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Phase I/II intrathecal AAV.GTX-GLUT1 gene therapy study for GLUT1 deficiency
jRCT2033240749Phase 2Recruiting
- Sponsor
- Japan Agency for Medical Research and Development
- Start
- Jul 2025
- Modality
- Gene Therapies
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of AAV.GTX-GLUT1 in Glucose Transporter Type 1 Deficiency Syndrome (Glut1DS), sponsored by Japan Agency for Medical Research and Development. Primary endpoints: Occurrence of adverse events and defects, Laboratory findings and 2 more. Started Jul 2025; status: recruiting.
02
Drugs
AAV.GTX-GLUT1
03
Conditions
Glucose Transporter Type 1 Deficiency Syndrome (Glut1DS)
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