Safety And Efficacy Of Sildenafil In Children With Pulmonary Arterial Hypertension

• Sponsor: Pfizer
• 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

Participant Flow

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
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

Baseline Characteristics

Arm/Group Title		Sildenafil
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
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%

Outcome Measures

1. Primary Outcome

Title	Change From Baseline in Pulmonary Vascular Resistance Index (PVRI) at Week 16
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

Outcome Measure Data

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
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
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

Outcome Measure Data

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
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
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

Outcome Measure Data

Analysis Population Description

Efficacy analysis set included all participants who received at least 1 dose of study drug.

Arm/Group Title	Sildenafil
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
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

Outcome Measure Data

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
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
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

Outcome Measure Data

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
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
Description	BNP is produced by ventricular cardiomyocytes. It causes reduction in preload and blood pressure by vasodilatation.
Time Frame	Baseline, Week 16

Outcome Measure Data

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
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
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

Outcome Measure Data

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
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
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

Outcome Measure Data

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
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)
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)

Outcome Measure Data

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
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)
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)

Outcome Measure Data

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
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)
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)

Outcome Measure Data

Analysis Population Description

Safety analysis set included all participants who received at least 1 dose of study drug.

Arm/Group Title	Sildenafil
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)
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)

Outcome Measure Data

Analysis Population Description

Safety analysis set included all participants who received at least 1 dose of study drug.

Arm/Group Title	Sildenafil
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
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

Outcome Measure Data

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
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
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

Outcome Measure Data

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
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
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)

Outcome Measure Data

Analysis Population Description

Safety analysis set included all participants who received at least 1 dose of study drug.

Arm/Group Title	Sildenafil
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
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)

Outcome Measure Data

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
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
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)

Outcome Measure Data

Analysis Population Description

Safety analysis set included all participants who received at least 1 dose of study drug.

Arm/Group Title	Sildenafil
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
Description	[Not Specified]
Time Frame	Baseline, Week 16

Outcome Measure Data

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
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
Description	[Not Specified]
Time Frame	Baseline, Week 16

Outcome Measure Data

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
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
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

Outcome Measure Data

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
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
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

Outcome Measure Data

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
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
Description	PCWP was measured by pulmonary artery catheterization and provided an indirect measure of left atrial pressure.
Time Frame	Baseline, Week 16

Outcome Measure Data

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
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
Description	Cardiac output is simply the amount of blood pumped by the heart per minute.
Time Frame	Baseline, Week 16

Outcome Measure Data

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
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
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

Outcome Measure Data

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
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
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

Outcome Measure Data

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
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
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

Outcome Measure Data

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
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
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

Outcome Measure Data

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
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
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

Outcome Measure Data

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
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
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

Outcome Measure Data

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
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
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

Outcome Measure Data

Analysis Population Description

PK parameter analysis set included all participants who have at least 1 of PK parameters of interest.

Arm/Group Title	Sildenafil
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
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

Outcome Measure Data

Analysis Population Description

PK parameter analysis set included all participants who have at least 1 of PK parameters of interest.

Arm/Group Title	Sildenafil
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
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

Outcome Measure Data

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
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
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

Outcome Measure Data

Analysis Population Description

PK parameter analysis set included all participants who have at least 1 of PK parameters of interest.

Arm/Group Title	Sildenafil
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
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

Outcome Measure Data

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
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
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

Outcome Measure Data

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
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
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

Outcome Measure Data

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
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
Description	[Not Specified]
Time Frame	Baseline, Week 16

Outcome Measure Data

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
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
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

Outcome Measure Data

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
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
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

Outcome Measure Data

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
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
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

Outcome Measure Data

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
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
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

Outcome Measure Data

Analysis Population Description

Efficacy analysis set included all participants who received at least 1 dose of study drug.

Arm/Group Title	Sildenafil
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
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

Outcome Measure Data

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
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)

Adverse Events

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
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%)
Serious Adverse Events
	Sildenafil
	Affected / at Risk (%)
Total	0/6 (0.00%)
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.

Limitations and Caveats

[Not Specified]

More Information

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

Name/Title: Pfizer ClinicalTrials.gov Call Center

Organization: Pfizer, Inc.

Phone: 1-800-718-1021

EMail: ClinicalTrials.gov_Inquiries@pfizer.com

Responsible Party:	Pfizer
ClinicalTrials.gov Identifier:	NCT01642407 History of Changes
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