New drug hope for kids with rare lung disease

NCT ID NCT05587712

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a drug called sotatercept in 42 children aged 1 to 18 who have pulmonary arterial hypertension (PAH), a serious lung condition that makes the heart work too hard. The drug is given as a shot every 3 weeks, and the main goal is to see if it is safe and how the body processes it. Researchers will also check if it helps children walk farther and improves heart function.

What this could mean

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

Active substance
sotatercept (a drug given as an injection under the skin every 3 weeks)
What this could lead to
If it works, this could provide a new treatment option for children with pulmonary arterial hypertension, helping control the disease and improve daily life.
What could go wrong
This is an early phase 2 study with only 42 children, so results may not apply to all patients. The drug may cause side effects or not work better than current treatments.

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.

As listed by the trial registrant

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

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Ankara Bilkent Şehir Hastanesi. ( Site 1403)

    RECRUITING

    Ankara, 06800, Turkey (Türkiye)

    Contact Phone: •••-•••-••••

  • Assistance Publique Hôpitaux de Marseille - Hôpital de la Timone ( Site 0303)

    RECRUITING

    Marseille, Provence-Alpes-Côte d'Azur Region, 13005, France

    Contact Phone: •••-•••-••••

  • CHU de Toulouse - Hôpital des Enfants ( Site 0302)

    RECRUITING

    Toulouse, Haute-Garonne, 31059, France

    Contact Phone: •••-•••-••••

  • Centrum Zdrowia Dziecka w Warszawie-Klinika Kardiologii ( Site 1103)

    RECRUITING

    Warsaw, Masovian Voivodeship, 04-730, Poland

    Contact Phone: •••-•••-••••

  • Children's Hospital Colorado ( Site 1609)

    RECRUITING

    Aurora, Colorado, 80045, United States

    Contact Phone: •••-•••-••••

  • Children's Hospital of Philadelphia (CHOP) ( Site 1608)

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

    Contact Phone: •••-•••-••••

  • Children's National Medical Center ( Site 1600)

    RECRUITING

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

    Contact Phone: •••-•••-••••

  • Children's Wisconsin ( Site 1610)

    RECRUITING

    Milwaukee, Wisconsin, 53226, United States

    Contact Phone: •••-•••-••••

  • Cincinnati Children's Hospital Medical Center ( Site 1602)

    RECRUITING

    Cincinnati, Ohio, 45229, United States

    Contact Phone: •••-•••-••••

  • Clinica Somer-Unidad de Investigacion y Docencia ( Site 0205)

    RECRUITING

    Rionegro, Antioquia, 054040, Colombia

    Contact Phone: •••-•••-••••

  • Clínica Imbanaco S.A.S ( Site 0203)

    RECRUITING

    Cali, Valle del Cauca Department, 760042, Colombia

    Contact Phone: •••-•••-••••

  • Fundación Valle del Lili ( Site 0200)

    RECRUITING

    Cali, Valle del Cauca Department, 760032, Colombia

    Contact Phone: •••-•••-••••

  • Gazi University Health Research and Application Center Gazi -Çocuk Sağlığı ve Hastalıkları Anabilim ( Site 1402)

    RECRUITING

    Ankara, 06560, Turkey (Türkiye)

    Contact Phone: •••-•••-••••

  • Great Ormond Street Hospital For Children NHS Foundation Trust ( Site 1500)

    RECRUITING

    London, London, City of, WC1N 3JH, United Kingdom

    Contact Phone: •••-•••-••••

  • HOSPITAL GENERAL UNIVERSITARIO GREGORIO MARAÑON ( Site 1301)

    RECRUITING

    Madrid, 28007, Spain

    Contact Phone: •••-•••-••••

  • Hacettepe Universite Hastaneleri ( Site 1400)

    RECRUITING

    Ankara, 06100, Turkey (Türkiye)

    Contact Phone: •••-•••-••••

  • Hospital Universitari Vall d'Hebron ( Site 1302)

    RECRUITING

    Barcelona, 08035, Spain

    Contact Phone: •••-•••-••••

  • Hospital Universitari i Politecnic La Fe ( Site 1303)

    RECRUITING

    Valencia, Valencia, 46026, Spain

    Contact Phone: •••-•••-••••

  • Hospital Universitario Ramón y Cajal ( Site 1300)

    RECRUITING

    Madrid, Madrid, Comunidad de, 28034, Spain

    Contact Phone: •••-•••-••••

  • Hôpital Universitaire Necker Enfants Malades ( Site 0300)

    RECRUITING

    Paris, 75015, France

    Contact Phone: •••-•••-••••

  • Klinikum der Universität München Großhadern ( Site 0404)

    RECRUITING

    München, Bavaria, 81337, Germany

    Contact Phone: •••-•••-••••

  • Medizinische Hochschule Hannover ( Site 0405)

    RECRUITING

    Hanover, Lower Saxony, 30625, Germany

    Contact Phone: •••-•••-••••

  • Mehmet Akif Ersoy Research and Training Hospital ( Site 1404)

    RECRUITING

    Istanbul, 34303, Turkey (Türkiye)

    Contact Phone: •••-•••-••••

  • Monroe Carell Jr. Children's Hospital ( Site 1601)

    COMPLETED

    Nashville, Tennessee, 37232, United States

  • Schneider Children's Medical Center ( Site 0603)

    RECRUITING

    Petah Tikva, 4920235, Israel

    Contact Phone: •••-•••-••••

  • Seattle Children's Hospital ( Site 1605)

    RECRUITING

    Seattle, Washington, 98105, United States

    Contact Phone: •••-•••-••••

  • Sheba Medical Center ( Site 0601)

    RECRUITING

    Ramat Gan, 5265601, Israel

    Contact Phone: •••-•••-••••

  • Stanford University School of Medicine ( Site 1603)

    RECRUITING

    Palo Alto, California, 94304, United States

    Contact Phone: •••-•••-••••

  • The Children's Hospital at Westmead ( Site 0001)

    RECRUITING

    Westmead, New South Wales, 2145, Australia

    Contact Phone: •••-•••-••••

  • The Regents of the University of California - Los Angeles (UCLA Pediatrics) ( Site 1606)

    RECRUITING

    Los Angeles, California, 90095, United States

    Contact Phone: •••-•••-••••

  • UCSF Benioff Children's Hospital San Francisco ( Site 1611)

    RECRUITING

    San Francisco, California, 94158, United States

    Contact Phone: •••-•••-••••

  • Universitaetsklinikum Heidelberg ( Site 0401)

    RECRUITING

    Heidelberg, Baden-Wurttemberg, 69120, Germany

    Contact Phone: •••-•••-••••

  • University Medical Center Groningen ( Site 0900)

    RECRUITING

    Groningen, 9713 GZ, Netherlands

    Contact Phone: •••-•••-••••

  • Uniwersyteckie Centrum Kliniczne-Klinika Kardiologii Dziecięcej i Wad Wrodzonych Serca ( Site 1102)

    RECRUITING

    Gdansk, Pomeranian Voivodeship, 80-952, Poland

    Contact Phone: •••-•••-••••

  • Wits Clinical Research-Chris Hani Baragwanath Hospital ( Site 1201)

    RECRUITING

    Johannesburg, Soweto, Gauteng, 2013, South Africa

    Contact Phone: •••-•••-••••

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