A 16-Week, International, Multicenter, Double-Blind, Randomized, Placebo-Controlled Comparison of the Efficacy and Safety of Oral UT-15C Sustained Release Tablets in Combination With an Endothelin Receptor Antagonist and/or a Phosphodiesterase-5 Inhibitor in Subjects With Pulmonary Arterial Hypertension
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 354
- 试验地点
- 72
- 主要终点
- Six Minute Walk Distance (6MWD)
研究概览
简要总结
This study was an international, multi-center, randomized, double-blind, placebo-controlled study in subjects with PAH who were currently receiving approved therapy for their PAH (i.e., endothelin receptor antagonist and/or phosphodiesterase-5 inhibitor). Study visits occurred at 4 week intervals for 16 weeks; the key measure of efficacy was the 6-minute walk test. Study procedures included routine blood tests, medical history, physical exams, disease evaluation, and exercise tests. One optional substudy was also a part of FREEDOM-C at select centers - a hemodynamic substudy with a right heart catheterization at Baseline and Week 16.
Patients who completed all assessments for 16-weeks were also eligible to enter an open-label, extension phase study (FREEDOM - EXT).
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 70 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Between 12 and 70 years of age, inclusive.
- •Body weight at least 45 kg (approximately 100 pounds).
- •PAH that is either idiopathic/heritable (including PAH associated with appetite suppressant/toxin use); PAH associated with repaired congenital systemic-to-pulmonary shunts (repaired ≥ 5 years); PAH associated with collagen vascular disease; or PAH associated with HIV.
- •Baseline 6-minute walk distance between 150 and 450 meters, inclusive.
- •Currently receiving an approved endothelin receptor antagonist and/or an approved phosphodiesterase-5 inhibitor for at least 90 days and on a stable dose for at least the last 30 days.
- •Previous testing (e.g., right heart catheterization, echocardiography) consistent with the diagnosis of PAH.
- •Reliable and cooperative with protocol requirements.
排除标准
- •Nursing or pregnant.
- •Received a prostacyclin within the past 30 days.
- •PAH due to conditions other than noted in the above inclusion criteria.
- •History of uncontrolled sleep apnea, renal insufficiency, anemia, left sided heart disease, uncontrolled systemic hypertension, or parenchymal lung disease.
- •Use of an investigational drug within 30 days of Baseline.
研究组 & 干预措施
Active
Subjects assigned to active therapy with UT-15C 0.25, 0.5, 1, or 5 mg oral tablets.
干预措施: Oral treprostinil (UT-15C) sustained release tablets (Drug)
Placebo Arm
Subjects assigned to placebo 0.25, 0.5, 1, or 5 mg oral tablets.
干预措施: Placebo (Drug)
结局指标
主要结局
Six Minute Walk Distance (6MWD)
时间窗: Baseline and 16 Weeks
Placebo corrected change in six minute walk distance (6MWD) from Baseline to Week 16, correlates with the historical clinical standard for assessing patient functional status in the treatment of PAH and is considered an objective measure of patient functional status by the American Thoracic Society (ATS). The six minute walk test was to be conducted 3 to 6 hours after the previous dose of study drug.
次要结局
- Borg Dyspnea Score(Baseline and 16 Weeks)
- Clinical Worsening Assessment(Baseline and 16 Weeks)
- Dyspnea-Fatigue Index(Baseline and 16 Weeks)
- World Health Organization Functional Classification for PAH(Week 16)
- Six Minute Walk Distance (6MWD)(Baseline and 4 weeks)
- Change in Symptoms of PAH From Baseline to Week 16(Baseline and 16 weeks)