First human trial of targeted therapy for devastating childhood brain disease
NCT ID NCT07363603
Summary
This study is testing a new drug called Tianasen for children with a rare and severe genetic brain disorder called GNAO1-encephalopathy. The goal is to see if the drug can safely reduce seizures and movement problems and possibly slow the disease's progression. All participants will receive the drug through spinal injections over a year, and researchers will closely monitor their symptoms and safety.
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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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Veltischev Research and Clinical Institute for Pediatrics and Pediatric Surgery of the Pirogov Russian National Research Medical University
RECRUITINGMoscow, Russia
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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