Clinical trial • Phase III • Cardiology

MACITENTAN for Pulmonary arterial hypertension

Phase III trial of MACITENTAN for Pulmonary arterial hypertension.

Overview

Trial Therapeutic Area
Cardiology
Trial Disease
Pulmonary arterial hypertension
Trial Stage
Phase III
Drug Modality
Small molecule
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
28-06-2024
First CTIS Authorization Date
30-07-2024

Trial design

Randomised, open-label, macitentan 10 mg (opsumit 10 mg film-coated tablets) oral; compared with macitentan 75 mg in a double-dummy randomized design (matching placebos used).-controlled, adaptive Phase III trial across 44 sites in Belgium, France, Poland and others.

Randomised
Yes
Open Label
Yes
Comparator
Macitentan 10 mg (Opsumit 10 mg film-coated tablets) oral; compared with macitentan 75 mg in a double-dummy randomized design (matching placebos used).
Adaptive
True; study described as group-sequential, adaptive, event-driven — i.e. a group-sequential/event-driven design with interim analyses to allow decision-making based on event accumulation (no further adaptive rules/detail provided in the public summary).
Target Sample Size
790

Eligibility

Recruits 790 Vulnerable-population handling: participants must sign an informed consent form (ICF) or their legally acceptable representative must sign if applicable ("Must sign an ICF (or their legally acceptable representative must sign) indicating that he or she understands the purpose of, and procedures required for, the study and is willing to participate in the study."). Separate ICF required for optional biomarker/sample collection; refusal of optional biomarker consent does not exclude participation. Country-specific ICF and language versions provided (multiple country/language-specific consent documents are listed in the trial documents)..

Pregnancy Exclusion
A female participant of childbearing potential must have a negative highly sensitive serum (β-human chorionic gonadotropin [β-hCG]) test at Screening and a negative urine pregnancy test prior to receiving their first dose of study intervention (i.e. either at beginning of the run-in period or prior to randomization [see Section 4.1]).
Vulnerable Population
Vulnerable-population handling: participants must sign an informed consent form (ICF) or their legally acceptable representative must sign if applicable ("Must sign an ICF (or their legally acceptable representative must sign) indicating that he or she understands the purpose of, and procedures required for, the study and is willing to participate in the study."). Separate ICF required for optional biomarker/sample collection; refusal of optional biomarker consent does not exclude participation. Country-specific ICF and language versions provided (multiple country/language-specific consent documents are listed in the trial documents).

Inclusion criteria

  • {"criterion_text":"- Target population: ≥ 18 (or the legal age of consent in the jurisdiction in which the study is taking place) years of age.\n- A female participant of childbearing potential must have a negative highly sensitive serum (β-human chorionic gonadotropin [β-hCG]) test at Screening and a negative urine pregnancy test prior to receiving their first dose of study intervention (i.e. either at beginning of the run-in period or prior to randomization [see Section 4.1]).\n- A female participant must be (as defined in Appendix 6 (Contraceptive and Barrier Guidance and Collection ) a) Not of childbearing potential, b) Of childbearing potential and - Practicing a highly effective, preferably user-independent method of contraception (failure rate of < 1% per year when used consistently and correctly) and agrees to remain on a highly effective method while receiving study intervention and until 30 days after last dose - the end of relevant systemic exposure. Examples of highly effective methods of contraception are located in Appendix 6. (Contraceptive and Barrier Guidance and Collection )\n- Willing and able to adhere to the lifestyle restrictions specified in this protocol.\n- A Belgium-specific inclusion criterion is detailed in Section 10.19 (see Appendix 19: Country/Territory-Specific Requirements).\n- Target population: Symptomatic PAH in WHO FC II, III, or IV.\n- Must sign an ICF (or their legally acceptable representative must sign) indicating that he or she understands the purpose of, and procedures required for, the study and is willing to participate in the study.\n- Target population: PAH subtype falling in one of the below classifications: - Idiopathic - Heritable - Drug- or toxin-induced - Related to: *Connective tissue disease, *HIV infection, *Portal hypertension *Congenital heart disease with o small/coincidental cardiac defect with systemic-to-pulmonary shunt (eg, atrial septal defect, ventricular septal defect, patent ductus arteriosus, atrioventricular septal defect) which does not account for the elevated PVR or o persistent PAH documented by an RHC ≥ 1 year after simple systemicto pulmonary shunt repair.\n- PAH diagnosis confirmed by hemodynamic evaluation at rest at any time prior to Screening: - Mean pulmonary artery pressure (mPAP) > 20 mm Hg, AND - Pulmonary artery wedge pressure (PAWP) or left ventricular end diastolic pressure (LVEDP) ≤ 15 mm Hg, AND - PVR ≥ 3 Wood Units (ie, ≥ 240 dyn∙sec∙cm−5).\n- Negative vasoreactivity test in idiopathic, heritable, and drug/toxininduced PAH. Patients for whom no vasoreactivity test was performed at diagnosis and currently treated with PAH therapy for more than 3 months, must have a confirmatory PAH diagnosis documented by hemodynamic evaluation at least 3 months after introduction of their PAH therapy.\n- Able to perform the 6MWT with a minimum distance of 50 m and maximum distance of 440 m at Screening. Patients able to walk more than 440 m at screening are eligible if they are in WHO FC III or IV and NT-proBNP level is ≥ 300 ng/L at screening, based on central laboratory results.\n- Patients already receiving PAH therapies (mono or combination therapies) must be on a stable regimen b for at least 3 months prior to screening visit and planned to be: - If on ERA therapy: discontinued at randomization or start of run-in (ie, last dose of ERA taken the day before initiating study intervention), - If on PAH therapy other than ERA: maintained on top of the study intervention.\n- Must sign a separate informed consent form (or their legallyacceptable representative must sign) if he or she agrees to provide optional samples for biomarker research (where local regulations permit). Refusal to give consent for the optional biomarker research samples does not exclude a participant from participation in the study."}

Exclusion criteria

  • {"criterion_text":"- Treatment with a strong CYP3A4 inducer (eg, rifabutin, rifampin, rifampicin, rifapentin, carbamazepine, phenobarbital, phenytoin, St. John's Wort) within 1 month prior to randomization or start of run-in, if applicable.\n- Treatment with a strong CYP3A4 inhibitor or a moderate dual CYP3A4/CYP2C9 inhibitor, or co-administration of a combination of moderate CYP3A4 and moderate CYP2C9 inhibitors in the 1-month period prior to randomization, or start of run-in, if applicable. External use (cream, shampoo, etc) per approved label is permitted.\n- Known moderate to severe hepatic impairment, defined as Child- Pugh Class B or C (see Appendix 5), based on records that confirm documented medical history.\n- Serum aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT)> 1.5 X upper limit of normal (ULN) at Screening.\n- Hemoglobin < 100 g/L (< 10 g/dL) at Screening.\n- Severe renal impairment as defined with an estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73m2 (Chronic Kidney Disease Epidemiology Collaboration [CKD EPI] 2009 equation) at screening\n- Systemic hypotension (systolic blood pressure [SBP] < 90 or diastolic blood pressure [DBP] < 50 mm Hg) at Screening.\n- For selected sites taking part to the cardiac MRI sub-study only: participants must not be considered for this sub-study in case of MRIincompatible permanent cardiac pacemaker, automatic internal cardioverter, metallic implant (eg, defibrillator, neurostimulator, hearing aid, permanent use of infusion device), multiple premature ventricular or atrial contractions, or any other condition that may confound cardiac MRI assessment or for which, in the opinion of the investigator, participation would not be in the best interests of the participant (eg, compromise well-being).\n- For participants involved in the cardiac remodeling and/or hemodynamic substudies only: Diuretic treatment initiated or dose changed within 1 week prior to the MRI or RHC assessment.\n- Known presence of three or more of the following risk factors for heart failure with preserved ejection fraction at Screening, based on records that confirm documented medical history: - Body mass index (BMI) > 30 kg/m2, - Diabetes mellitus of any type, - Essential hypertension (even if well controlled), - Coronary artery disease, ie, any of the following: *History of stable angina, or *Known more than 50% stenosis in a coronary artery, or *History of myocardial infarction, or *History of or planned coronary artery bypass grafting and/or coronary artery stenting.\n- Presence of moderate or severe obstructive lung disease (forced expiratory volume in 1 second [FEV1] / forced vital capacity [FVC] < 70%; and FEV1 < 60% of predicted after bronchodilator administration) in participants with a known or suspected history of significant lung disease, as documented by a spirometry test performed within 1 year prior to Screening.\n- Presence of moderate or severe restrictive lung disease (eg, total lung capacity [TLC] or FVC < 60% of normal predicted value) in participants with a known or suspected history of significant lung disease, as documented by a spirometry test performed within 1 year prior to Screening.\n- Significant unrepaired structural left heart valvular disease (ie, moderate or severe aortic or mitral stenosis or regurgitation); pericardial constriction; restrictive or congestive left-sided cardiomyopathy; life-threatening cardiac arrhythmias; significant left ventricular dysfunction; or left ventricular outflow obstruction.\n- Permanent atrial fibrillation or atrial flutter, in the opinion of the investigator.\n- Known or suspected pulmonary veno-occlusive disease (PVOD)."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Time to first CEC-adjudicated M/M event on-treatment (ie, up to 7 days after the last dose of double-blind (DB) study intervention)","definition_or_measurement_approach":"Time-to-event measure: time from randomization (or start of on-treatment period) to the first morbidity or mortality (M/M) event adjudicated by the Clinical Events Committee (CEC) while on-treatment; events counted up to 7 days after the last dose of the double-blind study intervention."}

Recruitment

Planned Sample Size
790
Recruitment Window Months
109
Consent Approach
Participants (≥18 years) must provide written informed consent; if unable to consent a legally acceptable representative may sign ("Must sign an ICF (or their legally acceptable representative must sign)"). Separate informed consent is required for optional biomarker/sample collection and for optional substudies; refusal to consent to optional samples does not exclude participation. Country/language-specific subject information sheets and ICFs are provided (multiple translated ICFs and country addenda listed in the trial documents).

Geography

Total Number Of Sites
44
Total Number Of Participants
110

Belgium

Earliest CTIS Part Ii Submission Date
17-07-2024
Latest Decision Or Authorization Date
11-05-2026
Processing Time Days
663
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
UZ Leuven
Department Name
Pneumology
Principal Investigator Name
Marion Delcroix
Principal Investigator Email
marion.delcroix@uzleuven.be
Contact Person Name
Marion Delcroix
Contact Person Email
marion.delcroix@uzleuven.be

France

Earliest CTIS Part Ii Submission Date
17-07-2024
Latest Decision Or Authorization Date
12-05-2026
Processing Time Days
664
Number Of Sites
4
Number Of Participants
7

Sites

Site Name
Assistance Publique Hopitaux De Paris
Department Name
Pneumology Department
Principal Investigator Name
Olivier SITBON
Principal Investigator Email
olivier.sitbon@u-psud.fr
Contact Person Name
Olivier SITBON
Contact Person Email
olivier.sitbon@u-psud.fr
Site Name
Centre Hospitalier Universitaire De Saint Etienne
Department Name
Vascular Medecine Department
Principal Investigator Name
Laurent BERTOLETTI
Principal Investigator Email
laurent.bertoletti@chu-st-etienne.fr
Contact Person Name
Laurent BERTOLETTI
Site Name
Centre Hospitalier Universitaire De Toulouse
Department Name
Pneumology Department
Principal Investigator Name
Grégoire PREVOT
Principal Investigator Email
prevot.g@chu-toulouse.fr
Contact Person Name
Grégoire PREVOT
Contact Person Email
prevot.g@chu-toulouse.fr
Site Name
Centre Hospitalier Universitaire De Nice
Department Name
Cardiology Department
Principal Investigator Name
Delphine BAUDOUY
Principal Investigator Email
baudouy.d@chu-nice.fr
Contact Person Name
Delphine BAUDOUY
Contact Person Email
baudouy.d@chu-nice.fr

Poland

Earliest CTIS Part Ii Submission Date
17-07-2024
Latest Decision Or Authorization Date
08-05-2026
Processing Time Days
660
Number Of Sites
6
Number Of Participants
16

Sites

Site Name
Uniwersytecki Szpital Kliniczny W Bialymstoku
Department Name
Klinika Kardiologii z Oddzialem Intensywnego Nadzoru Kardiologicznego
Principal Investigator Name
Karol Kamiński
Principal Investigator Email
kardio@uskwb.pl
Contact Person Name
Karol Kamiński
Contact Person Email
kardio@uskwb.pl
Site Name
Gornoslaskie Centrum Medyczne Im Prof. Leszka Gieca Sląskiego Uniwersytetu Medycznego W Katowicach
Department Name
I Katedra i Klinika Kardiologii SUM I Oddział Kardiologii
Principal Investigator Name
Katarzyna Mizia-Stec
Principal Investigator Email
badania.gcm@gmail.com
Contact Person Name
Katarzyna Mizia-Stec
Contact Person Email
badania.gcm@gmail.com
Site Name
Panstwowy Instytut Medyczny Ministerstwa Spraw Wewnetrznych I Administracji
Department Name
Klinika Kardiologii Inwazyjnej
Principal Investigator Name
Agnieszka Pawlak
Principal Investigator Email
kardiologia@cskmswia.gov.pl
Contact Person Name
Agnieszka Pawlak
Contact Person Email
kardiologia@cskmswia.gov.pl
Site Name
Krakowski Szpital Specjalistyczny Im. Sw. Jana Pawla II
Department Name
Oddzial Kliniczny Chorob Serca i Naczyn z Pododdziałem Intensywnego Nadzoru Kardiologicznego
Principal Investigator Name
Grzegorz Kopeć
Principal Investigator Email
sekr_kard@szpitaljp2.krakow.pl
Contact Person Name
Grzegorz Kopeć
Contact Person Email
sekr_kard@szpitaljp2.krakow.pl
Site Name
Uniwersytecki Szpital Kliniczny Nr 2 Pum W Szczecinie
Department Name
Klinika Kardiologii z Intensywnym Nadzorem Kardiologicznym
Principal Investigator Name
Małgorzata Peregud-Pogorzelska
Principal Investigator Email
kardiologia@spsk2-szczecin.pl
Contact Person Name
Małgorzata Peregud-Pogorzelska
Contact Person Email
kardiologia@spsk2-szczecin.pl
Site Name
Uniwersyteckie Centrum Kliniczne
Department Name
I Klinik Kardiologii Oddzial Intensywnej Terapii Kardiologicznej
Principal Investigator Name
Wiesław Puchalski
Principal Investigator Email
puchalski@uck.gda.pl
Contact Person Name
Wiesław Puchalski
Contact Person Email
puchalski@uck.gda.pl

Germany

Earliest CTIS Part Ii Submission Date
17-07-2024
Latest Decision Or Authorization Date
08-05-2026
Processing Time Days
660
Number Of Sites
3
Number Of Participants
7

Sites

Site Name
Universitaetsklinikum Regensburg AöR
Department Name
Klinik und Poliklinik für Innere Medizin II
Principal Investigator Name
Stefan Stadler
Principal Investigator Email
Stefan.Stadler@klinik.uni-regensburg.de
Contact Person Name
Stefan Stadler
Site Name
Medizinische Hochschule Hannover
Department Name
Abt. für Pulmonologie
Principal Investigator Name
Karen Olsson
Principal Investigator Email
Olsson.karen@mh-hannover.de
Contact Person Name
Karen Olsson
Contact Person Email
Olsson.karen@mh-hannover.de
Site Name
Klinikum der Universitaet Muenchen AöR
Department Name
Medizinische Klinik und Poliklinik V (Pneumologie)
Principal Investigator Name
Katrin Milger-Kneidinger
Principal Investigator Email
Katrin.Milger@med.uni-muenchen.de
Contact Person Name
Katrin Milger-Kneidinger

Denmark

Earliest CTIS Part Ii Submission Date
17-07-2024
Latest Decision Or Authorization Date
07-05-2026
Processing Time Days
659
Number Of Sites
1
Number Of Participants
3

Sites

Site Name
Aarhus Universitetshospital
Department Name
Afdeling B for Hjertesygdomme
Principal Investigator Name
Soren Mellemkjaer
Principal Investigator Email
Soren.Mellemkjaer@rm.dk
Contact Person Name
Soren Mellemkjaer
Contact Person Email
Soren.Mellemkjaer@rm.dk

Czechia

Earliest CTIS Part Ii Submission Date
17-07-2024
Latest Decision Or Authorization Date
11-05-2026
Processing Time Days
663
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
Vseobecna Fakultni Nemocnice V Praze
Department Name
2. interni klinika
Principal Investigator Name
Pavel Jansa
Principal Investigator Email
pavel.jansa@vfn.cz
Contact Person Name
Pavel Jansa
Contact Person Email
pavel.jansa@vfn.cz

Bulgaria

Earliest CTIS Part Ii Submission Date
17-07-2024
Latest Decision Or Authorization Date
11-05-2026
Processing Time Days
663
Number Of Sites
2
Number Of Participants
7

Sites

Site Name
University Hospital St. Anna
Department Name
Clinic of cardiology
Principal Investigator Name
Sarkis Kalustian
Principal Investigator Email
d_rkalustian@abv.bg
Contact Person Name
Sarkis Kalustian
Contact Person Email
d_rkalustian@abv.bg
Site Name
MHAT National Heart Hospital EAD
Department Name
Clinic of cardiology
Principal Investigator Name
Danail Avramov
Principal Investigator Email
dungobest@hotmail.com
Contact Person Name
Danail Avramov
Contact Person Email
dungobest@hotmail.com

Austria

Earliest CTIS Part Ii Submission Date
17-07-2024
Latest Decision Or Authorization Date
11-05-2026
Processing Time Days
663
Number Of Sites
3
Number Of Participants
3

Sites

Site Name
University Hospital Graz
Department Name
Universitätsklinik für Innere Medizin, klinische Abteilung für Pulmonologie
Principal Investigator Name
Gabor Kovacs
Principal Investigator Email
gabor.kovacs@uniklinikum.kages.at
Contact Person Name
Gabor Kovacs
Site Name
Medical University Of Vienna
Department Name
Universitätsklinik für Innere Medizin II, Klinische Abteilung für Kardiologie
Principal Investigator Name
Irene Marthe Lang
Principal Investigator Email
irene.lang@meduniwien.ac.at
Contact Person Name
Irene Marthe Lang
Contact Person Email
irene.lang@meduniwien.ac.at
Site Name
Ordensklinikum Linz GmbH
Principal Investigator Name
Regina Steringer-Mascherbauer
Contact Person Name
Regina Steringer-Mascherbauer

Greece

Earliest CTIS Part Ii Submission Date
17-07-2024
Latest Decision Or Authorization Date
07-05-2026
Processing Time Days
659
Number Of Sites
3
Number Of Participants
4

Sites

Site Name
Alexandra Hospital
Department Name
Heart Failure and Cardio-oncology Clinic/ Therapeutic Clinic
Principal Investigator Name
Alexandros Briasoulis
Principal Investigator Email
abriasoulis@med.uoa.gr
Contact Person Name
Alexandros Briasoulis
Contact Person Email
abriasoulis@med.uoa.gr
Site Name
University General Hospital Of Thessaloniki Ahepa
Department Name
Cardiology Clinic, Congenital Heart diseases and Pulmonary Arterial Hypertension
Principal Investigator Name
Georgios Giannakoulas
Principal Investigator Email
g.giannakoulas@gmail.com
Contact Person Name
Georgios Giannakoulas
Contact Person Email
g.giannakoulas@gmail.com
Site Name
General University Hospital Of Larissa
Department Name
University Cardiology Clinic
Principal Investigator Name
Ioannnis Skoularingis
Principal Investigator Email
iskoular@med.uth.gr
Contact Person Name
Ioannnis Skoularingis
Contact Person Email
iskoular@med.uth.gr

Sweden

Earliest CTIS Part Ii Submission Date
17-07-2024
Latest Decision Or Authorization Date
07-05-2026
Processing Time Days
659
Number Of Sites
1
Number Of Participants
1

Sites

Site Name
Sahlgrenska University Hospital-Vaestra Goetalandsregionen
Department Name
Kardiologens forskningsenhet
Principal Investigator Name
Sven-Erik Bartfay
Principal Investigator Email
sven-erik.bartfay@vgregion.se
Contact Person Name
Sven-Erik Bartfay
Contact Person Email
sven-erik.bartfay@vgregion.se

Portugal

Earliest CTIS Part Ii Submission Date
17-07-2024
Latest Decision Or Authorization Date
11-05-2026
Processing Time Days
663
Number Of Sites
2
Number Of Participants
15

Sites

Site Name
Unidade Local De Saude De Santa Maria E.P.E.
Department Name
Departamento de Coracao e Vasos
Principal Investigator Name
Rui Plácido
Principal Investigator Email
20808@chln.min-saude.pt
Contact Person Name
Rui Plácido
Contact Person Email
20808@chln.min-saude.pt
Site Name
Unidade Local De Saude De Coimbra E.P.E.
Department Name
Serviço de Cardiologia - Departamento Coracao e Vasos
Principal Investigator Name
Maria da Graca Castro
Principal Investigator Email
castro2406@gmail.com
Contact Person Name
Maria da Graca Castro
Contact Person Email
castro2406@gmail.com

Italy

Earliest CTIS Part Ii Submission Date
17-07-2024
Latest Decision Or Authorization Date
08-05-2026
Processing Time Days
660
Number Of Sites
4
Number Of Participants
5

Sites

Site Name
Fondazione IRCCS Policlinico San Matteo
Department Name
U.O. Cardiologia
Principal Investigator Name
Stefano Ghio
Principal Investigator Email
s.ghio@smatteo.pv.it
Contact Person Name
Stefano Ghio
Contact Person Email
s.ghio@smatteo.pv.it
Site Name
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Department Name
U.O.C. Pneumologia
Principal Investigator Name
Fabiana Baldi
Principal Investigator Email
fabiana.baldi@policlinicogemelli.it
Contact Person Name
Fabiana Baldi
Site Name
ASST Grande Ospedale Metropolitano Niguarda
Department Name
Cardiologia 2 - Insufficienza Cardiaca e Trapianti
Principal Investigator Name
Andrea Garascia
Principal Investigator Email
andrea.garascia@ospedaleniguarda.it
Contact Person Name
Andrea Garascia
Site Name
Fondazione Toscana Gabriele Monasterio
Department Name
Dipartimento Cardiotoracico U.O.S.V.D. Pneumologia
Principal Investigator Name
Edoardo Airò
Principal Investigator Email
edoardo.airo@ftgm.it
Contact Person Name
Edoardo Airò
Contact Person Email
edoardo.airo@ftgm.it

Spain

Earliest CTIS Part Ii Submission Date
17-07-2024
Latest Decision Or Authorization Date
11-05-2026
Processing Time Days
663
Number Of Sites
5
Number Of Participants
12

Sites

Site Name
Hospital Universitario 12 De Octubre
Department Name
Cardiology
Principal Investigator Name
Pilar Escribano
Principal Investigator Email
hipertensionpulmonar.hdoc@salud.madrid.org
Contact Person Name
Pilar Escribano
Site Name
Hospital Costa Del Sol
Department Name
Cardiology
Principal Investigator Name
Rafael Bravo Marques
Principal Investigator Email
rafael.bravo.sspa@juntadeandalucia.es
Contact Person Name
Rafael Bravo Marques
Site Name
Hospital Universitario Puerta De Hierro De Majadahonda
Department Name
Cardiology
Principal Investigator Name
Javier Segovia Cubero
Principal Investigator Email
jsecu@telefonica.net
Contact Person Name
Javier Segovia Cubero
Contact Person Email
jsecu@telefonica.net
Site Name
Hospital Universitario La Paz
Department Name
Pneumology
Principal Investigator Name
Sergio Alcolea
Principal Investigator Email
alcobatres@yahoo.es
Contact Person Name
Sergio Alcolea
Contact Person Email
alcobatres@yahoo.es
Site Name
Hospital Universitario De Salamanca
Department Name
Pneumology
Principal Investigator Name
Sergio Cadenas
Principal Investigator Email
sercam.2007@gmail.com
Contact Person Name
Sergio Cadenas
Contact Person Email
sercam.2007@gmail.com

Hungary

Earliest CTIS Part Ii Submission Date
17-07-2024
Latest Decision Or Authorization Date
11-05-2026
Processing Time Days
663
Number Of Sites
3
Number Of Participants
15

Sites

Site Name
University Of Pecs
Principal Investigator Name
Réka Faludi
Principal Investigator Email
faludi.reka@pte.hu
Contact Person Name
Réka Faludi
Contact Person Email
faludi.reka@pte.hu
Site Name
Clinic Of Pulmonology Semmelweis University
Principal Investigator Name
Kristóf Karlócai
Principal Investigator Email
kristof@karlocai.hu
Contact Person Name
Kristóf Karlócai
Contact Person Email
kristof@karlocai.hu
Site Name
University Of Szeged
Principal Investigator Name
Albert Varga
Principal Investigator Email
varga.albert@med.u-szeged.hu
Contact Person Name
Albert Varga
Contact Person Email
varga.albert@med.u-szeged.hu

Netherlands

Earliest CTIS Part Ii Submission Date
17-07-2024
Latest Decision Or Authorization Date
07-05-2026
Processing Time Days
659
Number Of Sites
3
Number Of Participants
8

Sites

Site Name
Sint Antonius Ziekenhuis Stichting
Department Name
Cardiology
Principal Investigator Name
Marco Post
Principal Investigator Email
m.post@antoniusziekenhuis.nl
Contact Person Name
Marco Post
Contact Person Email
m.post@antoniusziekenhuis.nl
Site Name
Stichting Amsterdam UMC
Department Name
Pneumology
Principal Investigator Name
Harm Jan Bogaard
Principal Investigator Email
hj.bogaard@amsterdamumc.nl
Contact Person Name
Harm Jan Bogaard
Contact Person Email
hj.bogaard@amsterdamumc.nl
Site Name
Stichting Radboud universitair medisch centrum
Department Name
Cardiology
Principal Investigator Name
Arie van Dijk
Principal Investigator Email
Arie.vanDijk@radboudumc.nl
Contact Person Name
Arie van Dijk
Contact Person Email
Arie.vanDijk@radboudumc.nl

Slovakia

Earliest CTIS Part Ii Submission Date
17-07-2024
Latest Decision Or Authorization Date
06-05-2026
Processing Time Days
658
Number Of Sites
2
Number Of Participants
3

Sites

Site Name
Národny ustav srdcovych a cievnych chorob a.s.
Department Name
Oddelenie zlyhávania a transplantácie
Principal Investigator Name
Eva Goncalvesová
Principal Investigator Email
EVA.GONCALVESOVA@nusch.sk
Contact Person Name
Eva Goncalvesová
Contact Person Email
EVA.GONCALVESOVA@nusch.sk
Site Name
Vychodoslovensky Ustav Srdcovych A Cievnych Chorob a.s.
Department Name
Oddelenie kardiológie
Principal Investigator Name
Bibiana Kafková
Principal Investigator Email
bibakafkova@gmail.com
Contact Person Name
Bibiana Kafková
Contact Person Email
bibakafkova@gmail.com

Sponsor

Primary sponsor

Full Name
Actelion Pharmaceuticals Ltd.
Organisation Type
Pharmaceutical company
Country Of Registered Address
Switzerland

Contract research organisations

Name
Iqvia Inc.
Responsibilities
Country & Site Management
Name
Perceptive Informatics Inc.
Responsibilities
cMRI Central reading
Name
Pharmaceutical Research Associates Group B.V.
Responsibilities
PK analysis, ET-1
Name
Labcorp Central Laboratory Services LP
Responsibilities
Central laboratory services: Glomerular Filtration Rate, NT-proBNP
Name
Eresearchtechnology Inc.
Responsibilities
central ECG reading, ePRO
Name
WCG Clinical Inc.
Responsibilities
Central Adjudication of Event

Third parties

  • {"country":"United States","full_name":"Perceptive Informatics Inc.","duties_or_roles":"cMRI Central reading","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Iqvia Inc.","duties_or_roles":"Country & Site Management","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"central ECG reading, ePRO","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Signant Health Global LLC","duties_or_roles":"IVRS – treatment randomisation","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Frontier Science & Technology Research Foundation Inc.","duties_or_roles":"interaction with IDMC","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"Glomerular Filtration Rate, NT-proBNP","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"EPL Pathology Archives LLC","duties_or_roles":"Biostorage service for optional biomarker research","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"WCG Clinical Inc.","duties_or_roles":"Central Adjudication of Event","organisation_type":"Pharmaceutical company"}
  • {"country":"Netherlands","full_name":"Pharmaceutical Research Associates Group B.V.","duties_or_roles":"PK analysis, ET-1","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
JNJ 67896062 (macitentan 75 mg film-coated tablet)
Active Substance
MACITENTAN
Modality
Small molecule
Routes Of Administration
ORAL
Route
ORAL
Dose Levels
75 mg
Maximum Dose
75 mg
Investigational Product Name
JNJ 67896062 (macitentan 37 mg film-coated tablet)
Active Substance
MACITENTAN
Modality
Small molecule
Routes Of Administration
ORAL
Route
ORAL
Dose Levels
37 mg
Maximum Dose
37 mg
Investigational Product Name
Opsumit 10 mg film-coated tablets
Active Substance
MACITENTAN
Modality
Small molecule
Routes Of Administration
ORAL USE
Route
ORAL USE
Authorisation Status
Marketing authorisation EU/1/13/893/001
Orphan Designation
Yes
Starting Dose
10 mg
Dose Levels
10 mg
Maximum Dose
10 mg
Investigational Product Name
Matching Placebo for Macitentan 75 mg
Modality
Other
Investigational Product Name
Matching Placebo for Macitentan 37.5 mg
Modality
Other
Investigational Product Name
Matching Placebo for Macitentan 10 mg
Modality
Other
Combination Treatment
Yes

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