Trial Outcomes & Findings for Clinical Study of Macitentan in Patients With PAH to Psychometrically Validate PAH-SYMPACT Instrument (NCT NCT01847014)
NCT ID: NCT01847014
Last Updated: 2018-08-22
Results Overview
Safety events are reported and documented as defined in study protocol from baseline to end of treatment period including safety follow-up visit.
TERMINATED
PHASE3
4 participants
From Visit 1 to Post-treatment safety follow-up visit (30 days after discontinuation of the study drug).
2018-08-22
Participant Flow
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.
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.
Participant milestones
| Measure |
Macitentan
Macitentan tablet, dose of 10 mg, once daily.
|
|---|---|
|
Baseline Extension Visit
STARTED
|
4
|
|
Baseline Extension Visit
COMPLETED
|
4
|
|
Baseline Extension Visit
NOT COMPLETED
|
0
|
|
Completion of Extension Study
STARTED
|
4
|
|
Completion of Extension Study
COMPLETED
|
0
|
|
Completion of Extension Study
NOT COMPLETED
|
4
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Clinical Study of Macitentan in Patients With PAH to Psychometrically Validate PAH-SYMPACT Instrument
Baseline characteristics by cohort
| Measure |
Macitentan
n=4 Participants
Macitentan tablet, dose of 10 mg, once daily.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=5 Participants
|
|
Age, Continuous
|
60.8 Years
n=5 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 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.
Safety events are reported and documented as defined in study protocol from baseline to end of treatment period including safety follow-up visit.
Outcome measures
| Measure |
Macitentan
n=4 Participants
Macitentan tablet, dose of 10 mg, once daily.
|
|---|---|
|
Incident Rate of Adverse Events (AEs).
Participants with severe intensity AEs
|
1 Participants
|
|
Incident Rate of Adverse Events (AEs).
Participants with AEs
|
3 Participants
|
Adverse Events
Macitentan
Serious adverse events
| Measure |
Macitentan
n=4 participants at risk
Macitentan tablet, dose of 10 mg, once daily.
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Chronic Obstructive Pulmonary Disease Exacerbation
|
25.0%
1/4 • Number of events 1
|
|
Cardiac disorders
Pulmonary Arterial Hypertension Exacerbation
|
25.0%
1/4 • Number of events 1
|
|
Cardiac disorders
Pulmonary Hypertension Exacerbation
|
25.0%
1/4 • Number of events 1
|
|
Cardiac disorders
Right Heart Failure
|
25.0%
1/4 • Number of events 1
|
Other adverse events
| Measure |
Macitentan
n=4 participants at risk
Macitentan tablet, dose of 10 mg, once daily.
|
|---|---|
|
Metabolism and nutrition disorders
Hyperlipidaemia
|
25.0%
1/4 • Number of events 1
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
25.0%
1/4 • Number of events 1
|
|
Metabolism and nutrition disorders
Type 2 Diabetes Mellitus
|
25.0%
1/4 • Number of events 1
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
25.0%
1/4 • Number of events 1
|
|
Cardiac disorders
Right Heart Failure
|
25.0%
1/4 • Number of events 1
|
|
Ear and labyrinth disorders
Vertigo
|
25.0%
1/4 • Number of events 1
|
|
Gastrointestinal disorders
Diarrhoea
|
25.0%
1/4 • Number of events 1
|
|
Gastrointestinal disorders
Nausea
|
25.0%
1/4 • Number of events 1
|
|
Gastrointestinal disorders
Vomiting
|
25.0%
1/4 • Number of events 1
|
|
General disorders
Chest Pain
|
25.0%
1/4 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Pain In Jaw
|
25.0%
1/4 • Number of events 1
|
|
Nervous system disorders
Headache
|
25.0%
1/4 • Number of events 1
|
|
Renal and urinary disorders
Renal Impairment
|
25.0%
1/4 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Chronic Obstructive Pulmonary Disease Exacerbation
|
25.0%
1/4 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary Arterial Hypertension Exacerbation
|
25.0%
1/4 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary Hypertension Exacerbation
|
25.0%
1/4 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
25.0%
1/4 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
25.0%
1/4 • Number of events 1
|
|
Vascular disorders
Flushing
|
25.0%
1/4 • Number of events 1
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Any study-related publication written independently by investigators must be submitted to Actelion for review at least 30 days prior to submission for publication or presentation. Upon review, Actelion provide comments, and may also request alterations and/or deletions for the sole purpose of protecting its confidential information an/or patient rights. Neither the institution nor the investigator should permit publication during such a review period.
- Publication restrictions are in place
Restriction type: OTHER