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

NCT05179876 | Recruiting Phase 3

• 4 other identifiers: CR109121NOPRODPAPUH30012021-002297-112023-506791-27-00
• Study Type: interventional
• Target: 280
• Locations: 12 countries
• Sites: 43

Brief 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.

Conditions

Hypertension, Pulmonary

Keywords

Chronic thromboembolic pulmonary hypertension (CTEPH); pulmonary arterial hypertension (PAH)

Outcome Measures

Primary Outcomes (4)

• Frequency of Treatment Emergent Adverse Events (TEAEs)

  An adverse event (AE) is any untoward medical occurrence in a clinical study participant administered a pharmaceutical (investigational or non-investigational) product. An AE does not necessarily have a causal relationship with the intervention. TEAEs are defined as a treatment-emergent AE is any AE temporally associated with the use of study treatment (from start of treatment in the PLATYPUS protocol until 30 days after study treatment discontinuation) whether or not considered by the investigator as related to study treatment.

  Baseline until End of Study (EOS) (up to 84 months)

• Frequency of TEAEs Leading to Discontinuation

  Frequency of TEAEs leading to discontinuation of study intervention will be reported. An AE is any untoward medical occurrence in a clinical study participant administered a pharmaceutical (investigational or non-investigational) product. An AE does not necessarily have a causal relationship with the intervention. TEAEs are defined as a treatment-emergent AE is any AE temporally associated with the use of study treatment (from start of treatment in the PLATYPUS protocol until 30 days after study treatment discontinuation) whether or not considered by the investigator as related to study treatment.

  Baseline until EOS (up to 84 months)

• Frequency of Serious Adverse Events (SAEs)

  SAE is any untoward medical occurrence that at any dose 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 and is medically important.

  Baseline until EOS (up to 84 months)

• Frequency of Deaths

  Frequency of deaths will be reported.

  Baseline until EOS (up to 84 months)

Study Arms (3)

Macitentan

EXPERIMENTAL

Participants who have completed a parent study, benefit from their study intervention maintenance and have no adequate alternative local treatment option will be enrolled in this study and will continue to receive study drug macitentan orally during the course of the study. For adult participants study visits will be scheduled every 6 months and for pediatric participants study visits will be scheduled every 3 months. The study includes on-site visits to collect efficacy and safety information until participant discontinuation/withdrawal, or the respective study intervention is made commercially available in the country/territory or an equivalent approved therapy becomes available, or the sponsor decides to terminate the study prematurely.

Drug: Macitentan

Selexipag

EXPERIMENTAL

Participants who have completed a parent study, benefit from their study intervention maintenance and have no adequate alternative local treatment option will be enrolled in this study and will continue to receive study drug selexipag orally during the course of the study. Study visits are scheduled every 6 months to collect efficacy and safety information until participant discontinuation/withdrawal, or the respective study intervention is made commercially available in the country/territory or an equivalent approved therapy becomes available, or the sponsor decides to terminate the study prematurely.

Drug: Selexipag

Macitentan/Tadalafil FDC

EXPERIMENTAL

Participants who have completed a parent study, benefit from their study intervention maintenance and have no adequate alternative local treatment option will be enrolled in this study and will continue to receive drug Macitentan and Tadalafil fixed dose combination (FDC) orally during the course of the study. Study visits are scheduled every 6 months to collect efficacy and safety information until participant discontinuation/withdrawal, or the respective study intervention is made commercially available in the country/territory or an equivalent approved therapy becomes available, or the sponsor decides to terminate the study prematurely.

Drug: Macitentan/Tadalafil FDC

Interventions

Macitentan DRUG

Adult participants will receive oral dose of macitentan 10 milligrams (mg) tablet once daily. Children greater than or equal to (\>=) 2 year to less than (\<) 18 years will be given an oral macitentan dose tailored to their body weight, ensuring an equivalent level of systemic exposure as in adults.

Also known as: JNJ-67896062

Macitentan

Selexipag DRUG

Adult Participant will receive oral dose of selexipag tablet twice daily at the dose strength corresponding to their maintenance dose at the end of their parent study. Available strengths: 200, 400, 600, 800, 1000, 1200, 1400 and 1600 micrograms (µg). Children with body weight category of \>=50 kg will use the tablets at the required dose strength as described for adults. Children with a body weight \< 50 kg will receive tablets for pediatric use (dose strengths: 100 and 150 mcg), twice daily to enable continuation of individually maximum tolerated dose of selexipag according to their body weight category.

Also known as: JNJ-67896049

Selexipag

Macitentan/Tadalafil FDC DRUG

Participants will receive oral FDC of macitentan 10 mg and tadalafil 40 mg once daily during the course of the study as already received in the parent studies.

Also known as: JNJ-68150420

Macitentan/Tadalafil FDC

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Participant must sign an informed consent form (ICF) (or their legally designated representative 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 sponsor parent study and: a) the indication of the parent study is included in the intervention-specific appendices (ISA) (pulmonary arterial hypertension \[PAH\]; b) participant has completed the parent study; c) no alternative means of access to study 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 2 years old for macitentan or selexipag
• A female participant of childbearing potential must: a) have a negative urine or serum pregnancy test prior to first intake of study intervention; b) agree to perform 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 days after 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/or parent(s)/ legally designated representative (LDR)(s) on the acceptable method of contraception

You may not qualify if:

• General:
• Participants prematurely discontinued from the study intervention in their parent study
• Female participant being pregnant, or breastfeeding, or planning to become pregnant while 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. For participants with hepatic impairment, Child-Pugh Class (Child-Pugh score) should be fully 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 the last 6 months, decompensated cardiac failure (if not under close medical supervision), severe arrhythmia, cerebrovascular events (for example, transient ischemic attack, stroke) within the last 3 months, or congenital or acquired valvular defects with clinically relevant myocardial function disorders not related to pulmonary hypertension (PH)
• Known and documented severe hepatic impairment that is, Child-Pugh Class C. For participants with hepatic impairment, Child-Pugh Class (Child-Pugh score) should be fully assessed and documented in the source documents at screening

Sponsors & Collaborators

• Actelion: lead

Study Sites (45)

The Republican Scientific-Practical Center ''Cardiology''

Minsk, 220036, Belarus

RECRUITING

Minsk Regional Clinical Hospital Of The Red Banner Of Labor

Minsk, Belarus

RECRUITING

UZ Leuven

Leuven, 3000, Belgium

COMPLETED

University Multiprofile Hospital for Active Treatment- UMHAT Sveta Anna AD

Sofia, 1750, Bulgaria

RECRUITING

Beijing Anzhen Hospital 1

Beijing, 100029, China

RECRUITING

Beijing Anzhen Hospital

Beijing, 100029, China

RECRUITING

The Second Xiangya Hospital of Central South Hospital

Changsha, 410011, China

RECRUITING

Jiangsu Province Hospital

Nanjing, China

RECRUITING

Qingdao Women and Children's Hospital

Qingdao, 266035, China

RECRUITING

Childrens Hospital of Shanghai

Shanghai, 200062, China

RECRUITING

The First Affiliated Hospital of Xian Jiaotong University

Xi'an, 710061, China

RECRUITING

Gottsegen György Országos Kardiológiai Intézet

Budapest, 1096, Hungary

RECRUITING

Klinika Kardiologii Z Oddzialem Intensywnego Nadzoru Kardiologicznego UM W Bialymstoku

Bialystok, 15 276, Poland

RECRUITING

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

Bydgoszcz, 85-168, Poland

COMPLETED

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

Katowice, 40 635, Poland

RECRUITING

Oddzial Kardiologii Wojewodzki Szpital Specjalistyczny im W Bieganskiego

Lodz, 91 347, Poland

RECRUITING

Wojewodzki Szpital Specjalistyczny im Stefana Kardynala Wyszynskiego SPZOZ

Lublin, 20 718, Poland

COMPLETED

SPSK2 PUM Klinika Kardiologii

Szczecin, 70 111, Poland

RECRUITING

Wojewodzki Szpital Specjalist Osrodek Badawczo Rozwojowy

Wroclaw, 51 124, Poland

COMPLETED

Wojewodzki Szpital Specjalistyczny we Wroclawiu

Wroclaw, 51 124, Poland

RECRUITING

Scientific and Research Institution of Cardiovascular Diseases Complex Problems

Kemerovo, 650002, Russia

COMPLETED

E.Meshalkin National Medical Research Center of the Ministry of Health of the Russian Federation

Novosibirsk, 630055, Russia

RECRUITING

Federal State Budgetary Institution

Saint Petersburg, 197341, Russia

COMPLETED

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

Tomsk, 634012, Russia

COMPLETED

Regional Clinical Hospital No1

Tyumen, 625000, Russia

RECRUITING

Abdullah, IA

Durban, 4001, South Africa

COMPLETED

Dr Kalla

Lenasia, 1820, South Africa

RECRUITING

Chungnam National University Hospital

Daejeon, 35015, South Korea

RECRUITING

Gachon University Gil Medical Center

Incheon, 21565, South Korea

RECRUITING

Seoul National University Hospital 1

Seoul, 03080, South Korea

RECRUITING

Seoul National University Hospital

Seoul, 03080, South Korea

RECRUITING

Asan Medical Center

Seoul, 05505, South Korea

RECRUITING

Samsung Medical Center

Seoul, 06351, South Korea

RECRUITING

The Catholic University of Korea Seoul St Marys Hospital

Seoul, 06591, South Korea

RECRUITING

Kaohsiung Veterans General Hospital

Kaohsiung City, 813, Taiwan

RECRUITING

National Cheng Kung University Hospital

Tainan, 704, Taiwan

RECRUITING

National Taiwan University Hospital

Taipei, 100, Taiwan

RECRUITING

Taipei Veterans General Hospital

Taipei, 112, Taiwan

RECRUITING

Chang-Gung Memorial Hospital, LinKou Branch

Taoyuan District, 333, Taiwan

RECRUITING

Maharaj Nakorn Chiang Mai hospital Faculty of Medicine

Chiang Mai, 50200, Thailand

RECRUITING

Municipal Inst. Of Dnipropetrovsk Region. Council

Dnipro, Ukraine

COMPLETED

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

Kyiv, 03680, Ukraine

COMPLETED

Hanoi Heart Hospital

Hanoi, Vietnam

RECRUITING

Hanoi Medical University Hospital

Hanoi, Vietnam

RECRUITING

Children's Hospital 1

Ho Chi Minh City, Vietnam

RECRUITING

MeSH Terms

Conditions

Hypertension, Pulmonary; Pulmonary Arterial Hypertension

Interventions

macitentan; selexipag

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Hypertension; Vascular Diseases; Cardiovascular Diseases

Study Officials

• Actelion Pharmaceuticals Ltd Clinical Trial

  Actelion

  STUDY DIRECTOR

Central Study Contacts

Study Contact

CONTACT

844-434-4210; Participate-In-This-Study1@its.jnj.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This is a prospective, open-label, extension study, with a platform-study design for long-term safety follow-up of participants using study intervention as in their respective pulmonary hypertension (PH) parent study (that is, pulmonary arterial hypertension \[PAH\] or chronic thromboembolic pulmonary hypertension \[CTEPH\]). Participants who have completed a parent study, that benefit from their study intervention maintenance and have no adequate alternative local treatment option (access to the study intervention or an equivalent approved therapy) will be enrolled in this study.

Study Record Dates

• First Submitted: December 17, 2021
• First Posted: January 5, 2022
• Study Start: May 4, 2022
• Primary Completion (Estimated): January 31, 2027
• Study Completion (Estimated): February 29, 2028
• Last Updated: April 13, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

CN (7); UA (2); TH (1); KR (7); ZA (2); VN (3); BE (1); HU (1); PL (8); RU (5); TW (5); BU (1)