EligibleTrials

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

Recruiting · NCT07363603 · Interventional (participants receive a specific treatment) · Lead sponsor: Pirogov Russian National Research Medical University

View the official record on ClinicalTrials.gov →
Epilepsy

Interventions studied

Antisense oligonucleotide treatment (ASO)

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.

Who can take part

Age range
1 Year to 14 Years
Sex
All (male and female)
Healthy volunteers
No - a diagnosis or condition is required
Phase
Phase 1, Phase 2
Study type
Interventional (participants receive a specific treatment)

Inclusion criteria

Exclusion criteria

Where it is running

1 location listed.

Read the full protocol, contacts and eligibility on ClinicalTrials.gov →

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