Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype

• Status: Terminated
• Trial end date: 2020-11-14
• Target enrollment: 0
• Participant gender: All

Summary

This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-814 in PiZZ subjects.

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

Details

• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Criteria

Key Inclusion Criteria:

• Subjects 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 RNAi therapy at any time previously

Other protocol defined Inclusion/Exclusion criteria may apply