New hope for rare muscle disease: ARGX-119 enters human testing
NCT ID NCT06436742
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-stage trial tests a new biologic drug called ARGX-119 in 16 adults with a rare genetic muscle weakness condition called DOK7-congenital myasthenic syndrome (CMS). The study aims to see if the drug is safe and how the body processes it. Participants will receive either ARGX-119 or a placebo by IV infusion, and researchers will monitor side effects and measure changes in walking distance and 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
ARGX-119 (a biologic drug given by IV infusion)
What this could lead to
If it works, this could point toward a treatment that improves muscle strength and daily function for people with DOK7-CMS.
What could go wrong
This is a very early Phase 1b trial with only 16 participants, so it is primarily testing safety. It may not show clear benefit, and results may not apply to all CMS types.
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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Ann and Robert H Lurie Childrens Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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CHU - Hospital de la Timone
COMPLETEDMarseille, 13385, France
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Clinical Trials Centre - South Eastern Health and Social Care Trust - The Ulster Hospital
RECRUITINGBelfast, BT16 1RH, United Kingdom
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Fondazione IRCCS Istituto Neurologico Carlo Besta
RECRUITINGMilan, 20133, Italy
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Group Hospitalier Pitie-Salpetriere
RECRUITINGParis, 75013, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
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John Radcliffe Hospital - Oxford University Hospitals NHS Foundation Trust
ACTIVE_NOT_RECRUITINGOxford, OX3 9DU, United Kingdom
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Ottawa Hospital Research Institute - Civic Campus
RECRUITINGOttawa, K1Y 4E9, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
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UC Davis Medical Center
ACTIVE_NOT_RECRUITINGSacramento, California, 95817, United States
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Universitat de Valencia - Hospital Universitari i Politecnic La Fe de Valencia (Hospital La Fe Bulevar Sur)
ACTIVE_NOT_RECRUITINGValencia, 46026, Spain