New hope for rare muscle disease: experimental drug ARGX-119 enters human testing
NCT ID NCT06436742
First seen Nov 01, 2025 · Last updated May 16, 2026 · Updated 30 times
Summary
This early-stage study tests a new medicine called ARGX-119 in 16 adults with a rare genetic condition (DOK7-CMS) that causes muscle weakness. The main goal is to check if the drug is safe and how the body handles it. Participants will receive either the drug or a placebo, and later all can receive the active drug. The study lasts about 38 months and also looks at whether the drug improves walking and daily function.
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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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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
Conditions
Explore the condition pages connected to this study.