A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype
Sponsored by Vertex Pharmaceuticals Incorporated
About this trial
Last updated 2 years ago
Study ID
Status
Type
Phase
Placebo
Accepting
Not accepting
Trial Timing
Ended 8 months ago
What is this trial about?
What are the Participation Requirements?
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.