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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| EFI/ACT-385781A | Experimental | EFI/ACT-385781 administered by continuous intravenous infusion via a central venous catheter using an ambulatory infusion pump |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| EFI/ACT-385781A | Drug |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in Pulmonary Vascular Resistance From Baseline to End of Treatment (EOT). | Right heart catheterization was performed for cardiac hemodynamic assessment at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. | Approximately 3 months |
| Change in Total Pulmonary Resistance From Baseline to End of Treatment (EOT). | Right heart catheterization was performed for cardiac hemodynamic assessment at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. | Approximately 3 months |
| Change in Mean Pulmonary Arterial Pressure From Baseline to End of Treatment (EOT). | Right heart catheterization was performed for cardiac hemodynamic assessment at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. | Approximately 3 months |
| Change in Mean Right Atrial Pressure From Baseline to End of Treatment (EOT). | Right heart catheterization was performed for cardiac hemodynamic assessment at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. | Approximately 3 months |
| Change in Pulmonary Capillary Wedge Pressure From Baseline to End of Treatment (EOT). | Right heart catheterization was performed for cardiac hemodynamic assessment at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. | Approximately 3 months |
| Change in Mean Cardiac Index From Baseline to End of Treatment (EOT). | Right heart catheterization was performed for cardiac hemodynamic assessment at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. |
Not provided
| Measure | Description | Time Frame |
|---|---|---|
| Change in 6-minute Walk Distance (6MWD) From Baseline to EOT. | The 6MWD was assessed at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. The 6-minute walk test is a non-encouraged test that measures the distance walked for the duration of 6 min. | Approximately 3 months |
| Change in Borg Dyspnea Score From Baseline to EOT. |
Inclusion Criteria:
Exclusion Criteria:
Patients with respiratory and/or cardiovascular distress in need of emergency care
Known or suspicion of pulmonary veno-occlusive disease (PVOD)
Current use of IV inotropic agents
Current use of any prostacyclin or prostacyclin analog other than Flolan®
Tachycardia with heart rate > 120 beats/min at rest
PAH related to any condition other than those specified in the inclusion criteria
Known hypersensitivity to the formulations EFI/ACT-385781A or any of its excipients, and Flolan® or any of its excipients
Cerebrovascular events (e.g., transient ischemic attack or stroke) within 6 months of screening
History of myocardial infarction
History of left-sided heart disease, including any of the following:
Chronic bleeding disorders
Central venous line infection within 90 days prior to screening and/or a history of recurring line infections
Women who are pregnant or breast-feeding
Participation in another clinical trial, except observational, or receipt of an investigational product within 30 days prior to randomization
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
Known concomitant life-threatening disease other than PAH with a life expectancy < 12 months
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UZ Gasthuisberg | Leuven | 3000 | Belgium | |||
| University Health Network, Toronto TGH - 10 EN - 220 |
Patients must have been treated with Flolan for at least 12 months and on a stable dose for at least 3 months prior to enrollment. There was a screening period of up to 14 days.
Patients were enrolled at eight centers in the European Union and Canada. First patient, first visit was 15 March 2011 and last patient, last visit was 2 February 2012.
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| ID | Title | Description |
|---|---|---|
| FG000 | Epoprostenol for Injection (EFI/ACT-385781A) | EFI/ACT-385781A administered by continuous intravenous infusion via a central venous catheter using an ambulatory infusion pump. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
All-treated set (One patient enrolled in the study but withdrew consent prior completing baseline assessment and receiving study drug).
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| ID | Title | Description |
|---|---|---|
| BG000 | EFI/ACT-385781A | EFI/ACT-385781A administered by continuous intravenous infusion via a central venous catheter using an ambulatory infusion pump. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change in Pulmonary Vascular Resistance From Baseline to End of Treatment (EOT). | Right heart catheterization was performed for cardiac hemodynamic assessment at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. | All treated set without imputation for missing values. Data was missing for 5 patients. | Posted | Mean | Standard Deviation | dyn/sec/cm^5 | Approximately 3 months |
|
|
All adverse events (AEs) that occurred from the start of study treatment until 24 h after the end of study treatment were recorded. In addition, all serious AEs that occurred up to 30 days after the end of study treatment were also recorded.
All treated set
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | EFI/ACT-385781A | EFI/ACT-385781A administered by continuous intravenous infusion via a central venous catheter using an ambulatory infusion pump. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diverticulitis | Infections and infestations | MedDRA Version 14.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| HEADACHE | Nervous system disorders | MedDRA Version 14.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Thomas Pfister, PhD, Senior Clinical Scientist | Actelion Pharmaceuticals Ltd | +41 61 565 5932 | thomas.pfister@actelion.com |
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| ID | Term |
|---|---|
| D000081029 | Pulmonary Arterial Hypertension |
| ID | Term |
|---|---|
| D006976 | Hypertension, Pulmonary |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
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| ID | Term |
|---|---|
| D011464 | Epoprostenol |
| D007267 | Injections |
| ID | Term |
|---|---|
| D044062 | Prostaglandins I |
| D011453 | Prostaglandins |
| D015777 | Eicosanoids |
| D005231 | Fatty Acids, Unsaturated |
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| Approximately 3 months |
The Borg dyspnea score was assessed at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. The Borg scale is a category-ratio scale, commonly used to evaluate the effects of exercise on dyspnea. The original and modified scales have ratio properties ranging from 0 = nothing at all to 10 = very, very severe, with descriptors from 0 to 10. Descriptors have been modified by others so that 10 has been labelled "extremely severe," or "the worst possible dyspnea imaginable." |
| Approximately 3 months |
| Number of Participants With Improved, No Change, or Worsening of New York Heart Association Functional Class (NYHA FC) From Baseline to EOT. | NYHA FC was assessed at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. Disease severity was assessed by NYHA classification of pulmonary arterial hypertension criteria: Class I: no limitation of physical activity (PA). Ordinary PA: no undue dyspnea/fatigue, chest pain, near syncope. Class II: slight limitation of PA. Comfortable at rest. Ordinary PA: undue dyspnea/fatigue, chest pain, near syncope. Class III: marked limitation of PA. Comfortable at rest. Less than ordinary PA: undue dyspnea/fatigue, chest pain, near syncope. Class IV: inability to carry out PA without symptoms. Right heart failure. Dyspnea/fatigue may even have been present at rest. Discomfort increased by any PA. | Approximately 3 months |
| Change in N-terminal Pro-B-type Natriuretic Peptide (NT proBNP) From Baseline to EOT. | Blood sampling for NT proBNP was performed at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. | Approximately 3 months |
| Change in Effectiveness Score of the Abbreviated Treatment Satisfaction Questionnaire for Medication (TSQM-9) From Baseline to EOT. | Patients were required to complete the TSQM-9 questionnaire at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. The TSQM-9 is a validated instrument to assess patients' satisfaction with medication, including a three question effectiveness scale. The TSQM-9 domain scores range from 0 to 100 with higher scores representing higher satisfaction on that domain. | Approximately 3 months |
| Change in Convenience Score of the Abbreviated Treatment Satisfaction Questionnaire for Medication (TSQM-9) From Baseline to EOT. | Patients were required to complete the TSQM-9 questionnaire at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. The TSQM-9 is a validated instrument to assess patients' satisfaction with medication, including a three question convenience scale. The TSQM-9 domain scores range from 0 to 100 with higher scores representing higher satisfaction on that domain. | Approximately 3 months |
| Change in Global Satisfaction Score of the Abbreviated Treatment Satisfaction Questionnaire for Medication (TSQM-9) From Baseline to EOT. | Patients were required to complete the TSQM-9 questionnaire at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. The TSQM-9 is a validated instrument to assess patients' satisfaction with medication, including a three question global satisfaction scale. The TSQM-9 domain scores range from 0 to 100 with higher scores representing higher satisfaction on that domain. | Approximately 3 months |
| Number of Participants With Adverse Events Leading to Discontinuation of Study Drug From Baseline to EOT. | Adverse events that led to discontinuation of study drug from the start of study treatment until the end of study treatment were recorded. | Approximately 3 months |
| Toronto |
| Ontario |
| M5G 2N2 |
| Canada |
| Sir Mortimer B Davis Jewish General Hospital | Montreal | Quebec | H3T 1 E2 | Canada |
| Centre Hospitalier Universitaire | Caen | 14033 | France |
| Hôpital Antoine Béclère | Clamart | 92141 | France |
| Orsola Malpighi | Bologna | 40138 | Italy |
| VU Medisch Centrum (VUMC) | Amsterdam | 1081 HV | Netherlands |
| Hospital Vall d'Hebron | Barcelona | 8035 | Spain |
| years |
|
| Age, Categorical | Count of Participants | Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Number | participants |
|
| Region of Enrollment | Number | participants |
|
| Participants |
|
|
| Other Pre-specified | Change in 6-minute Walk Distance (6MWD) From Baseline to EOT. | The 6MWD was assessed at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. The 6-minute walk test is a non-encouraged test that measures the distance walked for the duration of 6 min. | All-treated set who had both a baseline and an EOT assessment. | Posted | Mean | Standard Deviation | m | Approximately 3 months |
|
|
|
| Primary | Change in Total Pulmonary Resistance From Baseline to End of Treatment (EOT). | Right heart catheterization was performed for cardiac hemodynamic assessment at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. | All treated set without imputation for missing values | Posted | Mean | Standard Deviation | dyn/sec/cm^5 | Approximately 3 months |
|
|
|
| Primary | Change in Mean Pulmonary Arterial Pressure From Baseline to End of Treatment (EOT). | Right heart catheterization was performed for cardiac hemodynamic assessment at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. | All treated set without imputation for missing values | Posted | Mean | Standard Deviation | mmHg | Approximately 3 months |
|
|
|
| Primary | Change in Mean Right Atrial Pressure From Baseline to End of Treatment (EOT). | Right heart catheterization was performed for cardiac hemodynamic assessment at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. | All treated set without imputation for missing values | Posted | Mean | Standard Deviation | mmHg | Approximately 3 months |
|
|
|
| Primary | Change in Pulmonary Capillary Wedge Pressure From Baseline to End of Treatment (EOT). | Right heart catheterization was performed for cardiac hemodynamic assessment at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. | All treated set without imputation for missing values. Data was missing for 5 patients. | Posted | Mean | Standard Deviation | mmHg | Approximately 3 months |
|
|
|
| Primary | Change in Mean Cardiac Index From Baseline to End of Treatment (EOT). | Right heart catheterization was performed for cardiac hemodynamic assessment at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. | All treated set without imputation for missing values | Posted | Mean | Standard Deviation | L/min/m^2 | Approximately 3 months |
|
|
|
| Other Pre-specified | Change in Borg Dyspnea Score From Baseline to EOT. | The Borg dyspnea score was assessed at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. The Borg scale is a category-ratio scale, commonly used to evaluate the effects of exercise on dyspnea. The original and modified scales have ratio properties ranging from 0 = nothing at all to 10 = very, very severe, with descriptors from 0 to 10. Descriptors have been modified by others so that 10 has been labelled "extremely severe," or "the worst possible dyspnea imaginable." | All-treated set who had both a baseline and an EOT assessment. | Posted | Mean | Standard Deviation | units on a scale | Approximately 3 months |
|
|
|
| Other Pre-specified | Number of Participants With Improved, No Change, or Worsening of New York Heart Association Functional Class (NYHA FC) From Baseline to EOT. | NYHA FC was assessed at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. Disease severity was assessed by NYHA classification of pulmonary arterial hypertension criteria: Class I: no limitation of physical activity (PA). Ordinary PA: no undue dyspnea/fatigue, chest pain, near syncope. Class II: slight limitation of PA. Comfortable at rest. Ordinary PA: undue dyspnea/fatigue, chest pain, near syncope. Class III: marked limitation of PA. Comfortable at rest. Less than ordinary PA: undue dyspnea/fatigue, chest pain, near syncope. Class IV: inability to carry out PA without symptoms. Right heart failure. Dyspnea/fatigue may even have been present at rest. Discomfort increased by any PA. | All-treated set who had both a baseline and an EOT assessment. | Posted | Number | participants | Approximately 3 months |
|
|
|
| Other Pre-specified | Change in N-terminal Pro-B-type Natriuretic Peptide (NT proBNP) From Baseline to EOT. | Blood sampling for NT proBNP was performed at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. | All-treated set who had both a baseline and an EOT assessment. | Posted | Mean | Standard Deviation | ng/L | Approximately 3 months |
|
|
|
| Other Pre-specified | Change in Effectiveness Score of the Abbreviated Treatment Satisfaction Questionnaire for Medication (TSQM-9) From Baseline to EOT. | Patients were required to complete the TSQM-9 questionnaire at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. The TSQM-9 is a validated instrument to assess patients' satisfaction with medication, including a three question effectiveness scale. The TSQM-9 domain scores range from 0 to 100 with higher scores representing higher satisfaction on that domain. | All-treated set who had both a baseline and an EOT assessment. | Posted | Mean | Standard Deviation | units on a scale | Approximately 3 months |
|
|
|
| Other Pre-specified | Change in Convenience Score of the Abbreviated Treatment Satisfaction Questionnaire for Medication (TSQM-9) From Baseline to EOT. | Patients were required to complete the TSQM-9 questionnaire at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. The TSQM-9 is a validated instrument to assess patients' satisfaction with medication, including a three question convenience scale. The TSQM-9 domain scores range from 0 to 100 with higher scores representing higher satisfaction on that domain. | All-treated set who had both a baseline and an EOT assessment. | Posted | Mean | Standard Deviation | units on a scale | Approximately 3 months |
|
|
|
| Other Pre-specified | Change in Global Satisfaction Score of the Abbreviated Treatment Satisfaction Questionnaire for Medication (TSQM-9) From Baseline to EOT. | Patients were required to complete the TSQM-9 questionnaire at Screening or Day 1, prior to switch from Flolan® to EFI/ACT-385781A and at EOT. The TSQM-9 is a validated instrument to assess patients' satisfaction with medication, including a three question global satisfaction scale. The TSQM-9 domain scores range from 0 to 100 with higher scores representing higher satisfaction on that domain. | All-treated set who had both a baseline and an EOT assessment. | Posted | Mean | Standard Deviation | units on a scale | Approximately 3 months |
|
|
|
| Other Pre-specified | Number of Participants With Adverse Events Leading to Discontinuation of Study Drug From Baseline to EOT. | Adverse events that led to discontinuation of study drug from the start of study treatment until the end of study treatment were recorded. | All-treated set. | Posted | Number | participants | Approximately 3 months |
|
|
|
| 7 |
| 41 |
| 31 |
| 41 |
| Device-related infection (pseudomonas) | Infections and infestations | MedDRA Version 14.0 | Systematic Assessment |
|
| Device connection issue | General disorders | MedDRA Version 14.0 | Systematic Assessment |
|
| Right ventricular failure | Cardiac disorders | MedDRA Version 14.0 | Systematic Assessment |
|
| Device damage | General disorders | MedDRA Version 14.0 | Systematic Assessment |
|
| Device dislocation | General disorders | MedDRA Version 14.0 | Systematic Assessment |
|
| NASOPHARYNGITIS | Infections and infestations | MedDRA Version 14.0 | Systematic Assessment |
|
| PAIN IN JAW | Musculoskeletal and connective tissue disorders | MedDRA Version 14.0 | Systematic Assessment |
|
| FLUSHING | Vascular disorders | MedDRA Version 14.0 | Systematic Assessment |
|
| DYSPNOEA | Respiratory, thoracic and mediastinal disorders | MedDRA Version 14.0 | Systematic Assessment |
|
| EPISTAXIS | Respiratory, thoracic and mediastinal disorders | MedDRA Version 14.0 | Systematic Assessment |
|
| PAIN IN EXTREMITY | Musculoskeletal and connective tissue disorders | MedDRA Version 14.0 | Systematic Assessment |
|
| PALPITATIONS | Cardiac disorders | MedDRA Version 14.0 | Systematic Assessment |
|
Not provided
| D005227 |
| Fatty Acids |
| D008055 | Lipids |
| D012898 | Autacoids |
| D018836 | Inflammation Mediators |
| D001685 | Biological Factors |
| D004333 | Drug Administration Routes |
| D004358 | Drug Therapy |
| D013812 | Therapeutics |
| Title | Measurements |
|---|
|