A Randomized, Double-Blind, Placebo-Controlled Study of Sildenafil in Children With Pulmonary Arterial Hypertension.

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00159913
First received: September 8, 2005
Last updated: January 23, 2014
Last verified: January 2014
Results First Received: June 2, 2009  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator);   Primary Purpose: Treatment
Condition: Pulmonary Arterial Hypertension, Children
Interventions: Drug: Sildenafil citrate
Drug: Placebo

  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
The study was conducted at 32 centers in North, Latin and South America, Europe and Asia.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Of the 324 subjects screened, 235 subjects were randomized. 234 received treatment. One subject(sildenafil medium dose group) withdrew prior to taking any study treatment as the hemodynamic entrance criteria were not met.

Reporting Groups
  Description
Sildenafil Low Dose

Day 1-7 10 mg, followed by 10 mg TID (3 times daily) for body weights > 20-45 kg and > 45 kg, through Day 112.

Modeling of the plasma concentrations for each dose level showed that the low and medium doses were predicted to be similar for the 8 to 20 kg subjects (ie, subjects would receive the same dose because of the available tablet strengths); consequently there was no low dose for the >= 8-20 kg weight group.

Sildenafil Medium Dose Day 1-7 10 mg, followed by 10, 20, 40 mg TID (3 times daily) for body weights >= 8-20 kg, > 20-45 kg, > 45 kg respectively, through Day 112
Sildenafil High Dose Day 1-7 10 mg, followed by 20, 40, 80 mg TID (3 times daily) for body weights >= 8-20 kg, > 20-45 kg, > 45 kg respectively, through Day 112
Placebo Subjects randomized to this arm recieved placebo TID (three times daily) for 112 days.

Participant Flow:   Overall Study
    Sildenafil Low Dose     Sildenafil Medium Dose     Sildenafil High Dose     Placebo  
STARTED     42     55     77     60  
COMPLETED     40     55     75     58  
NOT COMPLETED     2     0     2     2  
Adverse Event                 1                 0                 1                 0  
Withdrawal by Subject                 0                 0                 0                 1  
Lost to Follow-up                 0                 0                 0                 1  
Protocol Violation                 1                 0                 1                 0  



  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.
No text entered.

Reporting Groups
  Description
Sildenafil Low Dose

Day 1-7 10 mg, followed by 10 mg TID (3 times daily) for body weights > 20-45 kg and > 45 kg, through Day 112.

Modeling of the plasma concentrations for each dose level showed that the low and medium doses were predicted to be similar for the 8 to 20 kg subjects (ie, subjects would receive the same dose because of the available tablet strengths); consequently there was no low dose for the >= 8-20 kg weight group.

Sildenafil Medium Dose Day 1-7 10 mg, followed by 10, 20, 40 mg TID (3 times daily) for body weights >= 8-20 kg, > 20-45 kg, > 45 kg respectively, through Day 112
Sildenafil High Dose Day 1-7 10 mg, followed by 20, 40, 80 mg TID (3 times daily) for body weights >= 8-20 kg, > 20-45 kg, > 45 kg respectively, through Day 112
Placebo Subjects randomized to this arm recieved placebo TID (three times daily) for 112 days.
Total Total of all reporting groups

Baseline Measures
    Sildenafil Low Dose     Sildenafil Medium Dose     Sildenafil High Dose     Placebo     Total  
Number of Participants  
[units: participants]
  42     55     77     60     234  
Age, Customized  
[units: participants]
         
1-4 Years     0     9     19     7     35  
5-12 Years     25     28     36     37     126  
13-17 Years     17     18     22     16     73  
>= 18 Years     0     0     0     0     0  
Gender  
[units: participants]
         
Female     25     31     51     38     145  
Male     17     24     26     22     89  



  Outcome Measures
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1.  Primary:   Percent Change From Baseline in Peak Volume of Oxygen (VO2) Consumed : Intent To Treat Population   [ Time Frame: Baseline, Week 16 ]

2.  Primary:   Percent Change From Baseline in Peak Volume of Oxygen (VO2) Consumed : Per Protocol Population   [ Time Frame: Baseline, Week 16 ]

3.  Secondary:   Change From Baseline to Week 16 in Mean Pulmonary Artery Pressure (mPAP)   [ Time Frame: Baseline, Week 16 ]

4.  Secondary:   Change From Baseline to Week 16 in Pulmonary Vascular Resistance Index (PVRI)   [ Time Frame: Baseline, Week 16 ]

5.  Secondary:   Percent Change From Baseline to Week 16 in: Respiratory Exchange Ratio (RER)   [ Time Frame: Baseline, Week 16 ]

6.  Secondary:   Percent Change From Baseline to Week 16 in Time to Maximum Volume of Oxygen Consumed (VO2)   [ Time Frame: Baseline, Week 16 ]

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

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

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

10.  Secondary:   Change From Baseline to Week 16 in Child Health Questionnaire Parent Form (CHQ-PF28), Physical Scale   [ Time Frame: Baseline, Week 16 ]

11.  Secondary:   Change From Baseline to Week 16 in Child Health Questionnaire Parent Form (CHQ-PF28), Psychosocial Scales   [ Time Frame: Baseline, Week 16 ]

12.  Secondary:   Change From Baseline to Week 16 in World Health Organization (WHO) Pulmonary Hypertension (PH) Functional Class   [ Time Frame: Baseline, Week 16 ]


  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.govCallCenter@pfizer.com


No publications provided by Pfizer

Publications automatically indexed to this study:

Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00159913     History of Changes
Other Study ID Numbers: A1481131
Study First Received: September 8, 2005
Results First Received: June 2, 2009
Last Updated: January 23, 2014
Health Authority: United States: Food and Drug Administration