A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype
- Registration Number
- NCT05643495
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
The purpose of this study is to evaluate the efficacy and safety of VX-864 in participants with the PiZZ genotype over 48 weeks.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 20
- Participants must have a PiZZ genotype confirmed at screening
- Plasma AAT levels indicating severe deficiency at screening
Key
- 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 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
Other protocol defined Inclusion/Exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Group A VX-864 Participants will receive VX-864 every 12 hours (q12h) for 48 weeks. Group B VX-864 Participants will undergo a liver biopsy done before receiving VX-864 q12h for 48 weeks, and will undergo a second liver biopsy at either Week 24 or Week 48.
- Primary Outcome Measures
Name Time Method Change in Blood Functional Alpha-1 Antitrypsin (AAT) Levels From Baseline at Week 48
- Secondary Outcome Measures
Name Time Method Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) Day 1 up to Week 52 Change in Blood Functional AAT Levels From Baseline up to Week 48 Change in Blood Antigenic AAT Levels From Baseline up to Week 48 Change in Blood Z-polymer Levels From Baseline up to Week 48 Part B: Change in Z-polymer Accumulation in the Liver From Baseline up to Week 48
Trial Locations
- Locations (13)
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States
University of Florida
🇺🇸Gainesville, Florida, United States
The University of Iowa Hospitals and Clinics: Adult Pulmonary Clinic
🇺🇸Iowa City, Iowa, United States
Inova Fairfax Medical Campus
🇺🇸Falls Church, Virginia, United States
University Hospital RWTH Aachen
🇩🇪Aachen, Germany
Hannibal Regional Healthcare System
🇺🇸Hannibal, Missouri, United States
Marsico Clinical Research Center at UNC Pulmonary Clinic
🇺🇸Chapel Hill, North Carolina, United States
Columbia University Irving Medical Center
🇺🇸New York, New York, United States
King's College Hospital
🇬🇧London, United Kingdom
Renovatio Clinical
🇺🇸Houston, Texas, United States
University of Utah Health
🇺🇸Salt Lake City, Utah, United States
Royal College of Surgeons in Ireland Clinical Research Centre, Beaumont Hospital
🇮🇪Beaumont, Ireland
Central Florida Pulmonary Group, P.A.
🇺🇸Orlando, Florida, United States