Clinical Study to Assess the Efficacy, Safety and Tolerability of Macitentan in Subjects With Inoperable Chronic Thromboembolic Pulmonary Hypertension

Phase 2

Completed

• Conditions: Chronic Thromboembolic Pulmonary Hypertension
• Interventions: Drug: Placebo; Drug: Macitentan

• Registration Number: NCT02021292
• Lead Sponsor: Actelion

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2013-12-20
• Study First Submitted QC: 2013-12-20
• Study First Posted: 2013-12-27
• Study Start: 2014-08-20
• Primary Completion: 2016-09-28
• Study Completion: 2016-09-28
• Results First Submitted: 2017-09-26
• Results First Submitted QC: 2017-12-20
• Results First Posted: 2018-01-23
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-28
• Last Update Posted: 2025-03-30

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 80

Inclusion Criteria

• Written informed consent
• Subject with CTEPH (WHO Group 4) judged as inoperable due to the localization of the obstruction being surgically inaccessible (i.e., distal disease).
• Female of childbearing potential must have a negative pre-treatment serum pregnancy test, be advised on appropriate methods of contraception, and agree to use 2 reliable methods of contraception.

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
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Matching placebo oral tablet, to be taken once daily.
Macitentan	Macitentan	Macitentan 10 mg, oral tablet, to be taken once daily.

Primary Outcome Measures

Name	Time	Method
Change From Baseline to Week 16 in Pulmonary Vascular Resistance (PVR) at Rest.	From baseline to Week 16	The primary efficacy endpoint is defined as the PVR at rest at Week 16 expressed as percent of baseline PVR at rest.

Secondary Outcome Measures

Name	Time	Method
Change From Baseline to Week 24 in Exercise Capacity, as Measured by the 6-minute Walk Distance (6MWD).	From baseline to Week 24	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 24 in Borg Dyspnea Index Collected at the End of the 6-minute Walk Test (6MWT).	From baseline to Week 24	This outcome measures the difference in the Borg dyspnea index collected at the end of the 6-minute walk test (6MWT) at Week 24 compared to baseline. The Borg dyspnea index rates the severity of dyspnea (difficult or labored breathing) on a scale from 0 ('Nothing at all') to 10 ('Very, very severe - maximal'). A decrease in the Borg dyspnea index indicates an improvement.
Proportion of Subjects With Worsening in WHO Functional Class (FC) From Baseline to Week 24	From baseline to Week 24	WHO functional classes are defined as follows: 1) class I: no symptoms with exercise or at rest. No limitation of activity. 2) 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). 3) class III: may not have symptoms at rest but activities greatly limited by shortness of breath, fatigue, or near fainting. 4) class IV: symptoms at rest (such as dyspnea and/or fatigue) and inability to carry out any physical activity without symptoms (e.g. may faint especially while bending over with their heads lowered). Patients in class IV manifest signs of right heart failure. Shifting to a higher class (e.g. from class III to class IV) represents a 'worsening' while shifting to a lower class (e.g. from class III to class II) means an 'improvement'.

Trial Locations

Locations (36)

University Hospital Gasthuisberg / Interne Geneeskunde - I.G. Pneumologie; 🇧🇪 Leuven, Belgium

Beijing Chao-Yang Hospital-Department of Respiration; 🇨🇳 Beijing, China

Cardiovascular institute & Fuwai Hospital- Thrombus Center; 🇨🇳 Beijing, China

The first affiliated hospital of guangzhou medical university-respiratory department; 🇨🇳 Guangzhou, China

ngShanghai Pulmonary Hospital, Department of Pulmonary Circulation; 🇨🇳 Shanghai, China

The General Hospital of Shenyang Military Region,Congenital Heart Disease Department; 🇨🇳 Shenyang, China

Wuhan Asia Heart Hospital; 🇨🇳 Wuhan, China

Centre for PPH, Charles University , II Interni klinika1.LF a VFN; 🇨🇿 Praha, Czechia

CHU de Bicêtre; 🇫🇷 Le Kremlin-Bicêtre cedex, France

Hôpital Européen Georges Pompidou Service de Pneumologie, soins intensifs et endoscopies bronchiques; 🇫🇷 Paris cedex 15, France

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