NCT07363603 Tianasen (ASO-GNAO1) for GNAO1-Encephalopathy With Epilepsy and Movement Disorders.
| NCT ID | NCT07363603 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Pirogov Russian National Research Medical University |
| Condition | GNAO1 |
| Study Type | INTERVENTIONAL |
| Enrollment | 5 participants |
| Start Date | 2025-09-09 |
| Primary Completion | 2026-12-31 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 5 participants in total. It began in 2025-09-09 with a primary completion date of 2026-12-31.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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.
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 1 year and older (≥1 year) until 14 years at the time of informed consent signing. * Diagnosis: A confirmed a c.607G\>A variant in of GNAO1 gene based on genetic testing, and a clinical presentation that includes both epilepsy and movement disorders. * Treatment Resistance: * For seizures: Documented resistance to antiseizure medications prior to screening, defined as the persistence of seizures despite adequate trials of at least two appropriately dosed antiseizure medications. * For non-epileptic hyperkinesias/dystonia: Documented resistance to anti-hyperkinetic medications prior to screening, defined as the persistence of debilitating hyperkinesias or dystonic attacks despite adequate trials of at least two appropriately dosed anti-hyperkinetic medications. * Contraception (for females of reproductive potential): For post- menarche female adolescents, a negative serum or urine pregnancy test at screening and agreement to use highly effective methods of contraception (e.g., hormonal implants, combined oral contraceptives, intrauterine device) throughout the study participation period. Exclusion Criteria: * Unacceptable Risk: Any concurrent severe medical, neurological, or psychiatric condition, or any other significant circumstance (e.g., unstable clinical status) that, in the judgment of the Investigator, could significantly increase the risk associated with study participation or the administration of the investigational product, or could interfere with the interpretation of study results. * Impossibility of Intervention: Any anatomical abnormality, coagulation disorder, active infection, or other condition that constitutes a contraindication to or precludes the safe performance of repeated lumbar punctures for intrathecal administration of the study drug. * Pregnancy or Lactation: Pregnancy, lactation, or intention to become pregnant during the study period. * Concurrent Experimental Therapy: Receipt of any other investigational drug, device, or biological product within 1 month prior to screening or within a period of at least 5 half-lives of that product (whichever is longer). * Protocol Compliance: Any other disease, condition, or behavioral factor that, in the opinion of the Investigator, could compromise the patient's safety, preclude adherence to the protocol schedule, or interfere with the study conduct and endpoint assessments. Age: 14 years and older
Contact & Investigator
Elena D Belousova, Prof
PRINCIPAL INVESTIGATOR
Pirogov Russian National Research Medical University
Frequently Asked Questions
Who can join the NCT07363603 clinical trial?
This trial is open to participants of all sexes, aged 1 Year or older, up to 14 Years, studying GNAO1. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07363603 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT07363603 currently recruiting?
Yes, NCT07363603 is actively recruiting participants. Contact the research team at a.sharkov@pedklin.ru for enrollment information.
Where is the NCT07363603 trial being conducted?
This trial is being conducted at Moscow, Russia.
Who is sponsoring the NCT07363603 clinical trial?
NCT07363603 is sponsored by Pirogov Russian National Research Medical University. The principal investigator is Elena D Belousova, Prof at Pirogov Russian National Research Medical University. The trial plans to enroll 5 participants.