Functional Single Ventricle Heart Disease
Conditions
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.
Interventions
DRUGUdenafil
Active drug
Sponsors
National Heart, Lung, and Blood Institute (NHLBI)
Mezzion Pharma Co. Ltd
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to 18 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Treatment-Emergent Adverse Events | 52 Weeks | Summary of Treatment Emergent Adverse Events |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Log-transformed Reactive Hyperemia Index (lnRHI) | 52 Weeks | Change from baseline to 52 weeks in Log-transformed Reactive Hyperemia Index (lnRHI). The Reactive Hyperemia Index (RHI) is the ratio of the amount of blood flow after the release of a period of vessel occlusion relative to baseline amount of blood flow. Because the amount of blood does not decrease after release of vessel occlusion, the minimum value for the RHI is 1.0, which would indicate no change in blood flow. There is no upper limit for this ratio, and higher values are generally thought to represent a healthier endothelial response. In this study, as in other studies, we used the natural log of RHI (lnRHI) to normalize the data. |
| Change in Serum BNP | 52 Weeks | Change in Serum BNP from baseline to 52 weeks |
| Change in Myocardial Performance Index (MPI) | 52 Weeks | Change in Myocardial Performance Index from baseline to 52 weeks |
| Change in PedsQL Physical Functioning (Child Reported) | 52 Weeks | Change in PedsQL Physical Functioning (Child Reported) from baseline to 52 weeks. The Pediatric Quality of Life Inventory (PedsQL) - Physical Function Scale is calculated from a child's answers to a short questionnaire. The range of possible scores is 0-100, and higher scores indicate better reported physical function. |
| Change in Maximal VO2 | 52 Weeks | Change in Maximal VO2 from baseline to 52 weeks |
| Change in PedsQL Psychosocial Health Summary Score (Child Reported) | 52 Weeks | Change in PedsQL Psychosocial Health Summary Score (Child reported) from baseline to 52 weeks. The Pediatric Quality of Life Inventory (PedsQL) - Psychosocial Health Summary Scale is calculated from a child's answers to a short questionnaire. The range of possible scores is 0-100, and higher scores indicate better reported psychosocial health. |
| Change in PedsQL Psychosocial Health Summary Score (Parent Reported) | 52 Weeks | Change in PedsQL Psychosocial Health Summary Score (Parent reported) from baseline to 52 weeks. The Pediatric Quality of Life Inventory (PedsQL) - Psychosocial Health Summary Scale is calculated from parent's answers to a short questionnaire. The range of possible scores is 0-100, and higher scores indicate better reported psychosocial health. |
| Change in PedsQL Functional Health Status Score (Child Reported) | 52 Weeks | Change in PedsQL Functional Health Status Score (Child reported) from baseline to 52 weeks. The Pediatric Quality of Life Inventory (PedsQL) - Functional Health Status Score is calculated from a child's answers to a short questionnaire. The range of possible scores is 0-100, and higher scores indicate better functional health status. |
| Change in PedsQL Functional Health Status Score (Parent Reported) | 52 Weeks | Change in PedsQL Functional Health Status Score (Parent reported) from baseline to 52 weeks. The Pediatric Quality of Life Inventory (PedsQL) - Functional Health Status Score is calculated from parent's answers to a short questionnaire. The range of possible scores is 0-100, and higher scores indicate better functional health status. |
| Change in PedsQL Physical Functioning (Parent Reported) | 52 Weeks | Change in PedsQL Physical Functioning (Parent Reported) from baseline to 52 weeks. The Pediatric Quality of Life Inventory (PedsQL) - Physical Function Scale is calculated from parent's answers to a short questionnaire. The range of possible scores is 0-100, and higher scores indicate better reported physical function. |
Countries
Canada, South Korea, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| In PHN-Udenafil-02 (FUEL) Received Udenafil Participants in this study arm had received Udenafil in PHN-Udenafil-02 study. | 128 |
| In PHN-Udenafil-02 (FUEL) Received Placebo Participants in this study arm had received Placebo in PHN-Udenafil-02 study. | 122 |
| De Novo Did not participate in PHN-Udenafil-02 study. | 51 |
| Total | 301 |
Baseline characteristics
| Characteristic | Total | De Novo | In PHN-Udenafil-02 (FUEL) Received Placebo | In PHN-Udenafil-02 (FUEL) Received Udenafil |
|---|---|---|---|---|
| Age, Customized | 15.9 Years STANDARD_DEVIATION 2 | 14.9 Years STANDARD_DEVIATION 1.89 | 16.2 Years STANDARD_DEVIATION 1.94 | 16.1 Years STANDARD_DEVIATION 2 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 35 Participants | 4 Participants | 14 Participants | 17 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 264 Participants | 47 Participants | 107 Participants | 110 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 2 Participants | 0 Participants | 1 Participants | 1 Participants |
| Height | 164.9 centimeter STANDARD_DEVIATION 9.52 | 163.2 centimeter STANDARD_DEVIATION 9.08 | 166.3 centimeter STANDARD_DEVIATION 8.84 | 164.2 centimeter STANDARD_DEVIATION 10.18 |
| Log-transformed Reactive Hyperemia Index (lnRHI) | 1.745 Natural log-transformed RHI (lnRHI) STANDARD_DEVIATION 0.5117 | 1.585 Natural log-transformed RHI (lnRHI) STANDARD_DEVIATION 0.3987 | 1.757 Natural log-transformed RHI (lnRHI) STANDARD_DEVIATION 0.5403 | 1.748 Natural log-transformed RHI (lnRHI) STANDARD_DEVIATION 0.494 |
| Maximal VO2 | 1610.4 mL/min STANDARD_DEVIATION 449.13 | 1421.7 mL/min STANDARD_DEVIATION 261.32 | 1637.6 mL/min STANDARD_DEVIATION 444.26 | 1607.3 mL/min STANDARD_DEVIATION 470.8 |
| Myocardial Performance Index (MPI) | 0.45 Ratio STANDARD_DEVIATION 0.146 | 0.59 Ratio STANDARD_DEVIATION 0.326 | 0.46 Ratio STANDARD_DEVIATION 0.152 | 0.43 Ratio STANDARD_DEVIATION 0.118 |
| Pediatric Quality of Life (PedsQL)-Functional Health Status Score-Child reported | 73.26 score on a scale STANDARD_DEVIATION 13.795 | 71.53 score on a scale STANDARD_DEVIATION 12.802 | 73.56 score on a scale STANDARD_DEVIATION 14.755 | 73.50 score on a scale STANDARD_DEVIATION 13.108 |
| Pediatric Quality of Life (PedsQL)-Functional Health Status Score-Parent reported | 72.85 score on a scale STANDARD_DEVIATION 15.122 | 68.20 score on a scale STANDARD_DEVIATION 15.488 | 74.75 score on a scale STANDARD_DEVIATION 14.248 | 72.49 score on a scale STANDARD_DEVIATION 15.621 |
| Pediatric Quality of Life (PedsQL)-Physical Functioning-Child reported | 74.04 score on a scale STANDARD_DEVIATION 14.848 | 73.99 score on a scale STANDARD_DEVIATION 13.722 | 74.40 score on a scale STANDARD_DEVIATION 16.142 | 73.68 score on a scale STANDARD_DEVIATION 13.845 |
| Pediatric Quality of Life (PedsQL)-Physical Functioning-Parent reported | 76.18 score on a scale STANDARD_DEVIATION 16.324 | 73.24 score on a scale STANDARD_DEVIATION 18.112 | 76.90 score on a scale STANDARD_DEVIATION 14.948 | 76.43 score on a scale STANDARD_DEVIATION 17.064 |
| Pediatric Quality of Life (PedsQL)-Psychosocial Health Summary Score-Child reported | 72.83 score on a scale STANDARD_DEVIATION 15.327 | 70.23 score on a scale STANDARD_DEVIATION 14.429 | 73.11 score on a scale STANDARD_DEVIATION 16.306 | 73.40 score on a scale STANDARD_DEVIATION 14.562 |
| Pediatric Quality of Life (PedsQL)-Psychosocial Health Summary Score-Parent reported | 71.10 score on a scale STANDARD_DEVIATION 17.139 | 65.50 score on a scale STANDARD_DEVIATION 17.458 | 73.64 score on a scale STANDARD_DEVIATION 16.558 | 70.40 score on a scale STANDARD_DEVIATION 17.258 |
| Race (NIH/OMB) American Indian or Alaska Native | 2 Participants | 2 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 6 Participants | 2 Participants | 2 Participants | 2 Participants |
| Race (NIH/OMB) Black or African American | 14 Participants | 6 Participants | 5 Participants | 3 Participants |
| Race (NIH/OMB) More than one race | 5 Participants | 1 Participants | 2 Participants | 2 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 10 Participants | 2 Participants | 6 Participants | 2 Participants |
| Race (NIH/OMB) White | 264 Participants | 38 Participants | 107 Participants | 119 Participants |
| Serum BNP | 16.85 pg/mL STANDARD_DEVIATION 16.891 | — | 15.58 pg/mL STANDARD_DEVIATION 18.638 | 18.25 pg/mL STANDARD_DEVIATION 14.796 |
| Sex: Female, Male Female | 118 Participants | 19 Participants | 44 Participants | 55 Participants |
| Sex: Female, Male Male | 183 Participants | 32 Participants | 78 Participants | 73 Participants |
| Weight | 59.5 kilogram STANDARD_DEVIATION 13.86 | 57.7 kilogram STANDARD_DEVIATION 15.1 | 60.7 kilogram STANDARD_DEVIATION 13.58 | 59.2 kilogram STANDARD_DEVIATION 13.62 |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 301 |
| other Total, other adverse events | 257 / 301 |
| serious Total, serious adverse events | 54 / 301 |
Outcome results
Primary
Treatment-Emergent Adverse Events
Summary of Treatment Emergent Adverse Events
Time frame: 52 Weeks
Population: Safety Population
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Total | Treatment-Emergent Adverse Events | Subjects with one or more TEAEs adverse event (TEAE) | 257 Participants |
| Total | Treatment-Emergent Adverse Events | Subjects with one or more drug-related TEAEs | 206 Participants |
| Total | Treatment-Emergent Adverse Events | Subjects with one or more TEAEs of Grade >=3 | 31 Participants |
| Total | Treatment-Emergent Adverse Events | Subjects with one or more SAEs | 54 Participants |
| Total | Treatment-Emergent Adverse Events | Subjects with one or more drug-related SAEs | 15 Participants |
| Total | Treatment-Emergent Adverse Events | Subjects who temporarily stopped study drug for one or more TEAEs | 35 Participants |
| Total | Treatment-Emergent Adverse Events | Subjects who permanently discontinued study drug for one or more TEAEs | 21 Participants |
Secondary
Change in Log-transformed Reactive Hyperemia Index (lnRHI)
Change from baseline to 52 weeks in Log-transformed Reactive Hyperemia Index (lnRHI). The Reactive Hyperemia Index (RHI) is the ratio of the amount of blood flow after the release of a period of vessel occlusion relative to baseline amount of blood flow. Because the amount of blood does not decrease after release of vessel occlusion, the minimum value for the RHI is 1.0, which would indicate no change in blood flow. There is no upper limit for this ratio, and higher values are generally thought to represent a healthier endothelial response. In this study, as in other studies, we used the natural log of RHI (lnRHI) to normalize the data.
Time frame: 52 Weeks
Population: Results presented only for subjects who have data at baseline and 52 weeks (i.e., paired-data points).
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Total | Change in Log-transformed Reactive Hyperemia Index (lnRHI) | Baseline | 1.748 Natural log-transformed RHI (lnRHI) | Standard Deviation 0.494 |
| Total | Change in Log-transformed Reactive Hyperemia Index (lnRHI) | 52 weeks | 1.759 Natural log-transformed RHI (lnRHI) | Standard Deviation 0.5171 |
| In PHN-Udenafil-02 (FUEL) Received Placebo | Change in Log-transformed Reactive Hyperemia Index (lnRHI) | 52 weeks | 2.037 Natural log-transformed RHI (lnRHI) | Standard Deviation 0.6405 |
| In PHN-Udenafil-02 (FUEL) Received Placebo | Change in Log-transformed Reactive Hyperemia Index (lnRHI) | Baseline | 1.757 Natural log-transformed RHI (lnRHI) | Standard Deviation 0.5403 |
| De Novo | Change in Log-transformed Reactive Hyperemia Index (lnRHI) | Baseline | 1.585 Natural log-transformed RHI (lnRHI) | Standard Deviation 0.3987 |
| De Novo | Change in Log-transformed Reactive Hyperemia Index (lnRHI) | 52 weeks | 1.960 Natural log-transformed RHI (lnRHI) | Standard Deviation 0.5315 |
| Total | Change in Log-transformed Reactive Hyperemia Index (lnRHI) | Baseline | 1.745 Natural log-transformed RHI (lnRHI) | Standard Deviation 0.5117 |
| Total | Change in Log-transformed Reactive Hyperemia Index (lnRHI) | 52 weeks | 1.902 Natural log-transformed RHI (lnRHI) | Standard Deviation 0.5931 |
Secondary
Change in Maximal VO2
Change in Maximal VO2 from baseline to 52 weeks
Time frame: 52 Weeks
Population: Results presented only for subjects who have data at baseline and 52 weeks (i.e., paired-data points). VO2 has been presented in mL/min (i.e., not adjusted for body weight).
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Total | Change in Maximal VO2 | Baseline | 1607.3 mL/min | Standard Deviation 470.8 |
| Total | Change in Maximal VO2 | 52 weeks | 1646.1 mL/min | Standard Deviation 433.94 |
| In PHN-Udenafil-02 (FUEL) Received Placebo | Change in Maximal VO2 | 52 weeks | 1703.0 mL/min | Standard Deviation 434.31 |
| In PHN-Udenafil-02 (FUEL) Received Placebo | Change in Maximal VO2 | Baseline | 1637.6 mL/min | Standard Deviation 444.26 |
| De Novo | Change in Maximal VO2 | Baseline | 1421.7 mL/min | Standard Deviation 261.32 |
| De Novo | Change in Maximal VO2 | 52 weeks | 1425.0 mL/min | Standard Deviation 269.12 |
| Total | Change in Maximal VO2 | Baseline | 1610.4 mL/min | Standard Deviation 449.13 |
| Total | Change in Maximal VO2 | 52 weeks | 1659.6 mL/min | Standard Deviation 429.34 |
Secondary
Change in Myocardial Performance Index (MPI)
Change in Myocardial Performance Index from baseline to 52 weeks
Time frame: 52 Weeks
Population: Efficacy population
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Total | Change in Myocardial Performance Index (MPI) | Baseline | 0.43 mL/min | Standard Deviation 0.118 |
| Total | Change in Myocardial Performance Index (MPI) | 52 weeks | 0.43 mL/min | Standard Deviation 0.14 |
| In PHN-Udenafil-02 (FUEL) Received Placebo | Change in Myocardial Performance Index (MPI) | 52 weeks | 0.41 mL/min | Standard Deviation 0.14 |
| In PHN-Udenafil-02 (FUEL) Received Placebo | Change in Myocardial Performance Index (MPI) | Baseline | 0.46 mL/min | Standard Deviation 0.152 |
| De Novo | Change in Myocardial Performance Index (MPI) | Baseline | 0.59 mL/min | Standard Deviation 0.326 |
| De Novo | Change in Myocardial Performance Index (MPI) | 52 weeks | 0.53 mL/min | Standard Deviation 0.148 |
| Total | Change in Myocardial Performance Index (MPI) | Baseline | 0.45 mL/min | Standard Deviation 0.146 |
| Total | Change in Myocardial Performance Index (MPI) | 52 weeks | 0.42 mL/min | Standard Deviation 0.141 |
Secondary
Change in PedsQL Functional Health Status Score (Child Reported)
Change in PedsQL Functional Health Status Score (Child reported) from baseline to 52 weeks. The Pediatric Quality of Life Inventory (PedsQL) - Functional Health Status Score is calculated from a child's answers to a short questionnaire. The range of possible scores is 0-100, and higher scores indicate better functional health status.
Time frame: 52 Weeks
Population: Results presented only for subjects who have data at baseline and 52 weeks (i.e., paired-data points).
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Total | Change in PedsQL Functional Health Status Score (Child Reported) | Baseline | 73.50 score on a scale | Standard Deviation 13.108 |
| Total | Change in PedsQL Functional Health Status Score (Child Reported) | 52 weeks | 73.64 score on a scale | Standard Deviation 15.794 |
| In PHN-Udenafil-02 (FUEL) Received Placebo | Change in PedsQL Functional Health Status Score (Child Reported) | 52 weeks | 72.98 score on a scale | Standard Deviation 15.735 |
| In PHN-Udenafil-02 (FUEL) Received Placebo | Change in PedsQL Functional Health Status Score (Child Reported) | Baseline | 73.56 score on a scale | Standard Deviation 14.755 |
| De Novo | Change in PedsQL Functional Health Status Score (Child Reported) | Baseline | 71.53 score on a scale | Standard Deviation 12.802 |
| De Novo | Change in PedsQL Functional Health Status Score (Child Reported) | 52 weeks | 72.09 score on a scale | Standard Deviation 14.762 |
| Total | Change in PedsQL Functional Health Status Score (Child Reported) | Baseline | 73.26 score on a scale | Standard Deviation 13.795 |
| Total | Change in PedsQL Functional Health Status Score (Child Reported) | 52 weeks | 73.13 score on a scale | Standard Deviation 15.58 |
Secondary
Change in PedsQL Functional Health Status Score (Parent Reported)
Change in PedsQL Functional Health Status Score (Parent reported) from baseline to 52 weeks. The Pediatric Quality of Life Inventory (PedsQL) - Functional Health Status Score is calculated from parent's answers to a short questionnaire. The range of possible scores is 0-100, and higher scores indicate better functional health status.
Time frame: 52 Weeks
Population: Results presented only for subjects who have data at baseline and 52 weeks (i.e., paired-data points).
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Total | Change in PedsQL Functional Health Status Score (Parent Reported) | Baseline | 72.49 score on a scale | Standard Deviation 15.621 |
| Total | Change in PedsQL Functional Health Status Score (Parent Reported) | 52 weeks | 72.85 score on a scale | Standard Deviation 16.638 |
| In PHN-Udenafil-02 (FUEL) Received Placebo | Change in PedsQL Functional Health Status Score (Parent Reported) | 52 weeks | 73.63 score on a scale | Standard Deviation 15.044 |
| In PHN-Udenafil-02 (FUEL) Received Placebo | Change in PedsQL Functional Health Status Score (Parent Reported) | Baseline | 74.75 score on a scale | Standard Deviation 14.248 |
| De Novo | Change in PedsQL Functional Health Status Score (Parent Reported) | Baseline | 68.20 score on a scale | Standard Deviation 15.488 |
| De Novo | Change in PedsQL Functional Health Status Score (Parent Reported) | 52 weeks | 70.28 score on a scale | Standard Deviation 13.181 |
| Total | Change in PedsQL Functional Health Status Score (Parent Reported) | Baseline | 72.85 score on a scale | Standard Deviation 15.122 |
| Total | Change in PedsQL Functional Health Status Score (Parent Reported) | 52 weeks | 72.82 score on a scale | Standard Deviation 15.491 |
Secondary
Change in PedsQL Physical Functioning (Child Reported)
Change in PedsQL Physical Functioning (Child Reported) from baseline to 52 weeks. The Pediatric Quality of Life Inventory (PedsQL) - Physical Function Scale is calculated from a child's answers to a short questionnaire. The range of possible scores is 0-100, and higher scores indicate better reported physical function.
Time frame: 52 Weeks
Population: Results presented only for subjects who have data at baseline and 52 weeks (i.e., paired-data points).
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Total | Change in PedsQL Physical Functioning (Child Reported) | Baseline | 73.68 score on a scale | Standard Deviation 13.845 |
| Total | Change in PedsQL Physical Functioning (Child Reported) | 52 weeks | 74.75 score on a scale | Standard Deviation 15.823 |
| In PHN-Udenafil-02 (FUEL) Received Placebo | Change in PedsQL Physical Functioning (Child Reported) | 52 weeks | 74.10 score on a scale | Standard Deviation 17.691 |
| In PHN-Udenafil-02 (FUEL) Received Placebo | Change in PedsQL Physical Functioning (Child Reported) | Baseline | 74.40 score on a scale | Standard Deviation 16.142 |
| De Novo | Change in PedsQL Physical Functioning (Child Reported) | Baseline | 73.99 score on a scale | Standard Deviation 13.722 |
| De Novo | Change in PedsQL Physical Functioning (Child Reported) | 52 weeks | 73.56 score on a scale | Standard Deviation 14.669 |
| Total | Change in PedsQL Physical Functioning (Child Reported) | Baseline | 74.04 score on a scale | Standard Deviation 14.848 |
| Total | Change in PedsQL Physical Functioning (Child Reported) | 52 weeks | 74.30 score on a scale | Standard Deviation 16.482 |
Secondary
Change in PedsQL Physical Functioning (Parent Reported)
Change in PedsQL Physical Functioning (Parent Reported) from baseline to 52 weeks. The Pediatric Quality of Life Inventory (PedsQL) - Physical Function Scale is calculated from parent's answers to a short questionnaire. The range of possible scores is 0-100, and higher scores indicate better reported physical function.
Time frame: 52 Weeks
Population: Results presented only for subjects who have data at baseline and 52 weeks (i.e., paired-data points).
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Total | Change in PedsQL Physical Functioning (Parent Reported) | Baseline | 76.43 score on a scale | Standard Deviation 17.064 |
| Total | Change in PedsQL Physical Functioning (Parent Reported) | 52 weeks | 75.99 score on a scale | Standard Deviation 18.205 |
| In PHN-Udenafil-02 (FUEL) Received Placebo | Change in PedsQL Physical Functioning (Parent Reported) | 52 weeks | 76.10 score on a scale | Standard Deviation 15.772 |
| In PHN-Udenafil-02 (FUEL) Received Placebo | Change in PedsQL Physical Functioning (Parent Reported) | Baseline | 76.90 score on a scale | Standard Deviation 14.948 |
| De Novo | Change in PedsQL Physical Functioning (Parent Reported) | Baseline | 73.24 score on a scale | Standard Deviation 18.112 |
| De Novo | Change in PedsQL Physical Functioning (Parent Reported) | 52 weeks | 74.11 score on a scale | Standard Deviation 13.946 |
| Total | Change in PedsQL Physical Functioning (Parent Reported) | Baseline | 76.18 score on a scale | Standard Deviation 16.324 |
| Total | Change in PedsQL Physical Functioning (Parent Reported) | 52 weeks | 75.77 score on a scale | Standard Deviation 16.582 |
Secondary
Change in PedsQL Psychosocial Health Summary Score (Child Reported)
Change in PedsQL Psychosocial Health Summary Score (Child reported) from baseline to 52 weeks. The Pediatric Quality of Life Inventory (PedsQL) - Psychosocial Health Summary Scale is calculated from a child's answers to a short questionnaire. The range of possible scores is 0-100, and higher scores indicate better reported psychosocial health.
Time frame: 52 Weeks
Population: Results presented only for subjects who have data at baseline and 52 weeks (i.e., paired-data points).
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Total | Change in PedsQL Psychosocial Health Summary Score (Child Reported) | Baseline | 73.40 score on a scale | Standard Deviation 14.562 |
| Total | Change in PedsQL Psychosocial Health Summary Score (Child Reported) | 52 weeks | 73.01 score on a scale | Standard Deviation 17.451 |
| In PHN-Udenafil-02 (FUEL) Received Placebo | Change in PedsQL Psychosocial Health Summary Score (Child Reported) | 52 weeks | 72.37 score on a scale | Standard Deviation 17.174 |
| In PHN-Udenafil-02 (FUEL) Received Placebo | Change in PedsQL Psychosocial Health Summary Score (Child Reported) | Baseline | 73.11 score on a scale | Standard Deviation 16.306 |
| De Novo | Change in PedsQL Psychosocial Health Summary Score (Child Reported) | Baseline | 70.23 score on a scale | Standard Deviation 14.429 |
| De Novo | Change in PedsQL Psychosocial Health Summary Score (Child Reported) | 52 weeks | 71.31 score on a scale | Standard Deviation 17.015 |
| Total | Change in PedsQL Psychosocial Health Summary Score (Child Reported) | Baseline | 72.83 score on a scale | Standard Deviation 15.327 |
| Total | Change in PedsQL Psychosocial Health Summary Score (Child Reported) | 52 weeks | 72.49 score on a scale | Standard Deviation 17.212 |
Secondary
Change in PedsQL Psychosocial Health Summary Score (Parent Reported)
Change in PedsQL Psychosocial Health Summary Score (Parent reported) from baseline to 52 weeks. The Pediatric Quality of Life Inventory (PedsQL) - Psychosocial Health Summary Scale is calculated from parent's answers to a short questionnaire. The range of possible scores is 0-100, and higher scores indicate better reported psychosocial health.
Time frame: 52 Weeks
Population: Results presented only for subjects who have data at baseline and 52 weeks (i.e., paired-data points).
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Total | Change in PedsQL Psychosocial Health Summary Score (Parent Reported) | Baseline | 70.40 score on a scale | Standard Deviation 17.258 |
| Total | Change in PedsQL Psychosocial Health Summary Score (Parent Reported) | 52 weeks | 71.18 score on a scale | Standard Deviation 18.027 |
| In PHN-Udenafil-02 (FUEL) Received Placebo | Change in PedsQL Psychosocial Health Summary Score (Parent Reported) | 52 weeks | 72.32 score on a scale | Standard Deviation 17.099 |
| In PHN-Udenafil-02 (FUEL) Received Placebo | Change in PedsQL Psychosocial Health Summary Score (Parent Reported) | Baseline | 73.64 score on a scale | Standard Deviation 16.558 |
| De Novo | Change in PedsQL Psychosocial Health Summary Score (Parent Reported) | Baseline | 65.50 score on a scale | Standard Deviation 17.458 |
| De Novo | Change in PedsQL Psychosocial Health Summary Score (Parent Reported) | 52 weeks | 68.24 score on a scale | Standard Deviation 14.73 |
| Total | Change in PedsQL Psychosocial Health Summary Score (Parent Reported) | Baseline | 71.10 score on a scale | Standard Deviation 17.139 |
| Total | Change in PedsQL Psychosocial Health Summary Score (Parent Reported) | 52 weeks | 71.25 score on a scale | Standard Deviation 17.184 |
Secondary
Change in Serum BNP
Change in Serum BNP from baseline to 52 weeks
Time frame: 52 Weeks
Population: Results presented only for subjects who have data at baseline and 52 weeks (i.e., paired-data points). The de novo population did not have any paired-data.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Total | Change in Serum BNP | Baseline | 18.25 pg/mL | Standard Deviation 14.796 |
| Total | Change in Serum BNP | 52 weeks | 26.18 pg/mL | Standard Deviation 24.241 |
| In PHN-Udenafil-02 (FUEL) Received Placebo | Change in Serum BNP | Baseline | 15.58 pg/mL | Standard Deviation 18.638 |
| In PHN-Udenafil-02 (FUEL) Received Placebo | Change in Serum BNP | 52 weeks | 23.13 pg/mL | Standard Deviation 24.584 |
| Total | Change in Serum BNP | Baseline | 16.85 pg/mL | Standard Deviation 16.891 |
| Total | Change in Serum BNP | 52 weeks | 24.58 pg/mL | Standard Deviation 24.347 |