Clinical Study to Evaluate the Effects of Macitentan on Exercise Capacity in Subjects With Eisenmenger Syndrome

Completed · Phase 3 · Has a placebo group

Conditions studied: Pulmonary Arterial Hypertension

In brief

Clinical study to assess the efficacy, safety, and tolerability of macitentan in subjects with Eisenmenger Syndrome.

Key facts

Study ID

NCT01743001

Run by

Actelion

People needed

226

Starts

2013-05-21

Expected to finish

2016-12-01

Last updated by the study team

2018-02-23

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Subjects:
• not participating in the hemodynamic sub-study: males or females ≥ 12 years of age.
• participating in the hemodynamic sub-study: males or females ≥ 18 years of age.
• Subjects (including those with Down Syndrome [DS]) with confirmed Eisenmenger Syndrome [ES] (European Society of Cardiology [ESC] and the European Respiratory Society [ERS] guidelines):
• Established by echocardiography as:
• Large congenital shunting defect at atrial, ventricular or arterial level*
• and right to left shunt or bi-directional shunt with prevalent right to left direction.
• Resting peripheral oxygen saturation (SpO2) ≤ 90% and > 70% (pulse oximetry, room air).
• The lower limit is 65% if a subject is living at an altitude greater than 2500 m above sea level.
• Subjects with any of the following open defects are eligible for the study either as an isolated defect or in combination:
• atrial septal defect (ASD)
• ventricular septal defect (VSD)
• partial or complete atrioventricular septal defect (AVSD)
• patent ductus arteriosus (PDA)
• aortopulmonary window (AP window)
• total or partial anomalous pulmonary venous return (TAPVR, PAPVR) The defects may be either unoperated or previously palliated surgically (provided significant residual defect remains).
• The Steering Committee will review the echocardiography data of all subjects (main study and sub study) to confirm eligibility prior to Randomization.
• Subjects with the following findings at cardiac catheterization:
• Mean resting pulmonary arterial pressure (mPAP) > 25 mmHg
• Pulmonary capillary wedge pressure (PCWP) or mean left atrial pressure (LAP) or left ventricular end diastolic pressure (LVED) ≤ 15 mmHg
• Pulmonary vascular resistance (PVR) ≥ 800 dyn∙s/cm5 or ≥ 10 Wood units
• Subjects with WHO functional class ≥ II.
• Subjects able to reliably perform the the 6-minute walk test (6MWT) with a minimum distance of 50 m and a maximum distance of 450 m.

You may not qualify if…

• Main study and hemodynamic sub-study: Any of the following conditions previously known or identified via cardiac catheterization or echocardiography:
• Pulmonary arterial or venous stenosis > 25% size of native pulmonary artery (PA) or pulmonary vein
• Severe tricuspid regurgitation in the setting of left to right shunt at the ventricular or atrial level
• Greater than mild tricuspid stenosis
• Intracavitary RV outflow obstruction
• Greater than mild mitral stenosis
• Intracavitary LV outflow obstruction
• Subvalvular or supravalvular aortic stenosis
• Aortic coarctation
• Greater than moderate mitral regurgitation
• Recognized extracardiac systemic venous collaterals to the pulmonary venous circulation
• Recognized hepatic wedge pressure-inferior vena cava pressure gradient >12 mm Hg
• PCWP "v" waves >20 mmHg
• Tetralogy of Fallot
• Truncus arteriosus
• Interrupted aortic arch
• Transposition of great arteries
• Single ventricle defects: absent AV connection (mitral or tricuspid atresia), double inlet AV connections left or right ventricle, functional univentricular heart (unbalanced AVSD, hypoplastic RV, double outlet RV), hypoplastic left heart syndrome
• Ebstein's anomaly
• Severe aortic regurgitation
• Pulmonary atresia
• PAPVR or TAPVR, ONLY if there is lung hypoplasia or if documentation confirming the absence of lung hypoplasia does not exist.
• For subjects participating in the hemodynamic sub-study the following will also be considered exclusion criteria:
• SVC stenosis >25% size of native vessel
• PDA, AP window, TAPVR, PAPVR, or ASD sinus venosus with anomalous pulmonary veins

Where it is running

• Stanford Hospital and Clinic — Palo Alto, California, United States
• Emory University Hospital/the Emory Clinic — Atlanta, Georgia, United States
• Barnes-Jewish Hosp/Wash Univ School of Med — St Louis, Missouri, United States
• Children'S Heart Center Nevada — Las Vegas, Nevada, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Texas Children'S Hosp - Dept of Cardiology — Houston, Texas, United States
• Gen Hosp Univ Vienna Dept Cardiology — Vienna, Austria
• Mhat Nat Card Hosp - Cardiology Clinic — Sofia, Bulgaria
• Mhat Nat Card Hosp - Pediatric Clin / Ped Card Dept — Sofia, Bulgaria
• Mhat Sveta Anna Clin Card — Sofia, Bulgaria
• Inst Nat Torax, Unidad Cardiopatia Congenitas Del Adulto — Providencia, Chile
• Clinica Tabancura - Cardio Unit — Santiago, Chile
• Guangdong General Hospital, Cardiology Dpt — Guangzhou, Guangdong, China
• Wu Han Asia Heart Hosp — Wuhan, Hubei, China
• The General Hosp of Shenyang Military Region — Shenyang, Liaoning, China
• Beijing Anzhen Hospital, Cardiology Dpt — Beijing, China
• Cardiovascular Institute&Fuwai Hospital — Beijing, China
• Shanghai Pulmonary Hospital, Dept of Pulmonary Circulation — Shanghai, China
• Hosp La Timone - Dept Pediatric Cardiology — Marseille, France
• Hosp Laennec - Dept Cardiology — Nantes, France
• Hosp Pompidou - Dept Congenital Cardiac Diseases — Paris, France
• Hosp Cardiology Haut Leveque - Dept Congenital Diseases — Pessac, France
• Herzzentrum Berlin, Ped Cardiology — Berlin, Germany
• Universitätsklinikum Giessen - Pediatric Heart Center — Giessen, Germany
• Ahmanson/UCLA Heart Disease Center — Los Angeles, California, United States

Full record on ClinicalTrials.gov

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