New hope for rare muscle disease: ARGX-119 enters human testing
NCT ID NCT06436742
First seen Jun 24, 2026 · Last updated Jul 24, 2026 · Updated 2 times
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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
Locations
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Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, Illinois, 60611, United States
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CHU - Hospital de la Timone
Marseille, 13385, France
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Clinical Trials Centre - South Eastern Health and Social Care Trust - The Ulster Hospital
Belfast, BT16 1RH, United Kingdom
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Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, 20133, Italy
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Group Hospitalier Pitie-Salpetriere
Paris, 75013, France
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John Radcliffe Hospital - Oxford University Hospitals NHS Foundation Trust
Oxford, OX3 9DU, United Kingdom
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Ottawa Hospital Research Institute - Civic Campus
Ottawa, K1Y 4E9, Canada
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UC Davis Medical Center
Sacramento, California, 95817, United States
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Universitat de Valencia - Hospital Universitari i Politecnic La Fe de Valencia (Hospital La Fe Bulevar Sur)
Valencia, 46026, Spain
More trials for these conditions
Other studies related to the condition(s) this trial covers.