Pulmonary Arterial Hypertension
Conditions
Keywords
Pulmonary Arterial Hypertension (PAH), psychometric instrument, PAH-SYMPACT
Brief summary
SYMPHONY Extension is an extension of AC-055-401, a multi-center, open-label, single-arm, Phase 3b study of macitentan in patients with Pulmonary Arterial Hypertension to psychometrically validate the PAH-SYMPACT instrument. The objective is to assess the long-term safety of macitentan in subjects with PAH beyond the treatment in the AC-055-401 study.
Interventions
DRUGMacitentan
Macitentan tablet, dose of 10 mg, once daily.
Sponsors
Actelion
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
* Signed informed consent prior to any study-mandated procedure. * Patients with PAH who completed study AC-055-401 * Women of childbearing potential must: * Have a negative urine pregnancy test at Visit 1 and agree to perform monthly serum pregnancy tests. * Agree to use two methods of contraception from Visit 1 until 1 month after study drug discontinuation.
Exclusion criteria
* Patients who prematurely discontinued study drug in study AC-055-401 * Females who are lactating or pregnant (positive Visit 1 pregnancy test) or plan to become pregnant during the study * Known hypersensitivity to macitentan or its excipients or drugs of the same class
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incident Rate of Adverse Events (AEs). | From Visit 1 to Post-treatment safety follow-up visit (30 days after discontinuation of the study drug). | Safety events are reported and documented as defined in study protocol from baseline to end of treatment period including safety follow-up visit. |
Countries
United States
Participant flow
Recruitment details
All sites activated on the SYMPHONY AC-055-401 clinical trial were invited to participate in this open-label extension study. Due to meeting the study closure milestone, defined as the marketing of Opsumit on 18 OCT 2013, only two sites received activation. Recruitment occurred between Quarter 2 2013 and Quarter 3 2015.
Pre-assignment details
Subjects who completed the sixteen week treatment of macitentan on the SYMPHONY AC-055-401 clinical trial were eligible to enter this open-label extension study.
Participants by arm
| Arm | Count |
|---|---|
| Macitentan Macitentan tablet, dose of 10 mg, once daily. | 4 |
| Total | 4 |
Baseline characteristics
| Characteristic | Macitentan |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 1 Participants |
| Age, Categorical Between 18 and 65 years | 3 Participants |
| Age, Continuous | 60.8 Years |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 4 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 3 Participants |
| Sex: Female, Male Female | 4 Participants |
| Sex: Female, Male Male | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 4 |
| other Total, other adverse events | 3 / 4 |
| serious Total, serious adverse events | 1 / 4 |
Outcome results
Primary
Incident Rate of Adverse Events (AEs).
Safety events are reported and documented as defined in study protocol from baseline to end of treatment period including safety follow-up visit.
Time frame: From Visit 1 to Post-treatment safety follow-up visit (30 days after discontinuation of the study drug).
Population: Four participants represent the Extension Analysis Set, who transitioned from completion of SYMPHONY AC-055-401 to this open-label extension clinical trial.
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Macitentan | Incident Rate of Adverse Events (AEs). | Participants with severe intensity AEs | 1 Participants |
| Macitentan | Incident Rate of Adverse Events (AEs). | Participants with AEs | 3 Participants |