Actively Recruiting
Tianasen (ASO-GNAO1) for GNAO1-Encephalopathy With Epilepsy and Movement Disorders.
Led by Pirogov Russian National Research Medical University · Updated on 2026-01-23
5
Participants Needed
1
Research Sites
68 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Tianasen (ASO-GNAO1) for GNAO1-Encephalopathy With Epilepsy and Movement Disorders.
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Written informed consent from parent(s) or legal guardian(s) and child's assent when appropriate
- Male or female children aged 1 year to less than 14 years at consent
- Confirmed c.607G>A mutation in GNAO1 gene with epilepsy and movement disorders
- Documented resistance to at least two antiseizure medications for seizures
- Documented resistance to at least two anti-hyperkinetic medications for hyperkinesias or dystonia
- Negative pregnancy test and agreement to use effective contraception for post-menarche females
You will not qualify if you...
- Any severe medical, neurological, or psychiatric condition increasing risk or affecting study results
- Anatomical abnormalities, coagulation disorders, infections, or other issues preventing safe lumbar puncture
- Pregnancy, lactation, or intention to become pregnant during study
- Use of other investigational drugs or devices within 1 month or 5 half-lives before screening
- Any condition that compromises safety, protocol adherence, or study assessments
- Age 14 years or older
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Veltischev Research and Clinical Institute for Pediatrics and Pediatric Surgery of the Pirogov Russian National Research Medical University
Moscow, Russia
Actively Recruiting
Research Team
A
Artem A Sharkov
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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