Statistical analysis
  • Udenafil vs Placebo · ANCOVA · p = 0.915 (LS mean was the estimated treatment difference from the analysis model.) · Mean difference (final values): 0.14Fixed factors: ventricular morphology and treatment group with a continuous covariate of Baseline maximal VO2. LOCF imputation.
SecondaryChange in Work Rate at Maximal Exercise Effort Between Baseline and Week 26 Using LOCF

Change in power at maximal exercise effort.

Time frame:
Week 26
Reported as:
Mean · Watts
Change in Work Rate at Maximal Exercise Effort Between Baseline and Week 26 Using LOCF
WattsUdenafilPlacebo
Change in Work Rate at Maximal Exercise Effort Between Baseline and Week 26 Using LOCF2.99 ± 14.3342.45 ± 13.503
Statistical analysis
  • Udenafil vs Placebo · ANCOVA · p = 0.891 (LS mean was the estimated treatment difference from the analysis model.) · Mean difference (final values): 0.19Fixed factors: ventricular morphology and treatment group with a continuous covariate of Baseline maximal VO2. LOCF imputation.
SecondaryChange in Pediatric Quality of Life (PedsQL) Generic Core Scales - Physical Functioning Subscale (Child Reported)

The PedsQL is a short questionnaire that has been used in multiple studies, including a previous study of Fontan patients. A 5-point Likert response scale (0-4) is used. This score was linearly transformed on a scale of 0-100 (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0). For an individual, a higher score is associated with a higher reported healthcare-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 weeks. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

Time frame:
26 weeks
Reported as:
Mean · Scores on a scale
Change in Pediatric Quality of Life (PedsQL) Generic Core Scales - Physical Functioning Subscale (Child Reported)
Scores on a scaleUdenafilPlacebo
Change in Pediatric Quality of Life (PedsQL) Generic Core Scales - Physical Functioning Subscale (Child Reported)2.08 ± 12.0061.53 ± 11.823
Statistical analysis
  • Udenafil vs Placebo · ANCOVA · p = 0.691 (LS mean was the estimated treatment difference from the analysis model.) · Mean difference (final values): 0.44Fixed factors: ventricular morphology and treatment group, with a continuous covariate of Baseline physical functioning (child reported)
SecondaryChange in Pediatric Quality of Life (PedsQL) Generic Core Scales - Physical Functioning Subscale (Parent Reported)

The PedsQL is a short questionnaire that has been used in multiple studies, including a previous study of Fontan patients. A 5-point Likert response scale (0-4) is used. This score was linearly transformed on a scale of 0-100 (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0). For an individual, a higher score is associated with a higher reported healthcare-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 weeks. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

Time frame:
26 weeks
Reported as:
Mean · Scores on a scale
Change in Pediatric Quality of Life (PedsQL) Generic Core Scales - Physical Functioning Subscale (Parent Reported)
Scores on a scaleUdenafilPlacebo
Change in Pediatric Quality of Life (PedsQL) Generic Core Scales - Physical Functioning Subscale (Parent Reported)2.74 ± 18.0111.94 ± 15.328
Statistical analysis
  • Udenafil vs Placebo · ANCOVA · p = 0.985 (LS mean was the estimated treatment difference from the analysis model.) · Mean difference (final values): 0.03Fixed factors: ventricular morphology and treatment group with a continuous covariate of Baseline physical functioning (parent reported).
SecondaryChange in Pediatric Quality of Life (PedsQL) Generic Core Scales - Psychosocial Health Subscale (Child Reported)

The PedsQL is a short questionnaire that has been used in multiple studies, including a previous study of Fontan patients. A 5-point Likert response scale (0-4) is used. This score was linearly transformed on a scale of 0-100 (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0). For an individual, a higher score is associated with a higher reported healthcare-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 weeks. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

Time frame:
26 weeks
Reported as:
Mean · Scores on a scale
Change in Pediatric Quality of Life (PedsQL) Generic Core Scales - Psychosocial Health Subscale (Child Reported)
Scores on a scaleUdenafilPlacebo
Change in Pediatric Quality of Life (PedsQL) Generic Core Scales - Psychosocial Health Subscale (Child Reported)2.84 ± 11.3561.74 ± 10.700
Statistical analysis
  • Udenafil vs Placebo · ANCOVA · p = 0.273 (LS mean was the estimated treatment difference from the analysis model.) · Mean difference (final values): 1.14Fixed factors: ventricular morphology and treatment group with a continuous covariate of Baseline psychosocial health summary score (child reported).
SecondaryChange in Pediatric Quality of Life (PedsQL) Generic Core Scales - Psychosocial Health Subscale (Parent Reported)

The PedsQL is a short questionnaire that has been used in multiple studies, including a previous study of Fontan patients. A 5-point Likert response scale (0-4) is used. This score was linearly transformed on a scale of 0-100 (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0). For an individual, a higher score is associated with a higher reported healthcare-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 weeks. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

Time frame:
26 weeks
Reported as:
Mean · Scores on a scale
Change in Pediatric Quality of Life (PedsQL) Generic Core Scales - Psychosocial Health Subscale (Parent Reported)
Scores on a scaleUdenafilPlacebo
Change in Pediatric Quality of Life (PedsQL) Generic Core Scales - Psychosocial Health Subscale (Parent Reported)2.64 ± 13.5462.15 ± 13.737
Statistical analysis
  • Udenafil vs Placebo · ANCOVA · p = 0.966 (LS mean was the estimated treatment difference from the analysis model.) · Mean difference (final values): -0.06Fixed factors: ventricular morphology and treatment group with a continuous covariate of Baseline psychosocial health summary score (parent reported).
SecondaryChange in Pediatric Quality of Life (PedsQL) Inventory - Cardiac Module Scale - Treatment II Subscale (Child-reported)

The PedsQL is a short questionnaire that has been used in multiple studies, including a previous study of Fontan patients. A 5-point Likert response scale (0-4) is used. This score was linearly transformed on a scale of 0-100 (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0). For an individual, a higher score is associated with a higher reported healthcare-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 weeks. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

Time frame:
26 weeks
Reported as:
Mean · Scores on a scale
Change in Pediatric Quality of Life (PedsQL) Inventory - Cardiac Module Scale - Treatment II Subscale (Child-reported)
Scores on a scaleUdenafilPlacebo
Change in Pediatric Quality of Life (PedsQL) Inventory - Cardiac Module Scale - Treatment II Subscale (Child-reported)0.24 ± 11.655-0.09 ± 9.046
Statistical analysis
  • Udenafil vs Placebo · ANCOVA · p = 0.706 (LS mean was the estimated treatment difference from the analysis model.) · Mean difference (final values): -0.38Fixed factors: ventricular morphology and treatment group with a continuous covariate of Baseline cardiac module scale (Treatment II).
SecondaryChange in Pediatric Quality of Life (PedsQL) Inventory - Cardiac Module Scale - Perceived Physical Appearance Subscale

The PedsQL is a short questionnaire that has been used in multiple studies, including a previous study of Fontan patients. A 5-point Likert response scale (0-4) is used. This score was linearly transformed on a scale of 0-100 (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0). For an individual, a higher score is associated with a higher reported healthcare-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 weeks. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

Time frame:
26 weeks
Reported as:
Mean · Scores on a scale
Change in Pediatric Quality of Life (PedsQL) Inventory - Cardiac Module Scale - Perceived Physical Appearance Subscale
Scores on a scaleUdenafilPlacebo
Change in Pediatric Quality of Life (PedsQL) Inventory - Cardiac Module Scale - Perceived Physical Appearance Subscale2.45 ± 20.8190.78 ± 17.396
Statistical analysis
  • Udenafil vs Placebo · ANCOVA · p = 0.382 (LS mean was the estimated treatment difference from the analysis model.) · Mean difference (final values): 1.50Fixed factors: ventricular morphology and treatment group with a continuous covariate of Baseline cardiac module scale (perceived physical appearance)
SecondaryChange in Pediatric Quality of Life (PedsQL) Inventory - Cardiac Module Scale - Treatment Anxiety Subscale

The PedsQL is a short questionnaire that has been used in multiple studies, including a previous study of Fontan patients. A 5-point Likert response scale (0-4) is used. This score was linearly transformed on a scale of 0-100 (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0). For an individual, a higher score is associated with a higher reported healthcare-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 weeks. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

Time frame:
26 weeks
Reported as:
Mean · Scores on a scale
Change in Pediatric Quality of Life (PedsQL) Inventory - Cardiac Module Scale - Treatment Anxiety Subscale
Scores on a scaleUdenafilPlacebo
Change in Pediatric Quality of Life (PedsQL) Inventory - Cardiac Module Scale - Treatment Anxiety Subscale3.37 ± 19.2581.66 ± 16.477
Statistical analysis
  • Udenafil vs Placebo · ANCOVA · p = 0.236 (LS mean was the estimated treatment difference from the analysis model.) · Mean difference (final values): 1.91Fixed factors: ventricular morphology and treatment group with a continuous covariate of Baseline cardiac module scale (treatment anxiety).
SecondaryChange in Pediatric Quality of Life (PedsQL) Inventory - Cardiac Module Scale - Cognitive Problems Subscale

The PedsQL is a short questionnaire that has been used in multiple studies, including a previous study of Fontan patients. A 5-point Likert response scale (0-4) is used. This score was linearly transformed on a scale of 0-100 (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0). For an individual, a higher score is associated with a higher reported healthcare-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 weeks. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

Time frame:
26 weeks
Reported as:
Mean · Scores on a scale
Change in Pediatric Quality of Life (PedsQL) Inventory - Cardiac Module Scale - Cognitive Problems Subscale
Scores on a scaleUdenafilPlacebo
Change in Pediatric Quality of Life (PedsQL) Inventory - Cardiac Module Scale - Cognitive Problems Subscale2.19 ± 16.8263.05 ± 17.027
Statistical analysis
  • Udenafil vs Placebo · ANCOVA · p = 0.543 (LS mean was the estimated treatment difference from the analysis model.) · Mean difference (final values): -0.99Fixed factors: ventricular morphology and treatment group with a continuous covariate of Baseline cardiac module scale (cognitive problems).
SecondaryChange in Pediatric Quality of Life (PedsQL) Inventory - Cardiac Module Scale - Communication Problems Subscale

The PedsQL is a short questionnaire that has been used in multiple studies, including a previous study of Fontan patients. A 5-point Likert response scale (0-4) is used. This score was linearly transformed on a scale of 0-100 (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0). For an individual, a higher score is associated with a higher reported healthcare-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 week. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

Time frame:
26 weeks
Reported as:
Mean · Scores on a scale
Change in Pediatric Quality of Life (PedsQL) Inventory - Cardiac Module Scale - Communication Problems Subscale
Scores on a scaleUdenafilPlacebo
Change in Pediatric Quality of Life (PedsQL) Inventory - Cardiac Module Scale - Communication Problems Subscale1.82 ± 20.4064.36 ± 17.954
Statistical analysis
  • Udenafil vs Placebo · ANCOVA · p = 0.352 (LS mean was the estimated treatment difference from the analysis model.) · Mean difference (final values): -1.66Fixed factors: ventricular morphology and treatment group with a continuous covariate of Baseline cardiac module scale (communication problems).
SecondaryChange in Pediatric Cardiac Quality of Life Inventory (PCQLI) Total Score-Ages 8 to 12 (Child Reported)

The PCLQI is a disease-specific measure of health-related quality of life for children and adolescents with congenital or acquired heart disease. The PCQLI score is calculated by adding together the scores from the two subscales: Disease Impact and Psychosocial Impact. Each subscale can score a maximum of 50 points, resulting in a total score with a maximum of 100. Higher scores indicate better perceived health-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 weeks. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

Time frame:
26 weeks
Reported as:
Mean · Scores on a scale
Change in Pediatric Cardiac Quality of Life Inventory (PCQLI) Total Score-Ages 8 to 12 (Child Reported)
Scores on a scaleUdenafilPlacebo
Change in Pediatric Cardiac Quality of Life Inventory (PCQLI) Total Score-Ages 8 to 12 (Child Reported)3.26 ± 12.287-2.37 ± 16.368
Statistical analysis
  • Udenafil vs Placebo · ANCOVA · p = 0.533 (LS mean was the estimated treatment difference from the analysis model.) · Mean difference (final values): 3.59Fixed factors of ventricular morphology and treatment group with a continuous covariate of Baseline total score (age 8-12, child reported).
SecondaryChange in Pediatric Cardiac Quality of Life Inventory (PCQLI) Total Score-Ages 8 to 12 (Parent Reported)

The PCLQI is a disease-specific measure of health-related quality of life for children and adolescents with congenital or acquired heart disease. The PCQLI score is calculated by adding together the scores from the two subscales: Disease Impact and Psychosocial Impact. Each subscale can score a maximum of 50 points, resulting in a total score with a maximum of 100. Higher scores indicate better perceived health-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 weeks. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

Time frame:
26 weeks
Reported as:
Mean · Scores on a scale
Change in Pediatric Cardiac Quality of Life Inventory (PCQLI) Total Score-Ages 8 to 12 (Parent Reported)
Scores on a scaleUdenafilPlacebo
Change in Pediatric Cardiac Quality of Life Inventory (PCQLI) Total Score-Ages 8 to 12 (Parent Reported)-0.91 ± 13.399-4.44 ± 6.389
Statistical analysis
  • Udenafil vs Placebo · ANCOVA · p = 0.654 (LS mean was the estimated treatment difference from the analysis model.) · Mean difference (final values): 1.84Fixed factors: ventricular morphology and treatment group with a continuous covariate of Baseline total score (age 8-12, parent reported).
SecondaryChange in Pediatric Cardiac Quality of Life Inventory (PCQLI) Total Score-Ages 13 to 18 (Child Reported)

The PCLQI is a disease-specific measure of health-related quality of life for children and adolescents with congenital or acquired heart disease. The PCQLI score is calculated by adding together the scores from the two subscales: Disease Impact and Psychosocial Impact. Each subscale can score a maximum of 50 points, resulting in a total score with a maximum of 100. Higher scores indicate better perceived health-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 weeks. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

Time frame:
26 weeks
Reported as:
Mean · Scores on a scale
Change in Pediatric Cardiac Quality of Life Inventory (PCQLI) Total Score-Ages 13 to 18 (Child Reported)
Scores on a scaleUdenafilPlacebo
Change in Pediatric Cardiac Quality of Life Inventory (PCQLI) Total Score-Ages 13 to 18 (Child Reported)-0.08 ± 11.0320.09 ± 9.626
Statistical analysis
  • Udenafil vs Placebo · ANCOVA · p = 0.963 (LS mean was the estimated treatment difference from the analysis model.) · Mean difference (final values): -0.05Fixed factors: ventricular morphology and treatment group with a continuous covariate of Baseline total score (ages 13-18, child reported)
SecondaryChange in Pediatric Cardiac Quality of Life Inventory (PCQLI) Total Score-Ages 13 to 18 (Parent Reported)

The PCLQI is a disease-specific measure of health-related quality of life for children and adolescents with congenital or acquired heart disease. The PCQLI score is calculated by adding together the scores from the two subscales: Disease Impact and Psychosocial Impact. Each subscale can score a maximum of 50 points, resulting in a total score with a maximum of 100. Higher scores indicate better perceived health-related quality of life. For each population (treatment and placebo), population means were calculated at baseline and 26 weeks. The outcome measure is the difference between week 26 and baseline (week 26 - baseline).

Time frame:
26 weeks
Reported as:
Mean · Scores on a scale
Change in Pediatric Cardiac Quality of Life Inventory (PCQLI) Total Score-Ages 13 to 18 (Parent Reported)
Scores on a scaleUdenafilPlacebo
Change in Pediatric Cardiac Quality of Life Inventory (PCQLI) Total Score-Ages 13 to 18 (Parent Reported)0.36 ± 11.722-1.60 ± 11.179
Statistical analysis
  • Udenafil vs Placebo · ANCOVA · p = 0.246 (LS mean was the estimated treatment difference from the analysis model.) · Mean difference (final values): 1.36Fixed factors of ventricular morphology and treatment group with a continuous covariate of Baseline total score (ages 13-18, parent reported).

Adverse events

Collected over Adverse Events were recorded over the 26 weeks of study period plus an additional 3 months of follow-up.. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Drug0/200 (0%)14/200 (7%)158/200 (79%)
Placebo0/200 (0%)10/200 (5%)135/200 (67.5%)
Most frequent serious events
Showing 10 of 27
Most frequent serious events
EventDrugPlacebo
DyspnoeaRespiratory, thoracic and mediastinal disorders2/2001/200
SyncopeNervous system disorders0/2002/200
Chest PainRespiratory, thoracic and mediastinal disorders2/2000/200
InfluenzaInfections and infestations2/2000/200
Retinal Artery ThrombosisEye disorders1/2000/200
DiplegiaNervous system disorders1/2000/200
PalpitationNervous system disorders1/2000/200
Gastrointestinal hemorhageGastrointestinal disorders1/2000/200
Protein-losing gastroenteropathyEndocrine disorders1/2000/200
ParalysisNervous system disorders1/2000/200
Most frequent other events
Most frequent other events
EventDrugPlacebo
HeadacheNervous system disorders79/20051/200
FlushingVascular disorders32/20012/200
Upper Respiratory Tract InfectinsInfections and infestations24/20022/200
EpistaxisRespiratory, thoracic and mediastinal disorders21/2005/200
DizzinessNervous system disorders16/20017/200
NauseaGastrointestinal disorders16/2009/200
Chest PainGeneral disorders9/20016/200
VomitingGastrointestinal disorders15/2005/200
NasopharyngitisInfections and infestations13/2009/200

Baseline characteristics

Age, Continuous
Age, Continuous(years)DrugPlaceboTotal
Mean15.4 ± 2.03315.6 ± 1.97815.5 ± 2.005
Sex: Female, Male
Sex: Female, Male(Participants)DrugPlaceboTotal
Female8972161
Male111128239
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)DrugPlaceboTotal
Hispanic or Latino312556
Not Hispanic or Latino168174342
Unknown or Not Reported112
Race (NIH/OMB)
Race (NIH/OMB)(Participants)DrugPlaceboTotal
American Indian or Alaska Native000
Asian172138
Native Hawaiian or Other Pacific Islander000
Black or African American101323
White169155324
More than one race224
Unknown or Not Reported2911
Region of Enrollment
Region of Enrollment(participants)DrugPlaceboTotal
Canada81119
South Korea141933
United States178170348
Peak VO2
Peak VO2(mL/kg/min)DrugPlaceboTotal
Mean27.84 ± 6.87728.01 ± 6.12827.92 ± 6.50
Weight
Weight(kg)DrugPlaceboTotal
Mean57.1 ± 13.92559.0 ± 13.18758.1 ± 13.577
Maximal V02 at ventilatory anaerobic threshold (VAT)
Maximal V02 at ventilatory anaerobic threshold (VAT)(mL/kig/min)DrugPlaceboTotal
Mean18.35 ± 4.63317.71 ± 4.30418.02 ± 4.471

21 further baseline measures are reported on the registry.

08

Study locations

30 sites
  • Phoenix Children's Hospital/Children's Heart Center at Phoenix Children's Hospital
    Phoenix, Arizona 85016, United States
  • Children's Hospital Los Angeles
    Los Angeles, California 90027, United States
  • Cedars/Sinai Heart Institute
    Los Angeles, California 90048, United States
  • Rady Children's Hospital
    San Diego, California 92123, United States
  • Children's Hospital Colorado
    Aurora, Colorado 80045, United States
  • Nemours Cardiac Center/Alfred I. duPont Hospital for Children
    Wilmington, Delaware 19803, United States
  • Children's National Medical Center
    Washington, District of Columbia 20008, United States
  • Johns Hopkins All Children's Heart Institute
    Saint Petersburg, Florida 33701, United States
  • Children's Healthcare of Atlanta
    Atlanta, Georgia 30322, United States
  • Riley Hospital for Children/Herman B. Wells Center for Pediatric Research
    Indianapolis, Indiana 46201, United States
  • Boston Children's Hospital
    Boston, Massachusetts 02115, United States
  • University of Michigan Congenital Heart Center/C.S. Mott Children's Hospital
    Ann Arbor, Michigan 48109-4204, United States
  • Children's Mercy Hospital Kansas City
    Kansas City, Missouri 64108, United States
  • Washington University St. Louis/St.Louis Children's Hospital
    Saint Louis, Missouri 63110, United States
  • University of Nebraska Children's Hospital and Medical Center
    Omaha, Nebraska 68114, United States
  • Children's Hospital of New York
    New York, New York 10032, United States
  • Duke University Medical Center
    Durham, North Carolina 27710, United States
  • Cincinnati Children's Hospital Medical Center
    Cincinnati, Ohio 45229, United States
  • Nationwide Children's Hosptial
    Columbus, Ohio 43205, United States
  • Children's Hospital of Philadelphia
    Philadelphia, Pennsylvania 19104, United States
  • Medical University of South Carolina
    Charleston, South Carolina 29425, United States
  • Vanderbilt University Medical Center
    Nashville, Tennessee 37232, United States
  • Texas Children's Hospital
    Houston, Texas 77030, United States
  • Primary Children's Medical Hospital/Dept. of Pediatric Cardiology
    Salt Lake City, Utah 84113, United States
  • Seattle Children's Hosptial
    Seattle, Washington 98105, United States
  • Children's Hospital of Wisconsin
    Milwaukee, Wisconsin 53226, United States
  • Stollery Children's Hospital - University of Alberta
    Edmonton, Alberta T6G 2B7, Canada
  • The Hospital for Sick Children
    Toronto, Ontario ON M5G 1X8, Canada
  • Sejong General Hospital
    Bucheon-si, Gyeonggi-do 14754, Korea, Republic of
  • Seoul National University Children's Hospital
    Seoul, 110-744, Korea, Republic of
09

References and documents

Publications

  • Di Maria MV, Goldberg DJ, Zak V, Hu C, Lubert AM, Dragulescu A, Mackie AS, McCrary A, Weingarten A, Parthiban A, Goot B, Goldstein BH, Taylor C, Lindblade C, Petit CJ, Spurney C, Harrild DM, Urbina EM, Schuchardt E, Beom Kim G, Kyoung Yoon J, Colombo JN, Files MD, Schoessling M, Ermis P, Wong PC, Garg R, Swanson SK, Menon SC, Srivastava S, Thorsson T, Johnson TR, Krishnan US, Paridon SM, Frommelt PC; Pediatric Heart Network. Impact of Udenafil on Echocardiographic Indices of Single Ventricle Size and Function in FUEL Study Participants. Circ Cardiovasc Imaging. 2022 Nov;15(11):e013676. doi: 10.1161/CIRCIMAGING.121.013676. Epub 2022 Nov 15. PubMed 36378780 ↗
  • Goldberg DJ, Zak V, Goldstein BH, Schumacher KR, Rhodes J, Penny DJ, Petit CJ, Ginde S, Menon SC, Kim SH, Kim GB, Nowlen TT, DiMaria MV, Frischhertz BP, Wagner JB, McHugh KE, McCrindle BW, Shillingford AJ, Sabati AA, Yetman AT, John AS, Richmond ME, Files MD, Payne RM, Mackie AS, Davis CK, Shahanavaz S, Hill KD, Garg R, Jacobs JP, Hamstra MS, Woyciechowski S, Rathge KA, McBride MG, Frommelt PC, Russell MW, Urbina EM, Yeager JL, Pemberton VL, Stylianou MP, Pearson GD, Paridon SM; Pediatric Heart Network Investigators. Results of the FUEL Trial. Circulation. 2020 Feb 25;141(8):641-651. doi: 10.1161/CIRCULATIONAHA.119.044352. Epub 2019 Nov 17. PubMed 31736357 ↗
  • 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. PubMed 29910047 ↗

Study documents

  • Study protocol · Feb 27, 2021
  • Statistical analysis plan · Jun 23, 2016

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jun 17, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT02741115
Lead sponsor
Mezzion Pharma Co. Ltd
Collaborators
National Heart, Lung, and Blood Institute (NHLBI)
Responsible party
Sponsor
First posted
Apr 18, 2016
Start date
Jul 22, 2016
Primary completion
Dec 27, 2018
Completion
Apr 30, 2019
Results posted
May 21, 2025
Last update
Jun 17, 2025

Study contacts

Steve Paridon, MD
principal investigator · Children's Hospital of Phildelphia

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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