Study to evaluate if macitentan is efficient, safe and tolerable enough to be used for treatment of inoperable chronic thromboembolic pulmonary hypertension (CTEPH)

Phase 1

Conditions: Inoperable chronic thromboembolic pulmonary hypertension (CTEPH)
MedDRA version: 19.0Level: LLTClassification code 10068739Term: Chronic thromboembolic pulmonary hypertensionSystem Organ Class: 100000004855
Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]

Registration Number: EUCTR2013-002950-56-HU

Lead Sponsor: Actelion Pharmaceuticals Ltd.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 78

Inclusion Criteria

- Male or female = 18 and = 80 years of age.
- Subject with CTEPH (WHO Group 4) judged as inoperable due to the localization of the obstruction being surgically inaccessible (i.e., distal disease).
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 47
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 31

Exclusion Criteria

- Previous pulmonary endarterectomy.
- Recurrent thromboembolism despite sufficient oral anticoagulants.
- Symptomatic acute pulmonary embolism in the 6-month period prior to randomization.
- Known moderate-to-severe restrictive lung disease (i.e., TLC < 60% of predicted value) or obstructive lung disease (i.e., FEV1 < 70% of predicted, with FEV1/FVC < 65%) or known significant chronic lung disease diagnosed by chest imaging (e.g., interstitial lung disease, emphysema).
- Acute or chronic conditions (other than dyspnea) that limit the ability to comply with study requirements in the 3-month period prior to Screening visit or during the Screening period.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the effect of macitentan 10 mg on pulmonary vascular resistance (PVR) at rest in comparison with placebo in subjects with inoperable chronic thromboembolic pulmonary hypertension (CTEPH).;Secondary Objective: • To evaluate the effects of macitentan 10 mg in comparison with placebo on:<br>– Exercise capacity<br>– Dyspnea (assessed by the Borg dyspnea index)<br>– WHO functional class (FC)<br><br>• To evaluate the safety and tolerability of macitentan 10 mg in this subject population.;Primary end point(s): PVR at rest at Week 16 expressed as percent of baseline PVR at rest;Timepoint(s) of evaluation of this end point: Baseline and Week 16

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): - Change from baseline to Week 24 in exercise capacity, as measured by the 6MWD.<br>- Change from baseline to Week 24 in Borg dyspnea index collected at the end of the 6MWT.<br>- Proportion of subjects with worsening in WHO FC from baseline to Week 24.;Timepoint(s) of evaluation of this end point: Baseline and Week 24