Effect of Tadalafil on Exercise Capacity in Pediatric Fontan Patients

NCT01291069 | Completed Phase 1 DMC

• 1 other identifier: IRB_00050085
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

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

Keywords

Fontan; Tadalafil; Exercise performance

Outcome Measures

Primary Outcomes (1)

• 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

  2 years

Secondary Outcomes (1)

• To evaluate the adverse effects of Tdalafil in patients (8-35 years)with the Fontan circulation and compare with the placebo group.

  2 years

Study Arms (2)

Tadalafil Citrate

EXPERIMENTAL

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.

Drug: Tadalafil Citrate

Sugar pill

PLACEBO COMPARATOR

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.

Drug: Sugar pill

Interventions

Tadalafil Citrate 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.

Also known as: Cialis

Tadalafil Citrate

Sugar pill 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

Also known as: Ora sweet

Sugar pill

Eligibility Criteria

• Age: 8 Years - 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Utah: lead

Study Sites (1)

Primary Childrens Medical Center

Salt Lake City, Utah, 84113, United States

Location

MeSH Terms

Conditions

Univentricular Heart

Interventions

Tadalafil; Sugars

Condition Hierarchy (Ancestors)

Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Carbolines; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Indole Alkaloids; Indoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds, 3-Ring; Carbohydrates

Study Officials

• Shaji C. Menon, MD

  University of Utah

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: February 4, 2011
• First Posted: February 7, 2011
• Study Start: September 1, 2011
• Primary Completion: February 1, 2014
• Study Completion: February 1, 2014
• Last Updated: April 17, 2017

Record last verified: 2017-04

Locations

US (1)