Trial · NCT01841762

Clinical Study of Macitentan in Patients With Pulmonary Arterial Hypertension to Psychometrically Validate the PAH-SYMPACT Instrument

NCT01841762, Phase 3, Completed, Actelion; results first posted 2018-10-31

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Clinical Study of Macitentan in Patients With Pulmonary Arterial Hypertension to Psychometrically Validate the PAH-SYMPACT Instrument. Trials, record NCT01841762. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01841762

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Clinical Study of Macitentan in Patients With Pulmonary Arterial Hypertension to Psychometrically Validate the PAH-SYMPACT Instrument
NCT number: NCT01841762
Status: Completed
Phases: Phase 3
Enrollment: 284 participants, actual
Lead sponsor: Actelion
Conditions: Pulmonary Arterial Hypertension
Interventions: Macitentan
First posted: 2013-04-29
Last updated: 2019-02-26
Results first posted: 2018-10-31

Source ClinicalTrials.gov

Registered outcomes5

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Development and Refinement of the Patient-reported Outcome Measure of Symptoms and Their Impact in PAH (the PAH-SYMPACT)	From Screening Visit (Day -14) to End of Treatment (EOT) Visit (Visit 4, Week 16)	Content validity of the PAH-SYMPACT was assessed using item performance, exploratory and confirmatory factor analysis. The final item content and domain structure of PAH-SYMPACT was determined based on these analyses from the Steering Committee (expert clinicians) and findings from the qualitative research done with patients previously.
Primary	Validation of the Patient-reported Outcome Measure of Symptoms and Their Impact in PAH (the PAH-SYMPACT), With Reliability Assessed Via Test-retest Reliability.	From ePRO period 1 (Days -14 to -8) to ePRO period 2 (Days -7 to -1) in screening period.	The reliability of the PAH-SYMPACT is assessed by test-retest reliability. Intra-class correlation coefficients (ICCs) assess test-retest reliability for the symptom and impact part scores as well as domains. ICCs equal to or greater than 0.70 are considered to demonstrate good test-retest reliability for total and domain scores.
Primary	Validation of the Patient-reported Outcome Measure of Symptoms and Their Impact in PAH (the PAH-SYMPACT), Assessing Internal Consistency Reliability.	From ePRO period 1 (Days -14 to -8) to ePRO period 2 (Days -7 to -1) in screening period.	The reliability of the PAH-SYMPACT is assessed by internal consistency reliability. This was determined using Cronbach's alpha-a value on an internal level scale from 0 to 1.0 with higher scores indicating a more-reliable (precise) instrument.
Secondary	Number of Participants With Treatment-emergent Adverse Events, Serious Adverse Events, and Adverse Events Resulting in Patient Study Drug Discontinuation Between Time Periods, BL to End of Study Visit (EoS, Week 16+30 Days for Follow-up Safety Visits)	From Day 1 (Baseline Visit) to End of Study visit (EoS).	Safety events are reported and documented as defined in study protocol.
Other	Assessment of the Sensitivity to Detect Change in the Symptoms and Impacts Domain Scores of the PAH-SYMPACT From Baseline to Week 16.	From Screening period (Days -7 to -1) to Week 16 (7-day period prior to Week 16 visit).	Sensitivity to change is an aspect of construct validity and represents the instrument's ability to detect underlying change. Sensitivity to change was examined to compare the difference in mean score in each domain of the PAH-SYMPACT. The symptoms and impacts domains consisted of 11 items each reported on a 7-point Likert Scale (from 0=no symptom/with no difficulty at all/not at all to 6=very severe symptoms/very much/extremely/not able at all). An average symptoms domain score is determined based on the daily scores of the containing items. An average impacts domain score is determined based on the items in the domain.

Source ClinicalTrials.gov

Outcomes5

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Development and Refinement of the Patient-reported Outcome Measure of Symptoms and Their Impact in PAH (the PAH-SYMPACT)	From Screening Visit (Day -14) to End of Treatment (EOT) Visit (Visit 4, Week 16)	Per Protocol Set (PPS) comprised all patients included in the Full Analysis Set (FAS) who had no major protocol violations.	Items	NUMBER
PRIMARY	Validation of the Patient-reported Outcome Measure of Symptoms and Their Impact in PAH (the PAH-SYMPACT), With Reliability Assessed Via Test-retest Reliability.	From ePRO period 1 (Days -14 to -8) to ePRO period 2 (Days -7 to -1) in screening period.	Per Protocol Set of 278 patients.	Intra-class correlation coefficient	NUMBER
PRIMARY	Validation of the Patient-reported Outcome Measure of Symptoms and Their Impact in PAH (the PAH-SYMPACT), Assessing Internal Consistency Reliability.	From ePRO period 1 (Days -14 to -8) to ePRO period 2 (Days -7 to -1) in screening period.	Per protocol set of 278.	Ratio of variance	NUMBER
SECONDARY	Number of Participants With Treatment-emergent Adverse Events, Serious Adverse Events, and Adverse Events Resulting in Patient Study Drug Discontinuation Between Time Periods, BL to End of Study Visit (EoS, Week 16+30 Days for Follow-up Safety Visits)	From Day 1 (Baseline Visit) to End of Study visit (EoS).	Safety set of 284 subjects.	Participants	COUNT_OF_PARTICIPANTS
OTHER_PRE_SPECIFIED	Assessment of the Sensitivity to Detect Change in the Symptoms and Impacts Domain Scores of the PAH-SYMPACT From Baseline to Week 16.	From Screening period (Days -7 to -1) to Week 16 (7-day period prior to Week 16 visit).	Per Protocol Set of 278 subjects.	Score on a scale	MEDIAN

Source ClinicalTrials.gov

Results by group28

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Units
Development and Refinement of the Patient-reported Outcome Measure of Symptoms and Their Impact in PAH (the PAH-SYMPACT)	Macitentan	Item number in symptoms part of baseline	16	Items
Development and Refinement of the Patient-reported Outcome Measure of Symptoms and Their Impact in PAH (the PAH-SYMPACT)	Macitentan	Item number in symptoms part at Week 16	11	Items
Development and Refinement of the Patient-reported Outcome Measure of Symptoms and Their Impact in PAH (the PAH-SYMPACT)	Macitentan	Item number in impacts part at baseline	25	Items
Development and Refinement of the Patient-reported Outcome Measure of Symptoms and Their Impact in PAH (the PAH-SYMPACT)	Macitentan	Item number in impacts part at Week 16	11	Items
Validation of the Patient-reported Outcome Measure of Symptoms and Their Impact in PAH (the PAH-SYMPACT), With Reliability Assessed Via Test-retest Reliability.	Macitentan	ICCs of Cardiopulmonary Symptoms domain	0.94	Intra-class correlation coefficient
Validation of the Patient-reported Outcome Measure of Symptoms and Their Impact in PAH (the PAH-SYMPACT), With Reliability Assessed Via Test-retest Reliability.	Macitentan	ICCs of Cardiovascular Symptoms domain	0.93	Intra-class correlation coefficient
Validation of the Patient-reported Outcome Measure of Symptoms and Their Impact in PAH (the PAH-SYMPACT), With Reliability Assessed Via Test-retest Reliability.	Macitentan	ICCs of Physical Impacts domain	0.91	Intra-class correlation coefficient
Validation of the Patient-reported Outcome Measure of Symptoms and Their Impact in PAH (the PAH-SYMPACT), With Reliability Assessed Via Test-retest Reliability.	Macitentan	ICCs of Cognitive/Emotional Impacts domain	0.84	Intra-class correlation coefficient
Validation of the Patient-reported Outcome Measure of Symptoms and Their Impact in PAH (the PAH-SYMPACT), Assessing Internal Consistency Reliability.	Macitentan	Cronbach's Alpha for Cardiopulmonary Symptoms	0.81	Ratio of variance
Validation of the Patient-reported Outcome Measure of Symptoms and Their Impact in PAH (the PAH-SYMPACT), Assessing Internal Consistency Reliability.	Macitentan	Cronbach's Alpha for Cardiovascular Symptoms	0.88	Ratio of variance

1 to 10 of 28Source ClinicalTrials.gov

See all 28 results by group

Participant flow9

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Screening Period	STARTED	Macitentan	335
Screening Period	COMPLETED	Macitentan	284
Screening Period	NOT COMPLETED	Macitentan	51
Treatment Period	STARTED	Macitentan	284
Treatment Period	COMPLETED	Macitentan	252
Treatment Period	NOT COMPLETED	Macitentan	32
Post-Treatment Safety Follow-Up Period	STARTED	Macitentan	284
Post-Treatment Safety Follow-Up Period	COMPLETED	Macitentan	269
Post-Treatment Safety Follow-Up Period	NOT COMPLETED	Macitentan	15

Source ClinicalTrials.gov

Adverse events30

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Oedema peripheral	Blood and lymphatic system disorders	Macitentan	50	284
other	Headache	Nervous system disorders	Macitentan	36	284
other	Dyspnoea	Respiratory, thoracic and mediastinal disorders	Macitentan	34	284
other	Cough	Respiratory, thoracic and mediastinal disorders	Macitentan	26	284
other	Nasal congestion	Respiratory, thoracic and mediastinal disorders	Macitentan	23	284
other	Fatigue	General disorders	Macitentan	22	284
other	Dizziness	Ear and labyrinth disorders	Macitentan	19	284
other	Upper Respiratory Tract Infection	Respiratory, thoracic and mediastinal disorders	Macitentan	19	284
other	Nausea	General disorders	Macitentan	17	284
other	Anaemia	Blood and lymphatic system disorders	Macitentan	16	284

1 to 10 of 30Source ClinicalTrials.gov

See all 30 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Macitentan	1	284
Total, other adverse events	Macitentan	228	284
Total, serious adverse events	Macitentan	49	284

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
29705220	DERIVED

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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