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Phase 2 study of VX-864 in subjects with Alpha-1 antitrypsin deficiency (PiZZ genotype)
2019-004881-16Phase 2Completed
- Sponsor
- Vertex Pharmaceuticals
- Start
- Jul 2020
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, randomised, double-blind trial in alpha-1 antitrypsin deficiency (AATD) comparing VX-864 with Placebo (2 arms). Primary endpoint: Change from baseline in plasma functional AAT levels at Day 28. Started Jul 2020; status: completed.
02
Drugs
VX-864
03
Conditions
alpha-1 antitrypsin deficiency (AATD)
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