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

Hypertension, Pulmonary Clinical Trial

— PLATYPUS  

Official title:

A Prospective, Open-label, Platform Study for Long-term Follow-up of Participants Using Study Intervention in Pulmonary Hypertension Parent Studies

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05179876
• Other study ID #: CR109121
• Secondary ID: 2021-002297-11NO
• Status: Recruiting
• Phase: Phase 3
• Start date: May 4, 2022
• Est. completion date: January 31, 2027

Study information

• Verified date: March 2024
• Source: Actelion
• Contact: Study Contact
• Phone: 844-434-4210
• Email: Participate-In-This-Study[@]its.jnj.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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 and NCT04565990]), 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 230
• Est. completion date: January 31, 2027
• Est. primary completion date: January 31, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years and older

Eligibility

Inclusion Criteria:

• Participant must sign an informed consent form (ICF) (or their legally acceptable 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] or chronic thromboembolic pulmonary hypertension [CTEPH] for adults, PAH for pediatric participants); 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 selexipag or macitentan/tadalafil FDC, and at least 2 years old for macitentan
• 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) agree to follow contraceptive methods until 30 days after the last intake of the study intervention Exclusion Criteria:

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

Macitentan/tadalafil FDC-specific:

• Known allergies, hypersensitivity, or intolerance to macitentan or tadalafil or their 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. For participants with hepatic impairment, Child-Pugh Class should be fully assessed and documented in the source documents at screening
• Severe renal impairment (estimated glomerular filtration rate [eGF]/creatinine clearance <30 milliliter per minute [mL/min])

Related Conditions & MeSH terms

• Hypertension
• Hypertension, Pulmonary

Intervention

Drug:
• Macitentan
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.
• Selexipag
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.
• Macitentan/Tadalafil FDC
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.

Locations

Country	Name	City	State
Belarus	Minsk Regional Clinical Hospital Of The Red Banner Of Labor	Minsk
Belarus	The Republican Scientific-Practical Center ''Cardiology''	Minsk
Belgium	UZ Leuven	Leuven
Bulgaria	University Multiprofile Hospital for Active Treatment- UMHAT Sveta Anna AD	Sofia
Korea, Republic of	Chungnam National University Hospital	Daejeon
Korea, Republic of	Gachon University Gil Medical Center	Incheon
Korea, Republic of	Asan Medical Center	Seoul
Korea, Republic of	Samsung Medical Center	Seoul
Korea, Republic of	Seoul National University Hospital	Seoul
Korea, Republic of	The Catholic University of Korea Seoul St. Mary's Hospital	Seoul
Poland	Szpital Uniwersytecki nr 2 im dr Jana Biziela w Bydgoszczy, Klinika Kardiologii	Bydgoszcz
Poland	Oddzial Kardiologii Wojewodzki Szpital Specjalistyczny im. W.Bieganskiego	Lodz
Poland	Wojewodzki Szpital Specjalistyczny im. Stefana Kardynala Wyszynskiego SPZOZ	Lublin
Poland	SPSK2 PUM, Klinika Kardiologii	Szczecin
Poland	Wojewodzki Szpital Specjalist, Osrodek Badawczo-Rozwojowy	Wroclaw
Russian Federation	Scientific and Research Institution of Cardiovascular Diseases Complex Problems	Kemerovo
Russian Federation	Federal State Budgetary Institution	St Petersburg
Russian Federation	Institute of Cardiology of Tomsk National Research Medical Center of Rus Academy of Sciences	Tomsk
South Africa	Abdullah, IA	Durban
Taiwan	Kaohsiung Veterans General Hospital	Kaohsiung
Taiwan	National Cheng Kung University Hospital	Tainan
Taiwan	National Taiwan University Hospital	Taipei
Taiwan	Taipei Veterans General Hospital	Taipei
Ukraine	Municipal Inst. Of Dnipropetrovsk Region. Council	Dnipro
Ukraine	State Institute Of Phthisiology And Pulmonology N.A. F.G. Yanovskiy Of Ams Ukraine	Kyiv

Sponsors (1)

Lead Sponsor	Collaborator
Actelion

Countries where clinical trial is conducted

Belarus,  Belgium,  Bulgaria,  Korea, Republic of,  Poland,  Russian Federation,  South Africa,  Taiwan,  Ukraine, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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 AEs with onset or worsening on or after date of first dose of study treatment.	Baseline until End of Study (EOS) (up to 57 months)
Primary	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 AEs with onset or worsening on or after date of first dose of study treatment.	Baseline until EOS (up to 57 months)
Primary	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 57 months)
Primary	Frequency of Deaths	Frequency of deaths will be reported.	Baseline until EOS (up to 57 months)