A Single-arm, Open Label Study Evaluating the Impact on Lifestyle of a New Thermo Stable Formulation of FLOLAN® in Subjects With Pulmonary Arterial Hypertension (PAH). (FLOLAN® is a Registered Trademark of the GlaxoSmithKline Group of Companies.)
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 16
- 试验地点
- 1
- 主要终点
- Change From Baseline in Medical Outcomes Study Short Form 36 (SF-36)
研究概览
简要总结
The purpose of this multicentre, open label, single-arm study in approximately 20 adult patients is to evaluate the Impact on lifestyle of a new thermo stable formulation of epoprostenol sodium in subjects with Pulmonary Arterial Hypertension (PAH).
详细描述
This is a multicentre, open label, single-arm study in approximately 20 adult patients (18 - 75 years old) designed to evaluate the Impact on lifestyle of a new thermo stable formulation of epoprostenol sodium in subjects with Pulmonary Arterial Hypertension (PAH). The co-primary objectives are 1) to describe the effect of the new thermo stable formulation of epoprostenol sodium on quality of life and 2) to determine the dose titration requirement in patients switching from the currently marketed FLOLAN (epoprostenol sodium) to the new thermo stable formulation. Secondary objectives include assessing the safety, tolerability and efficacy of the thermo stable formulation of epoprostenol sodium and the exploratory objective is to evaluate the effect of the new thermo stable formulation of epoprostenol sodium on haemodynamic parameters in a subset of subjects.
Subjects who are already receiving FLOLAN (epoprostenol sodium) for the treatment of PAH and have been on a stable dose for at least 3 months and on stable doses of other PAH treatments for at least 30 days prior to screening will be enrolled. After a screening visit, eligible subjects will have a 4-week run-in period with their existing FLOLAN (epoprostenol sodium) treatment. At the end of the 4-week period, they will be admitted to the clinic for baseline assessments and for switching to study medication (the new thermo stable formulation of epoprostenol sodium). Subjects will remain in hospital for a minimum of 6 hours to ensure clinical and hemodynamic stability prior to discharge. Subjects may stay in hospital for up to 24-48 hours after switching to the new thermo stable formulation of epoprostenol sodium at the discretion of the investigator. Dose titration requirement will be assessed at the time of discharge. Haemodynamic parameters will be obtained in a subgroup of subjects enrolled in centres where the collection of haemodynamic data is considered part of the standard of care. Subjects will receive the study medication as a continuous intravenous infusion for a 4-week treatment period. Those who complete the 4-week treatment period will have the option of entering an extension phase of the study to continue receiving the new formulation.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult male or female at least 18 to 75 years at the time of screening.
- •Subjects must have been on FLOLAN (epoprostenol sodium) therapy for pulmonary arterial hypertension (PAH) as approved in the product label.
- •Subjects must be on stable doses of their existing FLOLAN (epoprostenol sodium) treatment for a minimum of 3 months prior to screening.
- •Subjects must be on stable doses of any current PAH treatments other than FLOLAN (epoprostenol sodium) in the last 30 days.
- •Subjects must walk a distance of at least 150 meters during six-minute walk distance test (6MWD). This test must be completed during the Screening Visit.
- •A female subject is eligible to participate if she is of non-childbearing potential or of childbearing potential, has a negative pregnancy test at screen, and agrees to use one of the contraception methods listed in the protocol.
- •Subjects must be competent to understand the information given in the Institutional Review Board (IRB) or Independent Ethics Committee (IEC) approved informed consent form and must sign the form prior to the initiation of any study procedures.
排除标准
- •Subjects who are given FLOLAN (epoprostenol sodium) for a condition or in a manner that is outside the approved indication.
- •Subjects with congestive heart failure arising from severe left ventricular dysfunction.
- •Subjects, with or without supplemental oxygen, who have a resting arterial oxygen saturation (SaO2) <90% as measured by pulse oximetry at screening.
- •Subjects have been hospitalized as an emergency or visited the emergency room for a condition related to PAH or treatment for PAH in the last 3 months.
- •The subject's clinical condition is such that they are not expected to remain clinically stable for the duration of the study.
- •Female subjects who are pregnant or breastfeeding.
- •Subjects who have demonstrated noncompliance with previous medical regimens.
- •Subjects who have a history of abusing alcohol or illicit drugs within 1 year.
- •Subjects with a diagnosis of active hepatitis (hepatitis B surface antibody and hepatitis C antibody).
- •Subjects who have participated in a clinical study involving another investigational drug or device within four weeks before screening.
- •Subjects who had history malignancies within the past 5 years, with the exception of basal cell carcinoma of the skin or in situ carcinoma of the cervix.
- •Any concurrent condition that would affect the safety of the subject or in the opinion of the investigator it is not in the best interest of the patient to participate in the study.
研究组 & 干预措施
All subjects
Subjects enter the study already taking current marketed FLOLAN (epoprostenol sodium) and continue for 4 weeks (run-in). At baseline, they will be swopped to the new thermo stable formulation of epoprostenol sodium for 4 weeks (or longer if they continue in the extension phase of the study).
干预措施: current marketed FLOLAN (epoprostenol sodium) (Drug)
All subjects
Subjects enter the study already taking current marketed FLOLAN (epoprostenol sodium) and continue for 4 weeks (run-in). At baseline, they will be swopped to the new thermo stable formulation of epoprostenol sodium for 4 weeks (or longer if they continue in the extension phase of the study).
干预措施: new thermo stable formulation of epoprostenol sodium (Drug)
结局指标
主要结局
Change From Baseline in Medical Outcomes Study Short Form 36 (SF-36)
时间窗: Baseline (Visit 2 i.e. Day-14 [+ or - 7 days]) and Week 4 (Visit 3)
Subject-rated measure of health status comprised of 36 items: 8 subscale scores (physical functioning, role limitations due to physical health problems, bodily pain, general health, vitality, social functioning, role limitations due to emotional problems, and mental health), 2 summary scores (physical component and mental component), and a self-evaluated change in health status. Subscale and summary scores range: 0-100. Higher subscale and summary scores was considered as better health status. Change from Baseline was calculated as score at observation minus score at baseline. Baseline was defined as Visit 2 i.e. Day-14 (+ or - 7 days).
Change From Baseline in Study Specific Participant Acceptance Survey
时间窗: Baseline (Visit 2 i.e. Day-14 [+ or - 7 days]) and Week 4 (Visit 3).
Study-specific questionnaire comprised the pre-defined15 questions which included activities of daily living assessment. Participants rated the question on a scale of 1 to 10, where 1 was do not agree and 10 was strongly agree. Change from Baseline was calculated as score at observation minus score at Baseline. Changes from Baseline was assessed for Questions 2 to 12. Baseline was defined as Visit 2 i.e. Day-14 (+ or - 7 days).
Change From Baseline in Dose of Thermo Stable Epoprostenol Sodium at Week 4
时间窗: Baseline (Visit 2 i.e. Day-14 [+ or - 7 days]) and Week 4
Dose titration requirement was assessed at the time of discharge. Change from Baseline was calculated as score at observation minus score at Baseline. Units- nanogram per kilogram per minute (ng/kg/min). Baseline was Visit 2 i.e . Day-14 (+ or - 7 days).
次要结局
- Number of Participants With Any Treatment Emergent Adverse Events (AEs) and Treatment Emergent Serious Adverse Events(SAEs)(Up to visit 3 (Week 4))
- Number of Participants With Infusion Site Reactions During Treatment Period(Baseline visit (Visit 2) to Week 4 (Visit 3))
- Change From Baseline in Vital Signs at Week 4 : Systolic and Diastolic Blood Pressure(Baseline (Visit 2 i.e. Day-14 [+ or - 7 days]) and Week 4(Visit 3))
- Change From Baseline in Vital Signs at Week 4: Heart Rate(Baseline (Visit 2 i.e. Day-14 [+ or - 7 days]) to Week 4)
- Number of Participants With Abnormal Clinical Chemistry(Up to 1 week after Week 4 (Follow-up))
- Number of Participants With Abnormal Hematology(Up to 1 week after Week 4 (Follow-up))
- Number of Participants With Abnormal Urinalysis(Up to 1 week after Week 4 (Follow-up))
- Change From Baseline in Six Minute Walk Distance Test (6MWD) After 4-weeks of Treatment(Baseline (Visit 2 i.e. Day-14 [+ or - 7 days]) and Week 4)
- Breathlessness After 6MWD - Borg Dyspnoea Index (BDI)(Baseline (Visit 2 i.e. Day-14 [+ or - 7 days]) to Week 4)
- World Health Organization [WHO] Functional Class at Baseline and After 4- Weeks of Treatment(Baseline (Visit 2 i.e. Day-14 [+ or - 7 days]) and Week 4)
- Mean Oxygen Saturation in Blood Over Time(up to the treatment follow up (1 week after Visit 3 [Week 4]))
- Number of Participants With Urine Pregnancy Test Positive(up to the treatment follow up (1 week after Visit 3 [Week 4]))