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Trial intelligence / Profile preview
Long-term observational study of Gene Therapy for GLUT1 deficiency
jRCT2033250658Active, not recruiting
- Sponsor
- Japan Agency for Medical Research and Development
- Start
- Jan 2026
- Countries
- 1
01
Overview
Open-label, single-arm trial 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 evidence and 1 more. Started Jan 2026; status: active, not recruiting.
02
Conditions
Glucose Transporter Type 1 Deficiency Syndrome (Glut1DS)
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