NCT03154476

Brief Summary

The purpose of this study is to determine if the medication, sildenafil (also known as Revatio), can slow or stop the progression of liver disease in patients who previously had a Fontan operation.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Jul 2017

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

May 12, 2017

Completed
4 days until next milestone

First Posted

Study publicly available on registry

May 16, 2017

Completed
2 months until next milestone

Study Start

First participant enrolled

July 5, 2017

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 30, 2019

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 30, 2019

Completed
2.1 years until next milestone

Results Posted

Study results publicly available

July 28, 2021

Completed
Last Updated

August 23, 2021

Status Verified

July 1, 2021

Enrollment Period

2 years

First QC Date

May 12, 2017

Results QC Date

July 7, 2021

Last Update Submit

July 28, 2021

Conditions

CirrhosisCongenital Heart Disease

Outcome Measures

Primary Outcomes (1)

  • Liver Stiffness as Measured by Magnetic Resonance Elastography (MRE)

    Measured using magnetic resonance imaging derived liver stiffness (MRE-LS) reported in kilopascal (kPa).

    Baseline, 12 months (52 weeks), 24 months (104 weeks)

Study Arms (2)

Sildenafil

EXPERIMENTAL

Subjects randomized to this arm will receive sildenafil 5 mg 3 times per day for the first week, and titrated to 10 mg 3 times per day for the second week, and 20 mg 3 times per day from the third week to the end of the study period, 12 months.

Drug: Sildenafil

Placebo

PLACEBO COMPARATOR

Subjects will receive placebo times per day for 12 months.

Drug: Placebo

Interventions

SildenafilDRUG

Sildenafil will be initiated at 5 mg 3 times per day for the first week, and titrated to 10 mg 3 times per day for the second week and 20 mg 3 times per day from the third week to the end of the study period, 12 months.

Also known as: Revatio
Sildenafil
PlaceboDRUG

Placebo capsules matching study drug

Placebo

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • All adult Fontan patients who have no contraindications for magnetic resonance imaging (MRI) will be eligible for the study.

You may not qualify if:

  • Subjects with implantable pacemakers
  • Residual cardiac lesions (severe ventricular dysfunction, severe atrioventricular valve regurgitation, Fontan baffle or conduit obstruction)
  • Viral hepatitis
  • Severe renal dysfunction
  • History of sildenafil use in the six months prior to study enrollment
  • Ongoing sildenafil therapy
  • Patients currently taking nitrates
  • Hypotension at baseline (BP \<90/50 mmHg)
  • Pulmonary veno-occlusive disease
  • Hearing/vision impairment
  • Pulmonary hypertension due to sickle cell disease
  • Women of child-bearing potential with a positive pregnancy test will additionally be excluded

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

Location

Related Links

MeSH Terms

Conditions

FibrosisHeart Defects, Congenital

Interventions

Sildenafil Citrate

Condition Hierarchy (Ancestors)

Pathologic ProcessesPathological Conditions, Signs and SymptomsCardiovascular AbnormalitiesCardiovascular DiseasesHeart DiseasesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

SulfonamidesAmidesOrganic ChemicalsSulfonesSulfur CompoundsPiperazinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsPurinesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-Ring

Results Point of Contact

Title
Dr. Alexander Egbe
Organization
Mayo Clinic
Egbe.Alexander@mayo.edu
507-284-3725

Study Officials

  • Alexander C Egbe

    Mayo Clinic

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Masking Details
Both the treatment arm (sildenafil) and the placebo arm will receive monthly package of similar pills. Equal number of patients will be randomly assigned to each arm.
Purpose
OTHER
Intervention Model
PARALLEL
Model Details: Randomized Double-blind Placebo-control study
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Assistant Professor of Medicine and Pediatrics

Study Record Dates

First Submitted

May 12, 2017

First Posted

May 16, 2017

Study Start

July 5, 2017

Primary Completion

June 30, 2019

Study Completion

June 30, 2019

Last Updated

August 23, 2021

Results First Posted

July 28, 2021

Record last verified: 2021-07

Data Sharing

IPD Sharing
Will share

The safety of the subject will be monitored throughout the duration of the study. During the first month of enrollment (initiation and dose titration), adverse events will be collected by subject report and by weekly telephone interview with dedicated research personnel. For the rest of the study period, adverse events will be collected by subject report and by monthly telephone interview with dedicated research personnel. All participants will be provided with a pamphlet containing all the side effects of sildenafil, and contact information of research team for reporting any adverse event of concerns about the study. The patients will be required to check their pulse rate and blood pressure daily during the first month of drug therapy. Patient who experience hypotension (blood pressure \< 90/60 plus symptoms such as dizziness) during dose titration will be asked to remain on the previous tolerated dose

Locations

US(1)