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

Janssen Pharmaceutical K.K.7 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Pulmonary Arterial Hypertension

Interventions

MacitentanDRUG

Macitentan will be administered orally as a tablet.

Eligibility

Sex: ALLMin age: 3 MonthsMax age: 15 Years

Medical Language ↔ Plain English

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

Locations (11)

Nagano Children's Hospital

Azumino-shi, Japan

Institute of Science Tokyo Hospital

Bunkyō City, Japan

Fukuoka Children's Hospital

Fukuoka, Japan

Outcomes

Primary Outcomes

Fold Change From Baseline at Week 24 in Pulmonary Vascular Resistance Index (PVRI)

PVRI fold change at Week 24 was calculated as 100\*(PVRI at Week 24 divided by PVRI at baseline). PVR was determined by right heart catheterization.

Time frame: Baseline (Day 1), Week 24

Secondary Outcomes

Change From Baseline at Week 24 in Hemodynamic Variables: Pulmonary Vascular Resistance (PVR)

Time frame: Baseline (Day 1), Week 24

Change From Baseline in Mean Right Atrial Pressure (mRAP)

Time frame: Baseline (Day 1), Week 24

Change From Baseline at Week 24 in Hemodynamic Variables: Mean Pulmonary Arterial Pressure (mPAP)

Time frame: Baseline (Day 1), Week 24

Change From Baseline at Week 24 in Hemodynamic Variables: Cardiac Index (CI)

Time frame: Baseline (Day 1), Week 24

Change From Baseline at Week 24 in Hemodynamic Variables: Cardiac Output (CO)

Time frame: Baseline (Day 1), Week 24

Change From Baseline at Week 24 in Hemodynamic Variables: Total Pulmonary Resistance (TPR)

Time frame: Baseline (Day 1), Week 24

Change From Baseline at Week 24 in Hemodynamic Variables: Mixed Venous Oxygen Saturation (SvO2)

Time frame: Baseline (Day 1), Week 24

Number of Participants With Change From Baseline at Weeks 4, 8, 12, 16, 20, 24, 28, 40, and 52 in World Health Organization (WHO) Functional Class (FC)

Data from ClinicalTrials.gov

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Okayama University Hospital

Toho University Medical Center Omori Hospital

Ōta-ku, Japan

Hokkaido University Hospital

Sapporo, Japan

National Center for Child Health and Development

Setagaya Ku, Japan

Tokyo Women's Medical University Hospital

Shinjuku-ku, Japan

National Cerebral and Cardiovascular Center

Suita-Shi, Japan

Osaka University Hospital

Suita-shi, Japan

...and 1 more locations

Time frame: Weeks 4, 8, 12, 16, 20, 24, 28, 40, 52

Number of Participants With Change From Baseline at Weeks 4, 8, 12, 16, 20, 24, 28, 40, and 52 in Panama FC

Time frame: Weeks 4, 8, 12, 16, 20, 24, 28, 40, 52

Change From Baseline to Weeks 24 and 52 in 6-minute Walk Distance (6MWD) as Measured by the 6-minute Walk Test (6MWT)

Time frame: Baseline (Day 1), Weeks 24 and 52

Change From Baseline to Week 12, 24, 28, 40, and 52 in N-terminal Pro-Brain Natriuretic Peptide (NT-proBNP)

Time frame: Baseline (Day 1), Week 12, 24, 28, 40, and 52

Change From Baseline at Weeks 12, 24, and 52 in Tricuspid Annular Plane Systolic Excursion (TAPSE)

Time frame: Baseline (Day 1), Weeks 12, 24, and 52

Change From Baseline at Week 12, 24, and 52 in Left Ventricular Eccentricity Index (LVEI)

Time frame: Baseline (Day 1), Weeks 12, 24 and 52

Change From Baseline to Week 12, 24, and 52 in Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core Scales Short Form (SF-15)

Time frame: Baseline (Day 1), Weeks 12, 24, and 52

Change From Baseline to Weeks 12, 24, and 52 in Physical Activity as Measured by Accelerometry: Number of Hours of Daytime Activity

Time frame: Baseline (Day 1), Weeks 12, 24, and 52

Change From Baseline to Weeks 12, 24, and 52 in Physical Activity as Measured by Accelerometry: Mean Count Per Minute of Daily Activity

Time frame: Baseline (Day 1), Weeks 12, 24, and 52

Change From Baseline to Weeks 12, 24, and 52 in Physical Activity as Measured by Accelerometry: Mean Daily Time Spent in Light Physical Activity

Time frame: Baseline (Day 1), Weeks 12, 24, and 52

Change From Baseline to Weeks 12, 24, and 52 in Physical Activity as Measured by Accelerometry: Mean Daily Time Spent in Moderate to Vigorous Physical Activity

Time frame: Baseline (Day 1), Weeks 12, 24, and 52

Change From Baseline to Week 24 and Week 52 in Borg Dyspnea Index

Time frame: Baseline (Day 1), Weeks 24 and 52

Number of Participants With Treatment-emergent Adverse Events (TEAEs)

Time frame: From Baseline (Day 1) up to Week 56

Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs)

Time frame: From Baseline (Day 1) up to Week 56

Number of Participants With AEs Leading to Premature Discontinuation of Macitentan

Time frame: From Baseline (Day 1) up to Week 52

Number of Participants With AEs of Special Interest

Time frame: From Baseline (Day 1) up to Week 56

Number of Participants With Postbaseline Markedly Abnormal Hematology Laboratory Values

Time frame: Weeks 4, 8, 12, 16, 20, 24, 28, 40, 52

Number of Participants With Postbaseline Markedly Abnormal Clinical Chemistry Laboratory Values

Time frame: Weeks 4, 8, 12, 16, 20, 24, 28, 40, 52

Change From Baseline in Hematology Parameters: Platelets, Leukocytes, Neutrophils Band Form, Lymphocytes, Monocytes, Eosinophils, and Basophils