A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype
NCT ID: NCT05643495
Last Updated: 2025-09-08
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE2
14 participants
INTERVENTIONAL
2023-02-23
2024-08-19
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Group A
Participants received VX-864 every 12 hours (q12h) for 48 weeks or until study drug dosing was terminated.
VX-864
Tablets for oral administration.
Group B
Participants undergo a liver biopsy before receiving VX-864 q12h for 48 weeks or until study drug dosing was terminated, and undergo a second liver biopsy at either Week 24 or Week 48.
VX-864
Tablets for oral administration.
Interventions
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VX-864
Tablets for oral administration.
Eligibility Criteria
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Inclusion Criteria
* Plasma AAT levels indicating severe deficiency at screening
Exclusion Criteria
* 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
18 Years
80 Years
ALL
No
Sponsors
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Vertex Pharmaceuticals Incorporated
INDUSTRY
Responsible Party
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Locations
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University of Florida
Gainesville, Florida, United States
Central Florida Pulmonary Group, P.A.
Orlando, Florida, United States
The University of Iowa Hospitals and Clinics: Adult Pulmonary Clinic
Iowa City, Iowa, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Hannibal Regional Healthcare System
Hannibal, Missouri, United States
Columbia University Irving Medical Center
New York, New York, United States
Marsico Clinical Research Center at UNC Pulmonary Clinic
Chapel Hill, North Carolina, United States
Renovatio Clinical
Houston, Texas, United States
University of Utah Health
Salt Lake City, Utah, United States
Inova Fairfax Medical Campus
Falls Church, Virginia, United States
University Hospital RWTH Aachen
Aachen, , Germany
Royal College of Surgeons in Ireland Clinical Research Centre, Beaumont Hospital
Beaumont, , Ireland
King's College Hospital
London, , United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2022-002746-40
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
VX22-864-108
Identifier Type: -
Identifier Source: org_study_id
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