Back to resultsEffect of Tadalafil on Exercise Capacity in Pediatric Fontan Patients
Primary Purpose
Single Ventricle
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Tadalafil Citrate
Sugar pill
Sponsored by
About this trial
This is an interventional treatment trial for Single Ventricle focused on measuring Fontan, Tadalafil, Exercise performance
Eligibility Criteria
Inclusion Criteria:
- Patients who have undergone the Fontan procedure (regardless of type of Fontan connection or presence or absence of a fenestration)
- Age 8.0 to 35.0 years
- Stable clinical condition over the last 3 months (i.e. No change in medication, treatments, or development of new symptoms)
- Ability to perform exercise testing
- Consent and assent (as appropriate to participate in the study after receiving information concerning procedures, risks, and possible clinical benefits) o Patients will be enrolled without regard to gender, race, and ethnicity. All patients meeting study eligibility will be approached for consent.
Exclusion Criteria:
- Severe heart failure (New York Heart Association functional class III or IV)
- Presence of liver or renal dysfunction based on the latest lab test results
- Presence of hearing or visual deficit
- Transcutaneous arterial blood oxygen saturation (SaO2) <80% at rest
- History of echocardiographic, MRI, or angiographic evidence of Fontan pathway obstruction
- History of exercise-induced life-threatening arrhythmias or unstable rhythm(i.e. atrial flutter)
- Known or suspected pregnancy. Females in the reproductive age group will be screened for pregnancy using serum beta hCG prior to administering study drug.
- Patients on open label sildenafil or tadalafil
Sites / Locations
- Primary Childrens Medical Center
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Tadalafil Citrate
Sugar pill
Arm Description
The study subjects will be given Tadalfil citrate, encapsulated, 0.8-1 mg/kg/day in 1 dose orally. Max dose 40 mg. All patients will receive either study drug or placebo for a total of 20 days.
If allocated to the placebo arm, the child will be given a similar appearing medication; the placebo will be a sugar pill. All patients will receive either study drug or placebo for a total of 20 days.
Outcomes
Primary Outcome Measures
To compare the effects of Tadalafil versus placebo on exercise capacity (maximal oxygen consumption: VO2 max) for patients (8-35 years)with the Fontan circulation (8 -18 years).
Short-term treatment with Tadalafil will augment cardiac output and improve exercise capacity (maximal oxygen consumption: VO2 max) in patients with the Fontan circulation. Primary outcome variables include the change in peak exercise capacity (VO2 max), using cycle ergometry after one week of tadalafil therapy
Secondary Outcome Measures
To evaluate the adverse effects of Tdalafil in patients (8-35 years)with the Fontan circulation and compare with the placebo group.
Hypothesis: Short-term treatment with Tadalafil will be well tolerated by children with the Fontan circulation. Adverse effect forms will be used to compare the side effects between Tadalafil and treatment group.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01291069
Brief Title
Effect of Tadalafil on Exercise Capacity in Pediatric Fontan Patients
Official Title
Effect of Tadalafil on Exercise Capacity in Pediatric Fontan Patients
Study Type
Interventional
2. Study Status
Record Verification Date
April 2017
Overall Recruitment Status
Completed
Study Start Date
September 2011 (undefined)
Primary Completion Date
February 2014 (Actual)
Study Completion Date
February 2014 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University of Utah
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
This pilot study is aimed at assessing the short-term effects of Tadalafil on the hemodynamic response to exercise and exercise capacity in patients with Fontan circulation. Data regarding effect size and drug tolerability will be used in the design of a randomized multicenter trial. The long-term goal of this investigation is to systematically evaluate the effect of tadalafil therapy on exercise performance, quality of life, and delay of functional deterioration in patients with single ventricle physiology.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Single Ventricle
Keywords
Fontan, Tadalafil, Exercise performance
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
20 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Tadalafil Citrate
Arm Type
Experimental
Arm Description
The study subjects will be given Tadalfil citrate, encapsulated, 0.8-1 mg/kg/day in 1 dose orally. Max dose 40 mg. All patients will receive either study drug or placebo for a total of 20 days.
Arm Title
Sugar pill
Arm Type
Placebo Comparator
Arm Description
If allocated to the placebo arm, the child will be given a similar appearing medication; the placebo will be a sugar pill. All patients will receive either study drug or placebo for a total of 20 days.
Intervention Type
Drug
Intervention Name(s)
Tadalafil Citrate
Other Intervention Name(s)
Cialis
Intervention Description
If allocated to the treatment arm, the patient will be given tadalfil citrate, 0.8-1 mg/kg/day in 1 daily dose. Maximum dose 40 mg. All patients will receive either study drug or placebo for a total of 20 days.
Intervention Type
Drug
Intervention Name(s)
Sugar pill
Other Intervention Name(s)
Ora sweet
Intervention Description
If allocated to the placebo arm, the child will be given a similar appearing medication; the placebo will be a mixture of Ora-Sweet® and Ora-Plus® in a 1:1 ratio. All patients will receive drug for 20 days
Primary Outcome Measure Information:
Title
To compare the effects of Tadalafil versus placebo on exercise capacity (maximal oxygen consumption: VO2 max) for patients (8-35 years)with the Fontan circulation (8 -18 years).
Description
Short-term treatment with Tadalafil will augment cardiac output and improve exercise capacity (maximal oxygen consumption: VO2 max) in patients with the Fontan circulation. Primary outcome variables include the change in peak exercise capacity (VO2 max), using cycle ergometry after one week of tadalafil therapy
Time Frame
2 years
Secondary Outcome Measure Information:
Title
To evaluate the adverse effects of Tdalafil in patients (8-35 years)with the Fontan circulation and compare with the placebo group.
Description
Hypothesis: Short-term treatment with Tadalafil will be well tolerated by children with the Fontan circulation. Adverse effect forms will be used to compare the side effects between Tadalafil and treatment group.
Time Frame
2 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
8 Years
Maximum Age & Unit of Time
35 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Patients who have undergone the Fontan procedure (regardless of type of Fontan connection or presence or absence of a fenestration)
Age 8.0 to 35.0 years
Stable clinical condition over the last 3 months (i.e. No change in medication, treatments, or development of new symptoms)
Ability to perform exercise testing
Consent and assent (as appropriate to participate in the study after receiving information concerning procedures, risks, and possible clinical benefits) o Patients will be enrolled without regard to gender, race, and ethnicity. All patients meeting study eligibility will be approached for consent.
Exclusion Criteria:
Severe heart failure (New York Heart Association functional class III or IV)
Presence of liver or renal dysfunction based on the latest lab test results
Presence of hearing or visual deficit
Transcutaneous arterial blood oxygen saturation (SaO2) <80% at rest
History of echocardiographic, MRI, or angiographic evidence of Fontan pathway obstruction
History of exercise-induced life-threatening arrhythmias or unstable rhythm(i.e. atrial flutter)
Known or suspected pregnancy. Females in the reproductive age group will be screened for pregnancy using serum beta hCG prior to administering study drug.
Patients on open label sildenafil or tadalafil
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Shaji C. Menon, MD
Organizational Affiliation
University of Utah
Official's Role
Principal Investigator
Facility Information:
Facility Name
Primary Childrens Medical Center
City
Salt lake City
State/Province
Utah
ZIP/Postal Code
84113
Country
United States
12. IPD Sharing Statement
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Effect of Tadalafil on Exercise Capacity in Pediatric Fontan Patients
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