FREEDOM DR: An International, Multi-Center, 12-Week, Double Blind, Randomized, Placebo-Controlled, Parallel Group Study Followed by a 12-Week Open-Label Extension Study to Assess the Efficacy, Safety, and Dose Response of UT-15C SR in Subjects With Pulmonary Hypertension
试验速览
- 阶段
- 3 期
- 状态
- 撤回
- 发起方
- 试验地点
- 31
- 主要终点
- Change in six-minute walk distance from Baseline to Week 12
研究概览
简要总结
This study is an international, multi-center, randomized, double-blind, placebo-controlled study in subjects with PAH who are currently receiving approved therapy for their PAH (i.e., endothelin receptor antagonist and/or phosphodiesterase-5 inhibitor)or as a monotherapy treatment. Study visits will occur at 4 week intervals for 12 weeks with the key measure of efficacy being the 6-minute walk test. Study procedures include routine blood tests, medical history, physical exams, disease evaluation, and exercise tests. At the end of the first 12-weeks, the patient will be un-blinded. Patients will continue with another 12-Week open label portion with visits occuring at 4-week intervals.
Patients who complete all assessments for 24-weeks will also be eligible to enter a 36 month open-label, extension phase study (FREEDOM - EXT).
详细描述
This study is an international, multi-center, randomized, double-blind, placebo-controlled study in subjects with PAH who are currently receiving approved therapy for their PAH (i.e., endothelin receptor antagonist and/or phosphodiesterase-5 inhibitor)or as a monotherapy treatment. Study visits will occur at 4 week intervals for 12 weeks with the key measure of efficacy being the 6-minute walk test. Study procedures include routine blood tests, medical history, physical exams, disease evaluation, and exercise tests. At the end of the first 12-weeks, the patient will be un-blinded. Patients will continue with another 12-Week open label portion with visits occuring at 4-week intervals.
Patients who complete all assessments for 24-weeks will also be eligible to enter a 36 month 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
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Between 18 and 70 years of age, inclusive
- •Body weight at least 50 kilograms
- •PAH that is either idiopathic/familial; associated with repaired congenital systemic-to-pulmonary shunts (repaired ≥ 5 years); associated with collagen vascular disease; associated with HIV.
- •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 or not currently receiving approved PAH therapy.
- •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
Placebo
placebo
干预措施: Placebo (Drug)
UT-15C 0.25 mg
UT-15C 0.25 mg
干预措施: UT-15C 0.25 mg (Drug)
UT-15C 1 mg
UT-15C 1 mg
干预措施: UT-15C 1 mg (Drug)
UT-15C 5 mg
UT-15C 5 mg
干预措施: UT-15C 5 mg (Drug)
结局指标
主要结局
Change in six-minute walk distance from Baseline to Week 12
时间窗: 12 weeks
次要结局
- Trough 6-Minute Walk Distance(12 weeks)
- Trough Borg Dyspnea Score(12 weeks)
- Borg Dyspnea Score(12 weeks)
- Clinical Worsening Assessment(12 weeks)
- Dyspnea Fatigue Index(12 weeks)
- Symptoms of PAH(12 weeks)
- World Health Organization (WHO) Functional Class(12 weeks)
- Pro-B-type natriuretic peptide (Pro-BNP)(12 weeks)
- Optional hemodynamic parameters(12 weeks)
- Adverse events(12 weeks)
- Clinical Laboratory parameters(12 weeks)
- Electrocardiogram findings(12 weeks)