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New hope for kids with rare Muscle-Weakening disease

NCT ID NCT04833894

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

Summary

This study is testing a medication called efgartigimod in children and teenagers with generalized myasthenia gravis, a rare autoimmune disease that causes severe muscle weakness. The trial aims to find the right dose for young patients and see how safe and effective it is at controlling their symptoms. About 12 participants aged 2 to 18 will receive the treatment for 8 to 18 weeks while researchers monitor their health and disease activity.

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 - Hopital de la Timone

    RECRUITING

    Marseille, 13385, France

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

  • Alberta Childrens Hospital

    RECRUITING

    Calgary, T3B 6A8, Canada

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

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

    RECRUITING

    Chicago, Illinois, 60611, United States

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

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

    RECRUITING

    Paris, 75015, France

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

  • Azienda Ospedaliera Universitaria Policlinico Consorziale Di Bari

    RECRUITING

    Bari, 70124, Italy

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

  • Azienda Ospedaliero Universitaria A. Meyer

    RECRUITING

    Florence, 50139, Italy

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

  • British Columbia Children's Hospital

    RECRUITING

    Vancouver, V6H 3N1, Canada

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

  • Centralny Szpital Kliniczny - Uniwersyteckie Centrum Kliniczne WUM

    RECRUITING

    Warsaw, 02-097, Poland

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

  • Hospital Sant Joan de Deu

    RECRUITING

    Esplugues de Llobregat, 08950, Spain

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

  • Hospital Universitari i Politecnic La Fe de Valencia

    RECRUITING

    Valencia, 46026, Spain

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

  • Leids Universitair Medisch Centrum

    RECRUITING

    Leiden, 2333 ZA, Netherlands

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

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

    RECRUITING

    Manchester, M13 9WL, United Kingdom

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

  • Medizinische Universitat Wien

    COMPLETED

    Vienna, 1090, Austria

  • Ospedale Giannina Gaslini

    RECRUITING

    Genova, 16147, Italy

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

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

    RECRUITING

    Oxford, OX3 9DU, United Kingdom

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

  • Tbilisi State Medical University - Givi Zhvania Pediatric Academic Clinic

    COMPLETED

    Tbilisi, 159, Georgia

  • Universitair Ziekenhuis Antwerpen

    RECRUITING

    Antwerp, 2650, Belgium

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

  • University of North Carolina at Chapel Hill

    RECRUITING

    Chapel Hill, North Carolina, 27599, United States

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

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

    RECRUITING

    Charlottesville, Virginia, 22903, United States

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

  • Universitätsklinikum Essen

    COMPLETED

    Essen, 45147, Germany

  • Uniwersyteckie Centrum Kliniczne

    RECRUITING

    Gdansk, Woj. Pomorskie, 80-952, Poland

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

  • Vian - M. Iashvili Children's Central Hospital

    COMPLETED

    Tbilisi, 121, Georgia

  • Wielospecjalistyczna Poradnia Lekarska Synapsis

    RECRUITING

    Katowice, Woj. Slaskie, 40-123, Poland

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

Conditions

Explore the condition pages connected to this study.

GENERALIZED MYASTHENIA GRAVIS