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Guard1: AVR-RD-02 for Type 1 Gaucher Disease
NCT04145037Phase 2Terminated
- Sponsor
- AVROBIO, Inc.
- Start
- May 2019
- Primary completion
- Aug 2023
- Modality
- Gene Editing, Gene Silencing, Lentiviral Vectors
- Countries
- 2
01
Overview
Phase 2, open-label trial of AVR-RD-02 in Gaucher disease type 1 (GD1) (2 arms), sponsored by AVROBIO, Inc. Primary endpoints: Number of Clinically Significant Adverse Events (AEs) and Serious Adverse Events (SAEs) at 52 weeks, Vector Copy Number (VCN) in Peripheral Blood at 52 weeks and 6 more. Started May 2019; status: terminated.
02
Drugs
AVR-RD-02
03
Conditions
Gaucher disease type 1 (GD1)
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