New hope for kids with rare GNAO1 epilepsy: experimental drug trial launches
NCT ID NCT07363603
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests an experimental drug called ASO-GNAO1 (Tianasen) in 5 children aged 1 to 14 with a specific GNAO1 gene mutation that causes severe epilepsy and movement problems. The drug is given via spinal injections over 12 months to see if it can reduce seizures and improve symptoms. Because the disease is very rare and serious, all participants receive the active drug, and there is no placebo group.
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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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Veltischev Research and Clinical Institute for Pediatrics and Pediatric Surgery of the Pirogov Russian National Research Medical University
RECRUITINGMoscow, Russia
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