Effect of Tadalafil on Exercise Capacity in Pediatric Fontan Patients
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- 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).
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 8 Years 至 35 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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
研究组 & 干预措施
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.
干预措施: Tadalafil Citrate (Drug)
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.
干预措施: Sugar pill (Drug)
结局指标
主要结局
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).
时间窗: 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
次要结局
- To evaluate the adverse effects of Tdalafil in patients (8-35 years)with the Fontan circulation and compare with the placebo group.(2 years)
研究者
Shaji Menon
Assistant Professor
University of Utah