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Base Editing for Mutation Repair in HSPCs for X-linked CGD
NCT06325709Phase 2Recruiting
- Sponsor
- National Institute of Allergy and Infectious Diseases
- Start
- Apr 2024
- Primary completion
- Dec 2032
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial in X-linked chronic granulomatous disease (X-CGD), sponsored by National Institute of Allergy and Infectious Diseases. Primary endpoints: Safety of gene therapy using BE autologous HSPCs at 2 years and Efficacy of gene therapy at 12 months. Started Apr 2024; status: recruiting.
02
Conditions
X-linked chronic granulomatous disease (X-CGD)
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