The purpose of this study is to evaluate the efficacy and safety of VX-864 in participants with the PiZZ genotype over 48 weeks.
- Participants must have a PiZZ genotype confirmed at screening
- Plasma AAT levels indicating severe deficiency at screening
- 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
Interested in this trial?
About clinical trials
Volunteers who participate in clinical trials help make new medicines a reality. Clinical trials are carefully designed research studies in humans that evaluate the safety and efficacy of an investigational medicine.
If you’re interested in participating in a Vertex clinical trial, we encourage you to speak with your health care provider first about what’s involved and your eligibility.
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