New Gene-Targeting drug hopes to tame rare childhood epilepsy
NCT ID NCT07363603
First seen Jan 23, 2026 · Last updated May 24, 2026 · Updated 21 times
Summary
This early-stage trial tests an experimental drug called ASO-GNAO1 (Tianasen) in 5 children aged 1-14 with a specific GNAO1 gene mutation that causes severe epilepsy and uncontrolled movements. The drug is given via spinal injections over 12 months to see if it can reduce seizures and movement episodes. Because the disease is very rare and serious, all participants receive the active drug, and the study focuses on safety and finding the right dose.
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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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Genom att skicka in godkänner du våra Användarvillkor
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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