Trial · NCT01743001

Clinical Study to Evaluate the Effects of Macitentan on Exercise Capacity in Subjects With Eisenmenger Syndrome

NCT01743001, Phase 3, Completed, Actelion; results first posted 2018-01-30

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Clinical Study to Evaluate the Effects of Macitentan on Exercise Capacity in Subjects With Eisenmenger Syndrome. Trials, record NCT01743001. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01743001

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Trial

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

Title: Clinical Study to Evaluate the Effects of Macitentan on Exercise Capacity in Subjects With Eisenmenger Syndrome
NCT number: NCT01743001
Status: Completed
Phases: Phase 3
Enrollment: 226 participants, actual
Lead sponsor: Actelion
Conditions: Pulmonary Arterial Hypertension
Interventions: Macitentan 10 mg, Placebo
First posted: 2012-12-06
Last updated: 2018-02-23
Results first posted: 2018-01-30

Source ClinicalTrials.gov

Registered outcomes4

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

List	Outcome	Time frame	Description
Primary	Change From Baseline to Week 16 in Exercise Capacity, as Measured by 6-minute Walk Distance (6MWD)	From baseline to Week 16	The purpose of the six minute walk is to test exercise tolerance and capacity. The test measures the distance an individual is able to walk over a total of six minutes on a hard, flat surface. The goal is for the individual to walk as far as possible in six minutes.
Secondary	Change From Baseline to Week 16 in WHO Functional Class	From baseline to Week 16	A shift in WHO functional classes is considered an 'improvement' when shifting to a lower class (e.g. from class III to class II) or a 'worsening' when shifting to a higher class (e.g. from class III to class IV). Definition of functional classes as follows - Class I: no symptoms with exercise or at rest. Class II: No symptoms at rest but uncomfortable and short of breath with normal activity such as climbing a flight of stairs, grocery shopping, or making the bed. Class III: May not have symptoms at rest but activities greatly limited by shortness of breath, fatigue, or near fainting (e.g. doing normal chores around the house, have to take breaks while doing activities of daily living). Class IV: Symptoms at rest and severe symptoms with any activity. Most patients also have edema in the feet and ankles as result of right heart failure.
Secondary	Change From Baseline to Week 16 in Dyspnea, Assessed by the Borg Dyspnea Index	From baseline to Week 16	This outcome measures the difference in the Borg dyspnea index collected at the end of the 6-minute walk test (6MWT) at Week 16 compared to baseline. The Borg dyspnea index rates the severity of dyspnea (difficult or labored breathing) on a scale from 0 ('Nothing at all') to 10 ('Very, very severe - maximal'). A decrease in the Borg dyspnea index indicates an improvement.
Secondary	Change From Baseline to Week 16 in Quality of Life (QoL), Assessed by the Short Form-36 (SF-36) Questionnaire	From baseline to Week 16	The SF-36 is a multi-purpose, short-form health survey with 36 questions. It yields an 8-scale profile of the functional health and well-being scores (i.e., physical functioning, role physical, bodily pain, general health, vitality, social functioning, role emotional and mental health), as well as psychometrically based physical and mental health summary measures and a preference-based health utility (health rated as much better now than one year ago to much worse now than one year ago). It is a generic measure, as opposed to one that targets a specific age, disease, or treatment group. For each of the domains and scores that the SF36 measures an aggregate percentage score is produced. The percentage scores range from 0% (lowest or worst possible level of functioning) to 100% (highest or best possible level of functioning). A higher score for the individual domains and summary component scores indicates a better condition of the subject.

Source ClinicalTrials.gov

Outcomes4

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change From Baseline to Week 16 in Exercise Capacity, as Measured by 6-minute Walk Distance (6MWD)	From baseline to Week 16	Full analysis set: The analysis was performed on the full analysis set (FAS), i.e., all randomized subjects in the treatment group to which they were randomized and by imputing missing values at Week 16 according to pre-defined rules.	meter	MEAN
SECONDARY	Change From Baseline to Week 16 in WHO Functional Class	From baseline to Week 16	Full analysis set: The analysis was performed on the full analysis set (FAS), i.e., all randomized subjects in the treatment group to which they were randomized and by imputing missing values at Week 16 according to pre-defined rules.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Change From Baseline to Week 16 in Dyspnea, Assessed by the Borg Dyspnea Index	From baseline to Week 16	Full analysis set: The analysis was performed on the full analysis set (FAS), i.e., all randomized subjects in the treatment group to which they were randomized and by imputing missing values at Week 16 according to pre-defined rules.	Score on a scale	MEAN
SECONDARY	Change From Baseline to Week 16 in Quality of Life (QoL), Assessed by the Short Form-36 (SF-36) Questionnaire	From baseline to Week 16	Full analysis set (excluding children \<14 years of age and Down Syndrome subjects)	Score on a scale	MEAN

Source ClinicalTrials.gov

Results by group134

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

Outcome	Group	Category	Value	Spread	Units
Change From Baseline to Week 16 in Exercise Capacity, as Measured by 6-minute Walk Distance (6MWD)	Macitentan	6MWD at baseline	368.7	Standard Deviation 74.5	meter
Change From Baseline to Week 16 in Exercise Capacity, as Measured by 6-minute Walk Distance (6MWD)	Placebo	6MWD at baseline	380.3	Standard Deviation 76.3	meter
Change From Baseline to Week 16 in Exercise Capacity, as Measured by 6-minute Walk Distance (6MWD)	Macitentan	6MWD at Week 16	387.1	Standard Deviation 101.8	meter
Change From Baseline to Week 16 in Exercise Capacity, as Measured by 6-minute Walk Distance (6MWD)	Placebo	6MWD at Week 16	399.9	Standard Deviation 79.5	meter
Change From Baseline to Week 16 in Exercise Capacity, as Measured by 6-minute Walk Distance (6MWD)	Macitentan	Change in 6MWD from baseline to Week 16	18.3	Standard Deviation 84.4	meter
Change From Baseline to Week 16 in Exercise Capacity, as Measured by 6-minute Walk Distance (6MWD)	Placebo	Change in 6MWD from baseline to Week 16	19.7	Standard Deviation 53.0	meter
Change From Baseline to Week 16 in WHO Functional Class	Macitentan	WHO functional class 1 at Week 16	3		Participants
Change From Baseline to Week 16 in WHO Functional Class	Placebo	WHO functional class 1 at Week 16	1		Participants
Change From Baseline to Week 16 in WHO Functional Class	Macitentan	WHO functional class 2 at Week 16	72		Participants
Change From Baseline to Week 16 in WHO Functional Class	Placebo	WHO functional class 2 at Week 16	79		Participants

1 to 10 of 134Source ClinicalTrials.gov

See all 134 results by group

Participant flow6

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Macitentan	114
Overall Study	STARTED	Placebo	112
Overall Study	COMPLETED	Macitentan	111
Overall Study	COMPLETED	Placebo	112
Overall Study	NOT COMPLETED	Macitentan	3
Overall Study	NOT COMPLETED	Placebo	0

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	Events
other	Bronchitis	Infections and infestations	Macitentan	6	114	6
other	Bronchitis	Infections and infestations	Placebo	3	112	3
other	Haemoptysis	Respiratory, thoracic and mediastinal disorders	Macitentan	0	114	0
other	Haemoptysis	Respiratory, thoracic and mediastinal disorders	Placebo	6	112	8
other	Headache	Nervous system disorders	Macitentan	13	114	15
other	Headache	Nervous system disorders	Placebo	5	112	5
other	Nasopharyngitis	Infections and infestations	Macitentan	3	114	4
other	Nasopharyngitis	Infections and infestations	Placebo	14	112	16
other	Upper respiratory tract infection	Infections and infestations	Macitentan	11	114	12
other	Upper respiratory tract infection	Infections and infestations	Placebo	7	112	10

1 to 10 of 30Source ClinicalTrials.gov

See all 30 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Macitentan	1	114
Total, all-cause mortality	Placebo	0	112
Total, other adverse events	Macitentan	31	114
Total, other adverse events	Placebo	30	112
Total, serious adverse events	Macitentan	7	114
Total, serious adverse events	Placebo	2	112

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
30586694	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 comparisons2

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

Comparison	Source
Macitentan vs Placebo: deaths from any cause	Trial comparisons
Macitentan vs Placebo: serious adverse events	Trial comparisons

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.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT01743001

GET https://conductscience.org/api/trials/v1/NCT01743001/results?page=2

API reference For AI assistants