NCT01743001已完成3 期
A Multi-center, Double-blind, Randomized, Placebo-controlled, Parallel-group, Phase 3 Study to Evaluate the Effects of Macitentan on Exercise Capacity in Subjects With Eisenmenger Syndrome
Actelion55 个研究点 分布在 18 个国家目标入组 226 人开始时间: 2013年5月21日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 226
- 试验地点
- 55
- 主要终点
- Change From Baseline to Week 16 in Exercise Capacity, as Measured by 6-minute Walk Distance (6MWD)
研究概览
简要总结
Clinical study to assess the efficacy, safety, and tolerability of macitentan in subjects with Eisenmenger Syndrome.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects:
- •not participating in the hemodynamic sub-study: males or females ≥ 12 years of age.
- •participating in the hemodynamic sub-study: males or females ≥ 18 years of age.
- •Subjects (including those with Down Syndrome [DS]) with confirmed Eisenmenger Syndrome [ES] (European Society of Cardiology [ESC] and the European Respiratory Society [ERS] guidelines):
- •Established by echocardiography as:
- •Large congenital shunting defect at atrial, ventricular or arterial level*
- •and right to left shunt or bi-directional shunt with prevalent right to left direction.
- •Resting peripheral oxygen saturation (SpO2) ≤ 90% and > 70% (pulse oximetry, room air).
- •The lower limit is 65% if a subject is living at an altitude greater than 2500 m above sea level.
- •*Subjects with any of the following open defects are eligible for the study either as an isolated defect or in combination:
- •atrial septal defect (ASD)
- •ventricular septal defect (VSD)
- •partial or complete atrioventricular septal defect (AVSD)
- •patent ductus arteriosus (PDA)
- •aortopulmonary window (AP window)
- •total or partial anomalous pulmonary venous return (TAPVR, PAPVR) The defects may be either unoperated or previously palliated surgically (provided significant residual defect remains).
- •The Steering Committee will review the echocardiography data of all subjects (main study and sub study) to confirm eligibility prior to Randomization.
- •Subjects with the following findings at cardiac catheterization:
- •Mean resting pulmonary arterial pressure (mPAP) > 25 mmHg
- •Pulmonary capillary wedge pressure (PCWP) or mean left atrial pressure (LAP) or left ventricular end diastolic pressure (LVED) ≤ 15 mmHg
- •Pulmonary vascular resistance (PVR) ≥ 800 dyn∙s/cm5 or ≥ 10 Wood units
- •Subjects with WHO functional class ≥ II.
- •Subjects able to reliably perform the the 6-minute walk test (6MWT) with a minimum distance of 50 m and a maximum distance of 450 m.
排除标准
- •Main study and hemodynamic sub-study: Any of the following conditions previously known or identified via cardiac catheterization or echocardiography:
- •Pulmonary arterial or venous stenosis > 25% size of native pulmonary artery (PA) or pulmonary vein
- •Severe tricuspid regurgitation in the setting of left to right shunt at the ventricular or atrial level
- •Greater than mild tricuspid stenosis
- •Intracavitary RV outflow obstruction
- •Greater than mild mitral stenosis
- •Intracavitary LV outflow obstruction
- •Subvalvular or supravalvular aortic stenosis
- •Aortic coarctation
- •Greater than moderate mitral regurgitation
- •Recognized extracardiac systemic venous collaterals to the pulmonary venous circulation
- •Recognized hepatic wedge pressure-inferior vena cava pressure gradient >12 mm Hg
- •PCWP "v" waves >20 mmHg
- •Tetralogy of Fallot
- •Truncus arteriosus
- •Interrupted aortic arch
- •Transposition of great arteries
- •Single ventricle defects: absent AV connection (mitral or tricuspid atresia), double inlet AV connections left or right ventricle, functional univentricular heart (unbalanced AVSD, hypoplastic RV, double outlet RV), hypoplastic left heart syndrome
- •Ebstein's anomaly
- •Severe aortic regurgitation
- •Pulmonary atresia
- •PAPVR or TAPVR, ONLY if there is lung hypoplasia or if documentation confirming the absence of lung hypoplasia does not exist.
- •For subjects participating in the hemodynamic sub-study the following will also be considered exclusion criteria:
- •SVC stenosis >25% size of native vessel
- •PDA, AP window, TAPVR, PAPVR, or ASD sinus venosus with anomalous pulmonary veins
- •Down Syndrome
- •Subjects with deterioration of their clinical status within 3 months prior to Screening or during the Screening period.
- •Known moderate-to-severe restrictive (i.e., total lung capacity [TLC] < 60% of predicted value) or obstructive lung disease (i.e., forced expiratory volume in one second [FEV1] < 80 % of predicted value, and with FEV1 / forced vital capacity [FVC] < 70%)
- •Treatment with prostanoids within 1 month prior to Randomization
- •Subjects who initiated a PDE-5 inhibitor within 1 month prior to Randomization or those on a PDE-5 inhibitor for whom the dose has not been stable within 1 month prior to Randomization
- •Treatment with endothelin receptor antagonists (ERAs) within 1 month prior to Randomization
- •Subjects who initiated diuretics within 1 week prior to Randomization or subjects whose diuretic treatment has not been stable for at least 1 week prior to Randomization
- •Subjects being considered for an organ transplant
研究组 & 干预措施
Macitentan
Experimental
Subjects receive macitentan 10 mg oral tablet once daily
干预措施: Macitentan 10 mg (Drug)
Placebo
Placebo Comparator
Subjects receive macitentan-matching placebo oral tablet once daily
干预措施: Placebo (Drug)
结局指标
主要结局
Change From Baseline to Week 16 in Exercise Capacity, as Measured by 6-minute Walk Distance (6MWD)
时间窗: From baseline to Week 16
The purpose of the six minute walk is to test exercise tolerance and capacity. The test measures the distance an individual is able to walk over a total of six minutes on a hard, flat surface. The goal is for the individual to walk as far as possible in six minutes.
次要结局
- Change From Baseline to Week 16 in Quality of Life (QoL), Assessed by the Short Form-36 (SF-36) Questionnaire(From baseline to Week 16)
- Change From Baseline to Week 16 in Dyspnea, Assessed by the Borg Dyspnea Index(From baseline to Week 16)
- Change From Baseline to Week 16 in WHO Functional Class(From baseline to Week 16)
研究者
研究点 (55)
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