Clinical trial NCT01431716: Epoprostenol for Injection (EFI/ACT-385781A) - Pulmonary Arterial Hypertension
Official title: A Multicenter, Single-arm, Open-label, Phase 3b Study to Assess the Effects of Switching From Flolan® to EFI/ACT-385781A in Patients With Pulmonary Arterial Hypertension
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Pulmonary Arterial Hypertension
Lead sponsor: Actelion
Sponsor class: INDUSTRY
First posted: 2011-09-12
Enrollment: 42
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study is investigating the effect of switching from Flolan® to Epoprostenol for Injection (EFI/ACT-385781A) in pulmonary arterial hypertension patients currently treated with Flolan®. For this purpose patients being treated for at least 12 months with Flolan® will be switched from Flolan® to EFI/ACT-385781A and followed-up for 90 days. During these 90 days safety and tolerability of EFI/ACT-385781A will closely be monitored in all treated patients. This 90 follow-up will provide clinical evidence on the safety, tolerability, efficacy and treatment satisfaction of switching from Flolan® to EFI/ACT-385781A in patients with pulmonary arterial hypertension.


Eligibility criteria: 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 Connective tissue diseases or 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

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 EFI/ACT-385781A 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 randomization
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

Primary outcomes:
measure: Change in Pulmonary Vascular Resistance From Baseline to End of Treatment (EOT).
timeFrame: Approximately 3 months
measure: Change in Total Pulmonary Resistance From Baseline to End of Treatment (EOT).
timeFrame: Approximately 3 months
measure: Change in Mean Pulmonary Arterial Pressure From Baseline to End of Treatment (EOT).
timeFrame: Approximately 3 months
measure: Change in Mean Right Atrial Pressure From Baseline to End of Treatment (EOT).
timeFrame: Approximately 3 months
measure: Change in Pulmonary Capillary Wedge Pressure From Baseline to End of Treatment (EOT).
timeFrame: Approximately 3 months
measure: Change in Mean Cardiac Index From Baseline to End of Treatment (EOT).
timeFrame: Approximately 3 months
Secondary outcomes:
Other outcomes:
measure: Change in 6-minute Walk Distance (6MWD) From Baseline to EOT.
timeFrame: Approximately 3 months
measure: Change in Borg Dyspnea Score From Baseline to EOT.
timeFrame: Approximately 3 months
measure: Number of Participants With Improved, No Change, or Worsening of New York Heart Association Functional Class (NYHA FC) From Baseline to EOT.
timeFrame: Approximately 3 months
measure: Change in N-terminal Pro-B-type Natriuretic Peptide (NT proBNP) From Baseline to EOT.
timeFrame: Approximately 3 months
measure: Change in Effectiveness Score of the Abbreviated Treatment Satisfaction Questionnaire for Medication (TSQM-9) From Baseline to EOT.
timeFrame: Approximately 3 months
measure: Change in Convenience Score of the Abbreviated Treatment Satisfaction Questionnaire for Medication (TSQM-9) From Baseline to EOT.
timeFrame: Approximately 3 months
measure: Change in Global Satisfaction Score of the Abbreviated Treatment Satisfaction Questionnaire for Medication (TSQM-9) From Baseline to EOT.
timeFrame: Approximately 3 months
measure: Number of Participants With Adverse Events Leading to Discontinuation of Study Drug From Baseline to EOT.
timeFrame: Approximately 3 months
Locations:
city: Leuven
country: Belgium
facility: UZ Gasthuisberg
city: Toronto
country: Canada
facility: University Health Network, Toronto TGH - 10 EN - 220
state: Ontario
city: Montreal
country: Canada
facility: Sir Mortimer B Davis Jewish General Hospital
state: Quebec
city: Caen
country: France
facility: Centre Hospitalier Universitaire
city: Clamart Cedex
country: France
facility: Hôpital Antoine Béclère
city: Bologna
country: Italy
facility: Orsola Malpighi
city: Amsterdam
country: Netherlands
facility: VU Medisch Centrum (VUMC)
city: Barcelona
country: Spain
facility: Hospital Vall d'Hebron
Study officials:
References: