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VGM-R02b Gene Therapy for Glutaric Acidemia Type I
NCT06217861Phase 1Recruiting
- Sponsor
- Shanghai Vitalgen BioPharma Co., Ltd.
- Start
- Apr 2024
- Primary completion
- Aug 2027
- Modality
- Gene Editing, Gene Silencing, AAV Vectors
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of VGM-R02b in glutaric acidemia type 1 (GA1), sponsored by Shanghai Vitalgen BioPharma Co., Ltd. Primary endpoints: Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs) at 52 weeks and Changes from baseline in vital signs, and clinical laboratory results at 52 weeks. Started Apr 2024; status: recruiting.
02
Drugs
VGM-R02b
03
Conditions
glutaric acidemia type 1 (GA1)
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