Phase 3 N=234 Randomized Treatment

A Long Term Extension Study Evaluating Safety Of Sildenafil Citrate When Used To Treat Pulmonary Arterial Hypertension (PAH) In Children

Pulmonary Arterial Hypertension

Bottom Line

View on ClinicalTrials.gov: NCT00159874 ↗

Enrolled (actual)

234

Serious AEs

43.2%

Results posted

Aug 2014

Primary outcome: Primary: Number of Participants Reporting at Least One Adverse Event — 41; 55; 73; 13 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Sildenafil citrate (Drug)
Age: Pediatric · 1+ yrs
Sex: All
Sponsor: Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Primary completion: Dec 2012

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants Reporting at Least One Adverse Event	41; 55; 73; 13; 19; 22	—
PRIMARY Number of Participants Reporting Treatment-related Adverse Events	20; 24; 41; 9; 9; 11	—
PRIMARY Number of Participants Reporting at Least One Serious Adverse Event	13; 33; 38; 1; 4; 10	—
PRIMARY Number of Participants Reporting Treatment-related Serious Adverse Events	1; 1; 4; 0; 0; 0	—
PRIMARY Number of Deaths Reported in the Study Prior to the Data Monitoring Committee (DMC) Recommendation of Dose Down Titration	5; 10; 22	—
PRIMARY Number of Deaths Reported During This Study	5; 13; 24	—
PRIMARY Discontinuation Due to Intolerability	2; 1; 3	—
PRIMARY Downtitration in Dose Due to Intolerability.	0; 0; 3; 0; 2; 1	—
PRIMARY Number of Participants With Deterioration Post Baseline in Visual Acuity Safety Tests	10; 11; 17; 0; 4; 4	—
PRIMARY Number of Participants With Deterioration Post Baseline in Color Vision Monitoring Safety Tests.	2; 2; 1; 0; 0; 1	—
PRIMARY Pediatric Cognitive Development Status at Week 16.	2; 4; 2; 0; 1; 1	—
PRIMARY Pediatric Cognitive Development Status at Week 52.	1; 1; 2; 0; 0; 0	—
PRIMARY Pediatric Motor Development Status at Week 16.	0; 0; 0; 0; 1; 0	—
PRIMARY Pediatric Motor Development Status at Week 52	0; 0; 0; 0; 0; 0	—
SECONDARY Peak Volume of Oxygen (VO2) Consumed at Year 1 Using a Bicycle Ergometry Cardiopulmonary Exercise Test (CPX)	19.97; 18.69; 17.93	0.253
SECONDARY Percentage Change From Baseline in Percent Predicted Peak VO2 at Year 1.	12.79; 7.65; 5.83; 8.70; 0.20; -6.13	—
SECONDARY Percent Change From Baseline in Time to Maximum VO2 at Year 1	25.47; 13.08; 7.70; 21.17; 36.68; -9.64	—
SECONDARY Percent Change From Baseline in Respiratory Exchange Ratio at Year 1	2.15; 5.63; 0.68; -3.69; 0.27; 10.75	—
SECONDARY Percent Change From Start of Sildenafil in Total Ventilation (VE) to Year 1	14.29; 12.38; 11.80	—
SECONDARY Percentage Change From Baseline in End Tidal Oxygen (O2) at Year 1.	0.59; -0.52; 0.08	—
SECONDARY Percentage Change From Baseline in End Tidal Carbon Dioxide (CO2) at Year 1.	7.83; 7.68; 13.16	—
SECONDARY Percentage Change From Baseline in Anaerobic Threshold at Year 1.	-1.22; 1.99; 3.28; 7.23; -3.59; 8.96	—
SECONDARY Summary of Shift in Changes From Start of Sildenafil in World Health Organization Pulmonary Hypertension (WHO PH) Functional Class by A1481156 Treatment Group at Year 1.	0; 0; 0; 1; 0; 1	—
SECONDARY Summary of Shift in Changes From Start of Sildenafil in WHO PH Functional Class by A1481156 Treatment Group at Year 2.	0; 0; 0; 0; 1; 1	—
SECONDARY Summary of Shift in Changes From Start of Sildenafil in WHO PH Functional Class by A1481156 Treatment Group at Year 3.	0; 0; 0; 1; 0; 1	—
SECONDARY Summary of Shift in Changes From Start of Sildenafil in WHO PH Functional Class by A1481156 Treatment Group at Year 4.	0; 0; 0; 0; 0; 2	—
SECONDARY Additions From Baseline in Background Therapy up to the End of Study	6; 5; 11; 2; 1; 2	—
SECONDARY Change From Baseline in Child Health Questionnaire-Parent Form (CHQ-PF28) as Assessed by the Psychosocial Scale at Year 1.	5.63; 3.92; 3.48; 13.74; 5.30; 4.27	—
SECONDARY Change From Baseline in Child Health Questionnaire-Parent Form (CHQ-PF28) as Assessed by the Physical Scale at Year 1.	14.29; 9.34; 5.91; 8.51; 9.86; 4.64	—
SECONDARY Participant (Parent) Global Assessment at Year 1	9; 14; 21; 13; 27; 26	—
SECONDARY Physician Global Assessment at Year 1	6; 6; 6; 8; 18; 27	—

Summary

Active treatment, dose-blinded extension study evaluating the safety and long term efficacy of sildenafil citrate in children with PAH.

Eligibility Criteria

Inclusion Criteria

Patients must complete the 16 Week double-blind efficacy study A1481131.

Exclusion Criteria

Any patient who did not complete Study A1481131.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00159874). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.