Clinical Trial: Tianasen (ASO-GNAO1) for GNAO1-Encephalopathy With Epilepsy and Movement Disorders.

U.S., Jan. 24 -- ClinicalTrials.gov registry received information related to the study (NCT07363603) titled 'Tianasen (ASO-GNAO1) for GNAO1-Encephalopathy With Epilepsy and Movement Disorders.' on Sept. 29, 2025.

Brief Summary: The goal of this clinical trial is to evaluate the efficacy and safety of the investigational drug ASO-GNAO1 (Tianasen) in pediatric patients with c.607G>A mutation in the GNAO1 gene associated with epilepsy and neurodevelopmental disorder. The main questions it aims to answer are:

1. Does intrathecal administration of ASO-GNAO1 slow or halt the progression of motor and cognitive symptoms?

2. Is ASO-GNAO1 safe and well-tolerated in this patient population?

3. What is the appropriate therapeutic dose?

This is an ...