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NCT05643495
A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype
Conditions: Alpha-1 Antitrypsin Deficiency
Sex: All
Ages: 18 Years – 80 Years
Phase: PHASE2
Enrollment: 20
Sponsor: Vertex Pharmaceuticals Incorporated
Location: United States
Summary
The purpose of this study is to evaluate the efficacy and safety of VX-864 in participants with the PiZZ genotype over 48 weeks.
Eligibility Criteria
Key Inclusion Criteria:Participants must have a PiZZ genotype confirmed at screeningPlasma AAT levels indicating severe deficiency at screeningKey Exclusion Criteria:History of a medical condition that could negatively impact the ability to complete the studySolid organ, or hematological transplantation or is currently on a transplant listHistory of use of gene therapy or Ribonucleic acid interference (RNAi) therapy at any time previouslyParticipants for whom discontinuation of augmentation therapy is not considered to be in their best interest, based on the clinical judgement of the treating physicianOther protocol defined Inclusion/Exclusion criteria may apply.
Source: ClinicalTrials.gov (NCT05643495). StuddyBuddy aggregates publicly available trial information.
