Study to evaluate if macitentan is efficient, safe and tolerable enough to be used for treatment of Eisenmenger syndrome.

Phase 1

• Conditions: Eisenmenger Syndrome; MedDRA version: 14.1Level: PTClassification code 10058554Term: Eisenmenger's syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]

• Registration Number: EUCTR2012-003335-33-HU
• Lead Sponsor: Actelion Pharmaceuticals Ltd.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-04-30
• Last Update Posted: 16 January 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 220

Inclusion Criteria

- Males or females = 18 and = 70 years of age.

- Subjects with confirmed ES (European Society of Cardiology [ESC] and the European Respiratory Society [ERS] guidelines):
• Established by echocardiography as:
o Isolated ASD > 2 cm in diameter,
o or isolated VSD > 1 cm in diameter,
o or presence of both ASD and VSD (with either VSD = 1 cm in diameter and ASD > 2 cm in diameter, or VSD > 1 cm in diameter and ASD = 2 cm in diameter, or VSD > 1 cm in diameter and ASD > 2 cm in diameter),
o and right to left shunt or bi-directional shunt with prevalent right to left direction.
• Unoperated, or previously palliated surgically for defects mentioned above (incomplete closure) and including fully repaired patent ductus arteriosus (PDA).
• Resting peripheral arterial oxygen saturation (SaO2) = 90% and >70% (pulse oximetry, room air).

- Cardiac catheterization measurements must show the following:
• 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, or subjects with WHO functional class III or IV in whom standard PAH therapy is not feasible or has failed.

- Subjects able to perform the 6-minute walk test (6MWT) with a minimum distance of 50 m and a maximum distance of 450 m.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 190
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 15

Exclusion Criteria

- Pulmonary arterial hypertension (PAH) not fulfilling the criteria of ES as described in inclusion criterion 3 (e.g., complex cardiac defects, single ventricle, and unrepaired or partially repaired PDA).

- Known moderate-to-severe restrictive (i.e., total lung capacity [TLC] < 60% of predicted value) or obstructive lung disease (i.e., forced expiratory volume in one second [FEV1] < 80 % of predicted, with FEV1 / forced vital capacity [FVC] < 70%).

- Down Syndrome.

- Treatment with phosphodiesterase-5 (PDE-5) inhibitors or prostanoids within 1 month prior to Randomization.

- Treatment with endothelin receptor antagonists (ERAs) within 1 month prior to Randomization.

- Subjects who initiated diuretics within 1 week prior to Randomization or subjects whose diuretic treatment has not been stable within 1 week prior to Randomization.

- Subjects being considered for an organ transplant.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified