Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

New hope for kids with rare lung disease: macitentan trial shows promise

NCT ID NCT02932410

Disease control Sponsor: Actelion 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested the drug macitentan in 165 children with pulmonary arterial hypertension (PAH), a serious lung condition. The children were randomly assigned to receive macitentan or standard care. The main goal was to see how the drug behaves in the body and whether it is safe and effective. The trial is now complete, and results may help doctors decide if macitentan is a good option for managing PAH in children.

What this could mean

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

Active substance
Macitentan (a drug taken by mouth as a dispersible tablet)
What this could lead to
If it works, this could provide a new treatment option to help control pulmonary arterial hypertension in children, potentially reducing hospitalizations and disease progression.
What could go wrong
This is a completed phase 3 trial, but results may not show a clear benefit over existing treatments. The study is open-label, which can introduce bias. Risks include side effects like headache, nausea, and potential liver issues.

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

165 people

The number who actually took part.

Started

Oct 2017

Finished

Nov 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

1 month to 17 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.

Key Inclusion Criteria: * Signed informed consent by the parent(s) or legally designated representative and assent from developmentally capable children prior to initiation of any study-mandated procedure * Males or females between greater than or equal to (\>=) 1 month and less than (\<) 18 years of age * Participants with body weight \>= 3.5 kilograms (kg) at randomization * Pulmonary arterial hypertension (PAH) diagnosis confirmed by historical RHC (mPAP greater than or equal to \[\>=\] 25 millimeters of mercury \[mmHg\], and Pulmonary artery wedge pressure \[PAWP\] less than or equal to \[\<=\] 15 mmHg, and Pulmonary vascular resistance index \[PVRi\] greater than \[\>\] 3 WU × m2), where in the absence of pulmonary vein obstruction and/or significant lung disease 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 * PAH belonging to the Nice 2013 Updated Classification Group 1 (including participants with Down Syndrome) and of following etiologies: idiopathic PAH; heritable PAH; PAH associated with congenital heart disease (CHD); Drug or toxin induced PAH; PAH associated with HIV; PAH associated with connective tissue diseases (PAH-aCTD); and World health organization (WHO) Functional class I to III * Females of childbearing potential must have a negative pregnancy test at Screening and at Baseline, and must agree to undertake monthly pregnancy tests, and to use a reliable method of contraception (if sexually active) up to the end of study (EOS) Key Exclusion Criteria: * Participants with PAH due to portal hypertension, schistosomiasis, or with pulmonary veno-occlusive disease and/or pulmonary capillary hemangiomatosis, and persistent pulmonary hypertension of the newborn * Participants with PAH associated with Eisenmenger syndrome, or with moderate to large left-to-right shunts * Participants receiving a combination of \> 2 PAH-specific treatments at randomization. * Treatment with intravenous (IV) or subcutaneous (SC) prostanoids within 4 weeks before randomization, unless given for vasoreactivity testing * Hemoglobin or hematocrit \<75 percent (%) of the lower limit of normal range * Serum Aspartate aminotransferase (AST) and/or Alanine aminotransferase (ALT) greater than (\>) 3 times the upper limit of normal range * Pregnancy (including family planning) or breastfeeding. * Any circumstances or conditions, which, in the opinion of the investigator, may affect full participation in the study or compliance with the protocol * Severe hepatic impairment, for example Child-Pugh Class C * Clinical signs of hypotension which in the investigator's judgment would preclude initiation of a PAH-specific therapy * Severe renal insufficiency (estimated creatinine clearance \<30 mL/min or serum creatinine \>221 micro-moles per liter \[micro-mol/L\]) * Participants with known diagnosis of bronchopulmonary dysplasia

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Pulmonary arterial hypertension are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

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

  • Bashkiria State Medical University

    Ufa, 450000, Russia

  • Beijing Anzhen Hospital of The Capital University of Medical Sciences

    Beijing, 100029, China

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • C.H. Regional Reina Sofia

    Córdoba, 14004, Spain

  • CICUM San Miguel

    Guadalajara, 44160, Mexico

  • Centro Hospitalar E Universitário de Coimbra EPE

    Coimbra, 3000-075, Portugal

  • Centro Hospitalar de Lisboa Ocidental, EPE - Hospital de Santa Cruz

    Carnaxide, 2790-134, Portugal

  • Centro Hospitalar de Sao Joao EPE

    Porto, 4200-319, Portugal

  • Centro Medico Imbanaco de Cali SA

    Cali, 760042, Colombia

  • Chaim Sheba Medical Center

    Ramat Gan, 52621, Israel

  • Children's Heart Center

    Las Vegas, Nevada, 89109, United States

  • Children's Hospital 1

    Ho Chi Minh City, 700000, Vietnam

  • Children's Hospital at Westmead

    Westmead, 2145, Australia

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010, United States

  • Childrens Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Childrens Hospital of Shanghai

    Shanghai, 200062, China

  • Childrens Hospital of Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Clinical Hospital №1

    Tyumen, 625023, Russia

  • Columbia University Medical Center - PIN

    New York, New York, 10032, United States

  • Detroit Medical Center

    Detroit, Michigan, 48201, United States

  • Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • East Carolina University

    Greenville, North Carolina, 27834, United States

  • Fundacion Santa Fe de Bogota

    Bogotá, 220246, Colombia

  • GBUZ Children's Hospital named after Bashlyaeva Z.A. Moscow

    Moscow, 125408, Russia

  • Gottsegen Gyorgy Orszagos Kardiologiai Intezet

    Budapest, 1096, Hungary

  • Groupe Hospitalier Necker Enfants Malades

    Paris, 75743, France

  • HUS Uusi lastensairaala

    Helsinki, 00290, Finland

  • Hanoi Heart Hospital

    Hanoi, 100000, Vietnam

  • Hanoi Medical University Hospital

    Hanoi, 100000, Vietnam

  • Hospital General Universitario Gregorio Marañon

    Madrid, 28009, Spain

  • Hospital Santa Marta

    Lisbon, 1169-024, Portugal

  • Hospital Universitari i Politecnic La Fe de Valencia

    Valencia, 46026, Spain

  • Hospital Universitario 12 de Octubre

    Madrid, 28041, Spain

  • Hospital Universitario La Paz

    Madrid, 28046, Spain

  • Hospital Universitario Ramon y Cajal

    Madrid, 28034, Spain

  • Hospital Universitario Vall d'Hebron - PPDS

    Barcelona, 08035, Spain

  • Hôpital Des Enfants

    Toulouse, 31059, France

  • Hôpital Haut-Lévêque - Hôpital cardiologique

    Pessac, 33604, France

  • Hôpital de la Timone Enfants

    Marseille, 13385, France

  • Inkosi Albert Luthuli Central Hospital

    Durban, 4001, South Africa

  • Institut Jantung Negara

    Kuala Lumpur, 50400, Malaysia

  • Instituto Nacional de Cardiologia Dr. Ignacio Chavez

    Mexico City, 14080, Mexico

  • Instituto Nacional de Pediatría

    Mexico City, 04530, Mexico

  • Instytut Pomnik - Centrum Zdrowia Dziecka

    Warsaw, 04-730, Poland

  • Irmandade Da Santa Casa de Misericórdia de São Paulo

    São Paulo, 01221-020, Brazil

  • Lady Cilento Children's Hospital

    South Brisbane, 4101, Australia

  • Landes Frauen Und Kinderklinik Linz

    Linz, 4020, Austria

  • MI Dnipropetrovsk Specialized Clin. Med. Center of Mother and Child n.a. prof. M.F. Rudnev of DRC

    Dnipro, 49000, Ukraine

  • MI Scientific Practical Medical Center for Children Cardiology and Cardiosurgery of MOH of Ukraine

    Kyiv, 04050, Ukraine

  • Maharaj Nakorn Chiang Mai Chiang Mai University

    Chiang Mai, 50200, Thailand

  • Makati Medical Center

    Makati City, 1229, Philippines

  • Mayo Clinic - PPDS

    Rochester, Minnesota, 55905, United States

  • Medizinische Universitat Wien

    Linz, 4020, Austria

  • Medizinische Universität Graz

    Graz, 8036, Austria

  • Montefiore Medical Center

    The Bronx, New York, 10467, United States

  • Municipal Institution of Health Care Regional Children's Clinical Hospital

    Kharkiv, 61093, Ukraine

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Novosibirsk Research Institue of Blood Circulation Pathology n.a. E.N. Meshalkin

    Novosibirsk, 630055, Russia

  • Operadora de Hospitales Angeles SA de CV Hospital Angeles Lomas

    México, 52763, Mexico

  • Philippine Heart Center

    Quezon City, 0850, Philippines

  • Phoenix Childrens Hospital

    Phoenix, Arizona, 85016, United States

  • Primary Children's Medical Center

    Salt Lake City, Utah, 84113, United States

  • Qingdao Women and Children's Hospital

    Qingdao, 26600, China

  • Rambam Medical Center - PPDS

    Haifa, 31096, Israel

  • Research Institute of Complex Cardiovascular Pathology

    Kemerovo, 650002, Russia

  • Riley Hospital For Children

    Indianapolis, Indiana, 46202, United States

  • Royal Children's Hospital Melbourne - PIN

    Parkville, 3052, Australia

  • Russian National Research Medical University n.a. N.I.Pirogov

    Moscow, 125412, Russia

  • Saint Petersburg State Pediatric Medical Academy

    Saint Petersburg, 194100, Russia

  • Sainte Justine Hospital

    Montreal, H3T 1C5, Canada

  • Schneider Children's Medical Center of Israel - PIN

    Petah Tikva, 49100, Israel

  • Seoul National University Hospital

    Seoul, 03080, South Korea

  • Severance Hospital Yonsei University Health System - PPDS

    Seoul, 03722, South Korea

  • Shanghai Children's Medical Center

    Shanghai, 200127, China

  • Szpital Kliniczny im. Karola Jonschera Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu

    Poznan, 60-572, Poland

  • Tam Duc Hospital

    Ho Chi Minh City, 700000, Vietnam

  • UCLA Children's Heart Center

    Los Angeles, California, 90095, United States

  • UCSF Medical Center

    San Francisco, California, 94143-2202, United States

  • Unidad de Investigación Clínica En Medicina SC

    Monterrey, 64716, Mexico

  • University Hospitals Cleveland Medical Center, Rainbow Babies and Childrens Hospital

    Cleveland, Ohio, 44106, United States

  • University Malaya Medical Centre

    Kuala Lumpur, 59100, Malaysia

  • University of The Free State

    Bloemfontein, 9300, South Africa

  • University of Virginia Health System

    Charlottesville, Virginia, 19803, United States

  • Wojewodzki Szpital Specjalistyczny we Wroclawiu Osrodek Badawczo-Rozwojowy

    Wroclaw, 51-124, Poland

More trials for these conditions

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