A Multi-center, Prospective Study to Observe the Safety, Tolerability, and Efficacy of Remodulin® in Patients With Pulmonary Arterial Hypertension
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- Excelsior
- 入组人数
- 20
- 主要终点
- Change from baseline in NT-proBNP
研究概览
简要总结
This is a multi-center, single-arm, prospective study to enroll patients diagnosed as pulmonary arterial hypertension. The objective in this study is to observe the efficacy and safety of treprostinil in subjects with pulmonary arterial hypertension.
详细描述
This is a multi-center, single-arm, prospective study to enroll patients diagnosed as pulmonary arterial hypertension. The expected study period will be 2 years. PAH patients, who met NHI criteria, to receive treprostinil subcutaneously/ intravenously, will be invited to join this study for observation. The dosage and administration schedule of study medication will be judged by investigators. If the eligibility criteria have been met and the subject have signed the informed consent, the subjects will be enrolled into this study. There will be 10 visits: Visit 1 (Screening visit, Week -2 ~ -1), Visit 2 (Week 1, initial of use treprostinil), Visit 3 to 9 (Week 13 to 85), Visit 10 (Week 97). The Day 1 in Week 1 should be the start day of treprostinil treatment. Subjects will be returned to the clinics every 3 months (i.e. 12 weeks) after Visit 2. The NT-proBNP level, WHO functional class, 6-minute walk distance, echocardiography, pulse oximetry, modified Borg Dyspnea Scale, SF-36 questionnaire, and dosage regimens of treprostinil (including dose adjustment) will be collected. For time to clinical worsening and death, it will be followed up during study period. All intercurrent events within 24 months will be recorded. Safety information, including vital signs, and physical exams will be checked. Adverse events of special interest (AESIs) will be reviewed every visit by using a predesignated AESI checklist.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with symptomatic idiopathic or heritable PAH classified as WHO functional class III and IV symptoms.
- •PAH patient, who met NHI criteria, will be scheduled to receive subcutaneous/intravenous treprostinil treatment.
- •Ability to adhere to the study visit schedule and understand and comply with all protocol requirements.
- •Will provide completed and signed written informed consents
排除标准
- •Active treatment with injectable prostanoids different from subcutaneous/intravenous treprostinil
结局指标
主要结局
Change from baseline in NT-proBNP
时间窗: Week 97
Calculate the \"N terminal pro B type natriuretic peptide\" change from baseline to Week 97
Change from baseline in WHO functional class
时间窗: Week 97
Calculate the World Health Organization functional class change from baseline to Week 97. The World Health Organization (WHO) functional class describes how severe a patient\'s pulmonary hypertension (PH) symptoms are. There are four different classes - I is the mildest and IV is the most severe form of PH.
次要结局
- Time to clinical worsening and death(96 weeks)
- Change from baseline in NT-proBNP(Week 13, 25, 37, 49, 61, 73, and 85)
- Change from baseline in 6-minute walk distance (6MWD)(Week 25, 49, 73, and 97)
- Trend of dose usage from initiation treatment to clinical worsening(96 weeks)
- Adverse events of special interest (AESIs)(96 weeks)
- Change from baseline in WHO functional class(Week 13, 25, 37, 49, 61, 73, and 85)
- Change from baseline in right atrium (RA) area(Week 25, 49, 73, and 97)
- Change from baseline in TAPSE/sPAP ratio.(Week 25, 49, 73, and 97)
- Change from baseline in Pericardial effusion.(Week 25, 49, 73, and 97)
- Change from baseline in modified Borg Dyspnea Scale(Week 25, 49, 73, and 97)
- Change from baseline in SF-36 questionnaire(Week 25, 49, 73, and 97)
- Intercurrent event(96 weeks)