Single Ventricle
Conditions
Keywords
Fontan, Tadalafil, Exercise performance
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.
Interventions
DRUGTadalafil Citrate
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.
DRUGSugar pill
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
Sponsors
University of Utah
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
ALL
Age
8 Years to 35 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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). | 2 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
| Measure | Time frame | Description |
|---|---|---|
| To evaluate the adverse effects of Tdalafil in patients (8-35 years)with the Fontan circulation and compare with the placebo group. | 2 years | 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. |
Countries
United States
Outcome results
None listed