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VX-864 in alpha-1 antitrypsin deficiency (PiZZ genotype)
2022-002746-40Phase 2Terminated
- Sponsor
- Vertex Pharmaceuticals
- Start
- Nov 2022
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of VX-864 in alpha-1 antitrypsin deficiency (AATD), sponsored by Vertex Pharmaceuticals. Primary endpoint: Change from baseline in blood levels of functional alpha-1 antitrypsin at Week 48. Started Nov 2022; status: terminated.
02
Drugs
VX-864
03
Conditions
alpha-1 antitrypsin deficiency (AATD)
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