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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

Phase 2
Completed
Conditions
Heart Failure With Preserved Ejection Fraction
Interventions
Drug: Placebo
Registration Number
NCT03153111
Lead Sponsor
Actelion
Brief 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.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
143
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

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PlaceboPlaceboSubjects randomized to the placebo arm received one tablet of placebo every day for at least 24 to maximum 52 weeks.
MacitentanMacitentanSubjects randomized to the macitentan arm receives one tablet of macitentan 10 mg every day for at least 24 to maximum 52 weeks.
Primary Outcome Measures
NameTimeMethod
Percent of Baseline N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) Assessed at Week 24Week 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 Outcome Measures
NameTimeMethod
Change From Baseline to Week 24 in Accelerometer-assessed Proportion of Time Spent in Light to Vigourous Physical ActivityBaseline 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.

Number of Participants With Worsening of Heart Failure (WHF) Events Over 52 WeeksWeeks 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.

Change From Baseline to Week 24 in the Clinical Summary Score Assessed by Kansas City Cardiomyopathy Questionnaire (KCCQ) ScoreBaseline 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.

Trial Locations

Locations (95)

Bnai Zion Medical Center

🇮🇱

Haifa, Israel

Wolfson Medical Center

🇮🇱

Holon, Israel

Hadassah Medical Center

🇮🇱

Jerusalem, Israel

Galilee Medical Center

🇮🇱

Nahariya, Israel

Rabin Medical Center Beilinson Campus

🇮🇱

Petah Tikva, Israel

Kaplan Medical Center

🇮🇱

Rehovot, Israel

Uniwersyteckie Centrum Kliniczne

🇵🇱

Gdansk, Poland

Krakowski Szpital Specjalityczny im Jana Pawla II Oddzial Kliniczny Chorob Serca i Naczyn

🇵🇱

Krakow, Poland

Wojewodzki Szpital Specjalistyczny im Stefana Kardynala Wyszynskiego SPZOZ

🇵🇱

Lublin, Poland

4 Wojskowy Szpital Kliniczny Z Poliklinika SP ZOZ

🇵🇱

Wroclaw, Poland

Institutul de urgenta pentru Boli Cardiovasculare Prof. Dr. C.C. Iliescu

🇷🇴

Bucuresti, Romania

Cardiomed

🇷🇴

Craiova, Romania

SAL MED Pitesti

🇷🇴

Pitesti, Romania

Cmi Dr Podoleanu Cristian

🇷🇴

Targu-Mures, Romania

Diagnostic Consulting Center I Sliven

🇧🇬

Sliven, Bulgaria

Multiprofile Hospital for Active Treatment - National Heart Hospital

🇧🇬

Sofia, Bulgaria

University Multiprofile Hospital for Active Treatment- UMHAT Sveta Anna AD

🇧🇬

Sofia, Bulgaria

Fakultni nemocnice Brno

🇨🇿

Brno, Czechia

Fakultni nemocnice Hradec Kralove

🇨🇿

Hradec Králové, Czechia

University Hospital Plzeň-Lochotín

🇨🇿

Plzen, Czechia

General University Hospital II.department of Internal Medicine-cardiology and angiology

🇨🇿

Praha 2, Czechia

IKEM

🇨🇿

Praha 4, Czechia

Aarhus Universitetshospital

🇩🇰

Aarhus, Denmark

Bispebjerg Og Frederiksberg Hospital

🇩🇰

Copenhagen, Denmark

Sydvestjysk Sygehus

🇩🇰

Esbjerg, Denmark

Hopital Henri Mondor

🇫🇷

Creteil Cedex, France

CHU de Grenoble Hopital Albert Michallon

🇫🇷

Grenoble Cedex 9, France

Hopital de Bicetre

🇫🇷

Le Kremlin Bicetre, France

Hopital Cardiologique

🇫🇷

Lille Cedex, France

CHU Rouen Hopital Charles Nicolle

🇫🇷

Rouen Cedex, France

Charite Universitatsmedizin Berlin Campus Virchow Klinikum

🇩🇪

Berlin, Germany

Universitatsklinikum Bonn

🇩🇪

Bonn, Germany

Universitätsklinikum Carl Gustav Carus Medizinische Klinik und Poliklinik 1-Pneumologie

🇩🇪

Dresden, Germany

Universitaetsklinikum Giessen

🇩🇪

Giessen, Germany

Universitaetsklinikum Schleswig Holstein Campus Kiel

🇩🇪

Kiel, Germany

Herzzentrum Uniklinik Köln Klinik III für Innere Medizin

🇩🇪

Köln, Germany

Klinikum Würzburg Mitte gGmbH Standort Missioklinik

🇩🇪

Würzburg, Germany

Budai Irgalmasrendi Korhaz

🇭🇺

Budapest, Hungary

Semmelweis Egyetem Varosmajor Sziv es Ergyogyaszati Klinika

🇭🇺

Budapest, Hungary

Magyar Honvedseg Egeszsegugyi Kozpont

🇭🇺

Budapest, Hungary

Barzilai Medical Center

🇮🇱

Ashkelon, Israel

Hillel Yaffe Medical Center

🇮🇱

Hadera, Israel

Ekaterinburg City Clinical Hospital #14

🇷🇺

Ekaterinburg, Russian Federation

Federal State Budget Scientific Institution

🇷🇺

Kemerovo, Russian Federation

Krasnodar Regional Clinical Hospital

🇷🇺

Krasnodar, Russian Federation

Moscow City Clinical Hospital No.51

🇷🇺

Moscow, Russian Federation

National Medical Research Center of Cardiology of MoH of Russian Federation

🇷🇺

Moscow, Russian Federation

State Healthcare Institution, Regional Clinical Cardiology Dispensary

🇷🇺

Saratov, Russian Federation

Federal State Budgetary Institution

🇷🇺

St Petersburg, Russian Federation

Hospital Universitario La Fe

🇪🇸

Valencia, Spain

Sahlgrenska Universitetsjukhuset

🇸🇪

Goteborg, Sweden

State Autonomous Healthcare Institution Of Yaroslavl Region

🇷🇺

Yaroslavl, Russian Federation

Hospital Vall d'Hebron

🇪🇸

Barcelona, Spain

Hospital General Universitario Gregorio Maranon

🇪🇸

Madrid, Spain

Hospital Clinico San Carlos

🇪🇸

Madrid, Spain

Hospital Clinico Universitario de Valencia

🇪🇸

Valencia, Spain

Vascular Medicine Institute University Of Pittsburgh

🇺🇸

Pittsburgh, Pennsylvania, United States

AnMed Health

🇺🇸

Anderson, South Carolina, United States

North Dallas Research Associates

🇺🇸

McKinney, Texas, United States

Baylor Scott and White Research Institute

🇺🇸

Temple, Texas, United States

Inova Heart and Vascular Institute

🇺🇸

Falls Church, Virginia, United States

Clinica Adventista Belgrano

🇦🇷

Caba, Argentina

Centro Médico Dra. De Salvo

🇦🇷

Ciudad Autónoma de Buenos Aires, Argentina

Instituto de Investigaciones Clinicas Mar del Plata

🇦🇷

Mar Del Plata, Argentina

Medizinische Universitaet Wien

🇦🇹

Vienna, Austria

Maestri E Kormann Consultoria Médico- Científica Ltda

🇧🇷

Blumenau, Brazil

Rede D or Sao Luiz S A Hospital do Coracao do Brasil

🇧🇷

Brasilia, Brazil

Instituto de Pesquisa Clinica de Campinas

🇧🇷

Campinas, Brazil

Instituto do Coracao de Marília

🇧🇷

Marilia, Brazil

Instituto de Moléstias Cardiovasculares Tatuí

🇧🇷

Tatuí, Brazil

Specialized Hospital for Active Treatment in Cardiology - Pleven

🇧🇬

Pleven, Bulgaria

Royal Papworth Hospital NHS Foundation Trust

🇬🇧

Cambridge, United Kingdom

Mayo Clinic Arizona

🇺🇸

Phoenix, Arizona, United States

Cedars Sinai Heart Institute

🇺🇸

Beverly Hills, California, United States

Sharp Memorial Hospital

🇺🇸

San Diego, California, United States

Harbor Ucla Medical Center

🇺🇸

Torrance, California, United States

South Denver Cardiology Associates PC

🇺🇸

Littleton, Colorado, United States

Northwestern University Feinberg School of Medicine

🇺🇸

Chicago, Illinois, United States

University Of Iowa - Hospitals & Clinics

🇺🇸

Iowa City, Iowa, United States

University of Maryland

🇺🇸

Baltimore, Maryland, United States

Massachusetts General Hospital

🇺🇸

Boston, Massachusetts, United States

Washington University School Of Medicine

🇺🇸

Saint Louis, Missouri, United States

Weill Cornell Medicine

🇺🇸

New York, New York, United States

Peak Clinical Trials

🇺🇸

Apex, North Carolina, United States

University of North Carolina (UNC) - School of Medicine

🇺🇸

Chapel Hill, North Carolina, United States

The Lindner Clinical Trial Center

🇺🇸

Cincinnati, Ohio, United States

Lehigh Valley Health Network

🇺🇸

Allentown, Pennsylvania, United States

Drexel University College of Medicine

🇺🇸

Philadelphia, Pennsylvania, United States

Allegheny

🇺🇸

Pittsburgh, Pennsylvania, United States

MultiCare Health System

🇺🇸

Tacoma, Washington, United States

Aurora Saint Lukes Medical Center

🇺🇸

Milwaukee, Wisconsin, United States

CCBR - Buenos Aires - AR

🇦🇷

Buenos Aires, Argentina

Castle Hill Hospital

🇬🇧

Cottingham, United Kingdom

Royal Free Hospital

🇬🇧

London, United Kingdom

Sheffield Teaching Hospitals NHS Foundation Trust Royal Hallamshire Hospital

🇬🇧

Sheffield, United Kingdom

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