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

NCT ID NCT05167825

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

7 people

The number who actually took part.

Started

Nov 2022

Finished

Mar 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

3 months to 15 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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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

More trials for these conditions

Other studies related to the condition(s) this trial covers.