Trial Outcomes & Findings for Sildenafil in Single Ventricle Patients (NCT NCT01169519)

Results Overview

Assessment of peak sildenafil plasma concentration.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

21 participants

Primary outcome timeframe

5 minutes after completion of sildenafil infusion

Results posted on

2013-12-05

Participant Flow

Patients recruited upon presentation for elective Pre-stage II or stage III catheterization. The first patient was recruited on 4/14/2011 and the final patient was enrolled on 11/29/2012

Enrolled subjects were excluded from trial participation if they demonstrated baseline hemodynamics suggesting that they would not tolerate sildenafil infusion (e.g. baseline hypotension)

Participant milestones

Participant milestones
Measure	Baseline/Sildenafil Assessment of baseline hemodynamics followed by sildenafil infusion with repeat assessment of hemodynamics
Overall Study STARTED	21
Overall Study COMPLETED	21
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Sildenafil in Single Ventricle Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Sildenafil n=21 Participants Pharmacokinetic and hemodynamic evaluation following sildenafil administration Sildenafil by injection : Sildenafil 0.45mg/kg by injection over 20min Sildenafil by injection : Sildenafil 0.25mg/kg injection over 20min Sildenafil by injection : Sildenafil 0.35mg/kg by injection over 20min Sildenafil by injection : Sildenafil 0.125mg/kg injection over 20min
Age, Categorical <=18 years	21 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age Continuous	3.4 years STANDARD_DEVIATION 2.6 • n=5 Participants
Sex: Female, Male Female	12 Participants n=5 Participants
Sex: Female, Male Male	9 Participants n=5 Participants
Region of Enrollment United States	21 participants n=5 Participants

PRIMARY outcome

Timeframe: 5 minutes after completion of sildenafil infusion

Population: 3 patients were enrolled in this group but in 1 participant, an inadequate plasma sample volume prevented accurate determination of sildenafil concentration.

Assessment of peak sildenafil plasma concentration.

Outcome measures

Outcome measures
Measure	Peak Sildenafil Level (ng/mL) for Dose = 0.125mg/kg n=2 Participants Maximum sildenafil plasma concentration measured 5 minutes after completion of sildenafil infusion	Peak Sildenafil Level (ng/mL) for Dose = 0.35mg/kg n=8 Participants Maximum sildenafil plasma concentration measured 5 minutes after completion of sildenafil infusion	Peak Sildenafil Level (ng/mL) for Dose = 0.45mg/kg n=5 Participants Maximum sildenafil plasma concentration measured 5 minutes after completion of sildenafil infusion	Peak Sildenafil Level (ng/mL) for Dose = 0.25mg/kg n=5 Participants Maximum sildenafil plasma concentration measured 5 minutes after completion of sildenafil infusionplasma concentration
Maximum Sildenafil Plasma Concentration	181 ng/mL Standard Deviation 126	515 ng/mL Standard Deviation 98	545 ng/mL Standard Deviation 185	202 ng/mL Standard Deviation 92

SECONDARY outcome

Timeframe: 10 minutes after completion of sildenafil infusion

Assessment of pulmonary vascular resistance

Outcome measures

Outcome measures
Measure	Peak Sildenafil Level (ng/mL) for Dose = 0.125mg/kg n=21 Participants Maximum sildenafil plasma concentration measured 5 minutes after completion of sildenafil infusion	Peak Sildenafil Level (ng/mL) for Dose = 0.35mg/kg n=21 Participants Maximum sildenafil plasma concentration measured 5 minutes after completion of sildenafil infusion	Peak Sildenafil Level (ng/mL) for Dose = 0.45mg/kg Maximum sildenafil plasma concentration measured 5 minutes after completion of sildenafil infusion	Peak Sildenafil Level (ng/mL) for Dose = 0.25mg/kg Maximum sildenafil plasma concentration measured 5 minutes after completion of sildenafil infusionplasma concentration
Hemodynamic Safety and Efficacy	2.3 Wood units * m^2 Interval 1.7 to 3.0	1.6 Wood units * m^2 Interval 1.2 to 2.0	—	—

Adverse Events

Dose = 0.125mg/kg

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Dose = 0.25mg/kg

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Dose = 0.35mg/kg

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Dose = 0.45mg/kg

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Kevin Hill

Duke University Medical Center

Phone: 919-668-8305

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place