Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype
A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 in PiZZ Subjects
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-864 in PiZZ subjects.
Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels
Change in Plasma Antigenic AAT Levels
Study Arms / Comparison Groups
Description: Participants received placebo matched to VX-864 in the treatment period for 28 days.
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
July 24, 2020
May 4, 2021
Primary Completion Date
May 4, 2021
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
18 Years - 80 Years
Accepts Healthy Volunteers
Vertex Pharmaceuticals Incorporated