Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype
Sponsored by Vertex Pharmaceuticals Incorporated
About this trial
Last updated 3 years ago
Study ID
VX19-864-101
Status
Completed
Type
Interventional
Phase
Phase 2
Placebo
Yes
Accepting
18 to 80 Years
All
Not accepting
Healthy Volunteers
Trial Timing
Ended 4 years ago
What is this trial about?
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-864 in PiZZ
subjects.
What are the Participation Requirements?
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