Recruiting
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
December 2022 - December 2024
Study type
Interventional
Intervention/Treatment
Drug : VX-864
Estimated enrollment
20

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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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Location
  • Recruiting
    1
    Hannibal Regional Healthcare System
    Hannibal Missouri 63401 United States
  • Recruiting
    1
    Renovatio Clinical
    Houston Texas 77380 United States