New hope for kids with rare muscle disease: drug trial launches
NCT ID NCT06392386
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a drug called efgartigimod in 12 children aged 2 to 18 with generalized myasthenia gravis, a condition that causes muscle weakness. The goal is to find the right dose and see if it is safe. Participants receive injections and are monitored for up to 14 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
What this could lead to
If successful, this could provide a new treatment option to control symptoms in children with generalized myasthenia gravis.
What could go wrong
This is a small, early-phase study 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- Hôpital de La Timone
RECRUITINGMarseille, 13385, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Carolinas HealthCare System Neurosciences Institute - Neurology
RECRUITINGCharlotte, North Carolina, 28207, United States
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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Childrens Hospital of Eastern Ontario
RECRUITINGOttawa, K1H 8L1, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Fakultni nemocnice Brno
RECRUITINGBrno, 613 00, Czechia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Fakultni nemocnice Ostrava
RECRUITINGOstrava, 708 00, Czechia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Hadassah Medical Center- Ein Kerem
RECRUITINGJerusalem, 9112001, Israel
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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Hôpital Nestlé
RECRUITINGLausanne, 1011, Switzerland
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Istituto G Gaslini Ospedale Pediatrico IRCCS
RECRUITINGGenova, 16147, Italy
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Leiden University Medical Center
RECRUITINGLeiden, 2333 ZA, Netherlands
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Neurologia Śląska Centrum Medyczne
RECRUITINGKatowice, 40-689, Poland
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Neurology Rare Disease Center
RECRUITINGDenton, Texas, 76208, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Oxford Children's Hospital
RECRUITINGOxford, OX3 9DU, United Kingdom
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Schneider Children's Medical Center of Israel
RECRUITINGPetah Tikvah, 4920235, Israel
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Tel Aviv Sourasky Medical Center Ichilov
RECRUITINGTel Aviv, 6423906, Israel
Contact Phone: •••-•••-•••• Email: •••••@•••••
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UZ Gent
RECRUITINGGhent, 9000, Belgium
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Virginia
RECRUITINGCharlottesville, Virginia, 22903, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Universitätsklinikum Essen
RECRUITINGEssen, 45147, Germany
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Uniwersyteckie Centrum Kliniczne w Gdansku
RECRUITINGGdansk, 80-211, Poland
Contact Phone: •••-•••-•••• Email: •••••@•••••