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

Phase 2 Terminated NCT05643495

Condition	Alpha-1 Antitrypsin Deficiency
Phase	Phase 2
Sponsor	Vertex Pharmaceuticals Incorporated
Status	Terminated
Enrollment	14 participants
Interventions	VX-864
Start date	Feb 23, 2023
Completion date	Aug 19, 2024
Results posted	Sep 8, 2025

Summary

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

Change in Blood Functional Alpha-1 Antitrypsin (AAT) Levels

From Baseline at Week 48

Open in BENITIONS View on ClinicalTrials.gov

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This page summarises publicly available data from ClinicalTrials.gov. It is not medical advice and not investment advice. Always consult a qualified healthcare professional.

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