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

NCT05167825 | Completed Phase 3 Results DMC FDA Drug

• 3 other identifiers: CR10912867896062PAH30012023-000984-30
• Study Type: interventional
• Target: 7
• Locations: 1 country
• Sites: 11

Brief Summary

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

Conditions

Pulmonary Arterial Hypertension

Outcome Measures

Primary Outcomes (2)

• 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.

  Baseline (Day 1), Week 24

• Change From Baseline in Hematology Parameter: Neutrophils Band Form (NBF)

  Change from baseline in hematology parameters: NBF was reported. Data for each parameters was planned to be reported at specified timepoints only.

  Baseline (Day 1), Weeks 8, 16, 20, 40, 52

Secondary Outcomes (43)

• Change From Baseline to Week 24 in Hemodynamic Variable: Pulmonary Vascular Resistance (PVR)

  Baseline (Day 1), Week 24

• Change From Baseline to Week 24 in Hemodynamic Variable: Mean Right Atrial Pressure (mRAP)

  Baseline (Day 1), Week 24

• Change From Baseline to Week 24 in Hemodynamic Variable: Mean Pulmonary Arterial Pressure (mPAP)

  Baseline (Day 1), Week 24

• Change From Baseline to Week 24 in Hemodynamic Variable: Cardiac Index (CI)

  Baseline (Day 1), Week 24

• Change From Baseline to Week 24 in Hemodynamic Variable: Cardiac Output (CO)

  Baseline (Day 1), Week 24

Study Arms (1)

Macitentan

EXPERIMENTAL

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

Drug: Macitentan

Interventions

Macitentan DRUG

Macitentan will be administered orally as a tablet.

Also known as: OPSUMIT, ZEPENDO

Macitentan

Eligibility Criteria

• Age: 3 Months - 15 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Janssen Pharmaceutical K.K.: lead

Study Sites (11)

Nagano Children's Hospital

Azumino-shi, 399-8288, Japan

Location

Institute of Science Tokyo Hospital

Bunkyō City, 113 8519, Japan

Location

Fukuoka Children's Hospital

Fukuoka, 813-0017, Japan

Location

Okayama University Hospital

Okayama, 700 8558, Japan

Location

Toho University Medical Center Omori Hospital

Ōta-ku, 143-8541, Japan

Location

Hokkaido University Hospital

Sapporo, 060-8648, Japan

Location

National Center for Child Health and Development

Setagaya Ku, 157 8535, Japan

Location

Tokyo Women's Medical University Hospital

Shinjuku-ku, 162-8666, Japan

Location

National Cerebral and Cardiovascular Center

Suita-Shi, 564-8565, Japan

Location

Osaka University Hospital

Suita-shi, 565-0871, Japan

Location

The University of Tokyo Hospital

Tokyo, 113-8655, Japan

Location

MeSH Terms

Conditions

Pulmonary Arterial Hypertension

Interventions

macitentan

Condition Hierarchy (Ancestors)

Hypertension, Pulmonary; Lung Diseases; Respiratory Tract Diseases

Limitations and Caveats

The interpretation of study results was limited by small sample size of only 7 participants. Study was not controlled, hence no comparison to Standard of Care (or placebo) was possible.

Results Point of Contact

• Title: Executive Medical Director CP
• Organization: Janssen Pharmaceutical K.K.

ClinicalTrialDisclosure@its.jnj.com

844-434-4210

Study Officials

• Janssen Pharmaceutical K.K. Clinical Trial

  Janssen Pharmaceutical K.K.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 24, 2021
• First Posted: December 22, 2021
• Study Start: November 14, 2022
• Primary Completion: August 23, 2024
• Study Completion: March 5, 2025
• Last Updated: May 7, 2026
• Results First Posted: October 21, 2025

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

JP (11)