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A Multicenter, Single-arm, Open-label, Phase 3b Study to Assess the Effects of Switching From Flolan® to ACT-385781A in Patients with Pulmonary Arterial Hypertension - EPITOME-2

Conditions
Pulmonary Arterial Hypertension
MedDRA version: 12.1Level: LLTClassification code 10064911Term: Pulmonary arterial hypertension
Registration Number
EUCTR2010-018322-40-NL
Lead Sponsor
ACTELION Pharmaceuticals Ltd
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
ot Recruiting
Sex
All
Target Recruitment
35
Inclusion Criteria

1. Male or female aged 18 years and above
2. Patients with the following types of pulmonary arterial hypertension (PAH) belonging to WHO Group I:
•Idiopathic (IPAH)
•Heritable (HPAH)
•Associated (APAH) with
o Connective tissue diseases
o Drugs and toxins
3. Patients treated with Flolan® for at least 12 months and on a stable dose for at least 3 months prior to enrollment
4. Patients who are currently treated with concomitant PAH therapy listed below must have been treated for at least 90 days and on a stable dose for 30 days prior to enrollment:
• Bosentan
• Ambrisentan
• Sitaxsentan
• Sildenafil
• Tadalafil
5. Women of childbearing potential must use a reliable method of contraception
6. Signed informed consent prior to initiation of any study mandated procedure

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Patients with respiratory and/or cardiovascular distress in need of emergency care
2. Known or suspicion of pulmonary veno-occlusive disease (PVOD)
3. Current use of IV inotropic agents
4. Current use of any prostacyclin or prostacyclin analog other than Flolan®
5. Tachycardia with heart rate > 120 beats/min at rest
6. PAH related to any condition other than those specified in the inclusion criteria
7. Known hypersensitivity to the formulations of EFI or any of its excipients, and Flolan® or any of its excipients
8. Cerebrovascular events (e.g., transient ischemic attack or stroke) within 6 months of screening
9. History of myocardial infarction
10. History of left-sided heart disease, including any of the following:
• hemodynamically significant aortic or mitral valve disease
• restrictive or congestive cardiomyopathy
• left ventricular ejection fraction < 40% by multigated radionucleotide angiogram (MUGA), angiography, or echocardiography
• unstable angina pectoris
• life-threatening cardiac arrhythmias
11. Chronic bleeding disorders
12. Central venous line infection within 90 days prior to screening and/or a history of recurring line infections
13. Women who are pregnant or breast-feeding
14. Participation in another clinical trial, except observational, or receipt of an investigational product within 30 days prior to enrollment
15. Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results such as drug or alcohol dependence or psychiatric disease
16. Known concomitant life-threatening disease other than PAH with a life expectancy < 12 months

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Main Objective: • To evaluate the change in cardiac hemodynamics from baseline to 3-month following switch from Flolan® to EFI in patients with pulmonary arterial hypertension (PAH).<br>• To evaluate the safety and tolerability of switching from Flolan® to EFI in patients with PAH.<br><br>;Secondary Objective: Not Applicable;Primary end point(s): Tolerability / Safety endpoints:<br>• Treatment-emergent adverse events (AEs) up to 24 hours post-EOT<br>• Change from baseline to EOT in vital signs [heart rate (HR) and blood pressure (BP)] and body weight<br>• AEs leading to premature discontinuation of study drug <br>• Treatment-emergent serious AEs (SAEs) up to 30 days post-EOT<br><br><br>Efficacy endpoint:<br>• Change from baseline to EOT in cardiac hemodynamics including:<br>o Pulmonary vascular resistance (PVR)<br>o Mean pulmonary arterial pressure (mPAP)<br>o Right atrial pressure (RAP)<br>o Pulmonary artery occlusion pressure (PAOP)<br>o Cardiac index (CI)<br><br><br>
Secondary Outcome Measures
NameTimeMethod
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