Effect of Tadalafil on Exercise Capacity in Pediatric Fontan Patients

Utah System of Higher Education (USHE)

Status and phase: Completed; Phase 1

Conditions: Single Ventricle

Treatments: Drug: Tadalafil Citrate; Drug: Sugar pill

Study type: Interventional

Funder types: Other

Identifiers

NCT01291069

IRB_00050085

Details and patient eligibility

About

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.

Enrollment: 20 patients

Sex: All

Ages: 8 to 35 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Triple Blind

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20 participants in 2 patient groups, including a placebo group

Tadalafil Citrate

Experimental group

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.

Treatment:

Drug: Tadalafil Citrate

Sugar pill

Placebo Comparator group

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.

Treatment:

Drug: Sugar pill