A Study Providing Treatment Access in Participants With Pulmonary Hypertension Completing a Parent Study and Having no Other Option

Last updated: May 22, 2025
Sponsor: Actelion
Overall Status: Active - Recruiting

Phase

3

Condition

Stress

Circulation Disorders

Williams Syndrome

Treatment

Macitentan/Tadalafil FDC

Macitentan

Selexipag

Clinical Study ID

NCT05179876
CR109121
2021-002297-11
NOPRODPAPUH3001
2023-506791-27-00
  • Ages > 2
  • All Genders

Study Summary

The purpose of the study is to enable participants with pulmonary hypertension (PH) currently treated with study intervention(s) in a clinical study (parent studies [NCT03422328, NCT03904693,NCT04565990, NCT02932410, NCT03492177, and NCT04175600]), to continue to benefit from the intervention after closure of the parent study in case they have no alternative means of access to the study intervention. This study will allow assessment of the long-term safety of each study intervention.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Participant must sign an informed consent form (ICF) (or their legally designatedrepresentative must sign) indicating that participant understands the purpose of,and procedures required for, the study and is willing to participate in the study

  • Participant treated with oral macitentan or selexipag or fixed dose combination (FDC) of macitentan 10 milligrams (mg) and tadalafil 40 mg at the end of a sponsorparent study and: a) the indication of the parent study is included in theintervention-specific appendices (ISA) (pulmonary arterial hypertension [PAH]; b)participant has completed the parent study; c) no alternative means of access tostudy intervention (or equivalent approved therapy) have been identified; d)participant may continue to benefit from treatment with the study intervention; e)Participant is at least 18 years old for macitentan/tadalafil FDC, and at least 2years old for macitentan or selexipag

  • A female participant of childbearing potential must: a) have a negative urine orserum pregnancy test prior to first intake of study intervention; b) agree toperform monthly urine pregnancy test up to the end of the safety follow-up period;c) If heterosexually active, agree to follow contraceptive methods until 30 daysafter the last intake of the study intervention. For pediatric female participants:It is the responsibility of the investigator to ensure appropriate counselling,including consultation with a specialist (if needed), to the participant and/orparent(s)/ legally designated representative (LDR)(s) on the acceptable method ofcontraception

Exclusion

Exclusion Criteria:

General:

  • Participants prematurely discontinued from the study intervention in their parentstudy

  • Female participant being pregnant, or breastfeeding, or planning to become pregnantwhile enrolled in this study

  • Planned or current treatment with another investigational treatment

Macitentan-specific:

  • Known allergies, hypersensitivity, or intolerance to macitentan or its excipients

  • Hemoglobin less than (<) 80 grams per liter (g/L)

  • Serum aspartate (AST) and/or alanine aminotransferases (ALT) greater than (>) 3*upper limit of normal (ULN)

  • Known and documented severe hepatic impairment that is, Child-Pugh Class C. Forparticipants with hepatic impairment, Child-Pugh Class (Child-Pugh score) should befully assessed and documented in the source documents at screening

Selexipag-specific:

  • Known allergies, hypersensitivity, or intolerance to selexipag or its excipients

  • Suspected or known pulmonary veno-occlusive disease (PVOD)

  • Uncontrolled thyroid disease

  • Severe coronary heart disease or unstable angina, myocardial infarction within thelast 6 months, decompensated cardiac failure (if not under close medicalsupervision), severe arrhythmia, cerebrovascular events (for example, transientischemic attack, stroke) within the last 3 months, or congenital or acquiredvalvular defects with clinically relevant myocardial function disorders not relatedto pulmonary hypertension (PH)

  • Known and documented severe hepatic impairment that is, Child-Pugh Class C. Forparticipants with hepatic impairment, Child-Pugh Class (Child-Pugh score) should befully assessed and documented in the source documents at screening

  • Children only: (a) Current suspicion of intussusception or ileus or gastrointestinalobstruction, per the investigator's judgment; (b) hemoglobin or hematocrit <75percent (%) of the lower limit of normal range

Macitentan/tadalafil FDC-specific:

  • Known allergies, hypersensitivity, or intolerance to macitentan or tadalafil ortheir excipients

  • Hemoglobin <80 g/L

  • Serum aspartate (AST) and/or alanine aminotransferases (ALT) >3* ULN range

  • Known and documented severe hepatic impairment that is, Child-Pugh Class C. Forparticipants with hepatic impairment, Child-Pugh Class should be fully assessed anddocumented in the source documents at screening

  • Severe renal impairment (estimated glomerular filtration rate [eGF]/creatinineclearance <30 milliliter per minute [mL/min])

Study Design

Total Participants: 280
Treatment Group(s): 4
Primary Treatment: Macitentan/Tadalafil FDC
Phase: 3
Study Start date:
May 04, 2022
Estimated Completion Date:
September 28, 2029

Connect with a study center

  • Minsk Regional Clinical Hospital Of The Red Banner Of Labor

    Minsk,
    Belarus

    Active - Recruiting

  • The Republican Scientific-Practical Center ''Cardiology''

    Minsk, 220036
    Belarus

    Active - Recruiting

  • UZ Leuven

    Leuven, 3000
    Belgium

    Completed

  • University Multiprofile Hospital for Active Treatment- UMHAT Sveta Anna AD

    Sofia, 1750
    Bulgaria

    Active - Recruiting

  • Beijing Anzhen Hospital

    Beijing, 100029
    China

    Active - Recruiting

  • The Second Xiangya Hospital of Central South Hospital

    Changsha, 410011
    China

    Active - Recruiting

  • Jiangsu Province Hospital

    Nanjing,
    China

    Active - Recruiting

  • The First Affiliated Hospital of Xian Jiaotong University

    Xi'An, 710061
    China

    Active - Recruiting

  • CHRU Besançon -Hôpital Jean Minjoz

    Besançon, 25030
    France

    Site Not Available

  • CHU de Bordeaux - Hospital Haut-Leveque

    Bordeaux (Pessac), 33604
    France

    Site Not Available

  • GH est - Hôpital Cardiovasculaire et Pneumologie Louis Pradel

    Bron Cedex, 69677
    France

    Site Not Available

  • Hôpital Côte de Nacre

    Caen Cedex,
    France

    Site Not Available

  • CHU Dijon

    Dijon Cedex, 21079
    France

    Site Not Available

  • CHU Grenoble

    Grenoble, 38700
    France

    Site Not Available

  • Hopital Bicêtre - Aphp Hôpitaux Universitaires Paris-Sud

    Le Kremlin Bicetre cedex, 94275
    France

    Site Not Available

  • Hôpital Cardiologique - Chru Lille

    Lille Cedex, 59037
    France

    Site Not Available

  • CHU de Limoges

    Limoges Cedex, 87042
    France

    Site Not Available

  • CHU de la Timone

    Marseille cedex 5, 13385
    France

    Site Not Available

  • CHU de Montpellier - Arnaud de Villeneuve

    Montpellier, 34295
    France

    Site Not Available

  • CHU Nantes - Hopital Nord Laënnec

    Nantes Cedex 1, 44093
    France

    Site Not Available

  • Centre Hospitalier Universitaire - de Nice - Hopital Pasteur

    Nice, 06001
    France

    Site Not Available

  • Hôpital Européen Georges Pompidou

    Paris Cedex 15, 75908
    France

    Site Not Available

  • CHU de Reims

    Reims, 51100
    France

    Site Not Available

  • Chu Rennes - Hopital Pontchaillou

    Rennes, 35033
    France

    Site Not Available

  • CHU Rouen - Hopital Charles Nicolle

    Rouen, 76031
    France

    Site Not Available

  • CHU Saint-Etienne - Hôpital Nord

    St Priest en Jarez Cedex, 42277
    France

    Site Not Available

  • Nouvel Hôpital Civil

    Strasbourg, 67091
    France

    Site Not Available

  • Hopital Larrey CHU de Toulouse

    Toulouse Cedex 9,
    France

    Site Not Available

  • CHU de Nancy - Hôpital de Brabois

    Vandoeuvre les Nancy Cedex, 54511
    France

    Site Not Available

  • Sanjivani Hospitals

    Ahmedabad, 380015
    India

    Site Not Available

  • Apollo Hospitals

    Chennai, 600006
    India

    Site Not Available

  • Chungnam National University Hospital

    Daejeon, 35015
    Korea, Republic of

    Active - Recruiting

  • Gachon University Gil Medical Center

    Incheon, 21565
    Korea, Republic of

    Active - Recruiting

  • Asan Medical Center

    Seoul, 05505
    Korea, Republic of

    Active - Recruiting

  • Samsung Medical Center

    Seoul, 06351
    Korea, Republic of

    Active - Recruiting

  • Seoul National University Hospital

    Seoul, 03080
    Korea, Republic of

    Active - Recruiting

  • The Catholic University of Korea Seoul St Marys Hospital

    Seoul, 06591
    Korea, Republic of

    Active - Recruiting

  • The Catholic University of Korea Seoul St. Mary's Hospital

    Seoul, 06591
    Korea, Republic of

    Active - Recruiting

  • Klinika Kardiologii Z Oddzialem Intensywnego Nadzoru Kardiologicznego UM W Bialymstoku

    Bialystok, 15 276
    Poland

    Active - Recruiting

  • Klinika Kardiologii z Oddzialem Intensywnego Nadzoru Kardiologicznego, UM w Białymstoku

    Białystok, 15-276
    Poland

    Site Not Available

  • Szpital Uniwersytecki nr 2 im dr Jana Biziela w Bydgoszczy, Klinika Kardiologii

    Bydgoszcz, 85-168
    Poland

    Completed

  • Uniwersyteckie Centrum Kliniczne

    Gdansk, 80-214
    Poland

    Site Not Available

  • SPSK nr 7 SUM w Katowicach Gornoslaskie Centrum Medyczne im Prof Leszka Gieca

    Katowice, 40 635
    Poland

    Active - Recruiting

  • SPSK nr 7 SUM w Katowicach Gornoslaskie Centrum Medyczne im. Prof. Leszka Gieca

    Katowice, 40-635
    Poland

    Active - Recruiting

  • Oddzial Kardiologii Wojewodzki Szpital Specjalistyczny im W Bieganskiego

    Lodz, 91 347
    Poland

    Active - Recruiting

  • Oddzial Kardiologii Wojewodzki Szpital Specjalistyczny im. W.Bieganskiego

    Lodz, 91-347
    Poland

    Active - Recruiting

  • Wojewodzki Szpital Specjalistyczny im Stefana Kardynala Wyszynskiego SPZOZ

    Lublin, 20 718
    Poland

    Active - Recruiting

  • Wojewodzki Szpital Specjalistyczny im. Stefana Kardynala Wyszynskiego SPZOZ

    Lublin, 20-718
    Poland

    Active - Recruiting

  • ECZ Otwock Klinika Kardiologii, Klinika Krążenia Płucnego Chorób Zakrzepowo-Zatorowych i Kardiologii

    Otwock, 05-400
    Poland

    Site Not Available

  • SPSK2 PUM Klinika Kardiologii

    Szczecin, 70 111
    Poland

    Active - Recruiting

  • SPSK2 PUM, Klinika Kardiologii

    Szczecin, 70-111
    Poland

    Active - Recruiting

  • Wojewodzki Szpital Specjalist Osrodek Badawczo Rozwojowy

    Wroclaw, 51 124
    Poland

    Active - Recruiting

  • Wojewodzki Szpital Specjalist, Osrodek Badawczo-Rozwojowy

    Wroclaw, 51-124
    Poland

    Active - Recruiting

  • Wojewodzki Szpital Specjalistyczny we Wroclawiu

    Wroclaw, 51 124
    Poland

    Active - Recruiting

  • Wojewodzki Szpital Specjalistyczny, Oddzial Kardiologiczny

    Wrocław, 51-124
    Poland

    Site Not Available

  • Institutul de pneumoftiziologie Marius Nasta

    Bucuresti, 050152
    Romania

    Site Not Available

  • Altay Regional Cardiological Dispensary

    Barnaul, 656055
    Russian Federation

    Site Not Available

  • Scientific and Research Institution of Cardiovascular Diseases Complex Problems

    Kemerovo, 650002
    Russian Federation

    Active - Recruiting

  • National Medical Research Center of Cardiology of MoH of Russian Federation

    Moscow, 121552
    Russian Federation

    Site Not Available

  • Federal State Budgetary Institution Of Ministry Of Health Of Russian Federation

    Novosibirsk, 630055
    Russian Federation

    Site Not Available

  • Samara Regional Clinical Cardiological Dispensary

    Samara, 443070
    Russian Federation

    Site Not Available

  • Federal State Budgetary Institution

    St Petersburg, 197341
    Russian Federation

    Completed

  • Institute of Cardiology of Tomsk National Research Medical Center of Rus Academy of Sciences

    Tomsk, 634012
    Russian Federation

    Completed

  • Abdullah, IA

    Durban, 4001
    South Africa

    Active - Recruiting

  • Dr Kalla

    Lenasia, 1820
    South Africa

    Active - Recruiting

  • Kaohsiung Veterans General Hospital

    Kaohsiung, 813
    Taiwan

    Active - Recruiting

  • National Cheng Kung University Hospital

    Tainan, 704
    Taiwan

    Active - Recruiting

  • National Taiwan University Hospital

    Taipei, 100
    Taiwan

    Active - Recruiting

  • Taipei Veterans General Hospital

    Taipei, 112
    Taiwan

    Active - Recruiting

  • Chang-Gung Memorial Hospital, LinKou Branch

    Taoyuan, 333
    Taiwan

    Active - Recruiting

  • Istanbul University Istanbul Medical Faculty

    Capa_Istanbul, 34093
    Turkey

    Site Not Available

  • Municipal Inst. Of Dnipropetrovsk Region. Council

    Dnipro,
    Ukraine

    Active - Recruiting

  • Health Care Municipal Institution City Clinical Hospital #13

    Kharkiv,
    Ukraine

    Site Not Available

  • State Institute Of Phthisiology And Pulmonology N.A. F.G. Yanovskiy Of Ams Ukraine

    Kyiv, 03680
    Ukraine

    Active - Recruiting

  • Lviv Regional Clinical Hospital

    Lviv, 79000
    Ukraine

    Site Not Available

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