New hope for teens with rare bleeding disorder: experimental drug enters final testing

NCT ID NCT07194850

First seen Jun 27, 2026 · Last updated Aug 13, 2026 · Updated 4 times

Summary

This study tests an IV drug called efgartigimod in teens aged 12 to 18 with chronic immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bruising and bleeding. The goal is to find the right dose and see if it safely raises platelet counts. Participants first receive either the drug or a placebo for up to 24 weeks, then everyone gets the drug for up to two years.

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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

    Email: •••••@•••••

Locations

  • A.O.U. Citta della Salute e della Scienza di Torino - Ospedale Infantile Regina Margherita

    RECRUITING

    Torino, 10126, Italy

  • Bambino Gesu Children's Hospital

    RECRUITING

    Roma, 165, Italy

  • Cardiff and Vale NHS Trust - University Hospital of Wales (UHW)

    RECRUITING

    Cardiff, CF14 4XW, United Kingdom

  • Centre Hospitalier Universitaire de Bordeaux Groupe Hospitalier Pellegrin Hopital des Enfants

    RECRUITING

    Bordeaux, 33000, France

  • Charite - Universitaetsmedizin Berlin - Campus Virchow-Klinikum (CVK)

    RECRUITING

    Berlin, 13353, Germany

  • Fondazione IRCCS San Gerardo dei Tintori

    RECRUITING

    Monza, 20900, Italy

  • Gaslini Children's Hospital

    RECRUITING

    Genoa, 16147, Italy

  • Hospital Infantil Universitario Nino Jesus (HIUNJS)

    RECRUITING

    Madrid, 28009, Spain

  • Hospital Materno-Infantil Universitario Gregorio Maranon

    RECRUITING

    Madrid, 28009, Spain

  • Hospital Sant Joan de Deu Barcelona

    RECRUITING

    Esplugues de Llobregat, 08950, Spain

  • Hospital Universitari Vall d'Hebron

    RECRUITING

    Barcelona, 08035, Spain

  • Hospital Universitario 12 de Octubre

    RECRUITING

    Madrid, 28041, Spain

  • Institute Of Mother And Child Healthcare Of Serbia Dr Vukan Cupic

    RECRUITING

    Belgrade, 11070, Serbia

  • Institutul Clinic Fundeni

    RECRUITING

    Bucharest, 022328, Romania

  • Royal Manchester Children's Hospital - Manchester University NHS Foundation Trust

    RECRUITING

    Manchester, M139WL, United Kingdom

  • Samodzielny Publiczny Zaklad Opieki Zdrowotnej Uniwersytecki Szpital Kliniczny nr 4

    RECRUITING

    Lodz, 92-213, Poland

  • The Royal Hospital for Children - Glasgow Health Board

    RECRUITING

    Glasgow, G51 4TF, United Kingdom

  • Universitaetsklinikum Freiburg Kinder - Kinder- und Jugendklinik

    RECRUITING

    Freiburg im Breisgau, 79106, Germany

  • University Clinical Centre of Kragujevac

    RECRUITING

    Kragujevac, 34000, Serbia

  • Uniwersytecki Szpital Dzieciecy w Lublinie

    RECRUITING

    Lublin, 20-093, Poland

  • Vilnius University Hospital Santaros Klinikos, Children's Hospital

    RECRUITING

    Vilnius, 8406, Lithuania

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