Summary

Eligibility
for people ages 18-80 (full criteria)
Location
at Sacramento, California and other locations
Dates
study started
estimated completion

Description

Summary

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

Official Title

A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-814 in PiZZ Subjects

Keywords

Alpha 1-Antitrypsin Deficiency

Eligibility

You can join if…

Open to people ages 18-80

  • Subjects must have a PiZZ genotype confirmed at screening
  • Plasma AAT levels indicating severe deficiency at screening

You CAN'T join if...

  • 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

Locations

  • University of California Davis Medical Center accepting new patients
    Sacramento California 95817 United States
  • Oregon Health & Science University accepting new patients
    Portland Oregon 97239 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Vertex Pharmaceuticals Incorporated
Links
Sign up for this study
ID
NCT04167345
Phase
Phase 2
Study Type
Interventional
Last Updated