PORtopulmonary Hypertension Treatment wIth maCitentan - a randOmized Clinical Trial

Phase 4

Completed

Conditions: Portopulmonary Hypertension

Interventions: Other: Placebo
Drug: Macitentan

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

Inclusion Criteria

Male or female of at least 18 years of age
Confirmed diagnosis of portopulmonary hypertension

Main

Exclusion Criteria

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): Mayo Clinic -Clinical Studies Unit
🇺🇸
Phoenix, Arizona, United States
UCSD
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La Jolla, California, United States
David Geffen School of Medicine, UCLA
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Los Angeles, California, United States
Keck School of Medicine
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Los Angeles, California, United States
UCSF
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San Francisco, California, United States
University of Colorado Health Sciences Center Aurora
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Aurora, Colorado, United States
University of Florida - Divison of Pulmonary Critical Care & Sleep
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Gainesville, Florida, United States
University of Florida College of Medicine
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Jacksonville, Florida, United States
Mayo Clinic Florida - Pulmonary Dept.
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Jacksonville, Florida, United States
University of Miami
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Miami, Florida, United States
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Mayo Clinic -Clinical Studies Unit
🇺🇸Phoenix, Arizona, United States