A Study to Evaluate Efficacy and Safety of Macitentan 75 mg in Inoperable or Persistent/Recurrent Chronic Thromboembolic Pulmonary Hypertension (MACiTEPH)

February 1, 2024 updated by: Actelion

A Prospective, Randomized, Double-blind, Multicenter, Placebo-controlled, Parallel Group, Adaptive Phase 3 Study With Open-label Extension to Evaluate Efficacy and Safety of Macitentan 75 mg in Inoperable or Persistent/Recurrent Chronic Thromboembolic Pulmonary Hypertension

The purpose of the study is to evaluate the effect of macitentan 75 mg versus placebo on exercise capacity at Week 28 in participants with chronic thromboembolic pulmonary hypertension (CTEPH).

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Detailed Description

CTEPH is one of the leading causes of severe pulmonary hypertension (PH), classified within World Health Organization (WHO) group 4 PH. It is a rare, progressive pulmonary vascular disease that if left untreated, leads to progressively increasing pulmonary vascular resistance (PVR) and eventually right ventricle failure and death. Histopathologic findings including endothelial cell dysfunction and distal pulmonary arterial remodeling are shared between PAH and CTEPH, and PH-specific therapies (that is, riociguat) have shown efficacy in inoperable and persistent/recurrent CTEPH. The endothelin receptor antagonist macitentan offers a different mode of action and addresses an important unmet medical need for an alternative treatment option in this indication. This study will assess the effect of macitentan 75 mg on exercise capacity in CTEPH. The total duration of the study is approximately 6 years. The study comprises of a screening period (at least 14 days and up to 60 days), a double-blind (DB) treatment period (28 weeks [minimum duration] up to 3.5 years), an open-label (OL) extension period (starts at end-of-DB-treatment [EODBT] and will end for all participants 104 weeks after the last participant has completed DB Week 28). The DB period consists of an 8-week up-titration phase and a maintenance phase. The maintenance phase is divided into a 28-week fixed duration part, at the end of which primary endpoint is assessed, and a variable duration part. The duration of the DB period for an individual participant depends on the timepoint of entry into the study and whether a CEC-confirmed clinical worsening event occurred. Participants who discontinue DB study intervention during the 28-week fixed duration part will be followed until Week 28 in a post-treatment observation period (PTOP).

Study Type

Interventional

Enrollment (Actual)

127

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Argentina
      • Buenos Aires, Argentina, C1039AAO
        • Sanatorio de la Trinidad Mitre
      • C.a.b.a., Argentina, C1180AAX
        • Sanatorio Güemes
      • Ciudad Autonoma de Buenos Aires, Argentina, C1006ACC
        • Nexo Salud Investigación Clínica
  • Australia
      • Chermside, Australia, 4032
        • Queensland Lung Transplant Service
      • Darlinghurst, Australia, 2010
        • St Vincent's Hospital
  • Austria
      • Graz, Austria, 8036
        • LKH-Univ. Klinikum Graz
      • Linz, Austria, 4020
        • Ordensklinikum Linz GmbH Elisabethinen
      • Vienna, Austria, 1090
        • Medizinische Universität Wien
  • Bulgaria
      • Sofia, Bulgaria, 1606
        • Military Medical Academy
      • Sofia, Bulgaria, 1750
        • University Multiprofile Hospital for Active Treatment- UMHAT Sveta Anna AD
  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T1Y 6J4
        • University Of Calgary - Peter Lougheed Centre
      • Edmonton, Alberta, Canada, T6G 2B7
        • University of Alberta Hospital
    • Ontario
      • Toronto, Ontario, Canada, M5G 2N2
        • University Health Network - Toronto General Hospital
  • China
      • Beijing, China, 100029
        • Beijing Anzhen Hospital
      • Beijing, China, 100029
        • China-Japan Friendship Hospital
      • Beijing, China, 100038
        • Beijing Shijitan Hospital
      • Beijing, China, 100020
        • Beijing ChaoYang Hospital
      • Chongqing, China, 400016
        • The First Affiliated Hospital of Chongqing Medical University
      • Guangzhou, China, 510140
        • The First Affiliated Hospital of Guangzhou Medical University
      • Hangzhou, China, 310016
        • Sir Run Run Shaw Hospital, Zhejiang University School of Medicine
      • Nanjing, China, 210009
        • Zhongda Hospital,Southeast University
      • Qingdao, China, 266003
        • The Affiliated Hospital of Medical College Qingdao University
      • Shanghai, China, 200433
        • Shanghai Pulmonary Hospital
      • Shanghai, China, 200040
        • Huashan Hospital of Fudan University
      • Shanghai, China
        • Zhongshan Hospital,Fudan University
      • Shenyang, China, 110000
        • The General Hospital of Northern Theater Command
      • Tian Jin, China, 300052
        • Tianjin Medical University General Hospital
      • Xi'An, China, 710061
        • The First Affiliated Hospital of Xian Jiaotong University
  • Colombia
      • Bogota, Colombia, 1101131
        • Fundación Neumológica Colombiana
      • Bogota, Colombia, 85369
        • Fundacion ABOOD Shaio
      • Cali, Colombia, 760042
        • Clínica Imbanaco S.A.S.
      • Medellin, Colombia, 681004
        • Centro Cardiovascular Colombiano Clínica Santa María
  • Czechia
      • Praha 2, Czechia, 128 08
        • General University Hospital II.department of Internal Medicine-cardiology and angiology
  • Denmark
      • Aarhus N, Denmark, 8200
        • Århus Universitetshospital, Skejby, Hjertemedicinsk Afdeling B
  • France
      • Brest, France, 29200
        • CHU de Brest - Hôpital de la Cavale Blanche
      • Grenoble Cedex 9, France, 38043
        • CHU de Grenoble Hopital Albert Michallon
      • Le Kremlin-Bicetre Cedex, France, 94275
        • Hopital Bicêtre - Aphp Hôpitaux Universitaires Paris-Sud
      • Lille Cedex, France, 59037
        • Hôpital Cardiologique - Chru Lille
      • Montpellier, France, 34295
        • CHU de Montpellier - Arnaud de Villeneuve
      • St Priest en Jarez Cedex, France, 42277
        • CHU Saint-Etienne - Hopital Nord
      • Toulouse Cedex 9, France
        • Hopital Larrey CHU de Toulouse
      • Vandoeuvre les Nancy Cedex, France, 54511
        • CHU de Nancy - Hopital de Brabois
  • Germany
      • Bonn, Germany, 53127
        • Universitätsklinikum Bonn
      • Dresden, Germany, 01307
        • Medizinische Fakultat Carl Gustav Carus Technische Universitat Dresden
      • Giessen, Germany, 35392
        • Universitaetsklinikum Giessen
      • Hamburg, Germany, 20246
        • Universitaetsklinikum Hamburg Eppendorf
      • Hannover, Germany, 30625
        • Medizinische Hochschule Hannover Zentrum Innere Medizin Klinik für Pneumologie
      • Heidelberg, Germany, 69126
        • Thoraxklinik Heidelberg
      • Homburg, Germany, 66421
        • Universitaetsklinikum des Saarlandes
      • Jena, Germany, 07747
        • Universitätsklinikum Jena
      • München, Germany, 80639
        • Krankenhaus Neuwittelsbach
  • Hungary
      • Budapest, Hungary, 1096
        • Gottsegen György Országos Kardiológiai Intézet, Felnőtt kardiológiai osztály
      • Szeged, Hungary, 6720
        • Szegedi Tudományegyetem, Általános Orvostudományi Kar, Családorvosi Intézet és rendelő
  • Israel
      • Tel Aviv, Israel, 6423906
        • Tel Aviv Medical Center
      • Tel-Hashomer, Israel, 5265601
        • The Chaim Sheba Medical Center
  • Italy
      • Chieti, Italy, 66100
        • Ospedale Ss. Annunziata
      • Pavia, Italy, 27100
        • Fondazione IRCCS Policlinico San Matteo
      • Pisa, Italy, 56124
        • Fondazione Toscana Gabriele Monasterio CNR
      • Roma, Italy, 00168
        • Policlinico Gemelli Universita Cattolica
      • Torino, Italy, 10126
        • A.O.U. Citta della Salute e della Scienza
  • Japan
      • Bunkyo, Japan, 113-8655
        • The University of Tokyo Hospital
      • Fukuoka, Japan, 812-8582
        • Kyushu University Hospital
      • Hiroshima, Japan, 737-8505
        • Kure Kyosai Hospital
      • Kanagawa, Japan, 216-8511
        • St.Marianna University Hospital
      • Kobe, Japan, 650-0017
        • Kobe University Hospital
      • Kyoto, Japan, 606-8507
        • Kyoto University Hospital
      • Kyoto, Japan, 602-8566
        • University Hospital Kyoto Perfectural University of Medicine
      • Matsumoto, Japan, 390-8621
        • Shinshu University Hospital
      • Meguro-ku, Japan, 153-8515
        • Toho University Medical Center, Ohashi Hospital
      • Mitaka, Japan, 181-8611
        • Kyorin University Hospital
      • Nagoya, Japan, 466-8560
        • Nagoya University Hospital
      • Okayama, Japan, 701-1192
        • National Hospital Organization Okayama Medical Center
      • Sapporo-shi, Japan, 060-8648
        • Hokkaido University Hospital
      • Suita-Shi, Japan, 564-8565
        • National Cerebral and Cardiovascular Center
      • Tokyo, Japan, 113-8431
        • Juntendo University Hospital
      • Tsukuba-City, Japan, 305-8576
        • University of Tsukuba Hospital
  • Korea, Republic of
      • Busan, Korea, Republic of, 49241
        • Pusan National University Hospital
      • Seoul, Korea, Republic of, 03080
        • Seoul National University Hospital
      • Seoul, Korea, Republic of, 05505
        • Asan Medical Center
      • Seoul, Korea, Republic of, 06351
        • Samsung Medical Center
      • Seoul, Korea, Republic of, 06591
        • The Catholic University of Korea Seoul St. Mary'S Hospital
  • Lithuania
      • Kaunas, Lithuania, 50161
        • Lietuvos Sveikatos Mokslu Universiteto Ligonine Kauno Klinik
      • Vilnius, Lithuania, LT-08661
        • Vilnius University Hospital Santariskiu Klinikos
  • Mexico
      • Ciudad De México, Mexico, 14080
        • Instituto Nacional de Cardiologia Dr. Ignacio Chavez
      • Mexico, Mexico, 52787
        • Operadora de Hospitales Angeles SA de CV Hospital Angeles Lomas
      • Monterrey, Mexico, 64718
        • Unidad de Investigacion Clinica en Medicina S.C. (UDICEM)
      • Morelia, Mexico, 58260
        • Centro de Investigacion Clinica Chapultepec
      • Nuevo Leon, Mexico, 64060
        • CRI Centro Regiomontano de Investigacion SC
  • Poland
      • Kraków, Poland, 31-202
        • Krakowski Szpital Specjalistyczny im Jana Pawla II
      • Lublin, Poland, 20-708
        • Wojewódzki Szpital Specjalistyczny im. Stefana Kardynała Wyszyńskiego, PZOZ
      • Otwock, Poland, 05-400
        • Europejskie Centrum Zdrowia Otwock Sp. z o.o
  • Portugal
      • Almada, Portugal, 2805-267
        • Hosp. Garcia de Orta
  • Romania
      • Bucuresti, Romania, 022328
        • Institutul de urgenta pentru Boli Cardiovasculare Prof. Dr. C.C. Iliescu
      • Tg. Mures, Romania, 540136
        • Spitalul Clinic Judetean de Urgenta
  • Russian Federation
      • Kazan, Russian Federation, 420101
        • State Autonomous HealthCare Institution 'Interregional Clinical Diagnostic Center'
      • Moscow, Russian Federation, 121552
        • National Medical Research Center of Cardiology of MoH of Russian Federation
      • Moscow, Russian Federation, 121309
        • Moscow City Clinical Hospital No.51
      • Saint-Petersburg, Russian Federation, 197341
        • National medical Research Center n.a. V.A.Almazov of MoH of Russian Federation
      • Volgograd, Russian Federation, 400008
        • Volgograd Regional Clinical Cardiology Center
  • Saudi Arabia
      • Riyadh, Saudi Arabia, 12713
        • King Faisal Specialist Hospital & Research Center
      • Riyadh, Saudi Arabia, 59046
        • King Fahad Medical City
  • Serbia
      • Belgrade, Serbia, 11000
        • University Clinical Center of Serbia
      • Sremska Kamenica, Serbia, 21204
        • Institute for Pulmonary Disease of Vojvodina
  • Singapore
      • Singapore, Singapore, 119228
        • National University Heart Centre, Singapore
      • Singapore, Singapore, 169609
        • National Heart Centre (NHC) Singapore
  • Slovakia
      • Bratislava, Slovakia, 833 48
        • Narodny ustav srdcovych a cievnych chorob
  • Spain
      • Barcelona, Spain, 08036
        • Hosp. Clinic de Barcelona
      • Madrid, Spain, 28041
        • Hosp. Univ. 12 de Octubre
      • Málaga, Spain, 29010
        • Hosp. Virgen de La Victoria
      • Toledo, Spain, 45007
        • Hosp. Gral. Univ. de Toledo
  • Taiwan
      • Kaohsiung, Taiwan, 813
        • Kaohsiung Veterans General Hospital
      • Taipei, Taiwan, 10002
        • National Taiwan University Hospital
      • Taipei, Taiwan, 112
        • Taipei Veterans General Hospital
      • Taoyuan, Taiwan, 333
        • Chang-Gung Memorial Hospital, LinKou Branch
  • Thailand
      • Chiang Mai, Thailand, 50200
        • Maharaj Nakorn Chiang Mai Hospital
      • Khon Kaen, Thailand, 40002
        • Srinagarind Hospital, Khon Kaen University
      • Pathumthani, Thailand, 12120
        • Thammasat Hospital
  • Turkey
      • Adana, Turkey, 01170
        • Adana City Hospital
      • Adana, Turkey, 01250
        • Cukurova University Medical Faculty
      • Ankara, Turkey, 06230
        • Hacettepe University Medical Faculty
      • Ankara, Turkey, 06800
        • Ankara Bilkent Sehir Hastanesi
      • Denizli, Turkey, 20070
        • Pamukkale University Medical Faculty
      • Eskisehir, Turkey, 26040
        • Eskisehir Osmangazi University Medical Faculty Hospital
      • Istanbul, Turkey, 34096
        • Istanbul University - Cerrahpasa Cardiology Institution
      • Istanbul, Turkey, 34899
        • Marmara University Medical Faculty
      • Istanbul, Turkey, 34668
        • Siyami Ersek Training and Research Hospital
      • Izmir, Turkey, 35100
        • Ege University Medical Faculty
      • Izmir, Turkey, 35340
        • Dokuz Eylul University Medical Faculty
      • Kartal Istanbul, Turkey, 34865
        • Kartal Kosuyolu Yuksek Ihtisas Egitim Ve Arastirma Hastanesi
      • Mersin, Turkey, 33110
        • Mersin University Medical Faculty
  • Ukraine
      • Cherkasy, Ukraine, 18009
        • CNE 'Cherkasy Regional Cardiological Center of Cherkasy Regional Council'
      • Dnipro, Ukraine, 49059
        • CE 'Dnipropetrovsk Regional Clinical Center of Cardiology and Cardiosurgery'
      • Kyiv, Ukraine, 03680
        • State Institute Of Phthisiology And Pulmonology N.A. F.G. Yanovskiy Of Ams Ukraine
      • Kyiv, Ukraine, 02000
        • SI National Scientific Center Institute of Cardiology of M.D. Strazhesko of NAMS of Ukraine
      • Lviv, Ukraine, 79010
        • Communal Noncommercial Enterprise of Lviv Regional Council 'Lviv Regional Clinical Hospital'
      • Ternopil, Ukraine, 46002
        • Municipal Non-commercial Enterprise Ternopil University Hospital of Ternopil Regional Council
  • United Kingdom
      • Cambridge, United Kingdom, CB2 0AY
        • Papworth Hospital NHS Trust
      • Glasgow, United Kingdom, G81 4DY
        • National Waiting Times Centre Board Golden Jubilee National Hospital
      • London, United Kingdom, NW3 2QG
        • Royal Free Hospital
      • London, United Kingdom, W12 0HS
        • Hammersmith Hospital
      • Newcastle Upon Tyne, United Kingdom, NE7 7DN
        • Freeman Hospital
      • Sheffield, United Kingdom, S10 2RX
        • Sheffield Teaching Hospitals NHS Foundation Trust Royal Hallamshire Hospital
  • United States
    • California
      • La Jolla, California, United States, 92037
        • University of California San Diego Medical Center
      • Los Angeles, California, United States, 90033
        • Keck School of Medicine of USC
      • Sacramento, California, United States, 95817-2201
        • UC Davis Medical Center
    • Colorado
      • Aurora, Colorado, United States, 80045
        • University of Colorado - Anschutz Medical Campus
    • Connecticut
      • New Haven, Connecticut, United States, 06519
        • Yale University School of Medicine
    • Florida
      • Gainesville, Florida, United States, 32608
        • University of Florida Health Jacksonville
    • Georgia
      • Atlanta, Georgia, United States, 30309
        • Piedmont Healthcare
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Northwestern University Feinberg School of Medicine
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Indiana University
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University Of Kansas Medical Center
    • Maryland
      • Baltimore, Maryland, United States, 21201
        • University of Maryland
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital
      • Boston, Massachusetts, United States, 21118
        • Tufts Medical Center
    • Nebraska
      • Omaha, Nebraska, United States, 68198-2265
        • University of Nebraska Medical Center
    • Nevada
      • Reno, Nevada, United States, 89509
        • VA Sierra Nevada Health Care System
    • New Mexico
      • Albuquerque, New Mexico, United States, 87131
        • University of New Mexico School of Medicine
    • New York
      • Syracuse, New York, United States, 13210
        • Syracuse VA Medical Center
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Cleveland Clinic
    • Oregon
      • Portland, Oregon, United States, 97210
        • Legacy Hospital
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15213
        • University of Pittsburgh Medical Center
    • Texas
      • Plano, Texas, United States, 75093
        • Baylor Scott White - Plano
    • Utah
      • Murray, Utah, United States, 84107
        • Intermountain Medical Center
      • Salt Lake City, Utah, United States, 84132
        • University of Utah Cardiovascular Center
    • Wisconsin
      • Madison, Wisconsin, United States, 53792-2442
        • University of Wisconsin Hospital and Clinics

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 80 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Chronic thromboembolic pulmonary hypertension (CTEPH) (World Health Organization [WHO] Group 4) fulfilling one of the following criteria: a) inoperable due to the localization of the obstruction being surgically inaccessible (that is, distal disease), b) persistent/recurrent CTEPH after balloon pulmonary angioplasty (BPA), and deemed inoperable due to the localization of the obstruction being surgically inaccessible (that is, distal disease), c) persistent/recurrent CTEPH after rescue pulmonary endarterectomy (PEA)
  • 6-minute walk distance (6MWD) greater than or equal to (>=) 100 meter (m) and less than or equal to (<=) 450 meters (m), documented by an eligibility and a baseline 6-minute walk test (6MWT). The baseline 6MWD must not differ by more than 15 percent (%) from the eligibility test
  • World Health Organization functional class (WHO FC) >= II
  • Participants are to receive riociguat as per local standard of care, unless it is contraindicated or unavailable

Exclusion Criteria:

  • Acute pulmonary embolism within 3 months prior to or during Screening
  • Planned balloon pulmonary angioplasty (BPA) during the fixed duration part of the double-blind period
  • Significant obstructive and restrictive lung disease
  • Acute or chronic conditions (other than dyspnea) that limit the ability to comply with study requirements, in particular with 6MWT (for example, intermittent claudication).
  • Symptomatic coronary artery disease requiring an intervention within 3 months prior to or during Screening or anticipated during the fixed duration part of the study
  • Decompensated cardiac failure if not under close supervision
  • Known and documented life-threatening cardiac arrhythmias
  • Acute myocardial infarction within 6 months prior to, or during Screening
  • Cerebrovascular events (including transient ischemic attack) within 3 months prior to, or during Screening
  • Known or suspicion of pulmonary veno-occlusive disease (PVOD)
  • Administration of ERAs, intravenous prostacyclins / prostacyclin analogs, or investigational treatment within 90 days prior to Randomization
  • Change in dose or initiation of Phosphodiesterase type-5 (PDE-5) inhibitors, oral, inhaled or subcutaneous (SC) prostacyclins / prostacyclin analogues, prostacyclin receptor agonists or riociguat, a) within 90 days prior to Randomization, or b) anticipated during the fixed duration part of the double-blind [DB] period
  • Hypotension, that is, systolic blood pressure (SBP) less than (<) 90 millimeters of mercury (mmHg) or diastolic blood pressure (DBP) <50 mmHg at Screening.
  • Severe renal dysfunction with an estimated Glomerular Filtration Rate <30 milliliters per minute per 1.73 meter square (mL/min/1.73 m^2) using the Chronic Kidney Disease Epidemiology Collaboration formula at Screening
  • Known moderate to severe hepatic impairment, defined as Child-Pugh Class B or C, based on records that confirm documented medical history
  • Serum aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) greater than or equal to (>=) 1.5*upper limit of normal (ULN) at Screening
  • Hemoglobin <100 g/L (<10 gram per deciliter [g/dL]) at Screening

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Macitentan
Participant will receive macitentan at a dose of 10 milligram (mg) once daily (OD) for 4 weeks, followed by a dose of macitentan 37.5 mg for another 4 weeks and continue with the target dose of macitentan 75 mg. Participants who have reached the target dose of 75 mg, completed the Double-blind (DB) period up to Week 28 (either on treatment or in Post-treatment observation period [PTOP]) at minimum, may be eligible for transitioning into the Open label (OL) extension period once all participants have completed the DB part of the study, or earlier if they experienced a Clinical event committee (CEC) confirmed clinical worsening event.
Drug: Macitentan
Participants will receive Macitentan film-coated tablets orally od.
Other Names:
  • Opsumit
  • ACT-064992,
Experimental: Placebo
Participants will receive placebo tablets matching the macitentan 10 mg, macitentan 37.5mg and macitentan 75 mg tablets, respectively. Participants who completed the DB period as per protocol either on treatment or in PTOP are eligible for transitioning to the OL extension period and will receive macitentan 75 mg after an 8-week double-dummy uptitration (macitentan 10 mg for 4 weeks, followed by 37.5 mg for another 4 weeks).
Drug: Macitentan
Participants will receive Macitentan film-coated tablets orally od.
Other Names:
  • Opsumit
  • ACT-064992,
Drug: Placebo
Participant will receive matching placebo tablets orally od.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline to Week 28 in 6- minute Walk Distance [6MWD]
Time Frame: Baseline up to Week 28
Change from baseline to week 28 in 6MWD as measured by 6-minute walk test [6MWT]) will be reported. The purpose of the six-minute walk test (6MWT) is to quantify exercise tolerance and capacity. This standardized 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 6 minutes.
Baseline up to Week 28

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Time to Clinical Worsening up to End-of double-blind-treatment (EODBT) Period
Time Frame: Up to EODBT (variable duration per participant, up to 3.5 years)
Time to first Clinical Event Committee (CEC) confirmed clinical worsening up to EODBT will be reported. Clinical worsening is defined as the occurrence of at least one of the following events: 1) All-cause death; 2) Heart and/or lung transplantation; 3) Unplanned pulmonary hypertension (PH)-related hospitalization; 4) PH-related deterioration from baseline identified by at least one of the following: a) Persistent increase in World Health Organization functional class (WHO FC) that cannot be explained by another cause (for example, viral infection); b) Persistent deterioration by at least 15 percent (%) in exercise capacity; as measured by the 6MWD; c) New or worsening signs or symptoms of right heart failure; 5) Rescue pulmonary endarterectomy (PEA) and/or balloon pulmonary angioplasty (BPA) procedure due to worsening of PH.
Up to EODBT (variable duration per participant, up to 3.5 years)
Number of Participants with Improvement in WHO Functional Class (WHO FC) From Baseline to Week 28
Time Frame: Baseline up to Week 28
Improvement in WHO FC from baseline to Week 28 will be calculated for each participant. WHO FC test is used to assess disease severity. Four functional classes (FC) are defined from FC I (no limitation of physical activity) to FC IV (inability to carry out any physical activity without symptoms). Improvement is considered when a participant changes from a higher class to a lower class.
Baseline up to Week 28
Change From Baseline to Week 28 in Pulmonary Arterial Hypertension - Symptoms Based on (PAH-SYMPACT) - Cardiopulmonary Symptom Domain Score
Time Frame: Baseline up to Week 28
The Cardiopulmonary Symptoms domain consists of 6 items (shortness of breath, fatigue, lack of energy, swelling in ankles or legs, swelling in stomach area and cough) reported on a 5-point Likert scale (from 0 to 4). The value 0 means "no symptom" and value 4 corresponds to "very severe symptoms". The symptoms part of the PAH-SYMPACT will be administered daily over a 7-day period. The recall period of symptom items is the last 24 hours. An average Cardiopulmonary Symptoms domain score is determined based on the daily scores of the 6 items. Questionnaires to be completed at site at screening for training purpose. PAH-SYMPACT to be performed at home, during the 7-day period prior to the scheduled visit.
Baseline up to Week 28
Change from Baseline to Week 28 in PAH-SYMPACT - Cardiovascular Symptom Domain Score
Time Frame: Baseline up to Week 28
The Cardiovascular Symptoms domain consists of 5 items (heart palpitations [fluttering], rapid heartbeat, chest pain, chest tightness, and lightheadedness) reported on a 5-point Likert scale (from 0 to 4). The value 0 corresponds to "no symptoms" and value 4 corresponds to "very severe symptoms". The symptoms part of the PAH-SYMPACT will be administered daily over a 7-day period. The recall period of symptom items is the last 24 hours. An average Cardiovascular Symptoms domain score is determined based on the daily scores of the 5 items. Questionnaires to be completed at site at screening for training purpose. PAH-SYMPACT to be performed at home, during the 7-day period prior to the scheduled visit.
Baseline up to Week 28
Change from baseline to Week 28 in Euro Quality of life-5-Dimension-5-Level (EQ-5D-5L) Utility Score
Time Frame: Baseline up to Week 28
The EQ-5D-5L is a generic measure of health status. The EQ-5D-5L is a 5-item questionnaire that assesses 5 domains including mobility, self-care, usual activities, pain/discomfort and anxiety/depression plus a visual analog scale rating "health today" with anchors ranging from 0 (worst imaginable health state) to 100 (best imaginable health state).
Baseline up to Week 28
Change From Baseline to Week 28 in Accelerometer-assessed Proportion of Time Spent in Moderate to Vigorous Physical Activity
Time Frame: Baseline up to Week 28
Change from baseline to week 28 in accelerometer-assessed proportion of time spent in moderate to vigorous physical activity will be assessed.
Baseline up to Week 28

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Actelion

Investigators

  • Study Director: Actelion Clinical Trial, Actelion

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 7, 2020

Primary Completion (Actual)

December 21, 2023

Study Completion (Actual)

December 21, 2023

Study Registration Dates

First Submitted

February 13, 2020

First Submitted That Met QC Criteria

February 13, 2020

First Posted (Actual)

February 17, 2020

Study Record Updates

Last Update Posted (Estimated)

February 2, 2024

Last Update Submitted That Met QC Criteria

February 1, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CR108742
  • 2019-004131-24 (EudraCT Number)
  • 67896062CTP3001 (Other Identifier: Actelion)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

The data sharing policy of the Janssen Pharmaceutical Companies of Johnson & Johnson is available at www.janssen.com/clinical-trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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