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

Study acronym: ASO-GNAO1
StatusRecruiting
PhasePhase 1/2
Started
View on ClinicalTrials.gov ↗

Record History

  1. Study First Submitted
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 open-label study without a placebo control group due to the rare and severe nature of the disease. All participants will receive the active drug. Participants will: Receive escalating doses of ASO-GNAO1 via intrathecal injection over a 12-month period. Undergo frequent neurological assessments, biomarker testing, and safety monitoring.
Trial Details
NCT Number NCT07363603
Lead Sponsor Pirogov Russian National Research Medical University
Conditions
(from sponsor)		 GNAO1, Epilepsy, Hyperkinesis
Related terms Epilepsy, Hyperkinesis
Enrollment 5 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov