Recruiting completed
Phase 2
A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype
The purpose of this study is to evaluate the efficacy and safety of VX-864 in participants with the PiZZ genotype over 48 weeks.
ClinicalTrials.gov Identifier
Eligibility criteria
Sex
Age
18 Years - 80 Years
Phase
2
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.
Trial details
Condition
Alpha-1 Antitrypsin Deficiency
Study duration
February 2023 - December 2024
Study type
Interventional
Intervention/Treatment
Drug : VX-864
Estimated enrollment
20
Interested in this trial?
About clinical trials
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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.
Location
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