NCT05643495

A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype

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

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 14 (actual)
• Sponsor: Vertex Pharmaceuticals Incorporated · Industry
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study evaluates the efficacy and safety of VX-864 in participants with the PiZZ genotype over 48 weeks.

Conditions

• Alpha-1 Antitrypsin Deficiency

Interventions

Type	Name	Description
DRUG	VX-864	Tablets for oral administration.

Timeline

• Start date: 2023-02-23
• Primary completion: 2024-08-19
• Completion: 2024-08-19
• First posted: 2022-12-08
• Last updated: 2025-09-08
• Results posted: 2025-09-08

Locations

13 sites across 4 countries: United States, Germany, Ireland, United Kingdom

Regulatory

• FDA-regulated drug study