A Clinical Study to Find Out if Macitentan is Effective and Safe in Japanese Patients With Chronic Thromboembolic Pulmonary Hypertension (CTEPH).

• Register: ClinicalTrials.gov
• Last refreshed on: 28 June 2021
• Main ID: NCT03809650
• Date of registration: 10/01/2019
• Prospective Registration: No
• Primary sponsor: Actelion
• Public title: A Clinical Study to Find Out if Macitentan is Effective and Safe in Japanese Patients With Chronic Thromboembolic Pulmonary Hypertension (CTEPH).
• Scientific title: A Prospective, Multicenter, Open-label, Single Arm, Phase III Study to Assess the Efficacy and Safety of Macitentan (ACT-064992) in Subjects With Chronic Thromboembolic Pulmonary Hypertension (CTEPH)
• Date of first enrolment: January 8, 2019
• Target sample size: 9
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT03809650
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Japan

Contacts

• Name: Yoshinari Yokoyama, PhD
• Affiliation: Actelion Japan

Key inclusion & exclusion criteria

Inclusion Criteria:

- Written informed consent to participate in the study obtained from the subject or
legal representative a) prior to initiation of any study mandated procedure

- Japanese subjects who have been diagnosed as having CTEPH:

1. Subjects who have not undergone balloon pulmonary angioplasty (BPA) and for whom
the investigator determines not to implement pulmonary endarterectomy (PEA) at
the time of the acquisition of informed consent due to the organized thrombosis
localized in the peripheral regions, high risk (complications, old age, etc.) or
for any other reasons.

2. Subjects who have postoperative persistent or recurrent pulmonary hypertension
(PH) after undergoing pulmonary endarterectomy (PEA) and/or BPA.

- PH subjects whose WHO FC is I to IV

- 6MWD measured during the screening period ranges from 150 m to 450 m

- Subjects who meet the following conditions according to the right heart
catheterization (RHC) performed during the screening period or within 8 weeks before
the acquisition of the informed consent:

1. Resting mean pulmonary arterial pressure (mPAP) = 25 mmHg

2. Pulmonary artery wedge pressure (PAWP) = 15 mmHg (if PAWP cannot be measured or
the value of PAWP is not reliable, left ventricular end-diastolic pressure = 13
mmHg)

3. Resting PVR = 400 dyn*sec/cm5

- Subjects treated with anti-coagulation agents, unfractionated heparin or low molecular
weight heparin at least 90 days prior to RHC at baseline

- Women with childbearing potential with negative serum pregnancy test results and able
to follow the appropriate contraceptive methods from the date of starting the study
drug administration up to 30 days after the discontinuation or completion of the study
drug administration. Fertile male subjects able to use condom during the same period.

Exclusion Criteria:

- BPA within 90 days prior to undergoing baseline RHC

- PEA within 180 days prior to undergoing baseline RHC

- Subjects with unstable pulmonary hemodynamics who have postoperative persistent or
recurrent PH after undergoing PEA and/or BPA

- Recurrent thromboembolism undergoing treatment with oral anti-coagulation agents

- Symptomatic acute pulmonary embolism within 180 days prior to the start of study drug
administration

- Known moderate-to-severe restrictive lung disease or obstructive lung disease or known
significant chronic lung disease diagnosed by chest imaging (e.g., interstitial lung
disease, emphysema)

- Acute myocardial infarction during Screening period

- Severe liver impairment.

- Systolic blood pressure (SBP) < 90 mmHg at screening.

- Any known factor or disease that may interfere with treatment compliance or full
participation in the study



Age minimum: 18 Years
Age maximum: 89 Years
Gender: All

Health Condition(s) or Problem(s) studied

Chronic Thromboembolic Pulmonary Hypertension (CTEPH)

Intervention(s)

Drug: macitentan 10 mg

Primary Outcome(s)

Evaluate the ratio in pulmonary vascular resistance (PVR) at rest from baseline to Week 16 [Time Frame: From Baseline to Week 16]

Secondary Outcome(s)

Change from baseline to Week 16 in pulmonary vascular resistance index (PVRI) at rest [Time Frame: From baseline to Week 16]

Change from baseline to Week 24 in 6-minute walk distance (6MWD) [Time Frame: From baseline to Week 24]

Change from baseline to Week 16 in PVR at rest [Time Frame: From baseline to Week 16]

Change from baseline to Week 24 in Borg dyspnea index [Time Frame: From baseline to Week 24]

Change from baseline to Week 24 in WHO functional class (WHO FC) [Time Frame: From baseline to Week 24]

Secondary ID(s)

AC-055E301

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Mitsubishi Logistics Corporation

EPS Corporation

General Laboratory, BML, Inc.

Imepro Inc.