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Safety And Efficacy Of Sildenafil In Children With Pulmonary Arterial Hypertension

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01642407
First Posted: July 17, 2012
Last Update Posted: May 10, 2017
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.
Information provided by (Responsible Party):
Pfizer
Results First Submitted: November 3, 2016  
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

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Sildenafil Participants received 10 milligram (mg) or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Week 4, 8 and 16. 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.

Participant Flow:   Overall Study
    Sildenafil
STARTED   6 
COMPLETED   4 
NOT COMPLETED   2 
Insufficient clinical response                2 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Efficacy analysis set included all participants who received at least 1 dose of study drug.

Reporting Groups
  Description
Sildenafil Participants received 10 milligram (mg) or 20 mg of Sildenafil (based on their body weight) orally, thrice daily on Day 1 (Baseline), Week 4, 8 and 16. 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.

Baseline Measures
   Sildenafil 
Overall Participants Analyzed 
[Units: Participants]
 6 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      6 100.0% 
Between 18 and 65 years      0   0.0% 
>=65 years      0   0.0% 
Age 
[Units: Years]
Mean (Standard Deviation)
 6.7  (5.4) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
FEMALE      3  50.0% 
MALE      3  50.0% 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Change From Baseline in Pulmonary Vascular Resistance Index (PVRI) at Week 16   [ Time Frame: Baseline, Week 16 ]

2.  Primary:   Change From Baseline in Mean Pulmonary Artery Pressure (mPAP) at Week 16   [ Time Frame: Baseline, Week 16 ]

3.  Primary:   Change From Baseline in World Health Organization (WHO) Functional Class in Participants With Pulmonary Arterial Hypertension (PAH) at Week 4   [ Time Frame: Baseline, Week 4 ]

4.  Primary:   Change From Baseline in World Health Organization (WHO) Functional Class in Participants With Pulmonary Arterial Hypertension (PAH) at Week 8   [ Time Frame: Baseline, Week 8 ]

5.  Primary:   Change From Baseline in World Health Organization (WHO) Functional Class in Participants With Pulmonary Arterial Hypertension (PAH) at Week 16   [ Time Frame: Baseline, Week 16 ]

6.  Primary:   Change From Baseline in Brain Natriuretic Peptide (BNP) at Week 16   [ Time Frame: Baseline, Week 16 ]

7.  Primary:   Change From Baseline in N-terminal Pro Brain Natriuretic Peptide (NT Pro-BNP) at Week 16   [ Time Frame: Baseline, Week 16 ]

8.  Secondary:   Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)   [ Time Frame: Baseline up to 28 days after last dose of study drug (up to 20 weeks) ]

9.  Secondary:   Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events (SAEs)   [ Time Frame: Baseline up to 28 days after last dose of study drug (up to 20 weeks) ]

10.  Secondary:   Change From Baseline in Systolic and Diastolic Blood Pressure (BP) at Week 4, 8 and 16   [ Time Frame: Baseline, Week 4, 8, 16 ]

11.  Secondary:   Change From Baseline in Heart Rate at Week 4, 8 and 16   [ Time Frame: Baseline, Week 4, 8, 16 ]

12.  Secondary:   Number of Participants With Laboratory Abnormalities   [ Time Frame: Baseline up to Week 16 ]

13.  Secondary:   Number of Participants With Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities   [ Time Frame: Baseline up to Week 16 ]

14.  Secondary:   Number of Participants With Ocular Examination Abnormalities   [ Time Frame: Baseline up to Week 16 ]

15.  Secondary:   Change From Baseline in Pulmonary Artery Systolic and Diastolic Pressure at Week 16   [ Time Frame: Baseline, Week 16 ]

16.  Secondary:   Change From Baseline in Systemic Artery Systolic and Diastolic Pressure at Week 16   [ Time Frame: Baseline, Week 16 ]

17.  Secondary:   Change From Baseline in Pulmonary Vascular Resistance (PVR) at Week 16   [ Time Frame: Baseline, Week 16 ]

18.  Secondary:   Change From Baseline in Right Atrial Pressure (RAP) at Week 16   [ Time Frame: Baseline, Week 16 ]

19.  Secondary:   Change From Baseline in Pulmonary Capillary Wedge Pressure (PCWP) at Week 16   [ Time Frame: Baseline, Week 16 ]

20.  Secondary:   Change From Baseline in Cardiac Output (CO) at Week 16   [ Time Frame: Baseline, Week 16 ]

21.  Secondary:   Change From Baseline in Cardiac Index (CI) at Week 16   [ Time Frame: Baseline, Week 16 ]

22.  Secondary:   Change From Baseline in Systemic Vascular Resistance (SVR) at Week 16   [ Time Frame: Baseline, Week 16 ]

23.  Secondary:   Change From Baseline in Systemic Vascular Resistance Index (SVRI) at Week 16   [ Time Frame: Baseline, Week 16 ]

24.  Secondary:   Change From Baseline in Mixed Venous Oxygen Saturation (SvO2) at Week 16   [ Time Frame: Baseline, Week 16 ]

25.  Secondary:   Change From Baseline in Arterial Oxygen Saturation (SaO2) at Week 16   [ Time Frame: Baseline, Week 16 ]

26.  Other Pre-specified:   Maximum Observed Plasma Concentration (Cmax) of Sildenafil and UK-103,320   [ Time Frame: Pre-dose (0 hour) on Week 4, 8, 16 and 1, 2, 4, 8 hours post-dose on Week 16 ]

27.  Other Pre-specified:   Area Under the Curve From Time Zero to End of Dosing Interval (AUCtau) of Sildenafil and UK-103,320   [ Time Frame: Pre-dose (0 hour) on Week 4, 8, 16 and 1, 2, 4, 8 hours post-dose on Week 16 ]

28.  Other Pre-specified:   Time to Reach Maximum Observed Plasma Concentration (Tmax) of Sildenafil and UK-103,320   [ Time Frame: Pre-dose (0 hour) on Week 4, 8, 16 and 1, 2, 4, 8 hours post-dose on Week 16 ]

29.  Other Pre-specified:   Terminal Half Life (t1/2) of Sildenafil and UK-103,320   [ Time Frame: Pre-dose (0 hour) on Week 4, 8, 16 and 1, 2, 4, 8 hours post-dose on Week 16 ]

30.  Other Pre-specified:   Apparent Oral Clearance (CL/F) of Sildenafil   [ Time Frame: Pre-dose (0 hour) on Week 4, 8, 16 and 1, 2, 4, 8 hours post-dose on Week 16 ]

31.  Other Pre-specified:   Apparent Volume of Distribution (Vz/F) of Sildenafil   [ Time Frame: Pre-dose (0 hour) on Week 4, 8, 16 and 1, 2, 4, 8 hours post-dose on Week 16 ]

32.  Other Pre-specified:   Change From Baseline in Ratio of Acceleration Time to Ejection Time (AcT/ET) at Week 16   [ Time Frame: Baseline, Week 16 ]

33.  Other Pre-specified:   Change From Baseline in Right Ventricular Tei Index at Week 16   [ Time Frame: Baseline, Week 16 ]

34.  Other Pre-specified:   Change From Baseline in Right Ventricular Size at Week 16   [ Time Frame: Baseline, Week 16 ]

35.  Other Pre-specified:   Change From Baseline in Tricuspid Valve Annulus Size at Week 16   [ Time Frame: Baseline, Week 16 ]

36.  Other Pre-specified:   Change From Baseline in Tricuspid Regurgitation - Pressure Gradient (TR-PG) Peak at Week 16   [ Time Frame: Baseline, Week 16 ]

37.  Other Pre-specified:   Change From Baseline in Pulmonary Regurgitation - Pressure Gradient (PR-PG) End-Diastole at Week 16   [ Time Frame: Baseline, Week 16 ]

38.  Other Pre-specified:   Number of Participants With Pericardial Effusion   [ Time Frame: Baseline up to Week 16 ]

39.  Other Pre-specified:   Change From Baseline in Tricuspid Annular Plane Systolic Excursion (TAPSE) at Week 16   [ Time Frame: Baseline, Week 16 ]

40.  Secondary:   Change From Baseline in World Health Organization (WHO) Functional Class in Participants With Pulmonary Arterial Hypertension (PAH) at Week 28, 40 and 52   [ Time Frame: Baseline, Week 28, 40, 52 ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

41.  Secondary:   Change From Baseline in Brain Natriuretic Peptide (BNP) at Week 52   [ Time Frame: Baseline, Week 52 ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

42.  Secondary:   Change From Baseline in N-terminal Pro Brain Natriuretic Peptide (NT Pro-BNP) at Week 52   [ Time Frame: Baseline, Week 52 ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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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.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Pfizer ClinicalTrials.gov Call Center
Organization: Pfizer, Inc.
phone: 1-800-718-1021
e-mail: 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: May 10, 2017