A Study Providing Treatment Access in Participants With P... | NCT05179876

ID	Type	Description
NOPRODPAPUH3001	Other Identifier	Janssen Research & Development, LLC
2021-002297-11	EudraCT Number
2023-506791-27-00	Registry Identifier	EUCT number

Status Module

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
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.

Interventions

Name	Type	Description	Arm Group Labels	Other Names
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.

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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)

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

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

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

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])

ID	Term
D006976	Hypertension, Pulmonary
D000081029	Pulmonary Arterial Hypertension

ID	Term
D008171	Lung Diseases
D012140	Respiratory Tract Diseases
D006973	Hypertension
D014652	Vascular Diseases
D002318	Cardiovascular Diseases