Tianasen (ASO-GNAO1) for GNAO1-Encephalopathy With Epilepsy and Movement Disorders.
Recruiting
Phase 1
Phase 2
Interventional Study
GNAO1
Epilepsy
Hyperkinesis
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 1 – 14
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 5 (estimated)
- Sponsor
- Pirogov Russian National Research Medical University · Other
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About This Trial
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…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Informed Consent: Written informed consent from the parent(s) or legal guardian(s) of the patient and the child's assent (where applicable based on age and cognitive ability), obtained prior to the initiation of any study-related procedures.
* Age: Male or female children aged…
Contacts