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VGN-R09b in AADC deficiency
NCT05765981Phase 1Recruiting
- Sponsor
- Shanghai Jiao Tong University School of Medicine
- Start
- Jan 2023
- Primary completion
- Feb 2025
- Modality
- Gene Editing, Gene Silencing, AAV Vectors
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of VGN-R09b in Aromatic L-amino acid decarboxylase deficiency (AADC), sponsored by Shanghai Jiao Tong University School of Medicine. Primary endpoints: Adverse Events at 52 weeks and Achievement Ratio of motor development milestones at 52 weeks. Started Jan 2023; status: recruiting.
02
Drugs
VGN-R09b
03
Conditions
Aromatic L-amino acid decarboxylase deficiency (AADC)
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