Trial · NCT03153111

A Study to Evaluate Whether Macitentan is an Effective and Safe Treatment for Patients With Heart Failure With Preserved Ejection Fraction and Pulmonary Vascular Disease

NCT03153111, Phase 2, Completed, Actelion; results first posted 2022-04-04

3 sourcestrials-2026-11-12-0.4.0

Cite this record

ConductScience Library. A Study to Evaluate Whether Macitentan is an Effective and Safe Treatment for Patients With Heart Failure With Preserved Ejection Fraction and Pulmonary Vascular Disease. Trials, record NCT03153111. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03153111

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Trial

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

Title: A Study to Evaluate Whether Macitentan is an Effective and Safe Treatment for Patients With Heart Failure With Preserved Ejection Fraction and Pulmonary Vascular Disease
NCT number: NCT03153111
Status: Completed
Phases: Phase 2
Enrollment: 143 participants, actual
Lead sponsor: Actelion
Conditions: Heart Failure With Preserved Ejection Fraction
Interventions: Macitentan, Placebo
First posted: 2017-05-15
Last updated: 2025-03-30
Other registries' IDs: eudract:2016-003653-15
Results first posted: 2022-04-04
Limitations and caveats: Recruitment was stopped prematurely in December 2019 due to slow enrollment which resulted in an underpowered study and impacted the meaningful interpretation of the results.

Source ClinicalTrials.gov

Registered outcomes4

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

List	Outcome	Time frame	Description
Primary	Percent of Baseline N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) Assessed at Week 24	Week 24	Percent of baseline NT-proBNP assessed at Week 24 was reported. Percent of baseline is calculated as the ratio of the Week 24 NT-proBNP value over baseline value, expressed in percentage. NT-proBNP is one of the best established cardiovascular response markers among all available surrogates in heart failure (HF).
Secondary	Change From Baseline to Week 24 in the Clinical Summary Score Assessed by Kansas City Cardiomyopathy Questionnaire (KCCQ) Score	Baseline to Week 24	The KCCQ is a validated health related quality of life measure for heart failure. The KCCQ is a 23-item, self-administered instrument that quantifies physical function, symptoms (frequency, severity and recent change), social function, self-efficacy and knowledge, and quality of life. Clinical summary score is one of the quality of life variable of interest derived from KCCQ. Clinical summary score is the mean of domains: physical limitations score (6 items) and total symptom score (2 items \[symptoms frequency and symptom burden\]). The score is calculated by summing domain responses and then transforming scores to a 0-100 unit scale with higher scores indicating better health status.
Secondary	Change From Baseline to Week 24 in Accelerometer-assessed Proportion of Time Spent in Light to Vigourous Physical Activity	Baseline to Week 24	Physical activity is assessed by accelerometer as the proportion of time spent in light to vigorous physical activity based on a threshold of greater than (\>)100 activity counts per minute and expressed as change from baseline to Week 24.
Secondary	Number of Participants With Worsening of Heart Failure (WHF) Events Over 52 Weeks	Weeks 16, 24, 36, 52	Number of participants with WHF events were reported. A WHF event includes HF death, hospitalization for WHF or an urgent visit for WHF.

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	Percent of Baseline N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) Assessed at Week 24	Week 24	Full analysis set (FAS) included participants which were randomized to double-blind study treatment.	percentage of baseline NT-proBNP	GEOMETRIC_MEAN
SECONDARY	Change From Baseline to Week 24 in the Clinical Summary Score Assessed by Kansas City Cardiomyopathy Questionnaire (KCCQ) Score	Baseline to Week 24	FAS included participants which were randomized to double-blind study treatment. Here, 'N' (number of participants analyzed) specifies all participants who were evaluated for this outcome measure (OM).	score on a scale	MEAN
SECONDARY	Change From Baseline to Week 24 in Accelerometer-assessed Proportion of Time Spent in Light to Vigourous Physical Activity	Baseline to Week 24	FAS included participants which were randomized to double-blind study treatment. Here, 'N' (number of participants analyzed) specifies all participants who were evaluated for this OM.	proportion of time spent	MEAN
SECONDARY	Number of Participants With Worsening of Heart Failure (WHF) Events Over 52 Weeks	Weeks 16, 24, 36, 52	FAS included participants which were randomized to double-blind study treatment.	Participants	COUNT_OF_PARTICIPANTS

Source ClinicalTrials.gov

Results by group14

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

Outcome	Group	Category	Value	Spread	Units
Percent of Baseline N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) Assessed at Week 24	Macitentan		108.39	Geometric Coefficient of Variation 0.65	percentage of baseline NT-proBNP
Percent of Baseline N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) Assessed at Week 24	Placebo		106.27	Geometric Coefficient of Variation 0.55	percentage of baseline NT-proBNP
Change From Baseline to Week 24 in the Clinical Summary Score Assessed by Kansas City Cardiomyopathy Questionnaire (KCCQ) Score	Macitentan		-2.37	Standard Deviation 16.12	score on a scale
Change From Baseline to Week 24 in the Clinical Summary Score Assessed by Kansas City Cardiomyopathy Questionnaire (KCCQ) Score	Placebo		0.89	Standard Deviation 17.72	score on a scale
Change From Baseline to Week 24 in Accelerometer-assessed Proportion of Time Spent in Light to Vigourous Physical Activity	Macitentan		-0.024	Standard Deviation 0.084	proportion of time spent
Change From Baseline to Week 24 in Accelerometer-assessed Proportion of Time Spent in Light to Vigourous Physical Activity	Placebo		-0.005	Standard Deviation 0.098	proportion of time spent
Number of Participants With Worsening of Heart Failure (WHF) Events Over 52 Weeks	Macitentan	Week 16	12		Participants
Number of Participants With Worsening of Heart Failure (WHF) Events Over 52 Weeks	Placebo	Week 16	5		Participants
Number of Participants With Worsening of Heart Failure (WHF) Events Over 52 Weeks	Macitentan	Week 24	14		Participants
Number of Participants With Worsening of Heart Failure (WHF) Events Over 52 Weeks	Placebo	Week 24	6		Participants

1 to 10 of 14Source ClinicalTrials.gov

See all 14 results by group

Participant flow6

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Macitentan	71
Overall Study	STARTED	Placebo	71
Overall Study	COMPLETED	Macitentan	60
Overall Study	COMPLETED	Placebo	62
Overall Study	NOT COMPLETED	Macitentan	11
Overall Study	NOT COMPLETED	Placebo	9

Source ClinicalTrials.gov

Adverse events310

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	Anaemia	Blood and lymphatic system disorders	Macitentan	5	71
other	Anaemia	Blood and lymphatic system disorders	Placebo	3	71
other	Blood Loss Anaemia	Blood and lymphatic system disorders	Macitentan	2	71
other	Blood Loss Anaemia	Blood and lymphatic system disorders	Placebo	1	71
other	Hypochromic Anaemia	Blood and lymphatic system disorders	Macitentan	2	71
other	Hypochromic Anaemia	Blood and lymphatic system disorders	Placebo	0	71
other	Iron Deficiency Anaemia	Blood and lymphatic system disorders	Macitentan	4	71
other	Iron Deficiency Anaemia	Blood and lymphatic system disorders	Placebo	1	71
other	Normocytic Anaemia	Blood and lymphatic system disorders	Macitentan	2	71
other	Normocytic Anaemia	Blood and lymphatic system disorders	Placebo	1	71

1 to 10 of 310Source ClinicalTrials.gov

See all 310 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Macitentan	2	71
Total, all-cause mortality	Placebo	5	71
Total, other adverse events	Macitentan	57	71
Total, other adverse events	Placebo	54	71
Total, serious adverse events	Macitentan	29	71
Total, serious adverse events	Placebo	23	71

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
40066571	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/NCT03153111

GET https://conductscience.org/api/trials/v1/NCT03153111/events?page=2

API reference For AI assistants