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

• Status: Not yet recruiting
• Trial end date: 2024-12-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Criteria

Key Inclusion Criteria:

• Participants must have a PiZZ genotype confirmed at screening

• Plasma AAT levels indicating severe deficiency at screening

Key Exclusion Criteria:

• History of a medical condition that could negatively impact the ability to complete the study

• Solid organ, or hematological transplantation or is currently on a transplant list

• History of use of gene therapy or Ribonucleic acid interference (RNAi) therapy at any time previously

• Participants for whom discontinuation of augmentation therapy is not considered to be in their best interest, based on the clinical judgement of the treating physician

Other protocol defined Inclusion/Exclusion criteria may apply.