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Safety And Efficacy Of Sildenafil In Children With Pulmonary Arterial Hypertension

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01642407
Recruitment Status : Completed
First Posted : July 17, 2012
Results First Posted : April 4, 2017
Last Update Posted : October 25, 2018
Sponsor:
Information provided by (Responsible Party):
Pfizer

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Pulmonary Arterial Hypertension
Hypertension, Pulmonary
Intervention Drug: Sildenafil
Enrollment 6
Recruitment Details  
Pre-assignment Details The study was conducted at 3 sites in Japan. Data reported is based on data cut-off date of 26 December 2016.
Arm/Group Title Sildenafil
Hide Arm/Group Description Participants received 10 milligram (mg) or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with less than or equal to (<=) 20 kilogram (kg) of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with greater than (>) 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Period Title: Part 1 (Screening Till Week 16)
Started 6
Completed 3
Not Completed 3
Reason Not Completed
Insufficient clinical response             3
Period Title: Part 2(Week17 to Maximum of 119.6 Weeks)
Started 3
Completed 1
Not Completed 2
Reason Not Completed
Coil Embolization             1
Change of treatment plan             1
Arm/Group Title Sildenafil
Hide Arm/Group Description Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Baseline Participants 6
Hide Baseline Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug.
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants
<=18 years
6
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 6 participants
6.7  (5.4)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants
Female
3
  50.0%
Male
3
  50.0%
1.Primary Outcome
Title Change From Baseline in Pulmonary Vascular Resistance Index (PVRI) at Week 16
Hide Description PVRI equals pulmonary vascular resistance (PVR) times body surface area (BSA) (PVRI = PVR*BSA). PVR is the resistance to blood flow through the pulmonary circulation and it was measured in Wood units. Wood unit =80 dyne*seconds per centimetre^5 (dyne*sec/cm^5).
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure at specified time points.”
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: wood units*meter^2
Baseline Number Analyzed 6 participants
18.567  (11.7629)
Change at Week 16 Number Analyzed 4 participants
-4.113  (6.3770)
2.Primary Outcome
Title Change From Baseline in Mean Pulmonary Artery Pressure (mPAP) at Week 16
Hide Description It was a hemodynamic parameter and measured using a pressure transducer positioned at the mid-axillary line with the participant in the supine position.
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure at specified time points.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: millimeter of mercury (mmHg)
Baseline Number Analyzed 6 participants
58.5  (22.94)
Change at Week 16 Number Analyzed 4 participants
-6.5  (15.15)
3.Primary Outcome
Title Change From Baseline in World Health Organization (WHO) Functional Class in Participants With Pulmonary Arterial Hypertension (PAH) at Week 4
Hide Description WHO functional classification for PAH range from Class I (no limitation in physical activity, no dyspnea with normal activity), Class II (slight limitation of physical activity), Class III (marked limitation of physical activity) and Class IV (cannot perform a physical activity without any symptoms, dyspnea at rest). The change from baseline in WHO functional class was classified into "Improved", "No change" and "Worsened". Improvement = reduction in functional class, worsened = increase in functional class and no change = no change in functional class. Change from baseline in number of participants in each functional class were reported.
Time Frame Baseline, Week 4
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Measure Type: Number
Unit of Measure: participants
Baseline: Class I 2
Baseline: Class II 3
Baseline: Class III 1
Baseline: Class IV 0
Week 4: Improved 1
Week 4: No change 5
Week 4: Worsened 0
4.Primary Outcome
Title Change From Baseline in World Health Organization (WHO) Functional Class in Participants With Pulmonary Arterial Hypertension (PAH) at Week 8
Hide Description WHO functional classification for PAH range from Class I (no limitation in physical activity, no dyspnea with normal activity), Class II (slight limitation of physical activity), Class III (marked limitation of physical activity) and Class IV (cannot perform a physical activity without any symptoms, dyspnea at rest). The change from baseline in WHO functional class was classified into "Improved", "No change" and "Worsened". Improvement = reduction in functional class, worsened = increase in functional class and no change = no change in functional class. Change from baseline in number of participants in each functional class were reported.
Time Frame Baseline, Week 8
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug. Here,'N' (Overall number of participants analyzed) signifies those participants who were evaluable for this measure.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: participants
Improved 1
No change 4
Worsened 0
5.Primary Outcome
Title Change From Baseline in World Health Organization (WHO) Functional Class in Participants With Pulmonary Arterial Hypertension (PAH) at Week 16
Hide Description WHO functional classification for PAH range from Class I (no limitation in physical activity, no dyspnea with normal activity), Class II (slight limitation of physical activity), Class III (marked limitation of physical activity) and Class IV (cannot perform a physical activity without any symptoms, dyspnea at rest). The change from baseline in WHO functional class was classified into "Improved", "No change" and "Worsened". Improvement = reduction in functional class, worsened = increase in functional class and no change = no change in functional class. Change from baseline in number of participants in each functional class were reported.
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug. Here, Overall Number of participants analyzed signifies those participants who were evaluable for this measure.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 4
Measure Type: Number
Unit of Measure: participants
Improved 1
No change 3
Worsened 0
6.Primary Outcome
Title Change From Baseline in Brain Natriuretic Peptide (BNP) at Week 16
Hide Description BNP is produced by ventricular cardiomyocytes. It causes reduction in preload and blood pressure by vasodilatation.
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure at specified time points.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: picogram per milliliter
Baseline Number Analyzed 6 participants
132.62  (135.080)
Change at Week 16 Number Analyzed 4 participants
-64.10  (129.638)
7.Primary Outcome
Title Change From Baseline in N-terminal Pro Brain Natriuretic Peptide (NT Pro-BNP) at Week 16
Hide Description NT pro-BNP is a cardiac marker, having the prognostic value for participants with heart failure or left ventricular dysfunction. Higher level of the marker was indicative of heart damage.
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure at specified time points.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: picogram per milliliter
Baseline Number Analyzed 6 participants
843.03  (1120.900)
Change at Week 16 Number Analyzed 4 participants
-546.85  (1107.621)
8.Secondary Outcome
Title Change From Baseline in World Health Organization (WHO) Functional Class in Participants With Pulmonary Arterial Hypertension (PAH) at Weeks 28, 40, 52, 64, 76, 88, 100, 112 and 124
Hide Description WHO functional classification for PAH range from Class I (no limitation in physical activity, no dyspnea with normal activity), Class II (slight limitation of physical activity), Class III (marked limitation of physical activity) and Class IV (cannot perform a physical activity without any symptoms, dyspnea at rest). The change from baseline in WHO functional class was classified into "Improved", "No change" and "Worsened". Improvement = reduction in functional class, worsened = increase in functional class and no change = no change in functional class. Change from baseline in number of participants in each functional class were reported.
Time Frame Baseline, Weeks 4, 8, 16, 28, 40, 52, 64, 76, 88, 100, 112 and 124
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set was used in this analysis. Here, 'Overall number of participants analyzed' specifies number of participants who completed Part 1 of the study and continued treatment with Sildenafil in Part 2 of the study and ‘Number analyzed’ = Participants evaluable for this outcome measure for specified categories.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 3
Measure Type: Count of Participants
Unit of Measure: Participants
Baseline: Class I Number Analyzed 3 participants
1
  33.3%
Baseline: Class II Number Analyzed 3 participants
2
  66.7%
Baseline: Class III Number Analyzed 3 participants
0
   0.0%
Baseline: Class IV Number Analyzed 3 participants
0
   0.0%
Week 28: Improved Number Analyzed 3 participants
1
  33.3%
Week 28: No Change Number Analyzed 3 participants
2
  66.7%
Week 28: Worsened Number Analyzed 3 participants
0
   0.0%
Week 40: Improved Number Analyzed 3 participants
1
  33.3%
Week 40: No Change Number Analyzed 3 participants
2
  66.7%
Week 40: Worsened Number Analyzed 3 participants
0
   0.0%
Week 52: Improved Number Analyzed 3 participants
1
  33.3%
Week 52: No Change Number Analyzed 3 participants
2
  66.7%
Week 52: Worsened Number Analyzed 3 participants
0
   0.0%
Week 64: Improved Number Analyzed 2 participants
1
  50.0%
Week 64: No Change Number Analyzed 2 participants
1
  50.0%
Week 64: Worsened Number Analyzed 2 participants
0
   0.0%
Week 76: Improved Number Analyzed 2 participants
1
  50.0%
Week 76: No Change Number Analyzed 2 participants
0
   0.0%
Week 76: Worsened Number Analyzed 2 participants
1
  50.0%
Week 88: Improved Number Analyzed 1 participants
1
 100.0%
Week 88: No Change Number Analyzed 1 participants
0
   0.0%
Week 88: Worsened Number Analyzed 1 participants
0
   0.0%
Week 100: Improved Number Analyzed 1 participants
1
 100.0%
Week 100: No Change Number Analyzed 1 participants
0
   0.0%
Week 100: Worsened Number Analyzed 1 participants
0
   0.0%
Week 112: Improved Number Analyzed 1 participants
1
 100.0%
Week 112: No Change Number Analyzed 1 participants
0
   0.0%
Week 112: Worsened Number Analyzed 1 participants
0
   0.0%
Week 124: Improved Number Analyzed 1 participants
1
 100.0%
Week 124: No Change Number Analyzed 1 participants
0
   0.0%
Week 124:Worsened Number Analyzed 1 participants
0
   0.0%
9.Secondary Outcome
Title Change From Baseline in Brain Natriuretic Peptide (BNP) at Week 52 and End of Treatment (EOT)
Hide Description BNP is produced by ventricular cardiomyocytes. It causes reduction in preload and blood pressure by vasodilatation.
Time Frame Baseline, Week 52 and End of treatment (maximum duration of treatment: 119.6 weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug.Here 'Overall number of participants analyzed' specifies number of participants who completed Part 1 of the study and continued treatment with Sildenafil in Part 2 of the study.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 3
Mean (Standard Deviation)
Unit of Measure: picograms per milliliter
Baseline 100.17  (151.478)
Change at Week 52 -85.17  (155.088)
Change at EoT -85.17  (155.088)
10.Secondary Outcome
Title Change From Baseline in N-terminal Pro Brain Natriuretic Peptide (NT Pro-BNP) at Week 52 and End of Treatment (EOT)
Hide Description NT pro-BNP is a cardiac marker, having the prognostic value for participants with heart failure or left ventricular dysfunction. Higher level of the marker was indicative of heart damage.
Time Frame Baseline, Week 52 and End of treatment (maximum duration of treatment: 119.6 weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
EEfficacy analysis set included all participants who received at least 1 dose of study drug.Here 'Overall number of participants analyzed' specifies number of participants who completed Part 1 of the study and continued treatment with Sildenafil in Part 2 of the study.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 3
Mean (Standard Deviation)
Unit of Measure: picograms per milliliter
Baseline 841.73  (1323.533)
Change at Week 52 -754.90  (1335.370)
Change at EoT -754.90  (1335.370)
11.Secondary Outcome
Title Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Hide Description An AE was any untoward medical occurrence attributed to study drug in a participant who received study drug. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent are events between first dose of study drug and up to 28 days after last dose that were absent before treatment or that worsened relative to pretreatment state. AEs included both SAEs and non-serious AEs.
Time Frame Baseline upto 28 days after last dose of study drug (maximum duration of treatment: 119.6 weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set included all participants who received at least 1 dose of study drug.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Measure Type: Count of Participants
Unit of Measure: Participants
AEs
6
 100.0%
SAEs
0
   0.0%
12.Secondary Outcome
Title Number of Participants With Treatment-Emergent Treatment-Related Adverse Events (AEs) and Serious Adverse Events (SAEs)
Hide Description Treatment-related AE was any untoward medical occurrence attributed to study drug in a participant who received study drug. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent are events between first dose of study drug and up to 28 days after last dose that were absent before treatment or that worsened relative to pre-treatment state. AEs included both SAEs and non-serious AEs.
Time Frame Baseline upto 28 days after last dose of study drug (maximum duration of treatment: 119.6 weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set included all participants who received at least 1 dose of study drug.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Measure Type: Count of Participants
Unit of Measure: Participants
AEs
3
  50.0%
SAEs
0
   0.0%
13.Secondary Outcome
Title Change From Baseline in Systolic and Diastolic Blood Pressure (BP) at Weeks 4, 8, 16, 28, 40, 52, 64, 76, 88, 100, 112 and 124
Hide Description BP measurement is recorded as supine and sitting systolic and diastolic systemic blood pressure: 1) Systolic blood pressure when heart is contracting and it is the maximum arterial pressure during contraction of left ventricle. 2) Diastolic BP when heart is relaxing and it is the minimum arterial pressure during relaxation and dilation of ventricles. Only those categories in which at least 1 participant had data were reported.
Time Frame Baseline, Weeks 4, 8, 16, 28, 40, 52, 64, 76, 88, 100, 112 and 124
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure for specified categories.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: mmHg
Baseline: Supine Systolic BP Number Analyzed 5 participants
95.4  (13.41)
Baseline: Supine Diastolic BP Number Analyzed 5 participants
55.8  (10.87)
Baseline: Sitting Systolic BP Number Analyzed 1 participants
110.0
Baseline: Sitting Diastolic BP Number Analyzed 1 participants
60.0
Change at Week 4: Supine Systolic BP Number Analyzed 5 participants
5.6  (9.91)
Change at Week 4: Supine Diastolic BP Number Analyzed 5 participants
1.6  (13.65)
Change at Week 4: Sitting Systolic BP Number Analyzed 1 participants
16.0
Change at Week 4: Sitting Diastolic BP Number Analyzed 1 participants
23.0
Change at Week 8: Supine Systolic BP Number Analyzed 4 participants
7.8  (14.93)
Change at Week 8: Supine Diastolic BP Number Analyzed 4 participants
2.3  (12.28)
Change at Week 8: Sitting Systolic BP Number Analyzed 1 participants
-6.0
Change at Week 8: Sitting Diastolic BP Number Analyzed 1 participants
-3.0
Change at Week 16: Supine Systolic BP Number Analyzed 3 participants
7.7  (17.62)
Change at Week 16: Supine Diastolic BP Number Analyzed 3 participants
-1.3  (12.22)
Change at Week 16: Sitting Systolic BP Number Analyzed 1 participants
8.0
Change at Week 16: Sitting Diastolic BP Number Analyzed 1 participants
10.0
Change at Week 28: Supine Systolic BP Number Analyzed 3 participants
7.0  (19.31)
Change at Week 28: Supine Diastolic BP Number Analyzed 3 participants
1.7  (9.29)
Change at Week 40: Supine Systolic BP Number Analyzed 2 participants
-7.5  (3.54)
Change at Week 40: Supine Diastolic BP Number Analyzed 2 participants
2.0  (0.00)
Change at Week 52: Supine Systolic BP Number Analyzed 2 participants
2.0  (15.56)
Change at Week 52: Supine Diastolic BP Number Analyzed 2 participants
10.0  (5.66)
Change at Week 64: Supine Systolic BP Number Analyzed 1 participants
1.0
Change at Week 64: Supine Diastolic BP Number Analyzed 1 participants
8.0
Change at Week 76: Supine Systolic BP Number Analyzed 1 participants
-13.0
Change at Week 76: Supine Diastolic BP Number Analyzed 1 participants
3.0
14.Secondary Outcome
Title Change From Baseline in Heart Rate at Weeks 4, 8, 16, 28, 40, 52, 64, 76, 88, 100, 112 and 124
Hide Description Only those categories in which at least 1 participant had data were reported.
Time Frame Baseline, Weeks 4, 8, 16, 28, 40, 52, 64, 76, 88, 100, 112 and 124
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure for specified categories.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: beats per minute (bpm)
Baseline: Supine Heart rate Number Analyzed 5 participants
100.2  (15.91)
Baseline: Sitting Heart rate Number Analyzed 1 participants
96.0
Change at Week 4: Supine Heart rate Number Analyzed 5 participants
-1.0  (12.08)
Change at Week 4: Sitting Heart rate Number Analyzed 1 participants
-22.0
Change at Week 8: Supine Heart rate Number Analyzed 4 participants
-0.5  (9.11)
Change at Week 8: Sitting Heart rate Number Analyzed 1 participants
0.0
Change at Week 16: Supine Heart rate Number Analyzed 3 participants
3.0  (28.69)
Change at Week 16: Sitting Heart rate Number Analyzed 1 participants
4.0
Change at Week 28: Supine Heart rate Number Analyzed 3 participants
6.7  (14.36)
Change at Week 40: Supine Heart rate Number Analyzed 2 participants
10.0  (16.97)
Change at Week 52: Supine Heart rate Number Analyzed 2 participants
-2.0  (11.31)
Change at Week 64: Supine Heart rate Number Analyzed 1 participants
17.0
Change at Week 76: Supine Heart rate Number Analyzed 1 participants
-8.0
15.Secondary Outcome
Title Number of Participants With Laboratory Abnormalities
Hide Description Laboratory abnormality criteria: Hematology (hemoglobin, hematocrit, red blood cell count [less than {<}]0.8*lower limit of normal [LLN]; platelets <0.5*LLN, greater than [>]1.75*upper limit of normal [ULN], white blood cells <0.6*LLN, >1.5*ULN; lymphocytes, neutrophils <0.8*LLN, >1.2*ULN, eosinophils, basophils, monocytes >1.2*ULN); liver function (total and direct bilirubin >1.5*ULN, aspartate aminotransferase, alanine aminotransferase, alkaline phosphatase >3.0*ULN, total protein, albumin <0.8*LLN, >1.2*ULN); renal (creatinine, blood urea nitrogen >1.3*ULN); electrolytes (sodium <0.95*LLN, >1.05*ULN, potassium, chloride <0.9*LLN, >1.1*ULN; other (glucose <0.6*LLN or >1.5*ULN ); urinalysis (dipstick) urine glucose, urine protein, urine blood/Hemoglobin, [greater than or equal to {>=}1].
Time Frame Baseline up-to End of treatment (maximum duration of treatment: 119.6 weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set included all participants who received at least 1 dose of study drug.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Measure Type: Count of Participants
Unit of Measure: Participants
4
  66.7%
16.Secondary Outcome
Title Number of Participants With Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities
Hide Description Criteria for clinically significant abnormality in ECG parameters: Maximum corrected QT interval (QTc) from 450 millisecond (msec) to less than (<) 480 msec, Maximum QTcB interval (Bazett's Correction) from 450 msec to <480 msec, Maximum QTcF interval (Fredericia's Correction) from 450 msec to <480 msec, maximum QTc interval increase from baseline of 30 msec to <60 msec and >=60 msec.
Time Frame Screening, Week 16, Week 52 and End of treatment (maximum duration of treatment: 119.6 weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure for specified categories.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Measure Type: Count of Participants
Unit of Measure: Participants
Screening Number Analyzed 6 participants
4
  66.7%
Week 16 Number Analyzed 6 participants
5
  83.3%
Week 52 Number Analyzed 2 participants
1
  50.0%
End of Treatment Number Analyzed 3 participants
1
  33.3%
17.Secondary Outcome
Title Number of Participants With Ocular Examination Abnormalities
Hide Description Ocular examination measures included external examination of the eye, funduscopy, assessments of visual acuity, and color vision. Ocular examination findings were considered abnormal based on investigator's decision.
Time Frame Screening up to end of treatment (maximum duration of treatment: 119.6 weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set included all participants who received at least 1 dose of study drug.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
18.Secondary Outcome
Title Change From Baseline in Pulmonary Artery Systolic and Diastolic Pressure at Week 16
Hide Description [Not Specified]
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure at specified time points.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: mmHg
Baseline: Systolic Pressure Number Analyzed 6 participants
82.5  (35.51)
Baseline: Diastolic Pressure Number Analyzed 6 participants
42.0  (18.19)
Change at Week 16: Systolic Pressure Number Analyzed 4 participants
-9.8  (18.01)
Change at Week 16: Diastolic Pressure Number Analyzed 4 participants
-3.5  (13.99)
19.Secondary Outcome
Title Change From Baseline in Systemic Artery Systolic and Diastolic Pressure at Week 16
Hide Description [Not Specified]
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure at specified time points.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: mmHg
Baseline: Systolic Pressure Number Analyzed 6 participants
95.3  (15.20)
Baseline: Diastolic Pressure Number Analyzed 6 participants
60.2  (19.30)
Change at Week 16: Systolic Pressure Number Analyzed 4 participants
0.3  (10.21)
Change at Week 16: Diastolic Pressure Number Analyzed 4 participants
-1.5  (14.25)
20.Secondary Outcome
Title Change From Baseline in Pulmonary Vascular Resistance (PVR) at Week 16
Hide Description The resistance to blood flow through the pulmonary circulation is known as PVR. It is largely influenced by the caliber of the pulmonary arteries and capillaries and was measured in terms of Wood units. Wood unit =80 dyne*seconds per centimetre^5 (dyne*sec/cm^5).
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure at specified time points.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: Wood units
Baseline Number Analyzed 6 participants
21.372  (11.3408)
Change at Week 16 Number Analyzed 4 participants
-6.145  (10.3499)
21.Secondary Outcome
Title Change From Baseline in Right Atrial Pressure (RAP) at Week 16
Hide Description RAP is the blood pressure in the right atrium of the heart. It reflects the amount of blood returning to the heart and the ability of the heart to pump the blood into the arterial system. RAP was measured using a pressure transducer positioned at the mid-axillary line with the participant in the supine position.
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure at specified time points.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: mmHg
Baseline Number Analyzed 6 participants
6.5  (2.88)
Change at Week 16 Number Analyzed 4 participants
1.3  (2.36)
22.Secondary Outcome
Title Change From Baseline in Pulmonary Capillary Wedge Pressure (PCWP) at Week 16
Hide Description PCWP was measured by pulmonary artery catheterization and provided an indirect measure of left atrial pressure.
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure at specified time points.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: mmHg
Baseline Number Analyzed 6 participants
8.5  (0.84)
Change at Week 16 Number Analyzed 4 participants
2.5  (1.00)
23.Secondary Outcome
Title Change From Baseline in Cardiac Output (CO) at Week 16
Hide Description Cardiac output is simply the amount of blood pumped by the heart per minute.
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure at specified time points.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: liter per minute
Baseline Number Analyzed 6 participants
2.620  (0.9879)
Change at Week 16 Number Analyzed 4 participants
0.420  (0.9076)
24.Secondary Outcome
Title Change From Baseline in Cardiac Index (CI) at Week 16
Hide Description Cardiac index is a hemodynamic parameter that relates the cardiac output from left ventricle in one minute to BSA, thus relating heart performance to the size of the individual. CI was calculated as cardiac output in systemic circulation divided by BSA.
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure at specified time points.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: liter per minute per meter square
Baseline Number Analyzed 6 participants
3.070  (0.7460)
Change at Week 16 Number Analyzed 4 participants
0.658  (1.8912)
25.Secondary Outcome
Title Change From Baseline in Systemic Vascular Resistance (SVR) at Week 16
Hide Description The resistance to blood flow through the systemic circulation is known as SVR. This can be used in measuring blood pressure, blood flow and cardiac function and measured in terms of Wood units. Wood unit =80 dyne*seconds per centimetre^5 (dyne*sec/cm^5).
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure at specified time points.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: Wood units
Baseline Number Analyzed 6 participants
26.545  (3.0768)
Change at Week 16 Number Analyzed 4 participants
-3.403  (4.4409)
26.Secondary Outcome
Title Change From Baseline in Systemic Vascular Resistance Index (SVRI) at Week 16
Hide Description SVRI equals systemic vascular resistance (SVR) times BSA. SVR is the resistance to blood flow through the systemic circulation and it was measured in Wood units. Wood unit =80 dyne*seconds per centimetre^5 (dyne*sec/cm^5).
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure at specified time points.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: Wood units*meter^2
Baseline Number Analyzed 6 participants
23.855  (12.1480)
Change at Week 16 Number Analyzed 4 participants
-2.378  (3.9096)
27.Secondary Outcome
Title Change From Baseline in Mixed Venous Oxygen Saturation (SvO2) at Week 16
Hide Description SvO2 is the percentage of mixed venous oxygen (amount of oxygen bound to hemoglobin in venous blood). Change from baseline in percentage of mixed venous oxygen was reported in this outcome measure.
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure at specified time points.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: percentage of mixed venous oxygen
Baseline Number Analyzed 6 participants
65.30  (8.549)
Change at Week 16 Number Analyzed 4 participants
5.38  (12.426)
28.Secondary Outcome
Title Change From Baseline in Arterial Oxygen Saturation (SaO2) at Week 16
Hide Description SaO2 is the percentage of arterial oxygen (amount of oxygen bound to hemoglobin in arterial blood). Change from baseline in percentage of arterial oxygen was reported in this outcome measure.
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure at specified time points.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: percentage of arterial oxygen
Baseline Number Analyzed 6 participants
95.08  (2.730)
Change at Week 16 Number Analyzed 4 participants
-0.80  (1.691)
29.Other Pre-specified Outcome
Title Maximum Observed Plasma Concentration (Cmax) of Sildenafil and UK-103,320
Hide Description UK-103,320 was the main metabolite of Sildenafil and was produced by cytochrome P450 3A4.
Time Frame Pre-dose (0 hour) on Week 4, 8, 16 and 1, 2, 4, 8 hours post-dose on Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic (PK) parameter analysis set included all participants who have at least 1 of PK parameters of interest.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: nanogram per milliliter (ng/mL)
Sildenafil
138.1
(73%)
UK-103,320
73.66
(48%)
30.Other Pre-specified Outcome
Title Area Under the Curve From Time Zero to End of Dosing Interval (AUCtau) of Sildenafil and UK-103,320
Hide Description UK-103,320 was the main metabolite of Sildenafil and was produced by cytochrome P450 3A4.
Time Frame Pre-dose (0 hour) on Week 4, 8, 16 and 1, 2, 4, 8 hours post-dose on Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
PK parameter analysis set included all participants who have at least 1 of PK parameters of interest.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: nanogram*hour per millimeter
Sildenafil
338.9
(54%)
UK-103,320
210.2
(74%)
31.Other Pre-specified Outcome
Title Time to Reach Maximum Observed Plasma Concentration (Tmax) of Sildenafil and UK-103,320
Hide Description UK-103,320 was the main metabolite of Sildenafil and was produced by cytochrome P450 3A4.
Time Frame Pre-dose (0 hour) on Week 4, 8, 16 and 1, 2, 4, 8 hours post-dose on Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
PK parameter analysis set included all participants who have at least 1 of PK parameters of interest.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Median (Full Range)
Unit of Measure: hour
Sildenafil
1.00
(1.00 to 1.97)
UK-103,320
1.00
(1.00 to 1.97)
32.Other Pre-specified Outcome
Title Terminal Half Life (t1/2) of Sildenafil and UK-103,320
Hide Description Terminal half-life is the time measured for the plasma concentration to decrease by one half of its original concentration. UK-103,320 was a main metabolite of sildenafil and was produced by cytochrome P450 3A4.
Time Frame Pre-dose (0 hour) on Week 4, 8, 16 and 1, 2, 4, 8 hours post-dose on Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
PK parameter analysis set included all participants who have at least 1 of PK parameters of interest. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure at specified time points.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Median (Full Range)
Unit of Measure: hour
Sildenafil Number Analyzed 2 participants
1.785
(1.63 to 1.94)
UK-103,320 Number Analyzed 3 participants
2.110
(2.04 to 3.26)
33.Other Pre-specified Outcome
Title Apparent Oral Clearance (CL/F) of Sildenafil
Hide Description Clearance of a drug is a measure of the rate at which a drug is metabolized or eliminated by normal biological processes.
Time Frame Pre-dose (0 hour) on Week 4, 8, 16 and 1, 2, 4, 8 hours post-dose on Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
PK parameter analysis set included all participants who have at least 1 of PK parameters of interest.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: liter per hour
41.73
(77%)
34.Other Pre-specified Outcome
Title Apparent Volume of Distribution (Vz/F) of Sildenafil
Hide Description Volume of distribution is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. Apparent volume of distribution after oral dose (Vz/F) is influenced by the fraction absorbed.
Time Frame Pre-dose (0 hour) on Week 4, 8, 16 and 1, 2, 4, 8 hours post-dose on Week 16
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Hide Analysis Population Description
PK parameter analysis set included all participants who have at least 1 of PK parameters of interest. Here, Overall Number of participants analyzed signifies those participants who were evaluable for this measure.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 2
Median (Full Range)
Unit of Measure: liter
77.90
(62.4 to 93.4)
35.Other Pre-specified Outcome
Title Change From Baseline in Ratio of Acceleration Time to Ejection Time (AcT/ET) at Week 16
Hide Description Acceleration time and ejection time are quantitative Doppler parameters and ratio of acceleration time to ejection time is a useful tool to evaluate the severity of aortic stenosis.
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure at specified time points.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: ratio
Baseline Number Analyzed 6 participants
0.3038  (0.09675)
Change at Week 16 Number Analyzed 4 participants
0.0175  (0.10261)
36.Other Pre-specified Outcome
Title Change From Baseline in Right Ventricular Tei Index at Week 16
Hide Description The right ventricular Tei Index is an index of myocardial performance. It is defined as the sum of isovolumic contraction time and isovolumic relaxation time divided by the ejection time.
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure at specified time points.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: ratio
Baseline Number Analyzed 3 participants
0.7540  (0.48602)
Change at Week 16 Number Analyzed 2 participants
-0.0440  (0.37618)
37.Other Pre-specified Outcome
Title Change From Baseline in Right Ventricular Size at Week 16
Hide Description [Not Specified]
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure at specified time points.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: centimeter (cm)
Baseline Number Analyzed 6 participants
3.37  (1.216)
Change at Week 16 Number Analyzed 4 participants
-0.40  (0.408)
38.Other Pre-specified Outcome
Title Change From Baseline in Tricuspid Valve Annulus Size at Week 16
Hide Description The tricuspid valve lies between the right atrium and the right ventricle and is placed in a more apical position than the mitral valve. The annulus separates the right atrium from the right ventricle. Change from baseline in tricuspid valve annulus size (in cm) was reported in this outcome measure.
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure at specified time points.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: cm
Baseline Number Analyzed 6 participants
2.190  (0.5636)
Change at Week 16 Number Analyzed 4 participants
-0.103  (0.3727)
39.Other Pre-specified Outcome
Title Change From Baseline in Tricuspid Regurgitation - Pressure Gradient (TR-PG) Peak at Week 16
Hide Description Tricuspid regurgitation (insufficiency) is the failure of the tricuspid valve to close properly during systole, leading to the leaking of blood from the right ventricle into the right atrium. Change from baseline in TR-PG peak (in mmHg) was reported in this outcome measure.
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure at specified time points.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: mmHg
Baseline Number Analyzed 5 participants
73.0  (39.31)
Change at Week 16 Number Analyzed 2 participants
-6.0  (33.94)
40.Other Pre-specified Outcome
Title Change From Baseline in Pulmonary Regurgitation - Pressure Gradient (PR-PG) End-Diastole at Week 16
Hide Description Pulmonary regurgitation (PR) or insufficiency is a valvular heart disease characterized by an incomplete closure of the pulmonary valve leading to a diastolic reflux into the right ventricle. Change from baseline in PR-PG end-diastole (in mmHg) was reported in this outcome measure.
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure at specified time points.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: mmHg
Baseline Number Analyzed 3 participants
29.0  (14.73)
Change at Week 16 Number Analyzed 2 participants
3.5  (13.44)
41.Other Pre-specified Outcome
Title Number of Participants With Pericardial Effusion
Hide Description Pericardial effusion is the presence of an abnormal amount of fluid in the pericardial cavity, as determined by echocardiography.
Time Frame Baseline up to Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Measure Type: Number
Unit of Measure: participants
0
42.Other Pre-specified Outcome
Title Change From Baseline in Tricuspid Annular Plane Systolic Excursion (TAPSE) at Week 16
Hide Description Tricuspid annular plane systolic excursion is a parameter depicting global right ventricular function. Change from baseline in TAPSE (in cm) was reported in this outcome measure.
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 dose of study drug. Here, ‘Number analyzed’ = Participants evaluable for this outcome measure at specified time points.
Arm/Group Title Sildenafil
Hide Arm/Group Description:
Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: cm
Baseline Number Analyzed 6 participants
1.47  (0.437)
Change at Week 16 Number Analyzed 4 participants
0.18  (0.320)
Time Frame Baseline up to 28 days after last dose of study drug (maximum duration of treatment: 119.6 weeks)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Sildenafil
Hide Arm/Group Description Participants received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Weeks 4, 8, and 16 in Part 1 of the study. Participants who completed Part 1 and required continuing treatment with Sildenafil, received 10 mg or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Weeks 28, 40, 52 and thereafter every 12 weeks until Sildenafil obtained marketing approval (up to a maximum of 119.6 weeks). Participants with <= 20 kg of body weight received 10 mg dose, thrice daily as powder for oral suspension and participants with > 20 kg of body weight received 20 mg dose, thrice daily as film-coated tablets.
All-Cause Mortality
Sildenafil
Affected / at Risk (%)
Total   0/6 (0.00%) 
Hide Serious Adverse Events
Sildenafil
Affected / at Risk (%)
Total   0/6 (0.00%) 
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Sildenafil
Affected / at Risk (%)
Total   6/6 (100.00%) 
Cardiac disorders   
Cardiac failure * 1  1/6 (16.67%) 
Eye disorders   
Conjunctivitis allergic * 1  1/6 (16.67%) 
Vision blurred * 1  1/6 (16.67%) 
Visual acuity reduced transiently * 1  1/6 (16.67%) 
Gastrointestinal disorders   
Dental caries * 1  1/6 (16.67%) 
Diarrhoea * 1  2/6 (33.33%) 
Vomiting * 1  1/6 (16.67%) 
Colitis * 1  1/6 (16.67%) 
General disorders   
Chest pain * 1  1/6 (16.67%) 
Feeling abnormal * 1  1/6 (16.67%) 
Infections and infestations   
Bronchitis * 1  3/6 (50.00%) 
Gastroenteritis * 1  2/6 (33.33%) 
Influenza * 1  1/6 (16.67%) 
Nasopharyngitis * 1  3/6 (50.00%) 
Upper respiratory tract infection * 1  3/6 (50.00%) 
Molluscum contagiosum * 1  1/6 (16.67%) 
Streptococcal infection * 1  1/6 (16.67%) 
Investigations   
Alanine aminotransferase increased * 1  1/6 (16.67%) 
Ammonia increased * 1  1/6 (16.67%) 
Aspartate aminotransferase increased * 1  1/6 (16.67%) 
Weight increased * 1  1/6 (16.67%) 
Blood urine present * 1  1/6 (16.67%) 
Musculoskeletal and connective tissue disorders   
Myalgia * 1  1/6 (16.67%) 
Nervous system disorders   
Headache * 1  2/6 (33.33%) 
Reproductive system and breast disorders   
Dysmenorrhoea * 1 [1]  1/6 (16.67%) 
Erection increased * 1 [1]  1/6 (16.67%) 
Respiratory, thoracic and mediastinal disorders   
Epistaxis * 1  2/6 (33.33%) 
Pulmonary arterial hypertension * 1  1/6 (16.67%) 
Rhinitis allergic * 1  1/6 (16.67%) 
Skin and subcutaneous tissue disorders   
Acne * 1  1/6 (16.67%) 
Dermatitis diaper * 1  1/6 (16.67%) 
Dry skin * 1  1/6 (16.67%) 
Eczema * 1  1/6 (16.67%) 
Rash * 1  1/6 (16.67%) 
Vascular disorders   
Flushing * 1  1/6 (16.67%) 
1
Term from vocabulary, MedDRA 20.1
*
Indicates events were collected by non-systematic assessment
[1]
As the event is gender specific, only female participants were evaluated.
[Not Specified]
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Pfizer ClinicalTrials.gov Call Center
Organization: Pfizer, Inc.
Phone: 1-800-718-1021
EMail: ClinicalTrials.gov_Inquiries@pfizer.com
Layout table for additonal information
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT01642407    
Other Study ID Numbers: A1481298
First Submitted: June 15, 2012
First Posted: July 17, 2012
Results First Submitted: November 3, 2016
Results First Posted: April 4, 2017
Last Update Posted: October 25, 2018