Phase 2 N=142 Randomized Quadruple-blind Treatment

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

Heart Failure With Preserved Ejection Fraction

Bottom Line

View on ClinicalTrials.gov: NCT03153111 ↗

Enrolled (actual)

142

Serious AEs

36.6%

Results posted

Apr 2022

Primary outcome: Primary: Percent of Baseline N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) Assessed at Week 24 — 108.39; 106.27 percentage of baseline NT-proBNP — p=0.7923

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Macitentan (Drug); Placebo (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Actelion
Primary completion: Mar 2021

Outcome Measures

Outcome	Result	p-value
PRIMARY Percent of Baseline N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) Assessed at Week 24	108.39; 106.27	0.7923
SECONDARY Change From Baseline to Week 24 in the Clinical Summary Score Assessed by Kansas City Cardiomyopathy Questionnaire (KCCQ) Score	-2.37; 0.89	0.2172
SECONDARY Change From Baseline to Week 24 in Accelerometer-assessed Proportion of Time Spent in Light to Vigourous Physical Activity	-0.024; -0.005	0.3665
SECONDARY Number of Participants With Worsening of Heart Failure (WHF) Events Over 52 Weeks	12; 5; 14; 6; 17; 9	—

Summary

This is a study to evaluate whether macitentan is an effective and safe treatment for patients with heart failure with preserved ejection fraction (HFpEF) and pulmonary vascular disease. The primary objective is to evaluate whether macitentan 10 mg reduces N-terminal pro-brain natriuretic peptide (NT-pro-BNP) as compared to placebo in these patients.

Eligibility Criteria

Inclusion Criteria

Signs or symptoms of Heart Failure (HF) (NYHA FC I I and II I ) requiring treatment with at least one oral diuretic (any type)
Left ventricular ejection fraction (LVEF) ≥ 40% (by echocardiography at Screening)
Structural heart disease consistent with heart failure with preserved ejection fraction (HFpEF) established by echocardiography at Screening
Elevated NT-proBNP
Pulmonary vascular disease or right ventricular dysfunction

Exclusion Criteria

Any prior valid measurement of LVEF < 40%. An echocardiogram is considered valid if its quality is sufficient to allow accurate assessment of LVEF and if it is reflective of the true status of the subject
Cardiovascular co-morbidities (e.g., significant unrepaired structural valvular heart disease; acute coronary syndrome, coronary artery bypass graft (CABG) or percutaneous coronary intervention (PCI) within 3 months of Screening; uncontrolled heart rate from atrial fibrillation or atrial flutter, history of serious life-threatening or hemodynamically significant arrhythmia; history of or anticipated heart transplant or ventricular assist device implantation, etc)
Systolic blood pressure (SBP) ≥ 180 mmHg, or diastolic blood pressure (DBP) ≥ 110 mmHg during Screening
Hemoglobin < 100g/L (< 10 g/dl) at Screening
Significant parenchymal lung disease (e.g., severe COPD, moderate or severe restrictive lung disease, diffuse interstitial fibrosis or alveolitis, pulmonary thromboembolism)
Severe renal dysfunction with an estimated Glomerular Filtration Rate (eGFR) < 30 mL/min per 1.73 m2
Severe hepatic impairment, e.g., Child Pugh Class C

Other protocol-defined inclusion/exclusion criteria may apply

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03153111). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.