PORtopulmonary Hypertension Treatment wIth maCitentan - a randOmized Clinical Trial
- Registration Number
- NCT02382016
- Lead Sponsor
- Actelion
- Brief Summary
24-week study to evaluate the efficacy and safety of macitentan for the treatment of portopulmonary hypertension.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 85
- Male or female of at least 18 years of age
- Confirmed diagnosis of portopulmonary hypertension
Main
- Severe hepatic impairment
- Severe obstructive or restrictive lung disease
- Pulmonary veno-occlusive disease
- Systolic blood pressure (SBP) < 90 mmHg at Screening
- ALT/AST >= 3 x ULN
- Bilirubin >= 3 mg/dL at Screening
- Any known factor or disease that might interfere with treatment compliance, study conduct, or interpretation of results
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebo Matching placebo tablet once daily. Investigational treatment Macitentan Macitentan film-coated tablet 10 mg once daily.
- Primary Outcome Measures
Name Time Method Relative Change From Baseline to Week 12 in Pulmonary Vascular Resistance (PVR). From enrollment/baseline to Week 12 in the Double Blind (DB) treatment period The relative change from baseline to Week 12 in PVR is expressed as a ratio of Week 12 to baseline PVR.
- Secondary Outcome Measures
Name Time Method Change From Baseline to Week 12 in the Biomarker N-terminal Pro B-type Natriuretic Peptide (NT-proBNP) From enrollment/baseline to Week 12 in the DB treatment period NT-proBNP functions as a strong indicator of prognosis in patients with pulmonary hypertension (PH). The relative change from baseline to Week 12 in NT-proBNP is expressed as a ratio of Week 12 to baseline NT-proBNP.
Change From Baseline to Week 12 in Mean Right Atrial Pressure (mRAP) From enrollment/baseline to Week 12 in the DB treatment period mRAP is the mean blood pressure in the right atrium of the heart.
Change From Baseline to Week 12 in Mixed Venous Oxygen Saturation (SVO2) From enrollment/baseline to Week 12 in the DB treatment period SVO2 help assess tissue oxygen delivery. It describes the percentage of oxygen bound to hemoglobin in the blood which returns to the heart. This reflects the amount of residual oxygen in the blood after oxygen extraction by the tissues throughout the body.
Change From Baseline to Week 12 in Cardiac Index From enrollment/baseline to Week 12 in the DB treatment period The cardiac index is an assessment of the function of the heart and relates the cardiac output to the patient's body size (the patient's body surface area).
Change From Baseline to Week 12 in Total Pulmonary Resistance (TPR) From enrollment/baseline to Week 12 in the DB treatment period TPR is the resistance the pulmonary circulation that must be overcome in order for the blood flow to occur. It takes into account the blood pressure in the pulmonary arteries and the cardiac output. It is an important measurement to monitor the function of the pulmonary circulation and detect disease progression or improvement.
Change From Baseline to Week 12 in 6-minute Walk Distance (6MWD) From enrollment/baseline to Week 12 in the DB treatment period 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.
Change From Baseline to Week 12 in WHO Functional Class (FC) From enrollment/baseline to Week 12 in the DB treatment period Changes from baseline to Week 12 in WHO FC were dichotomized as worsening (i.e., change \> 0) versus no change or improvement (i.e., change ≤ 0). 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 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. Class IV: symptoms at rest (e.g. dyspnea and/or fatigue) and inability to carry out any physical activity without symptoms. Patients in class IV manifest signs of right heart failure.
Change From Baseline to Week 12 in Mean Pulmonary Artery Pressure (mPAP) From enrollment/baseline to Week 12 in the DB treatment period mPAP is the mean blood pressure inside the pulmonary artery which moves the blood from the heart to the lungs. Monitoring of mPAP can detect small changes in the function of the heart.
Trial Locations
- Locations (51)
David Geffen School of Medicine, UCLA
🇺🇸Los Angeles, California, United States
Lékařská fakulta a Všeobecná fakultní nemocnice v Praze, II. Interní klinika kardiologie a angiologie U
🇨🇿Prague, Czechia
Hospital Universitario12 Octubre
🇪🇸Madrid, Spain
IKEM (Institut klinické a experimentální medicíny, Institute for Clinical and Experimental Medicine)
🇨🇿Prague, Czechia
The Royal Free Hospital/ Cardiology Department
🇬🇧London, United Kingdom
Sheffield Teaching Hospitals NHS Foundation Trust Royal Hallamshire Hospital
🇬🇧Sheffield, United Kingdom
Thoraxklinik des Universitätsklinikums Heidelberg Zentrum für pulmonale Hypertonie Studienkoordination
🇩🇪Heidelberg, Germany
Hospital Clinico i Provincial Servicio de Neumología
🇪🇸Barcelona, Spain
Boston University
🇺🇸Boston, Massachusetts, United States
Kentuckiana Pulmonary Associates
🇺🇸Louisville, Kentucky, United States
University of Miami
🇺🇸Miami, Florida, United States
Universitätsklinikum Carl Gustav Carus an der Technischen Universität Dresden
🇩🇪Dresden, Germany
Keck School of Medicine
🇺🇸Los Angeles, California, United States
UCSD
🇺🇸La Jolla, California, United States
Emory Healthcare
🇺🇸Atlanta, Georgia, United States
Piedmont Healthcare
🇺🇸Austell, Georgia, United States
Northwestern University
🇺🇸Chicago, Illinois, United States
Indiana University
🇺🇸Indianapolis, Indiana, United States
University of New Mexico Health Science Center
🇺🇸Albuquerque, New Mexico, United States
University of Pennsylvania
🇺🇸Philadelphia, Pennsylvania, United States
Baylor University Medical Center
🇺🇸Dallas, Texas, United States
Cleveland Clinic - Department of Cardiovascular Medicine
🇺🇸Cleveland, Ohio, United States
UPMC Montefiore
🇺🇸Pittsburgh, Pennsylvania, United States
University of Texas Southwestern Medical Center Division of NeuroCritical Care 75390-8550
🇺🇸Dallas, Texas, United States
University of Colorado Health Sciences Center Aurora
🇺🇸Aurora, Colorado, United States
Mayo Clinic -Clinical Studies Unit
🇺🇸Phoenix, Arizona, United States
UCSF
🇺🇸San Francisco, California, United States
Henry Ford Hospital
🇺🇸Detroit, Michigan, United States
University of Cincinnati
🇺🇸Cincinnati, Ohio, United States
Methodist Hospital
🇺🇸San Antonio, Texas, United States
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
Medizinische Hochschule Hannover, Abteilung Pneumologie
🇩🇪Hannover, Germany
CHU Toulouse - Hôpital Larrey Hôpital de Jour et Semaine
🇫🇷Toulouse, France
CHRU Hôpital Cavale Blanche Brest
🇫🇷Brest, Bretagne, France
Universitätsklinikum Giessen und Marburg GmbH Justus-Liebig Universität Giessen 35392
🇩🇪Giessen, Germany
Mayo Clinic Florida - Pulmonary Dept.
🇺🇸Jacksonville, Florida, United States
Hôpital Kremlin Bicêtre Service de Pneumologie
🇫🇷Paris, France
CHU Côte de Nacre, Service de Pneumologie
🇫🇷Caen, France
Hôpital cardiologique, Service de cardiologie 59037 Lille Cedex"
🇫🇷Lille, France
Hôpital Pontchaillou - CHU Rennes Service de Cardiologie et des Maladies Vasculaires Rennes Cedex 9 35033"
🇫🇷Rennes, France
University of Florida - Divison of Pulmonary Critical Care & Sleep
🇺🇸Gainesville, Florida, United States
Servico de Hipertensao Pulmonar - Complexo Hospitalar Santa Casa Irmandade Santa de Misericordia de Porto Alegre
🇧🇷Porto Alegre, Brazil
Universitätsklinikum Leipzig, Department Innere Medizin, Abteilung Pneumologie
🇩🇪Leipzig, Germany
University of Florida College of Medicine
🇺🇸Jacksonville, Florida, United States
South Miami Hospital
🇺🇸South Miami, Florida, United States
Hôpital Cardiologique et Pneumologique Louis Pradel 69677 Bron cedex"
🇫🇷Lyon, France
NHS Greater Glasgow and Clyde Trust
🇬🇧Glasgow, Scotland, United Kingdom
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo (HC-FMUSP)
🇧🇷Sao Paulo, Brazil
CHRU de Grenoble, Hôpital Albert Michallon
🇫🇷Grenoble, Rhône- Alpes, France
CHU Rouen
🇫🇷Rouen, France
University of Wisconsin-Madison
🇺🇸Madison, Wisconsin, United States