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New drug hope for kids with rare lung disease

NCT ID NCT05587712

Disease control Sponsor: Merck Sharp & Dohme LLC Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 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.

Study facts

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

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 42 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jan 2023

Expected to finish

Sep 2028

An estimate. End dates often move.

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 year 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.

Inclusion Criteria * Documented, historic diagnostic right heart catheterization (RHC) any time before Screening confirming the diagnosis of PAH WHO Group 1 in any of the following subtypes: * Idiopathic pulmonary arterial hypertension (IPAH) * Heritable PAH * Drug/toxin-induced PAH * PAH associated with connective tissue disease * PAH-congenital heart disease (CHD) with shunt closure \>6 months before Screening and subsequently confirmed by RHC before Screening * PAH with coincidental shunt. * Must be on a stable dose(s) of background PAH therapy (phosphdiesterase-5 (PDE5) inhibitors, endothelin receptor antagonists (ERAs), soluble guanylate cyclase stimulators (sGCS), or prostanoids \[including subcutaneous and intravenous\]) * If male, agree to the following during the intervention period and for at least 16 weeks (112 days) after the last dose of study intervention: * Abstains from heterosexual intercourse as their preferred and usual lifestyle (abstinent on a long-term and persistent basis) and agrees to remain abstinent or * Uses contraception unless confirmed to be azoospermic (vasectomized or secondary to medical cause, documented from the site personnel's review of the participant's medical records, medical examination, or medical history interview) as detailed below: * Uses a male condom plus partner use of an additional contraceptive method when having penile-vaginal intercourse with a woman of childbearing potential (WOCBP) who is not currently pregnant Note: Men with a pregnant or breastfeeding partner must agree to remain abstinent from penile-vaginal intercourse or use a male condom during each episode of penile-vaginal penetration. * If female, must be either not a WOCBP or use a contraceptive method that is highly effective or be abstinent from heterosexual intercourse during the intervention period and for at least 16 weeks (112 days) after the last dose of study intervention * If male, agrees to refrain from donating blood or sperm for the duration of the study and for 16 weeks (112 days) after the last dose of study intervention * If female, agrees to refrain from donating blood, eggs, or ovum for the duration of the study and for at least 16 weeks (112 days) after the last dose of study intervention Exclusion Criteria * History of left-sided heart disease, including valvular disease (eg, moderate or greater mitral or aortic regurgitation or stenosis), left ventricular outflow tract obstruction, and/or left heart failure (eg, restrictive or dilated cardiomyopathy) * Severe (as based on the opinion of the investigator) congenital or developmental abnormalities of the lung, thorax, and/or diaphragm * History of Eisenmenger syndrome, Potts shunt, or atrial septostomy * Unrepaired or residual cardiac shunt * Diagnosis of pulmonary veno-occlusive diseases, pulmonary capillary hemangiomatosis, or overt signs of capillary and/or venous involvement * PAH associated with portal hypertension * Known visceral (lung, liver, or brain) arteriovenous malformation(s) * History of full or partial pneumonectomy * Untreated more than mild obstructive sleep apnea * History of known pericardial constriction * Family history of sudden cardiac death or long QT syndrome * Any current or prior history of symptomatic coronary disease (myocardial infarction, percutaneous coronary intervention, coronary artery bypass graft surgery, or cardiac anginal chest pain) within 6 months before Screening * Cerebrovascular accident within 3 months before Screening * Prior exposure to sotatercept or luspatercept or has had an allergic reaction to any of their excipients

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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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    34 sites in 12 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Ankara Bilkent Şehir Hastanesi. ( Site 1403)

    RECRUITING

    Ankara, 06800, Turkey (Türkiye)

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

    RECRUITING

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

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

    RECRUITING

    Toulouse, Haute-Garonne, 31059, France

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

    RECRUITING

    Warsaw, Masovian Voivodeship, 04-730, Poland

  • Children's Hospital Colorado ( Site 1609)

    RECRUITING

    Aurora, Colorado, 80045, United States

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

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Children's National Medical Center ( Site 1600)

    RECRUITING

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

  • Children's Wisconsin ( Site 1610)

    RECRUITING

    Milwaukee, Wisconsin, 53226, United States

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

    RECRUITING

    Cincinnati, Ohio, 45229, United States

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

    RECRUITING

    Rionegro, Antioquia, 054040, Colombia

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

    RECRUITING

    Cali, Valle del Cauca Department, 760042, Colombia

  • Fundación Valle del Lili ( Site 0200)

    RECRUITING

    Cali, Valle del Cauca Department, 760032, Colombia

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

    RECRUITING

    Ankara, 06560, Turkey (Türkiye)

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

    RECRUITING

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

  • HOSPITAL GENERAL UNIVERSITARIO GREGORIO MARAÑON ( Site 1301)

    RECRUITING

    Madrid, 28007, Spain

  • Hacettepe Universite Hastaneleri ( Site 1400)

    RECRUITING

    Ankara, 06100, Turkey (Türkiye)

  • Hospital Universitari Vall d'Hebron ( Site 1302)

    RECRUITING

    Barcelona, 08035, Spain

  • Hospital Universitari i Politecnic La Fe ( Site 1303)

    RECRUITING

    Valencia, Valencia, 46026, Spain

  • Hospital Universitario Ramón y Cajal ( Site 1300)

    RECRUITING

    Madrid, Madrid, Comunidad de, 28034, Spain

  • Hôpital Universitaire Necker Enfants Malades ( Site 0300)

    RECRUITING

    Paris, 75015, France

  • Klinikum der Universität München Großhadern ( Site 0404)

    RECRUITING

    München, Bavaria, 81337, Germany

  • Medizinische Hochschule Hannover ( Site 0405)

    RECRUITING

    Hanover, Lower Saxony, 30625, Germany

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

    RECRUITING

    Istanbul, 34303, Turkey (Türkiye)

  • 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

  • Seattle Children's Hospital ( Site 1605)

    RECRUITING

    Seattle, Washington, 98105, United States

  • Sheba Medical Center ( Site 0601)

    RECRUITING

    Ramat Gan, 5265601, Israel

  • Stanford University School of Medicine ( Site 1603)

    RECRUITING

    Palo Alto, California, 94304, United States

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

    RECRUITING

    Westmead, New South Wales, 2145, Australia

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

    RECRUITING

    Los Angeles, California, 90095, United States

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

    RECRUITING

    San Francisco, California, 94158, United States

  • Universitaetsklinikum Heidelberg ( Site 0401)

    RECRUITING

    Heidelberg, Baden-Wurttemberg, 69120, Germany

  • University Medical Center Groningen ( Site 0900)

    RECRUITING

    Groningen, 9713 GZ, Netherlands

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

    RECRUITING

    Gdansk, Pomeranian Voivodeship, 80-952, Poland

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

    RECRUITING

    Johannesburg, Soweto, Gauteng, 2013, South Africa

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