A Study of Macitentan in Japanese Pediatric Participants With Pulmonary Arterial Hypertension

Janssen (J&J Innovative Medicine)

Status and phase: Completed; Phase 3

Conditions: Pulmonary Arterial Hypertension

Treatments: Drug: Macitentan

Study type: Interventional

Funder types: Industry

Identifiers

NCT05167825

67896062PAH3001 (Other Identifier)

2023-000984-30 (EudraCT Number)

CR109128

Details and patient eligibility

About

The purpose of this study is to evaluate the effect of macitentan on hemodynamic measures at Week 24 in pediatric populations.

Enrollment: 7 patients

Sex: All

Ages: 3 months to 15 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Pulmonary arterial hypertension (PAH) belonging to the nice 2013 updated classification group 1
• PAH diagnosis confirmed by historical right heart catheterization where in the absence of pulmonary vein obstruction and/or significant lung disease pulmonary artery wedge pressure (PAWP) can be replaced by left atrium pressure (LAP) or left ventricular end diastolic pressure (LVEDP) (in absence of mitral stenosis) assessed by heart catheterization
• World Health Organization (WHO) functional class (FC) I to IV
• PAH-specific treatment-naïve participants or participants on PAH-specific treatment
• A female of childbearing potential must have a negative highly sensitive serum beta-human chorionic gonadotropin (beta-hCG) test at screening and a negative urine pregnancy test at the first administration of study intervention
• A female participant must not get pregnant and must agree not to donate eggs during the study and for a period of up to 4 weeks following the end of study

Exclusion criteria

• Participants with PAH due to portal hypertension, schistosomiasis, pulmonary veno-occlusive disease, and/or pulmonary capillary hemangiomatosis, and persistent pulmonary hypertension of the newborn
• Participants with the following diseases: pulmonary vein stenosis; bronchopulmonary dysplasia
• Severe hepatic impairment, example, Child-Pugh Class C, at screening
• Pregnant, or breast-feeding, or planning to become pregnant while enrolled in this study or within 4 weeks after the last dose of study intervention
• Known allergies, hypersensitivity, or intolerance to macitentan or its excipients
• Participant with PAH associated with open shunts, with congenital cardiac abnormalities such as univentricular heart, with pulmonary hypertension due to lung disease, and renal dysfunction

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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7 participants in 1 patient group

Macitentan

Experimental group

Description:

Participants will receive oral dose of macitentan based on age and weight through Week 52.

Treatment:

Drug: Macitentan