A Phase 2, Open-label Study Evaluating Efficacy and Safety of VX-864 in Subjects With Alpha-1 Antitrypsin Deficiency Who Have the PiZZ Genotype, Over 48 Weeks
Latest Information Update: 06 Oct 2025
At a glance
- Drugs VX 864 (Primary)
- Indications Alpha 1-antitrypsin deficiency
- Focus Therapeutic Use
- Sponsors Vertex Pharmaceuticals
Most Recent Events
- 08 Sep 2025 Status changed from completed to discontinued.
- 19 Sep 2024 Status changed from active, no longer recruiting to completed.
- 21 Nov 2023 This trial has been discontinued in Ireland, according to the European Clinical Trials Database record.