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New hope for kids with rare muscle disease: drug trial launches

NCT ID NCT04833894

Recruiting now Disease control Sponsor: argenx Source: ClinicalTrials.gov ↗

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

Summary

This study is testing a drug called efgartigimod in children aged 2 to 17 with generalized myasthenia gravis, a condition that causes muscle weakness. The drug is given through an IV and aims to lower harmful antibodies. Researchers will check how the drug moves through the body, its safety, and whether it helps control symptoms. The trial involves 12 participants and lasts about 28 weeks.

What this could mean

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

Active substance
Efgartigimod (a drug given by IV infusion that lowers certain antibodies)
What this could lead to
If successful, this could provide a new treatment option for children with generalized myasthenia gravis, helping control muscle weakness.
What could go wrong
This is an early-phase trial with only 12 participants, so results may not apply to all children. The drug may cause side effects or not work as well as hoped.

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.

myasthenia gravis

As listed by the trial registrant

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

GENERALIZED MYASTHENIA GRAVIS

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • AP-HM - Hopital de la Timone

    RECRUITING

    Marseille, 13385, France

    Contact Email: •••••@•••••

  • Alberta Childrens Hospital

    RECRUITING

    Calgary, T3B 6A8, Canada

    Contact Email: •••••@•••••

  • Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital

    RECRUITING

    Chicago, Illinois, 60611, United States

    Contact Email: •••••@•••••

  • Assistance Publique Hopitaux de Paris (AP-HP) - Hopital Necker-Enfants Malades

    RECRUITING

    Paris, 75015, France

    Contact Email: •••••@•••••

  • Azienda Ospedaliera Universitaria Policlinico Consorziale Di Bari

    RECRUITING

    Bari, 70124, Italy

    Contact Email: •••••@•••••

  • Azienda Ospedaliero Universitaria A. Meyer

    RECRUITING

    Florence, 50139, Italy

    Contact Email: •••••@•••••

  • British Columbia Children's Hospital

    RECRUITING

    Vancouver, V6H 3N1, Canada

    Contact Email: •••••@•••••

  • Centralny Szpital Kliniczny - Uniwersyteckie Centrum Kliniczne WUM

    RECRUITING

    Warsaw, 02-097, Poland

    Contact Email: •••••@•••••

  • Charite Universitaetsmedizin Berlin - Campus Virchow-Klinikum - Sozialpadiatrisches Zentrum

    COMPLETED

    Berlin, 13353, Germany

  • Great Ormand Street Hospital for Children NHS Foundation Trust - Great Ormond Street Hospital

    RECRUITING

    London, WC1N 3JH, United Kingdom

    Contact Email: •••••@•••••

  • Hospital Sant Joan de Deu

    RECRUITING

    Esplugues de Llobregat, 08950, Spain

    Contact Email: •••••@•••••

  • Hospital Universitari i Politecnic La Fe de Valencia

    RECRUITING

    Valencia, 46026, Spain

    Contact Email: •••••@•••••

  • Leids Universitair Medisch Centrum

    RECRUITING

    Leiden, 2333 ZA, Netherlands

    Contact Email: •••••@•••••

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

    RECRUITING

    Manchester, M13 9WL, United Kingdom

    Contact Email: •••••@•••••

  • Medizinische Universitat Wien

    COMPLETED

    Vienna, 1090, Austria

  • Ospedale Giannina Gaslini

    RECRUITING

    Genova, 16147, Italy

    Contact Email: •••••@•••••

  • Oxford University Hospitals NHS Foundation Trust - John Radcliffe Hospital Children's Hospital

    RECRUITING

    Oxford, OX3 9DU, United Kingdom

    Contact Email: •••••@•••••

  • Tbilisi State Medical University - Givi Zhvania Pediatric Academic Clinic

    COMPLETED

    Tbilisi, 159, Georgia

  • Universitair Ziekenhuis Antwerpen

    RECRUITING

    Antwerp, 2650, Belgium

    Contact Email: •••••@•••••

  • University of North Carolina at Chapel Hill

    RECRUITING

    Chapel Hill, North Carolina, 27599, United States

    Contact Email: •••••@•••••

  • University of Virginia (UVA) Health - Developmental Pediatrics Clinic

    RECRUITING

    Charlottesville, Virginia, 22903, United States

    Contact Email: •••••@•••••

  • Universitätsklinikum Essen

    COMPLETED

    Essen, 45147, Germany

  • Uniwersyteckie Centrum Kliniczne

    RECRUITING

    Gdansk, Woj. Pomorskie, 80-952, Poland

    Contact Email: •••••@•••••

  • Vian - M. Iashvili Children's Central Hospital

    COMPLETED

    Tbilisi, 121, Georgia

  • Wielospecjalistyczna Poradnia Lekarska Synapsis

    RECRUITING

    Katowice, Woj. Slaskie, 40-123, Poland

    Contact Email: •••••@•••••

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