The purpose of this study evaluates the efficacy and safety of VX-864 in participants with the PiZZ genotype over 48 weeks.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
14
Tablets for oral administration.
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
Change in Blood Functional Alpha-1 Antitrypsin (AAT) Levels
Time frame: From Baseline at Week 48
Change in Blood Functional AAT Levels
Time frame: From Baseline up to Week 48
Change in Blood Antigenic AAT Levels
Time frame: From Baseline up to Week 48
Change in Blood Z-polymer Levels
Time frame: From Baseline up to Week 48
Group B: Change in Z-polymer Accumulation in the Liver
Time frame: From Baseline up to Week 48
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Day 1 up to Week 52
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
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
...and 3 more locations