Trial Outcomes & Findings for A Extension Study of Udenafil in Adolescents (NCT NCT03013751)
NCT ID: NCT03013751
Last Updated: 2025-12-09
Results Overview
Summary of Treatment Emergent Adverse Events
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
301 participants
Primary outcome timeframe
52 Weeks
Results posted on
2025-12-09
Participant Flow
Participant milestones
| Measure |
In PHN-Udenafil-02 (FUEL) Received Udenafil
Participants in this study arm had received Udenafil in PHN-Udenafil-02 study.
|
In PHN-Udenafil-02 (FUEL) Received Placebo
Participants in this study arm had received Placebo in PHN-Udenafil-02 study.
|
De novo
Did not participate in PHN-Udenafil-02 study.
|
|---|---|---|---|
|
Overall Study
STARTED
|
128
|
122
|
51
|
|
Overall Study
COMPLETED
|
128
|
122
|
51
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points). VO2 has been presented in mL/min (i.e., not adjusted for body weight).
Baseline characteristics by cohort
| Measure |
In PHN-Udenafil-02 (FUEL) Received Udenafil
n=128 Participants
Participants in this study arm had received Udenafil in PHN-Udenafil-02 study.
|
In PHN-Udenafil-02 (FUEL) Received Placebo
n=122 Participants
Participants in this study arm had received Placebo in PHN-Udenafil-02 study.
|
De Novo
n=51 Participants
Did not participate in PHN-Udenafil-02 study.
|
Total
n=301 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Customized
|
16.1 Years
STANDARD_DEVIATION 2.00 • n=128 Participants
|
16.2 Years
STANDARD_DEVIATION 1.94 • n=122 Participants
|
14.9 Years
STANDARD_DEVIATION 1.89 • n=51 Participants
|
15.9 Years
STANDARD_DEVIATION 2.00 • n=301 Participants
|
|
Sex: Female, Male
Female
|
55 Participants
n=128 Participants
|
44 Participants
n=122 Participants
|
19 Participants
n=51 Participants
|
118 Participants
n=301 Participants
|
|
Sex: Female, Male
Male
|
73 Participants
n=128 Participants
|
78 Participants
n=122 Participants
|
32 Participants
n=51 Participants
|
183 Participants
n=301 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
17 Participants
n=128 Participants
|
14 Participants
n=122 Participants
|
4 Participants
n=51 Participants
|
35 Participants
n=301 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
110 Participants
n=128 Participants
|
107 Participants
n=122 Participants
|
47 Participants
n=51 Participants
|
264 Participants
n=301 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=128 Participants
|
1 Participants
n=122 Participants
|
0 Participants
n=51 Participants
|
2 Participants
n=301 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=128 Participants
|
0 Participants
n=122 Participants
|
2 Participants
n=51 Participants
|
2 Participants
n=301 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=128 Participants
|
2 Participants
n=122 Participants
|
2 Participants
n=51 Participants
|
6 Participants
n=301 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=128 Participants
|
0 Participants
n=122 Participants
|
0 Participants
n=51 Participants
|
0 Participants
n=301 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=128 Participants
|
5 Participants
n=122 Participants
|
6 Participants
n=51 Participants
|
14 Participants
n=301 Participants
|
|
Race (NIH/OMB)
White
|
119 Participants
n=128 Participants
|
107 Participants
n=122 Participants
|
38 Participants
n=51 Participants
|
264 Participants
n=301 Participants
|
|
Race (NIH/OMB)
More than one race
|
2 Participants
n=128 Participants
|
2 Participants
n=122 Participants
|
1 Participants
n=51 Participants
|
5 Participants
n=301 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=128 Participants
|
6 Participants
n=122 Participants
|
2 Participants
n=51 Participants
|
10 Participants
n=301 Participants
|
|
Weight
|
59.2 kilogram
STANDARD_DEVIATION 13.62 • n=128 Participants
|
60.7 kilogram
STANDARD_DEVIATION 13.58 • n=122 Participants
|
57.7 kilogram
STANDARD_DEVIATION 15.10 • n=51 Participants
|
59.5 kilogram
STANDARD_DEVIATION 13.86 • n=301 Participants
|
|
Height
|
164.2 centimeter
STANDARD_DEVIATION 10.18 • n=128 Participants
|
166.3 centimeter
STANDARD_DEVIATION 8.84 • n=122 Participants
|
163.2 centimeter
STANDARD_DEVIATION 9.08 • n=51 Participants
|
164.9 centimeter
STANDARD_DEVIATION 9.52 • n=301 Participants
|
|
Maximal VO2
|
1607.3 mL/min
STANDARD_DEVIATION 470.80 • n=90 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points). VO2 has been presented in mL/min (i.e., not adjusted for body weight).
|
1637.6 mL/min
STANDARD_DEVIATION 444.26 • n=93 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points). VO2 has been presented in mL/min (i.e., not adjusted for body weight).
|
1421.7 mL/min
STANDARD_DEVIATION 261.32 • n=12 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points). VO2 has been presented in mL/min (i.e., not adjusted for body weight).
|
1610.4 mL/min
STANDARD_DEVIATION 449.13 • n=195 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points). VO2 has been presented in mL/min (i.e., not adjusted for body weight).
|
|
Myocardial Performance Index (MPI)
|
0.43 Ratio
STANDARD_DEVIATION 0.118 • n=59 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
0.46 Ratio
STANDARD_DEVIATION 0.152 • n=60 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
0.59 Ratio
STANDARD_DEVIATION 0.326 • n=4 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
0.45 Ratio
STANDARD_DEVIATION 0.146 • n=123 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
|
Log-transformed Reactive Hyperemia Index (lnRHI)
|
1.748 Natural log-transformed RHI (lnRHI)
STANDARD_DEVIATION 0.4940 • n=82 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
1.757 Natural log-transformed RHI (lnRHI)
STANDARD_DEVIATION 0.5403 • n=83 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
1.585 Natural log-transformed RHI (lnRHI)
STANDARD_DEVIATION 0.3987 • n=8 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
1.745 Natural log-transformed RHI (lnRHI)
STANDARD_DEVIATION 0.5117 • n=173 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
|
Serum BNP
|
18.25 pg/mL
STANDARD_DEVIATION 14.796 • n=48 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
15.58 pg/mL
STANDARD_DEVIATION 18.638 • n=53 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
—
|
16.85 pg/mL
STANDARD_DEVIATION 16.891 • n=101 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
|
Pediatric Quality of Life (PedsQL)-Physical Functioning-Child reported
|
73.68 score on a scale
STANDARD_DEVIATION 13.845 • n=111 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
74.40 score on a scale
STANDARD_DEVIATION 16.142 • n=119 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
73.99 score on a scale
STANDARD_DEVIATION 13.722 • n=37 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
74.04 score on a scale
STANDARD_DEVIATION 14.848 • n=267 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
|
Pediatric Quality of Life (PedsQL)-Physical Functioning-Parent reported
|
76.43 score on a scale
STANDARD_DEVIATION 17.064 • n=106 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
76.90 score on a scale
STANDARD_DEVIATION 14.948 • n=106 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
73.24 score on a scale
STANDARD_DEVIATION 18.112 • n=35 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
76.18 score on a scale
STANDARD_DEVIATION 16.324 • n=247 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
|
Pediatric Quality of Life (PedsQL)-Psychosocial Health Summary Score-Child reported
|
73.40 score on a scale
STANDARD_DEVIATION 14.562 • n=111 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
73.11 score on a scale
STANDARD_DEVIATION 16.306 • n=119 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
70.23 score on a scale
STANDARD_DEVIATION 14.429 • n=37 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
72.83 score on a scale
STANDARD_DEVIATION 15.327 • n=267 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
|
Pediatric Quality of Life (PedsQL)-Psychosocial Health Summary Score-Parent reported
|
70.40 score on a scale
STANDARD_DEVIATION 17.258 • n=106 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
73.64 score on a scale
STANDARD_DEVIATION 16.558 • n=106 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
65.50 score on a scale
STANDARD_DEVIATION 17.458 • n=35 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
71.10 score on a scale
STANDARD_DEVIATION 17.139 • n=247 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
|
Pediatric Quality of Life (PedsQL)-Functional Health Status Score-Child reported
|
73.50 score on a scale
STANDARD_DEVIATION 13.108 • n=111 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
73.56 score on a scale
STANDARD_DEVIATION 14.755 • n=119 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
71.53 score on a scale
STANDARD_DEVIATION 12.802 • n=37 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
73.26 score on a scale
STANDARD_DEVIATION 13.795 • n=267 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
|
Pediatric Quality of Life (PedsQL)-Functional Health Status Score-Parent reported
|
72.49 score on a scale
STANDARD_DEVIATION 15.621 • n=106 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
74.75 score on a scale
STANDARD_DEVIATION 14.248 • n=106 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
68.20 score on a scale
STANDARD_DEVIATION 15.488 • n=35 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
72.85 score on a scale
STANDARD_DEVIATION 15.122 • n=247 Participants • Baseline results only presented for subjects who have data at 52 weeks (i.e., paired-data points).
|
PRIMARY outcome
Timeframe: 52 WeeksPopulation: Safety Population
Summary of Treatment Emergent Adverse Events
Outcome measures
| Measure |
De Novo
Subjects not in the FUEL trial but joined the open-label trial de novo and received 52 weeks of udenafil.
|
Total
Total number of subjects who participated in the open-label extension study
|
In PHN-Udenafil-02 (FUEL) Received Placebo
Subjects received 26 weeks of placebo in the double-blind trial (FUEL) and then received 52 weeks of udenafil in the open-label trial.
|
In PHN-Udenafil-02 (FUEL) Received Udenafil
n=301 Participants
Subjects received 26 weeks of udenafil in the double-blind trial (FUEL) and then received 52 weeks of udenafil in the open-label trial.
|
|---|---|---|---|---|
|
Treatment-Emergent Adverse Events
Subjects with one or more TEAEs adverse event (TEAE)
|
—
|
—
|
—
|
257 Participants
|
|
Treatment-Emergent Adverse Events
Subjects with one or more drug-related TEAEs
|
—
|
—
|
—
|
206 Participants
|
|
Treatment-Emergent Adverse Events
Subjects with one or more TEAEs of Grade >=3
|
—
|
—
|
—
|
31 Participants
|
|
Treatment-Emergent Adverse Events
Subjects with one or more SAEs
|
—
|
—
|
—
|
54 Participants
|
|
Treatment-Emergent Adverse Events
Subjects with one or more drug-related SAEs
|
—
|
—
|
—
|
15 Participants
|
|
Treatment-Emergent Adverse Events
Subjects who temporarily stopped study drug for one or more TEAEs
|
—
|
—
|
—
|
35 Participants
|
|
Treatment-Emergent Adverse Events
Subjects who permanently discontinued study drug for one or more TEAEs
|
—
|
—
|
—
|
21 Participants
|
SECONDARY outcome
Timeframe: 52 WeeksPopulation: 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).
Change in Maximal VO2 from baseline to 52 weeks
Outcome measures
| Measure |
De Novo
n=12 Participants
Subjects not in the FUEL trial but joined the open-label trial de novo and received 52 weeks of udenafil.
|
Total
n=195 Participants
Total number of subjects who participated in the open-label extension study
|
In PHN-Udenafil-02 (FUEL) Received Placebo
n=93 Participants
Subjects received 26 weeks of placebo in the double-blind trial (FUEL) and then received 52 weeks of udenafil in the open-label trial.
|
In PHN-Udenafil-02 (FUEL) Received Udenafil
n=90 Participants
Subjects received 26 weeks of udenafil in the double-blind trial (FUEL) and then received 52 weeks of udenafil in the open-label trial.
|
|---|---|---|---|---|
|
Change in Maximal VO2
Baseline
|
1421.7 mL/min
Standard Deviation 261.32
|
1610.4 mL/min
Standard Deviation 449.13
|
1637.6 mL/min
Standard Deviation 444.26
|
1607.3 mL/min
Standard Deviation 470.80
|
|
Change in Maximal VO2
52 weeks
|
1425.0 mL/min
Standard Deviation 269.12
|
1659.6 mL/min
Standard Deviation 429.34
|
1703.0 mL/min
Standard Deviation 434.31
|
1646.1 mL/min
Standard Deviation 433.94
|
SECONDARY outcome
Timeframe: 52 WeeksPopulation: Results presented only for subjects who have data at baseline and 52 weeks (i.e., paired-data points).
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.
Outcome measures
| Measure |
De Novo
n=8 Participants
Subjects not in the FUEL trial but joined the open-label trial de novo and received 52 weeks of udenafil.
|
Total
n=173 Participants
Total number of subjects who participated in the open-label extension study
|
In PHN-Udenafil-02 (FUEL) Received Placebo
n=83 Participants
Subjects received 26 weeks of placebo in the double-blind trial (FUEL) and then received 52 weeks of udenafil in the open-label trial.
|
In PHN-Udenafil-02 (FUEL) Received Udenafil
n=82 Participants
Subjects received 26 weeks of udenafil in the double-blind trial (FUEL) and then received 52 weeks of udenafil in the open-label trial.
|
|---|---|---|---|---|
|
Change in Log-transformed Reactive Hyperemia Index (lnRHI)
Baseline
|
1.585 Natural log-transformed RHI (lnRHI)
Standard Deviation 0.3987
|
1.745 Natural log-transformed RHI (lnRHI)
Standard Deviation 0.5117
|
1.757 Natural log-transformed RHI (lnRHI)
Standard Deviation 0.5403
|
1.748 Natural log-transformed RHI (lnRHI)
Standard Deviation 0.4940
|
|
Change in Log-transformed Reactive Hyperemia Index (lnRHI)
52 weeks
|
1.960 Natural log-transformed RHI (lnRHI)
Standard Deviation 0.5315
|
1.902 Natural log-transformed RHI (lnRHI)
Standard Deviation 0.5931
|
2.037 Natural log-transformed RHI (lnRHI)
Standard Deviation 0.6405
|
1.759 Natural log-transformed RHI (lnRHI)
Standard Deviation 0.5171
|
SECONDARY outcome
Timeframe: 52 WeeksPopulation: 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.
Change in Serum BNP from baseline to 52 weeks
Outcome measures
| Measure |
De Novo
Subjects not in the FUEL trial but joined the open-label trial de novo and received 52 weeks of udenafil.
|
Total
n=101 Participants
Total number of subjects who participated in the open-label extension study
|
In PHN-Udenafil-02 (FUEL) Received Placebo
n=53 Participants
Subjects received 26 weeks of placebo in the double-blind trial (FUEL) and then received 52 weeks of udenafil in the open-label trial.
|
In PHN-Udenafil-02 (FUEL) Received Udenafil
n=48 Participants
Subjects received 26 weeks of udenafil in the double-blind trial (FUEL) and then received 52 weeks of udenafil in the open-label trial.
|
|---|---|---|---|---|
|
Change in Serum BNP
Baseline
|
—
|
16.85 pg/mL
Standard Deviation 16.891
|
15.58 pg/mL
Standard Deviation 18.638
|
18.25 pg/mL
Standard Deviation 14.796
|
|
Change in Serum BNP
52 weeks
|
—
|
24.58 pg/mL
Standard Deviation 24.347
|
23.13 pg/mL
Standard Deviation 24.584
|
26.18 pg/mL
Standard Deviation 24.241
|
SECONDARY outcome
Timeframe: 52 WeeksPopulation: Efficacy population
Change in Myocardial Performance Index from baseline to 52 weeks
Outcome measures
| Measure |
De Novo
n=4 Participants
Subjects not in the FUEL trial but joined the open-label trial de novo and received 52 weeks of udenafil.
|
Total
n=123 Participants
Total number of subjects who participated in the open-label extension study
|
In PHN-Udenafil-02 (FUEL) Received Placebo
n=60 Participants
Subjects received 26 weeks of placebo in the double-blind trial (FUEL) and then received 52 weeks of udenafil in the open-label trial.
|
In PHN-Udenafil-02 (FUEL) Received Udenafil
n=59 Participants
Subjects received 26 weeks of udenafil in the double-blind trial (FUEL) and then received 52 weeks of udenafil in the open-label trial.
|
|---|---|---|---|---|
|
Change in Myocardial Performance Index (MPI)
52 weeks
|
0.53 mL/min
Standard Deviation 0.148
|
0.42 mL/min
Standard Deviation 0.141
|
0.41 mL/min
Standard Deviation 0.140
|
0.43 mL/min
Standard Deviation 0.140
|
|
Change in Myocardial Performance Index (MPI)
Baseline
|
0.59 mL/min
Standard Deviation 0.326
|
0.45 mL/min
Standard Deviation 0.146
|
0.46 mL/min
Standard Deviation 0.152
|
0.43 mL/min
Standard Deviation 0.118
|
SECONDARY outcome
Timeframe: 52 WeeksPopulation: Results presented only for subjects who have data at baseline and 52 weeks (i.e., paired-data points).
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.
Outcome measures
| Measure |
De Novo
n=37 Participants
Subjects not in the FUEL trial but joined the open-label trial de novo and received 52 weeks of udenafil.
|
Total
n=267 Participants
Total number of subjects who participated in the open-label extension study
|
In PHN-Udenafil-02 (FUEL) Received Placebo
n=119 Participants
Subjects received 26 weeks of placebo in the double-blind trial (FUEL) and then received 52 weeks of udenafil in the open-label trial.
|
In PHN-Udenafil-02 (FUEL) Received Udenafil
n=111 Participants
Subjects received 26 weeks of udenafil in the double-blind trial (FUEL) and then received 52 weeks of udenafil in the open-label trial.
|
|---|---|---|---|---|
|
Change in PedsQL Physical Functioning (Child Reported)
Baseline
|
73.99 score on a scale
Standard Deviation 13.722
|
74.04 score on a scale
Standard Deviation 14.848
|
74.40 score on a scale
Standard Deviation 16.142
|
73.68 score on a scale
Standard Deviation 13.845
|
|
Change in PedsQL Physical Functioning (Child Reported)
52 weeks
|
73.56 score on a scale
Standard Deviation 14.669
|
74.30 score on a scale
Standard Deviation 16.482
|
74.10 score on a scale
Standard Deviation 17.691
|
74.75 score on a scale
Standard Deviation 15.823
|
SECONDARY outcome
Timeframe: 52 WeeksPopulation: Results presented only for subjects who have data at baseline and 52 weeks (i.e., paired-data points).
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.
Outcome measures
| Measure |
De Novo
n=35 Participants
Subjects not in the FUEL trial but joined the open-label trial de novo and received 52 weeks of udenafil.
|
Total
n=247 Participants
Total number of subjects who participated in the open-label extension study
|
In PHN-Udenafil-02 (FUEL) Received Placebo
n=106 Participants
Subjects received 26 weeks of placebo in the double-blind trial (FUEL) and then received 52 weeks of udenafil in the open-label trial.
|
In PHN-Udenafil-02 (FUEL) Received Udenafil
n=106 Participants
Subjects received 26 weeks of udenafil in the double-blind trial (FUEL) and then received 52 weeks of udenafil in the open-label trial.
|
|---|---|---|---|---|
|
Change in PedsQL Physical Functioning (Parent Reported)
52 weeks
|
74.11 score on a scale
Standard Deviation 13.946
|
75.77 score on a scale
Standard Deviation 16.582
|
76.10 score on a scale
Standard Deviation 15.772
|
75.99 score on a scale
Standard Deviation 18.205
|
|
Change in PedsQL Physical Functioning (Parent Reported)
Baseline
|
73.24 score on a scale
Standard Deviation 18.112
|
76.18 score on a scale
Standard Deviation 16.324
|
76.90 score on a scale
Standard Deviation 14.948
|
76.43 score on a scale
Standard Deviation 17.064
|
SECONDARY outcome
Timeframe: 52 WeeksPopulation: Results presented only for subjects who have data at baseline and 52 weeks (i.e., paired-data points).
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.
Outcome measures
| Measure |
De Novo
n=37 Participants
Subjects not in the FUEL trial but joined the open-label trial de novo and received 52 weeks of udenafil.
|
Total
n=267 Participants
Total number of subjects who participated in the open-label extension study
|
In PHN-Udenafil-02 (FUEL) Received Placebo
n=119 Participants
Subjects received 26 weeks of placebo in the double-blind trial (FUEL) and then received 52 weeks of udenafil in the open-label trial.
|
In PHN-Udenafil-02 (FUEL) Received Udenafil
n=111 Participants
Subjects received 26 weeks of udenafil in the double-blind trial (FUEL) and then received 52 weeks of udenafil in the open-label trial.
|
|---|---|---|---|---|
|
Change in PedsQL Psychosocial Health Summary Score (Child Reported)
Baseline
|
70.23 score on a scale
Standard Deviation 14.429
|
72.83 score on a scale
Standard Deviation 15.327
|
73.11 score on a scale
Standard Deviation 16.306
|
73.40 score on a scale
Standard Deviation 14.562
|
|
Change in PedsQL Psychosocial Health Summary Score (Child Reported)
52 weeks
|
71.31 score on a scale
Standard Deviation 17.015
|
72.49 score on a scale
Standard Deviation 17.212
|
72.37 score on a scale
Standard Deviation 17.174
|
73.01 score on a scale
Standard Deviation 17.451
|
SECONDARY outcome
Timeframe: 52 WeeksPopulation: Results presented only for subjects who have data at baseline and 52 weeks (i.e., paired-data points).
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.
Outcome measures
| Measure |
De Novo
n=35 Participants
Subjects not in the FUEL trial but joined the open-label trial de novo and received 52 weeks of udenafil.
|
Total
n=247 Participants
Total number of subjects who participated in the open-label extension study
|
In PHN-Udenafil-02 (FUEL) Received Placebo
n=106 Participants
Subjects received 26 weeks of placebo in the double-blind trial (FUEL) and then received 52 weeks of udenafil in the open-label trial.
|
In PHN-Udenafil-02 (FUEL) Received Udenafil
n=106 Participants
Subjects received 26 weeks of udenafil in the double-blind trial (FUEL) and then received 52 weeks of udenafil in the open-label trial.
|
|---|---|---|---|---|
|
Change in PedsQL Psychosocial Health Summary Score (Parent Reported)
Baseline
|
65.50 score on a scale
Standard Deviation 17.458
|
71.10 score on a scale
Standard Deviation 17.139
|
73.64 score on a scale
Standard Deviation 16.558
|
70.40 score on a scale
Standard Deviation 17.258
|
|
Change in PedsQL Psychosocial Health Summary Score (Parent Reported)
52 weeks
|
68.24 score on a scale
Standard Deviation 14.730
|
71.25 score on a scale
Standard Deviation 17.184
|
72.32 score on a scale
Standard Deviation 17.099
|
71.18 score on a scale
Standard Deviation 18.027
|
SECONDARY outcome
Timeframe: 52 WeeksPopulation: Results presented only for subjects who have data at baseline and 52 weeks (i.e., paired-data points).
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.
Outcome measures
| Measure |
De Novo
n=37 Participants
Subjects not in the FUEL trial but joined the open-label trial de novo and received 52 weeks of udenafil.
|
Total
n=267 Participants
Total number of subjects who participated in the open-label extension study
|
In PHN-Udenafil-02 (FUEL) Received Placebo
n=119 Participants
Subjects received 26 weeks of placebo in the double-blind trial (FUEL) and then received 52 weeks of udenafil in the open-label trial.
|
In PHN-Udenafil-02 (FUEL) Received Udenafil
n=111 Participants
Subjects received 26 weeks of udenafil in the double-blind trial (FUEL) and then received 52 weeks of udenafil in the open-label trial.
|
|---|---|---|---|---|
|
Change in PedsQL Functional Health Status Score (Child Reported)
Baseline
|
71.53 score on a scale
Standard Deviation 12.802
|
73.26 score on a scale
Standard Deviation 13.795
|
73.56 score on a scale
Standard Deviation 14.755
|
73.50 score on a scale
Standard Deviation 13.108
|
|
Change in PedsQL Functional Health Status Score (Child Reported)
52 weeks
|
72.09 score on a scale
Standard Deviation 14.762
|
73.13 score on a scale
Standard Deviation 15.580
|
72.98 score on a scale
Standard Deviation 15.735
|
73.64 score on a scale
Standard Deviation 15.794
|
SECONDARY outcome
Timeframe: 52 WeeksPopulation: Results presented only for subjects who have data at baseline and 52 weeks (i.e., paired-data points).
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.
Outcome measures
| Measure |
De Novo
n=35 Participants
Subjects not in the FUEL trial but joined the open-label trial de novo and received 52 weeks of udenafil.
|
Total
n=247 Participants
Total number of subjects who participated in the open-label extension study
|
In PHN-Udenafil-02 (FUEL) Received Placebo
n=106 Participants
Subjects received 26 weeks of placebo in the double-blind trial (FUEL) and then received 52 weeks of udenafil in the open-label trial.
|
In PHN-Udenafil-02 (FUEL) Received Udenafil
n=106 Participants
Subjects received 26 weeks of udenafil in the double-blind trial (FUEL) and then received 52 weeks of udenafil in the open-label trial.
|
|---|---|---|---|---|
|
Change in PedsQL Functional Health Status Score (Parent Reported)
Baseline
|
68.20 score on a scale
Standard Deviation 15.488
|
72.85 score on a scale
Standard Deviation 15.122
|
74.75 score on a scale
Standard Deviation 14.248
|
72.49 score on a scale
Standard Deviation 15.621
|
|
Change in PedsQL Functional Health Status Score (Parent Reported)
52 weeks
|
70.28 score on a scale
Standard Deviation 13.181
|
72.82 score on a scale
Standard Deviation 15.491
|
73.63 score on a scale
Standard Deviation 15.044
|
72.85 score on a scale
Standard Deviation 16.638
|
Adverse Events
Total
Serious events: 54 serious events
Other events: 257 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Total
n=301 participants at risk
Total number of subjects who participated in the open-label extension study (PHN-Udenafil-03)
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary artery stenosis
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary oedema
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Surgical and medical procedures
Angioplasty
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Surgical and medical procedures
Scoliosis surgery
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Surgical and medical procedures
Tympanoplasty
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Vascular disorders
Hyptension
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Blood and lymphatic system disorders
Anaemia
|
0.66%
2/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Cardiac disorders
Cardiac Failure
|
1.3%
4/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Cardiac disorders
Supraventricular tachycardia
|
0.66%
2/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Cardiac disorders
Ventricular tachycardia
|
0.66%
2/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Cardiac disorders
Atrial flutter
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Cardiac disorders
Atrial tachycardia
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Cardiac disorders
Cardiac failure congestive
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Cardiac disorders
Cardiac ventricular thrombosis
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Cardiac disorders
Palpitations
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Eye disorders
Blindness transient
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Gastrointestinal disorders
Protein-losing gastroenteropathy
|
1.00%
3/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Gastrointestinal disorders
Abdominal pain
|
0.66%
2/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Gastrointestinal disorders
Vomiting
|
0.66%
2/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Gastrointestinal disorders
Constipation
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Gastrointestinal disorders
Haematochezia
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Gastrointestinal disorders
Rectal haemorrhage
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Gastrointestinal disorders
Tooth Impacted
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
General disorders
Chest discomfort
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
General disorders
Chest pain
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
General disorders
Exercise tolerance decreased
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
General disorders
Oedema
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
General disorders
Pyrexia
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Hepatobiliary disorders
Cardiac cirrhosis
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Hepatobiliary disorders
Congestive hepatopathy
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Hepatobiliary disorders
Hepatic cirrhosis
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Hepatobiliary disorders
Hyperbilirubinaemia
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Infections and infestations
Appendicitis
|
0.66%
2/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Infections and infestations
Gastroenteritis
|
0.66%
2/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Infections and infestations
Influenza
|
0.66%
2/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Infections and infestations
Cellulitis
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Infections and infestations
Coronavirus infection
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Infections and infestations
Gastroenteritis viral
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Infections and infestations
Infectious mononucleosis
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Infections and infestations
Pneumonia
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Infections and infestations
Pneumonia bacterial
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Infections and infestations
Pneumonia respiratory syncytial viral
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Infections and infestations
Viral infection
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Injury, poisoning and procedural complications
Road traffic accident
|
0.66%
2/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Injury, poisoning and procedural complications
Ankle fracture
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Injury, poisoning and procedural complications
Procedural haemorrhage
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Metabolism and nutrition disorders
Dehydration
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Costochondritis
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Kyphosis
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Burkitt's Lymphoma
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Hepatocellular carcinoma
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Nervous system disorders
Transient ischaemic attack
|
1.00%
3/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Nervous system disorders
Syncope
|
0.66%
2/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Nervous system disorders
Cerebrovascular accident
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Nervous system disorders
Headache
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Nervous system disorders
Neuropathy peripheral
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Nervous system disorders
Presyncope
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Product Issues
Device malfunction
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Psychiatric disorders
Depression
|
1.00%
3/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Psychiatric disorders
Suicide attempt
|
0.66%
2/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Psychiatric disorders
Adjustment disorder with depressed mood
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Psychiatric disorders
Anxiety
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Psychiatric disorders
Hallucination, auditory
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Psychiatric disorders
Intentional self injury
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Renal and urinary disorders
Nephrolithiasis
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
0.33%
1/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
Other adverse events
| Measure |
Total
n=301 participants at risk
Total number of subjects who participated in the open-label extension study (PHN-Udenafil-03)
|
|---|---|
|
Nervous system disorders
Headache
|
40.5%
122/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Vascular disorders
Flushing
|
17.3%
52/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Nervous system disorders
Dizziness
|
14.0%
42/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Infections and infestations
Nasopharyngitis
|
13.6%
41/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
General disorders
Chest pain
|
11.6%
35/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
General disorders
Fatigue
|
10.3%
31/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Gastrointestinal disorders
Nausea
|
10.3%
31/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Infections and infestations
Upper respiratory tract infection
|
10.3%
31/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Nervous system disorders
Migraine
|
8.3%
25/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Infections and infestations
Influenza
|
7.6%
23/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
7.3%
22/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
General disorders
Pyrexia
|
7.3%
22/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
7.0%
21/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Infections and infestations
Sinusitis
|
7.0%
21/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Gastrointestinal disorders
Vomiting
|
7.0%
21/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
6.6%
20/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Gastrointestinal disorders
Abdominal pain upper
|
6.0%
18/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
6.0%
18/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Infections and infestations
Pharyngitis streptococcal
|
5.6%
17/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Infections and infestations
Ear infection
|
5.3%
16/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
5.3%
16/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Cardiac disorders
Palpitations
|
5.3%
16/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
|
Psychiatric disorders
Depression
|
5.0%
15/301 • 52 Weeks
Subjects taking the study drug for 52 weeks
|
Additional Information
Director of Clinical Operatons
Mezzion Pharmaceuticals, Inc
Phone: 3128980292
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place