New hope for kids with rare lung condition: macitentan shows promise in small study
NCT ID NCT05167825
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Fukuoka Children's Hospital
Fukuoka, 813-0017, Japan
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Hokkaido University Hospital
Sapporo, 060-8648, Japan
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Institute of Science Tokyo Hospital
Bunkyō City, 113 8519, Japan
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Nagano Children's Hospital
Azumino-shi, 399-8288, Japan
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National Center for Child Health and Development
Setagaya Ku, 157 8535, Japan
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National Cerebral and Cardiovascular Center
Suita-Shi, 564-8565, Japan
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Okayama University Hospital
Okayama, 700 8558, Japan
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Osaka University Hospital
Suita-shi, 565-0871, Japan
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The University of Tokyo Hospital
Tokyo, 113-8655, Japan
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Toho University Medical Center Omori Hospital
Ōta-ku, 143-8541, Japan
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Tokyo Women's Medical University Hospital
Shinjuku-ku, 162-8666, Japan