Heart DiseasePhase 1NCT01169519

Sildenafil in Single Ventricle Patients

A Phase 1 study testing Sildenafil by injection in people with Heart Disease, run by Duke University at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages 3 to 120 months
Conditions the study is for: Heart Disease
What is being tested?: Sildenafil by injection
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 21 participants
Where: 1 siteUnited States
Study dates: Apr 2011to Dec 2012
Phase: Phase 1
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 3 to 120 months
All sexes
No healthy volunteers
Child

Who may be able to join

Age ≥ 3 months; ≤120 months.
History of congenital heart disease with severe hypoplasia of a right or left ventricle.
Undergoing cardiac catheterization as part of standard clinical care.
Availability and willingness of the parent/legally authorized representative to provide written informed consent.

Who cannot join

History of serious adverse event related to sildenafil administration.
History of sildenafil exposure within 48 hours of the study.
Presence of pulmonary venous obstruction.
Treatment with organic nitrates or alpha blockade therapy.
Contraindication to cardiac catheterization as determined by the attending cardiologist and including:
Significant hemodynamic instability.

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Sepsis.
Need for Extra-Corporeal Membrane Oxygenation (ECMO) support.
Venous occlusion precluding adequate access.
Recent systemic illness.
Renal failure defined as serum creatinine > 2 times higher than the upper limit of normal.
Liver dysfunction defined as alanine aminotransferase or aspartate aminotransferase > 3 times higher than the upper limit of normal.
Thrombocytopenia defined as a platelet count < 50 000 cells/µL.
Leukopenia defined as white blood cells < 2500 cells/µL.

Trial locations and contacts

1 site in 1 country.

United States1 site

Duke University Medical Center
Durham, North Carolina, 27710

Other trials in United States

About this study

Official title: Safety, Pharmacokinetics and Hemodynamic Efficacy of Sildenafil in Single Ventricle Patients

Patients with single ventricle anatomy undergo staged surgical palliation. The result is an "in series" circulation with pulmonary blood flow and cardiac output directly related to pulmonary vascular resistance. While surgical outcomes have improved, the physiology of the single ventricle palliation results in continued long term attrition. Elevated pulmonary vascular resistance and impaired systemic ventricular function are important risk factors for failure of single ventricle palliation.

Sildenafil is a pulmonary vasodilator and has been shown to improve cardiac contractility in the pressure overloaded right ventricle.

The investigators will assess the safety, pharmacokinetics and hemodynamic efficacy of sildenafil in single ventricle patients following stage II and III surgical palliation.

Conditions: Heart Disease
Being tested: Sildenafil by injection
Keywords: Hypoplastic left heart syndrome, Hypoplastic right heart syndrome, Superior cavopulmonary anastomosis, Total cavopulmonary anastomosis, Children with single ventricle anatomy
Medical subject headings: Heart Diseases, Hypoplastic Left Heart Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Sildenafil by injectionDrug
Also called Revatio
Sildenafil 0.125mg/kg injection over 20min
Sildenafil by injectionDrug
Also called Revatio
Sildenafil 0.25mg/kg injection over 20min
Sildenafil by injectionDrug
Also called Revatio
Sildenafil 0.35mg/kg by injection over 20min
Sildenafil by injectionDrug
Also called Revatio
Sildenafil 0.45mg/kg by injection over 20min

Study groups in NCT01169519
Study group	Role	What they receive
Sildenafil	Active comparator	Pharmacokinetic and hemodynamic evaluation following sildenafil administration Receives: Drug: Sildenafil by injection

What is being measured

The primary outcome is the main question the study is designed to answer.

Maximum Sildenafil Plasma Concentration
Assessment of peak sildenafil plasma concentration.
Measured: 5 minutes after completion of sildenafil infusion

Secondary outcomes (1)

Hemodynamic Safety and Efficacy
Assessment of pulmonary vascular resistance
Measured: 10 minutes after completion of sildenafil infusion

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Apr 2011Dec 2012

First posted26 Jul 2010 (estimated)
Study startApr 2011
Primary completionDec 2012
Study completionDec 2012

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (2)Papers the sponsor has linked to this study.
Other trials for Heart DiseaseStudies of the same condition, open and under way.
More trials run by Duke UniversityThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 21 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01169519
Sponsor's protocol ID: Pro00025220
Status last verified: Nov 2012
First submitted: 7 Jul 2010
Last updated: 5 Dec 2013
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Publications

Hill KD, Tunks RD, Barker PC, Benjamin DK Jr, Cohen-Wolkowiez M, Fleming GA, Laughon M, Li JS. Sildenafil exposure and hemodynamic effect after stage II single-ventricle surgery. Pediatr Crit Care Med. 2013 Jul;14(6):593-600. doi: 10.1097/PCC.0b013e31828aa5ee.
PubMed 23823195
Tunks RD, Barker PC, Benjamin DK Jr, Cohen-Wolkowiez M, Fleming GA, Laughon M, Li JS, Hill KD. Sildenafil exposure and hemodynamic effect after Fontan surgery. Pediatr Crit Care Med. 2014 Jan;15(1):28-34. doi: 10.1097/PCC.0000000000000007.
PubMed 24201857

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 2/3
Efficacy, Safety, and Pharmacokinetics of Vericiguat in Pediatric Participants With Heart Failure Due to Left Ventricular Systolic Dysfunction (MK-1242-036)
Heart Failure, Left Ventricular Systolic Dysfunction
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Efficacy and Safety of a Half-dose Bolus of r-SAK Prior to Primary PCI in ST-elevation Myocardial Infarction
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Active, not recruitingPhase 4
Women's IschemiA TRial to Reduce Events In Non-ObstRuctive CAD
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VOLT CE Mark Study
Atrial Arrhythmia, Atrial Fibrillation
Recruiting
SYMmetric Valve fraME Expansion To Reduce hYpoattenuated Leaflet Thickening After Balloon-expandable TAVI: the SYMMETRY Trial
Aortic Valve Stenosis and/or Insufficiency, Hypoattenuated Leaflet Thickening

All Heart Disease trials

Study at a glance

From the registry record, last updated by the sponsor on 5 Dec 2013. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Safety, Pharmacokinetics and Hemodynamic Efficacy of Sildenafil in Single Ventricle Patients
ClinicalTrials.gov ID: NCT01169519
Acronym / sponsor ID: Pro00025220
Conditions: Heart Disease
Keywords: Hypoplastic left heart syndrome, Hypoplastic right heart syndrome, Superior cavopulmonary anastomosis, Total cavopulmonary anastomosis, Children with single ventricle anatomy
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 21 participants (actual)
Lead sponsor: Duke UniversityUniversity, hospital or other
Study start: Apr 2011
Primary completion: Dec 2012 (actual)
Study completion: Dec 2012 (actual)
First posted: 26 Jul 2010 (estimated)
Results: Posted 5 Dec 2013

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Sildenafil is a pulmonary vasodilator and has been shown to improve cardiac contractility in the pressure overloaded right ventricle.

The investigators will assess the safety, pharmacokinetics and hemodynamic efficacy of sildenafil in single ventricle patients following stage II and III surgical palliation.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Sildenafil	Active comparator	Drug: Sildenafil by injectionPharmacokinetic and hemodynamic evaluation following sildenafil administration

Who may qualify

Ages: 3 to 120 months
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (4 listed)

Age ≥ 3 months; ≤120 months.
History of congenital heart disease with severe hypoplasia of a right or left ventricle.
Undergoing cardiac catheterization as part of standard clinical care.
Availability and willingness of the parent/legally authorized representative to provide written informed consent.

Key exclusion criteria (14 listed)

History of serious adverse event related to sildenafil administration.
History of sildenafil exposure within 48 hours of the study.
Presence of pulmonary venous obstruction.
Treatment with organic nitrates or alpha blockade therapy.
Contraindication to cardiac catheterization as determined by the attending cardiologist and including:

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Sildenafil by injectionAlso: RevatioSildenafil 0.125mg/kg injection over 20min	Drug	Sildenafil
Sildenafil by injectionAlso: RevatioSildenafil 0.25mg/kg injection over 20min	Drug	Sildenafil
Sildenafil by injectionAlso: RevatioSildenafil 0.35mg/kg by injection over 20min	Drug	Sildenafil
Sildenafil by injectionAlso: RevatioSildenafil 0.45mg/kg by injection over 20min	Drug	Sildenafil

Primary outcome

Maximum Sildenafil Plasma Concentration
Time frame: 5 minutes after completion of sildenafil infusion
Assessment of peak sildenafil plasma concentration.

1 secondary outcome

Hemodynamic Safety and Efficacy
Time frame: 10 minutes after completion of sildenafil infusion

Results and safety

Results posted to the registry on 5 Dec 2013, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Baseline/Sildenafil	21	21

Primary outcome: Maximum Sildenafil Plasma Concentration

Time frame: 5 minutes after completion of sildenafil infusion. Mean (Standard Deviation). Unit: ng/mL

Measure	Peak Sildenafil Level (ng/mL) for Dose = 0.125mg/kg n = 2	Peak Sildenafil Level (ng/mL) for Dose = 0.35mg/kg n = 8	Peak Sildenafil Level (ng/mL) for Dose = 0.45mg/kg n = 5	Peak Sildenafil Level (ng/mL) for Dose = 0.25mg/kg n = 5
Value	181 (126)	515 (98)	545 (185)	202 (92)

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

1 secondary outcome is reported in the full results.

Adverse events

Other adverse events reported at 3% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Dose = 0.125mg/kg	0 of 3 (0%)	0 of 3 (0%)	Not reported
Dose = 0.25mg/kg	0 of 5 (0%)	0 of 5 (0%)	Not reported
Dose = 0.35mg/kg	0 of 8 (0%)	0 of 8 (0%)	Not reported
Dose = 0.45mg/kg	0 of 5 (0%)	0 of 5 (0%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 5 Dec 2013.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Results publication: PubMed 23823195Hill KD, Tunks RD, Barker PC, Benjamin DK Jr, Cohen-Wolkowiez M, Fleming GA, Laughon M, Li JS. Sildenafil exposure and hemodynamic effect after stage II single-ventricle surgery. Pediatr Crit Care Med. 2013 Jul;14(6):593-600. doi: 10.1097/PCC.0b013e31828aa5ee.
Derived from this study: PubMed 24201857Tunks RD, Barker PC, Benjamin DK Jr, Cohen-Wolkowiez M, Fleming GA, Laughon M, Li JS, Hill KD. Sildenafil exposure and hemodynamic effect after Fontan surgery. Pediatr Crit Care Med. 2014 Jan;15(1):28-34. doi: 10.1097/PCC.0000000000000007.

Related trials for Heart Disease

RecruitingPhase 2/3
Efficacy, Safety, and Pharmacokinetics of Vericiguat in Pediatric Participants With Heart Failure Due to Left Ventricular Systolic Dysfunction (MK-1242-036)
Heart Failure, Left Ventricular Systolic Dysfunction
RecruitingPhase 4
Efficacy and Safety of a Half-dose Bolus of r-SAK Prior to Primary PCI in ST-elevation Myocardial Infarction
ST Elevation Myocardial Infarction
Active, not recruitingPhase 4
Women's IschemiA TRial to Reduce Events In Non-ObstRuctive CAD
Coronary Artery Disease
Active, not recruiting
VOLT CE Mark Study
Atrial Arrhythmia, Atrial Fibrillation
Recruiting
SYMmetric Valve fraME Expansion To Reduce hYpoattenuated Leaflet Thickening After Balloon-expandable TAVI: the SYMMETRY Trial
Aortic Valve Stenosis and/or Insufficiency, Hypoattenuated Leaflet Thickening

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Kevin D Hill, MD principal investigator

Duke University

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Sildenafil by injection in people with Heart Disease, run by Duke University at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 5 Dec 2013. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 5 Dec 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Maximum Sildenafil Plasma Concentration (time frame: 5 minutes after completion of sildenafil infusion). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01169519, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Dec 2013.