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

NCT02060721 | Completed Phase 2 Results

• 2 other identifiers: AC-055E2022013-003457-25
• Study Type: interventional
• Target: 76
• Locations: 15 countries
• Sites: 33

Brief Summary

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

Conditions

Chronic Thromboembolic Pulmonary Hypertension

Keywords

Chronic thromboembolic pulmonary hypertension (CTEPH)

Outcome Measures

Primary Outcomes (8)

• Number of Participants With Treatment-emergent Adverse Events (TEAEs)

  An adverse event (AE) is any untoward medical occurrence in a participant participating in a clinical study that does not necessarily have a causal relationship with the pharmaceutical/ biological agent under study. TEAEs are those events that started after administration of the first dose and up to safety follow-up visit/end of study, that is, 30 days after the last dose of study medication.

  Up to 30 days after study treatment discontinuation (treatment exposure ranged from 1 to 82 months)

• Number of Participants With AEs Leading to Study Drug Discontinuation

  Number of participants with AEs leading to study drug discontinuation was reported.

  Up to 30 days after study treatment discontinuation (treatment exposure ranged from 1 to 82 months)

• Number of Participants With Treatment-emergent Serious Adverse Events (SAEs)

  A serious adverse event (SAE) is any untoward medical occurrence that at any dose resulting in any of following outcomes: results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, is a suspected transmission of any infectious agent via a medicinal product. Treatment-emergent SAEs were those events that started after administration of the first dose and up to safety follow-up visit/end of study, that is, 30 days after the last dose of study medication.

  Up to 30 days after study treatment discontinuation (treatment exposure ranged from 1 to 82 months)

• Number of Participants With Hemoglobin Abnormalities

  Number of participants with hemoglobin abnormalities were reported. It included hemoglobin less than (\<) 80 grams per liter (g/L), hemoglobin \<100 g/L, hemoglobin greater than or equal to (\>=) 80 g/L and \<100 g/L, hemoglobin \<100g/L and a decrease of \>20 g/L from baseline, decrease of \>20 g/L in hemoglobin from baseline, decrease of \>20 g/L and \<=50 g/L in hemoglobin from baseline, and decrease of \>50 g/L in hemoglobin from baseline.

  Up to 30 days after study treatment discontinuation (treatment exposure ranged from 1 to 82 months)

• Number of Participants With Liver Tests Abnormalities

  Number of participants with liver tests abnormalities were reported. It included alanine aminotransferase (ALT) or aspartate aminotransferase (AST): \>=3 x Upper limit of the normal range (ULN), \>=3 and \<5 x ULN, \>=5 ULN, and \>=5 and \<8 x ULN, \>= 8 x ULN, and total bilirubin \>=2 x ULN.

  Up to 30 days after study treatment discontinuation (treatment exposure ranged from 1 to 82 months)

• Change From Baseline in Blood Pressure at Month 6

  Change from baseline in blood pressure at Month 6 (both systolic blood pressure \[SBP\] and diastolic blood pressure \[DBP\]) was reported.

  Baseline and Month 6

• Change From Baseline in Pulse Rate at Month 6

  Change from baseline in pulse rate at Month 6 was reported.

  Baseline and Month 6

• Change From Baseline in Body Weight at Month 6

  Change from baseline in body weight at Month 6 was reported.

  Baseline and Month 6

Study Arms (1)

Macitentan

EXPERIMENTAL

Macitentan 10mg, oral tablet, once daily

Drug: Macitentan

Interventions

Macitentan DRUG

Macitentan 10mg, oral tablet, once daily

Also known as: ACT-064992

Macitentan

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Written informed consent
• Subject with CTEPH having completed the double-blind (DB) AC-055E201/ MERIT-1 study as scheduled (i.e., who remained in the DB study up to Week 24).
• Females 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.

You may not qualify if:

• Permanent discontinuation of DB study treatment due to an hepatic adverse event or liver aminotransferase abnormalities.
• Any known factor (e.g., drug or substance abuse) or disease (e.g., unstable psychiatric illness) that, in the opinion of the investigator, may interfere with treatment compliance or interpretation of the results, or that may influence the ability to comply with any of the study requirements.

Sponsors & Collaborators

• Actelion: lead

Study Sites (33)

Unknown Facility

Leuven, Belgium

Location

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Beijing, China

Location

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Guangzhou, China

Location

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Shanghai, China

Location

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Shenyang, China

Location

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Wuhan, China

Location

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Prague, Czechia

Location

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Le Kremlin-Bicêtre, France

Location

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Paris, France

Location

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Toulouse, France

Location

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Giessen, Germany

Location

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Heidelberg, Germany

Location

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Würzburg, Germany

Location

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Budapest, Hungary

Location

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Debrecen, Hungary

Location

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Kaunas, Lithuania

Location

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Mexico City, Mexico

Location

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Lublin, Poland

Location

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Wroclaw, Poland

Location

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Kemerovo, Russia

Location

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Moscow, Russia

Location

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Novosibirsk, Russia

Location

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Saint Petersburg, Russia

Location

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Tomsk, Russia

Location

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Zurich, Switzerland

Location

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Bangkok, Thailand

Location

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Chiang Mai, Thailand

Location

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Capa_Istanbul, Turkey (Türkiye)

Location

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Kyiv, Ukraine

Location

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Lviv, Ukraine

Location

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Cambridge, United Kingdom

Location

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London, United Kingdom

Location

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Sheffield, United Kingdom

Location

Related Publications (1)

• Kim NH, D'Armini AM, Howard LS, Jenkins DP, Jing ZC, Mayer E, Chamitava L, Lack G, Rofael H, Solonets M, Ghofrani HA. Long-Term Safety and Efficacy of Macitentan in Inoperable Chronic Thromboembolic Pulmonary Hypertension: Results from MERIT and its Open-Label Extension. Pulm Ther. 2025 Mar;11(1):101-116. doi: 10.1007/s41030-024-00276-w. Epub 2024 Nov 9.

  PMID: 39520648 DERIVED

MeSH Terms

Interventions

macitentan

Limitations and Caveats

Study limitations included the open-label (OL), uncontrolled design, and small sample size.

Results Point of Contact

• Title: Clinical Scientific Leader
• Organization: Actelion Pharmaceuticals Ltd.

ClinicalTrialDisclosure@its.jnj.com

844-434-4210

Study Officials

• Erin McGuire

  Actelion

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 2, 2014
• First Posted: February 12, 2014
• Study Start: February 3, 2015
• Primary Completion: March 21, 2022
• Study Completion: March 21, 2022
• Last Updated: March 30, 2025
• Results First Posted: April 11, 2023

Record last verified: 2025-03

Locations

DE (3); CN (5); PL (2); UA (2); CH (1); GB (3); HU (2); RU (5); LI (1); ME (1); CZ (1); FR (3); BE (1); TH (2); TU (1)