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New hope for kids with rare muscle disease: drug trial opens for ages 2-17

NCT ID NCT06392386

First seen Jan 06, 2026 · Last updated May 17, 2026 · Updated 20 times

Summary

This study is testing an investigational drug called efgartigimod in children aged 2 to 17 with generalized myasthenia gravis, a condition that causes muscle weakness. The goal is to find the right dose and see how safe it is. Participants will receive injections and be monitored for about 14 weeks.

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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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Contacts and locations

Study contacts

  • Contact

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

Locations

  • AP-HM- Hôpital de La Timone

    RECRUITING

    Marseille, 13385, France

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

  • Carolinas HealthCare System Neurosciences Institute - Neurology

    RECRUITING

    Charlotte, North Carolina, 28207, United States

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

  • Centralny Szpital Kliniczny - Uniwersyteckie Centrum Kliniczne WUM

    RECRUITING

    Warsaw, 02-097, Poland

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

  • Childrens Hospital of Eastern Ontario

    RECRUITING

    Ottawa, K1H 8L1, Canada

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

  • Fakultni nemocnice Brno

    RECRUITING

    Brno, 613 00, Czechia

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

  • Fakultni nemocnice Ostrava

    RECRUITING

    Ostrava, 708 00, Czechia

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

  • Hadassah Medical Center- Ein Kerem

    RECRUITING

    Jerusalem, 9112001, Israel

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

  • Hospital Universitari i Politecnic La Fe de Valencia

    RECRUITING

    Valencia, 46026, Spain

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

  • Hôpital Nestlé

    RECRUITING

    Lausanne, 1011, Switzerland

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

  • Istituto G Gaslini Ospedale Pediatrico IRCCS

    RECRUITING

    Genova, 16147, Italy

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

  • Leiden University Medical Center

    RECRUITING

    Leiden, 2333 ZA, Netherlands

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

  • Neurologia Śląska Centrum Medyczne

    RECRUITING

    Katowice, 40-689, Poland

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

  • Neurology Rare Disease Center

    RECRUITING

    Denton, Texas, 76208, United States

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

  • Oxford Children's Hospital

    RECRUITING

    Oxford, OX3 9DU, United Kingdom

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

  • Schneider Children's Medical Center of Israel

    RECRUITING

    Petah Tikvah, 4920235, Israel

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

  • Tel Aviv Sourasky Medical Center Ichilov

    RECRUITING

    Tel Aviv, 6423906, Israel

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

  • UZ Gent

    RECRUITING

    Ghent, 9000, Belgium

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

  • University of Virginia

    RECRUITING

    Charlottesville, Virginia, 22903, United States

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

  • Universitätsklinikum Essen

    RECRUITING

    Essen, 45147, Germany

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

  • Uniwersyteckie Centrum Kliniczne w Gdansku

    RECRUITING

    Gdansk, 80-211, Poland

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

Conditions

Explore the condition pages connected to this study.