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

NCT07363603 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2026-01-23

No results posted yet for this study

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 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

Interventions

BIOLOGICAL

Antisense oligonucleotide treatment (ASO)

Intrathecal escalating doses from 0.3 mg/kg to 1.5 mg/kg (single administration per dose level)

Sponsors & Collaborators

  • Pirogov Russian National Research Medical University

    lead OTHER

Principal Investigators

  • Elena D Belousova, Prof · Pirogov Russian National Research Medical University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Year
Max Age
14 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-09-09
Primary Completion
2026-12-31
Completion
2026-12-31

Countries

  • Russia

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07363603 on ClinicalTrials.gov