Pulmonary Arterial Hypertension
Conditions
Keywords
Pulmonary Arterial Hypertension, PAH, EPITOME-1
Brief summary
This is an open-label, non-randomized extension to study AC-066A401. The study will assess safety and tolerability of ACT-385781A and Flolan (epoprostenol sodium) while providing a means for continuing treatment after ending participation in study AC-0066A401.
Interventions
per Prescribing Information
DRUGFlolan®
per Prescribing Information
Sponsors
Actelion
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
1. Signed informed consent prior to initiation of any study-mandated procedure 2. Patients who completed participation in study AC-066A401 3. Patients who have not obtained authorization for commercial ACT-385781 and Flolan at the time of ending participation in study AC-066A401
Exclusion criteria
1. Patients who prematurely discontinued study drug in study AC-066A401 2. Patients for whom continued treatment with either ACT-385781 and Flolan is no longer considered appropriate 3. Known hypersensitivity to the investigational drug or comparative drug or drugs of the same class, or any of their excipients 4. 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 5. Known concomitant life-threatening disease with a life expectancy \< 12 months
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety and Tolerability of ACT-385781A and Flolan in Injectable Prostanoid Treatment-naïve Patients With Pulmonary Arterial Hypertension (PAH) - Number of Patients With Adverse Events Leading Discontinuation of Study Treatment | Up to 39 days. Day 1 - until patients transition from study medication to commercially-obtained medication |
| Safety and Tolerability of ACT-385781A and Flolan in Injectable Prostanoid Treatment-naïve Patients With PAH - Number of Deaths | Up to 39 days. Day 1 - until patients transition from study medication to commercially-obtained medication |
Countries
United States
Participant flow
Recruitment details
The study was conducted at Pulmonary Hypertension centers in the U.S.
Pre-assignment details
In this extension study patients could continue on the randomized treatment they received in the core study (AC-066A401) until commercial treatment became available.
Participants by arm
| Arm | Count |
|---|---|
| ACT-385781A (Epoprostenol for Injection) ACT-385781A (Actelion Epoprostenol)
ACT-385781A (Actelion Epoprostenol) : per Prescribing Information | 1 |
| Flolan® (Epoprostenol Sodium) for Injection Flolan®
Flolan® : per Prescribing Information | 1 |
| Total | 2 |
Baseline characteristics
| Characteristic | Flolan® (Epoprostenol Sodium) for Injection | ACT-385781A (Epoprostenol for Injection) | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 1 Participants | 1 Participants | 2 Participants |
| Age Continuous | 34 years STANDARD_DEVIATION 0 | 55 years STANDARD_DEVIATION 0 | 44.5 years STANDARD_DEVIATION 14.8 |
| Region of Enrollment United States | 1 participants | 1 participants | 2 participants |
| Sex: Female, Male Female | 1 Participants | 0 Participants | 1 Participants |
| Sex: Female, Male Male | 0 Participants | 1 Participants | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 1 / 1 | 1 / 1 |
| serious Total, serious adverse events | 0 / 1 | 1 / 1 |
Outcome results
Primary
Safety and Tolerability of ACT-385781A and Flolan in Injectable Prostanoid Treatment-naïve Patients With PAH - Number of Deaths
Time frame: Up to 39 days. Day 1 - until patients transition from study medication to commercially-obtained medication
Population: Study population
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| ACT-385781A (Epoprostenol for Injection) | Safety and Tolerability of ACT-385781A and Flolan in Injectable Prostanoid Treatment-naïve Patients With PAH - Number of Deaths | 0 participants |
| Flolan® (Epoprostenol Sodium) for Injection | Safety and Tolerability of ACT-385781A and Flolan in Injectable Prostanoid Treatment-naïve Patients With PAH - Number of Deaths | 0 participants |
Primary
Safety and Tolerability of ACT-385781A and Flolan in Injectable Prostanoid Treatment-naïve Patients With Pulmonary Arterial Hypertension (PAH) - Number of Patients With Adverse Events Leading Discontinuation of Study Treatment
Time frame: Up to 39 days. Day 1 - until patients transition from study medication to commercially-obtained medication
Population: Study population
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| ACT-385781A (Epoprostenol for Injection) | Safety and Tolerability of ACT-385781A and Flolan in Injectable Prostanoid Treatment-naïve Patients With Pulmonary Arterial Hypertension (PAH) - Number of Patients With Adverse Events Leading Discontinuation of Study Treatment | 0 participants |
| Flolan® (Epoprostenol Sodium) for Injection | Safety and Tolerability of ACT-385781A and Flolan in Injectable Prostanoid Treatment-naïve Patients With Pulmonary Arterial Hypertension (PAH) - Number of Patients With Adverse Events Leading Discontinuation of Study Treatment | 0 participants |