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

NCT ID NCT07194850

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

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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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

autoimmune thrombocytopenic purpura Purpura, Thrombocytopenic, Idiopathic thrombocytopenia due to immune destruction

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

  • Bambino Gesu Children's Hospital

    ACTIVE_NOT_RECRUITING

    Roma, 165, Italy

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

    RECRUITING

    Cardiff, CF14 4XW, United Kingdom

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

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

    RECRUITING

    Berlin, 13353, Germany

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

  • Gaslini Children's Hospital

    RECRUITING

    Genoa, 16147, Italy

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

  • Hospital Infantil Universitario Nino Jesus (HIUNJS)

    RECRUITING

    Madrid, 28009, Spain

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

  • Hospital Materno-Infantil Universitario Gregorio Maranon

    RECRUITING

    Madrid, 28009, Spain

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

  • Hospital Sant Joan de Deu Barcelona

    RECRUITING

    Esplugues de Llobregat, 08950, Spain

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

  • Hospital Universitari Vall d'Hebron

    RECRUITING

    Barcelona, 08035, Spain

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

  • Institutul Clinic Fundeni

    RECRUITING

    Bucharest, 022328, Romania

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

  • Uniwersytecki Szpital Dzieciecy w Lublinie

    RECRUITING

    Lublin, 20-093, Poland

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