A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of JTT-251 Administered for 24 Weeks to Participants With Pulmonary Arterial Hypertension (RELIEF-PAH)
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 主要终点
- Change in pulmonary vascular resistance (PVR) compared to baseline
研究概览
简要总结
Study to evaluate the efficacy, safety, tolerability and pharmacokinetics of JTT-251 administered for 24 weeks in participants with pulmonary arterial hypertension (PAH)
详细描述
This is a study to evaluate the efficacy, safety, tolerability and pharmacokinetics of JTT-251 administered for 24 weeks in participants with pulmonary arterial hypertension (PAH). Participants completing this study (RELIEF-PAH) will be eligible to enroll in an open-label extension study (RELIEF-PAH OLE) to evaluate the long-term efficacy, safety, tolerability and pharmacokinetics of JTT-251 in participants with PAH.
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 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of PAH as classified by idiopathic, heritable, drug and toxin- induced, congenital heart disease or associated with connective tissue disease (i.e., WHO Group 1)
- •Clinical diagnosis of PAH confirmed by RHC at any time prior to Visit 1
- •WHO functional status of Class II-IV at Visit 1
- •Two 6MWD test measurements between 100 and 450 meters with a relative difference of ≤15%. The baseline 6MWD test must be performed at Visit 2 before randomization.
- •Have a qualifying RHC performed between Visit 1 and Visit 2
- •On stable dose(s) of guideline-directed medical therapy for PAH (endothelin receptor antagonists, phosphodiesterase type-5 (PDE-5) inhibitors, soluble guanylate cyclase stimulators and prostacyclin pathway analogs) for at least 90 days prior to the qualifying RHC
排除标准
- •PAH associated with portal hypertension, human immunodeficiency virus (HIV), schistosomiasis or sickle cell disease as well as participants with pulmonary parenchymal disease or thromboembolic disease
- •Known significant left heart disease including: left ventricular dysfunction (i.e., left ventricular ejection fraction <35%); hemodynamically compromising, symptomatic, or severe aortic stenosis, aortic regurgitation, mitral stenosis, mitral regurgitation
- •Pulmonary hypertension belonging to WHO groups 2 to 5
- •Moderate to severe obstructive lung disease defined as forced expiratory volume in 1 second (FEV1) <55% of predicted value
- •Moderate to severe restrictive lung disease defined as total lung capacity (TLC) <60% of predicted value
- •Acute decompensated heart failure or hospital admission for worsening PAH symptoms within 30 days prior to the qualifying RHC
研究组 & 干预措施
JTT-251 Dose 1
One dose of study drug by mouth daily for 24 weeks
干预措施: JTT-251 (Drug)
JTT-251 Dose 2
One dose of study drug by mouth daily for 24 weeks
干预措施: JTT-251 (Drug)
JTT-251 Dose 3
One dose of study drug by mouth daily for 24 weeks
干预措施: JTT-251 (Drug)
Placebo
One dose of study drug by mouth daily for 24 weeks
干预措施: Placebo (Drug)
结局指标
主要结局
Change in pulmonary vascular resistance (PVR) compared to baseline
时间窗: 4, 12, 24 and 28 Weeks
Assessed by right heart catheterization (RHC)
Change in six-minute walk distance (6MWD) compared to baseline
时间窗: 24 Weeks
Change in World Health Organization (WHO) functional classification compared to baseline
时间窗: 24 Weeks
次要结局
- JTT-251 trough plasma concentrations(4, 12 and 24 Weeks)
- Number of adverse events(28 Weeks)