New hope for kids with rare GNAO1 epilepsy: experimental drug trial launches

NCT ID NCT07363603

Recruiting now Disease control Sponsor: Pirogov Russian National Research Medical University Source: ClinicalTrials.gov ↗

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.

Disclaimer Read more

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.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for EPILEPSY are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

developmental and epileptic encephalopathy, 17 epilepsy Hyperkinesis neurodevelopmental disorder with involuntary movements

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

EPILEPSY GNAO1 HYPERKINESIS

Contacts and locations

Show contact details

Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

Veltischev Research and Clinical Institute for Pediatrics and Pediatric Surgery of the Pirogov Russian National Research Medical University
RECRUITING

Moscow, Russia

Contact Phone: •••-•••-•••• Email: •••••@•••••