New hope for kids with rare Muscle-Weakening disease
NCT ID NCT06607627
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a drug called gefurulimab in 12 children aged 6 to 18 with generalized myasthenia gravis, a condition that causes muscle weakness. The drug works by blocking a part of the immune system to reduce symptoms. Participants receive weekly injections, and researchers will check how the drug moves through the body, its safety, and whether it improves muscle strength.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
gefurulimab (a drug that blocks a part of the immune system called complement C5)
What this could lead to
If successful, this could provide a new treatment option to control muscle weakness 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 patients. The drug requires lifelong use and carries infection risks, including meningococcal infection.
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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Research Site
RECRUITINGWashington D.C., District of Columbia, 20010, United States
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Research Site
RECRUITINGNorfolk, Virginia, 23507, United States
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Research Site
RECRUITINGJoinville, 89202-451, Brazil
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Research Site
RECRUITINGSalvador, 41253-190, Brazil
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Research Site
RECRUITINGSão José do Rio Preto, 15090-000, Brazil
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Research Site
RECRUITINGSão Paulo, 0438-002, Brazil
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Research Site
RECRUITINGSão Paulo, 05403-000, Brazil
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Research Site
RECRUITINGSaitama-Shi, 330-8777, Japan
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Research Site
WITHDRAWNBydgoszcz, 85-065, Poland
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Research Site
WITHDRAWNLodz, 93-338, Poland
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Research Site
RECRUITINGWarsaw, 02-097, Poland
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Research Site
RECRUITINGNew Taipei City, 23561, Taiwan
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Research Site
RECRUITINGTaipei, 11101, Taiwan