Trial · NCT01739400

Clinical Study to Assess the Long-term Safety, Tolerability, and Efficacy of Macitentan in Subjects With Eisenmenger Syndrome

NCT01739400, Phase 3, Terminated, Actelion; results first posted 2019-04-16

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Clinical Study to Assess the Long-term Safety, Tolerability, and Efficacy of Macitentan in Subjects With Eisenmenger Syndrome. Trials, record NCT01739400. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01739400

On this page

Trial

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

Title: Clinical Study to Assess the Long-term Safety, Tolerability, and Efficacy of Macitentan in Subjects With Eisenmenger Syndrome
NCT number: NCT01739400
Status: Terminated
Phases: Phase 3
Enrollment: 217 participants, actual
Lead sponsor: Actelion
Conditions: Pulmonary Arterial Hypertension
Interventions: Macitentan 10 mg tablet, once daily.
First posted: 2012-12-03
Last updated: 2019-04-16
Results first posted: 2019-04-16
Limitations and caveats: The MAESTRO parent study AC-055-305 (NCT01743001) did not meet its primary endpoint. The sponsor decided to prematurely terminate this OL study on January 12th 2018.

Source ClinicalTrials.gov

Registered outcomes4

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

List	Outcome	Time frame	Description
Primary	Change in Exercise Capacity as Measured by 6-minute Walking Distance (6MWD) Month 6 and 12	From baseline in DB parent study (AC-055-305, NCT01743001) up to month 12 in this OL study.	NOTE: The MAESTRO-OL study was exploratory in nature and no primary efficacy and safety endpoint were defined in the clinical protocol. This and the other exploratory efficacy outcome measures posted were selected to be reported as a primary endpoints. All efficacy analyses were considered exploratory. The analyses of the exploratory efficacy endpoints focused on the absolute values and on the change from DB baseline to Week 16 in the DB study and to Month 6 and Month 12 in the OL study. For missing 6MWD values in the OL study, the following imputation rules were applied: If the reason for missing data was death, a distance of zero (0) meters was imputed for all 6MWD visits from the date of death. For any other reasons, the last available value was carried forward.
Primary	Change in WHO Functional Class (FC) at Month 6 and 12	From baseline in DB parent study (AC-055-305, NCT01743001) up to month 12 in this OL study.	Class I: no symptoms with exercise or at rest. No limitation of activity. Class II: No symptoms at rest but slight limitation with ordinary activities causing symptoms (e.g. short of breath with climbing stairs). Class III: may not have symptoms at rest but activities greatly limited by shortness of breath, fatigue, or near fainting. Class IV: symptoms at rest and inability to carry out any physical activity without symptoms. Patients in class IV manifest signs of right heart failure. For missing WHO FC values in the OL study, the following imputation rules were applied: If the reason for missing data was death, class IV was imputed for all WHO visits from the date of death. For any other reasons, the last available value was carried forward.
Primary	Change in Borg Dyspnea Score at Month 6 and 12	From baseline in DB parent study (AC-055-305, NCT01743001) up to month 12 in this OL study.	The Borg dyspnea score rates the severity of dyspnea (difficult or labored breathing) on a scale from 0 ('Nothing at all') to 10 ('Very, very severe - maximal'). For missing Borg dyspnea index values in the OL study, the following imputation rules were applied: If the reason for missing data was death, a value of 10 was imputed for all Borg visits from the date of death. For any other reasons, the last available value was carried forward.
Primary	Change in Peripheral Oxygen Saturation (SpO2) at Rest at Month 6 and 12	From baseline in DB parent study (AC-055-305, NCT01743001) up to month 12 in this OL study.	No imputation of missing data for SpO2 was applied. Oxygen saturation assessed by pulse oximetry: peripheral oxygen saturation (SpO2) at rest before the 6-minute walk test (6MWT)

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 in Exercise Capacity as Measured by 6-minute Walking Distance (6MWD) Month 6 and 12	From baseline in DB parent study (AC-055-305, NCT01743001) up to month 12 in this OL study.		meter (m)	MEAN
PRIMARY	Change in WHO Functional Class (FC) at Month 6 and 12	From baseline in DB parent study (AC-055-305, NCT01743001) up to month 12 in this OL study.		Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Change in Borg Dyspnea Score at Month 6 and 12	From baseline in DB parent study (AC-055-305, NCT01743001) up to month 12 in this OL study.		score on a scale	MEAN
PRIMARY	Change in Peripheral Oxygen Saturation (SpO2) at Rest at Month 6 and 12	From baseline in DB parent study (AC-055-305, NCT01743001) up to month 12 in this OL study.	No imputation of missing data for oxygen saturation as assessed by SpO2 was applied. Therefore out of 109 subjects 104 subjects were analyzed at month 6 and 92 subjects at month 12 in the DB-macitentan group. In the DB-placebo group out of 108 subjects 103 subjects were analyzed at month 6 and 84 subjects at month 12.	% of oxygen saturation at rest	MEAN

Source ClinicalTrials.gov

Results by group86

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

Outcome	Group	Category	Value	Spread	Units
Change in Exercise Capacity as Measured by 6-minute Walking Distance (6MWD) Month 6 and 12	DB-macitentan	6MWD at DB study baseline	370.6	Standard Deviation 74.1	meter (m)
Change in Exercise Capacity as Measured by 6-minute Walking Distance (6MWD) Month 6 and 12	DB-placebo	6MWD at DB study baseline	381.6	Standard Deviation 76.7	meter (m)
Change in Exercise Capacity as Measured by 6-minute Walking Distance (6MWD) Month 6 and 12	DB-macitentan	6MWD at Week 16 in DB study	395.1	Standard Deviation 88.4	meter (m)
Change in Exercise Capacity as Measured by 6-minute Walking Distance (6MWD) Month 6 and 12	DB-placebo	6MWD at Week 16 in DB study	399.9	Standard Deviation 80.6	meter (m)
Change in Exercise Capacity as Measured by 6-minute Walking Distance (6MWD) Month 6 and 12	DB-macitentan	Change in 6MWD from DB study baseline to Week 16	24.4	Standard Deviation 71.0	meter (m)
Change in Exercise Capacity as Measured by 6-minute Walking Distance (6MWD) Month 6 and 12	DB-placebo	Change in 6MWD from DB study baseline to Week 16	18.2	Standard Deviation 53.0	meter (m)
Change in Exercise Capacity as Measured by 6-minute Walking Distance (6MWD) Month 6 and 12	DB-macitentan	6MWD at Month 6 in OL study	396.8	Standard Deviation 96.5	meter (m)
Change in Exercise Capacity as Measured by 6-minute Walking Distance (6MWD) Month 6 and 12	DB-placebo	6MWD at Month 6 in OL study	425.0	Standard Deviation 72.1	meter (m)
Change in Exercise Capacity as Measured by 6-minute Walking Distance (6MWD) Month 6 and 12	DB-macitentan	Change in 6MWD from DB study baseline to Month 6	26.2	Standard Deviation 77.9	meter (m)
Change in Exercise Capacity as Measured by 6-minute Walking Distance (6MWD) Month 6 and 12	DB-placebo	Change in 6MWD from DB study baseline to Month 6	43.4	Standard Deviation 51.5	meter (m)

1 to 10 of 86Source ClinicalTrials.gov

See all 86 results by group

Participant flow8

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

Period	Milestone	Group	Participants
Overall Study	STARTED	DB-macitentan	109
Overall Study	STARTED	DB-placebo	108
Overall Study	Treated With 10 mg Macitentan	DB-macitentan	109
Overall Study	Treated With 10 mg Macitentan	DB-placebo	108
Overall Study	COMPLETED	DB-macitentan	92
Overall Study	COMPLETED	DB-placebo	99
Overall Study	NOT COMPLETED	DB-macitentan	17
Overall Study	NOT COMPLETED	DB-placebo	9

Source ClinicalTrials.gov

Adverse events79

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	All-enrolled Analysis Set	20	217	27
other	Chest pain	General disorders	All-enrolled Analysis Set	13	217	13
other	Cough	Respiratory, thoracic and mediastinal disorders	All-enrolled Analysis Set	23	217	28
other	Diarrhoea	Gastrointestinal disorders	All-enrolled Analysis Set	18	217	20
other	Dizziness	Nervous system disorders	All-enrolled Analysis Set	18	217	20
other	Fatigue	General disorders	All-enrolled Analysis Set	11	217	11
other	Haemoglobin decreased	Investigations	All-enrolled Analysis Set	20	217	29
other	Haemoptysis	Respiratory, thoracic and mediastinal disorders	All-enrolled Analysis Set	24	217	42
other	Headache	Nervous system disorders	All-enrolled Analysis Set	29	217	34
other	Oedema peripheral	General disorders	All-enrolled Analysis Set	14	217	15

1 to 10 of 79Source ClinicalTrials.gov

See all 79 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	All-enrolled Analysis Set	7	217
Total, other adverse events	All-enrolled Analysis Set	152	217
Total, serious adverse events	All-enrolled Analysis Set	62	217

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

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.

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/NCT01739400

GET https://conductscience.org/api/trials/v1/NCT01739400/results?page=2

API reference For AI assistants