New drug tested for Long-Term safety in kids with rare muscle disease
NCT ID NCT05374590
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study looks at the long-term safety of efgartigimod in children with generalized myasthenia gravis, a disease that causes muscle weakness. The 12 participants are kids who already took part in earlier studies of this drug. They will continue to receive efgartigimod either through an IV or as a shot under the skin. The main goal is to track any side effects over time.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Efgartigimod (given intravenously or as a shot under the skin)
What this could lead to
If successful, this could confirm that efgartigimod is safe for long-term use in children with generalized myasthenia gravis, offering a continued treatment option.
What could go wrong
This is a small, open-label study with only 12 participants, so results may not apply to all children. It focuses on safety, not how well the drug works, and long-term risks are still unknown.
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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Locations
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AP-HM- Hôpital de La Timone
Marseille, 13005, France
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Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital
Chicago, Illinois, 60611, United States
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Atrium Health Neurology Specialty Care
Charlotte, North Carolina, 28207, United States
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Centralny Szpital Kliniczny - Uniwersyteckie Centrum Kliniczne WUM
Warsaw, 02-097, Poland
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Childrens Hospital of Eastern Ontario
Ottawa, K1H 8L1, Canada
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Great Ormand Street Hospital for Children NHS Foundation Trust - Great Ormond Street Hospital - Pediatric Neurology
London, WC1N 3JH, United Kingdom
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Hadassah Medical Center- Ein Kerem
Jerusalem, 9112001, Israel
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Hospital Universitari i Politecnic La Fe de Valencia
Valencia, 46026, Spain
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Leids Universitair Medisch Centrum
Leiden, 2333 ZA, Netherlands
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Oxford University Hospitals NHS Foundation Trust - John Radcliffe Hospital Children's Hospital
Oxford, OX3 9DU, United Kingdom
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UZ Gent
Ghent, 9000, Belgium
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Universitair Ziekenhuis Antwerpen
Edegem, 2650, Belgium
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University of Virginia (UVA) Health - Developmental Pediatrics Clinic
Charlottesville, Virginia, 22903, United States
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Universitätsklinikum Essen
Essen, 45147, Germany
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Uniwersyteckie Centrum Kliniczne w Gdansku
Gdansk, 80-211, Poland
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Wielospecjalistyczna Poradnia Lekarska Synapsis
Katowice, 40-123, Poland