New hope for kids with rare lung condition: macitentan shows promise in small study

NCT ID NCT05167825

Completed Disease control Sponsor: Janssen Pharmaceutical K.K. Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested the drug macitentan in 7 Japanese children aged 3 months to 15 years with pulmonary arterial hypertension (PAH), a condition where high blood pressure in the lungs strains the heart. The goal was to see if the drug improves blood flow and heart function after 24 weeks. Researchers also monitored safety and side effects. The study is complete, but results are not yet widely available.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: macitentan (also known as OPSUMIT, ZEPENDO)
What this could lead to: If successful, this could provide a treatment option for children with pulmonary arterial hypertension, potentially improving heart function and quality of life.
What could go wrong: This is a small, completed study with only 7 participants, so results may not apply to all children. The drug may cause side effects or not work as well in real-world use.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

pulmonary arterial hypertension

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PULMONARY ARTERIAL HYPERTENSION

Contacts and locations

Locations

Fukuoka Children's Hospital

Fukuoka, 813-0017, Japan
Hokkaido University Hospital

Sapporo, 060-8648, Japan
Institute of Science Tokyo Hospital

Bunkyō City, 113 8519, Japan
Nagano Children's Hospital

Azumino-shi, 399-8288, Japan
National Center for Child Health and Development

Setagaya Ku, 157 8535, Japan
National Cerebral and Cardiovascular Center

Suita-Shi, 564-8565, Japan
Okayama University Hospital

Okayama, 700 8558, Japan
Osaka University Hospital

Suita-shi, 565-0871, Japan
The University of Tokyo Hospital

Tokyo, 113-8655, Japan
Toho University Medical Center Omori Hospital

Ōta-ku, 143-8541, Japan
Tokyo Women's Medical University Hospital

Shinjuku-ku, 162-8666, Japan

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