New hope for kids with rare muscle disease: drug trial launches
NCT ID NCT04833894
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.
Disclaimer
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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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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AP-HM - Hopital de la Timone
RECRUITINGMarseille, 13385, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Alberta Childrens Hospital
RECRUITINGCalgary, T3B 6A8, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital
RECRUITINGChicago, Illinois, 60611, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Assistance Publique Hopitaux de Paris (AP-HP) - Hopital Necker-Enfants Malades
RECRUITINGParis, 75015, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Azienda Ospedaliera Universitaria Policlinico Consorziale Di Bari
RECRUITINGBari, 70124, Italy
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Azienda Ospedaliero Universitaria A. Meyer
RECRUITINGFlorence, 50139, Italy
Contact Phone: •••-•••-•••• Email: •••••@•••••
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British Columbia Children's Hospital
RECRUITINGVancouver, V6H 3N1, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Centralny Szpital Kliniczny - Uniwersyteckie Centrum Kliniczne WUM
RECRUITINGWarsaw, 02-097, Poland
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Charite Universitaetsmedizin Berlin - Campus Virchow-Klinikum - Sozialpadiatrisches Zentrum
COMPLETEDBerlin, 13353, Germany
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Great Ormand Street Hospital for Children NHS Foundation Trust - Great Ormond Street Hospital
RECRUITINGLondon, WC1N 3JH, United Kingdom
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Hospital Sant Joan de Deu
RECRUITINGEsplugues de Llobregat, 08950, Spain
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Hospital Universitari i Politecnic La Fe de Valencia
RECRUITINGValencia, 46026, Spain
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Leids Universitair Medisch Centrum
RECRUITINGLeiden, 2333 ZA, Netherlands
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Manchester University NHS Foundation Trust - Royal Manchester Children's Hospital
RECRUITINGManchester, M13 9WL, United Kingdom
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Medizinische Universitat Wien
COMPLETEDVienna, 1090, Austria
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Ospedale Giannina Gaslini
RECRUITINGGenova, 16147, Italy
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Oxford University Hospitals NHS Foundation Trust - John Radcliffe Hospital Children's Hospital
RECRUITINGOxford, OX3 9DU, United Kingdom
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Tbilisi State Medical University - Givi Zhvania Pediatric Academic Clinic
COMPLETEDTbilisi, 159, Georgia
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Universitair Ziekenhuis Antwerpen
RECRUITINGAntwerp, 2650, Belgium
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of North Carolina at Chapel Hill
RECRUITINGChapel Hill, North Carolina, 27599, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Virginia (UVA) Health - Developmental Pediatrics Clinic
RECRUITINGCharlottesville, Virginia, 22903, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Universitätsklinikum Essen
COMPLETEDEssen, 45147, Germany
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Uniwersyteckie Centrum Kliniczne
RECRUITINGGdansk, Woj. Pomorskie, 80-952, Poland
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Vian - M. Iashvili Children's Central Hospital
COMPLETEDTbilisi, 121, Georgia
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Wielospecjalistyczna Poradnia Lekarska Synapsis
RECRUITINGKatowice, Woj. Slaskie, 40-123, Poland
Contact Phone: •••-•••-•••• Email: •••••@•••••