Effect of Tadalafil on Exercise Capacity in Pediatric Fontan Patients

NCT ID: NCT01291069

Last Updated: 2017-04-17

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-09-30
• Study Completion Date: 2014-02-28

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.

Conditions

Single Ventricle

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

Tadalafil Citrate

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.

Group Type: EXPERIMENTAL

Tadalafil Citrate

Intervention Type: DRUG

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.

Sugar pill

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.

Group Type: PLACEBO_COMPARATOR

Sugar pill

Intervention Type: DRUG

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

Interventions

Tadalafil 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.

Intervention Type: DRUG

Sugar 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

Intervention Type: DRUG

Other Intervention Names

Cialis; Ora sweet

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

• Minimum Eligible Age: 8 Years
• Maximum Eligible Age: 35 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of Utah

OTHER

Sponsor Role: lead

Responsible Party

Shaji Menon

Assistant Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Shaji C. Menon, MD

Role: PRINCIPAL_INVESTIGATOR

University of Utah

Locations

Primary Childrens Medical Center

Salt Lake City, Utah, United States

Countries

United States

Other Identifiers

IRB_00050085

Identifier Type: -

Identifier Source: org_study_id