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AAV2/8.TBG.hARSB for MPS VI
NCT03173521Phase 2Completed
- Sponsor
- Fondazione Telethon
- Start
- Jul 2017
- Primary completion
- Jul 2021
- Modality
- Gene Editing, Gene Silencing, Gene Addition/Replacement
- Countries
- 2
01
Overview
Phase 2, open-label, single-arm trial of AAV8-hARSB in mucopolysaccharidosis type VI, sponsored by Fondazione Telethon. Primary endpoints: Safety and tolerability of the IMP administration at 5 years and Urinary GAG levels at 5 years. Started Jul 2017; status: completed.
02
Drugs
AAV8-hARSB
03
Conditions
mucopolysaccharidosis type VI
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