A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype
NCT05643495 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 14
Last updated 2025-09-08
Summary
The purpose of this study evaluates the efficacy and safety of VX-864 in participants with the PiZZ genotype over 48 weeks.
Conditions
- Alpha-1 Antitrypsin Deficiency
Interventions
- DRUG
-
VX-864
Tablets for oral administration.
Sponsors & Collaborators
-
Vertex Pharmaceuticals Incorporated
lead INDUSTRY
Study Design
- Allocation
- NON_RANDOMIZED
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- PARALLEL
Eligibility
- Min Age
- 18 Years
- Max Age
- 80 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2023-02-23
- Primary Completion
- 2024-08-19
- Completion
- 2024-08-19
- FDA Drug
- Yes
Countries
- United States
- Germany
- Ireland
- United Kingdom
Study Locations
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