Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype
NCT ID: NCT04474197
Last Updated: 2022-05-26
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
44 participants
INTERVENTIONAL
2020-07-24
2021-05-04
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Placebo
Participants received placebo matched to VX-864 in the treatment period for 28 days.
Placebo
Placebo matched to VX-864 for oral administration.
VX-864 100 mg
Participants received VX-864 100 milligrams (mg) every 12 hours (q12h) in the treatment period for 28 days.
VX-864
Tablets for oral administration.
VX-864 300 mg
Participants received VX-864 300 mg q12h in the treatment period for 28 days.
VX-864
Tablets for oral administration.
VX-864 500 mg
Participants received VX-864 500 mg q12h in the treatment period for 28 days.
VX-864
Tablets for oral administration.
Interventions
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VX-864
Tablets for oral administration.
Placebo
Placebo matched to VX-864 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 RNAi therapy at any time previously
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 Alabama at Birmingham
Birmingham, Alabama, United States
David Geffen School of Medicine at UCLA
Los Angeles, California, United States
University of California Davis Medical Center
Sacramento, California, United States
National Jewish Health
Denver, Colorado, United States
University of Florida
Gainesville, Florida, United States
University of Miami Miller School of Medicine
Miami, Florida, United States
Central Florida Pulmonary
Orlando, Florida, United States
The University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
University of Minnesota
Minneapolis, Minnesota, United States
Blessing Corporate Services, Inc., dba Blessing Health System
Hannibal, Missouri, United States
Cardinal Glennon Children's Hospital - St. Louis University
St Louis, Missouri, United States
University of North Carolina Medical Center
Chapel Hill, North Carolina, United States
Wake Forest University Baptist Medical Center
Winston-Salem, North Carolina, United States
University of Cincinnati
Cincinnati, Ohio, United States
Oregon Health & Science University
Portland, Oregon, United States
Temple University Hospital
Philadelphia, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
Renovatio Clinical
Houston, Texas, United States
The University of Texas Health Science Center at Tyler
Tyler, Texas, United States
University of Utah Health
Salt Lake City, Utah, United States
Inova Fairfax Medical Campus
Falls Church, Virginia, United States
Queen Elizabeth II Health Sciences Center
Halifax, , Canada
Inspiration Research Ltd
Toronto, , Canada
University Hospital RWTH Aachen
Aachen, , Germany
Universitätsklinikum Essen
Essen, , Germany
Medizinische Hochschule Hannover
Hanover, , Germany
Royal College of Surgeons in Ireland Clinical Research Centre, Beaumont Hospital
Beaumont, , Ireland
Skanes Universitetssjukhus, Malmo
Malmo, , Sweden
Staploe Medical Centre
Soham Ely, , 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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2019-004881-16
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
VX19-864-101
Identifier Type: -
Identifier Source: org_study_id
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