Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype
- Registration Number
- NCT04474197
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-864 in PiZZ subjects.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 44
- Subjects 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 RNAi therapy at any time previously
Other protocol defined Inclusion/Exclusion criteria may apply
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebo Participants received placebo matched to VX-864 in the treatment period for 28 days. VX-864 300 mg VX-864 Participants received VX-864 300 mg q12h in the treatment period for 28 days. VX-864 500 mg VX-864 Participants received VX-864 500 mg q12h in the treatment period for 28 days. VX-864 100 mg VX-864 Participants received VX-864 100 milligrams (mg) every 12 hours (q12h) in the treatment period for 28 days.
- Primary Outcome Measures
Name Time Method Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels From Baseline at Day 28 Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) Day 1 up to Week 8
- Secondary Outcome Measures
Name Time Method Observed Pre-dose Plasma Concentration (Ctrough) of VX-864 Pre-dose at Day 7, Day 14, Day 21 and Day 28 Change in Plasma Antigenic AAT Levels From Baseline at Day 28
Trial Locations
- Locations (30)
Blessing Corporate Services, Inc., dba Blessing Health System
🇺🇸Hannibal, Missouri, United States
Cardinal Glennon Children's Hospital - St. Louis University
🇺🇸Saint Louis, Missouri, United States
Universitätsklinikum Essen
🇩🇪Essen, Germany
Medizinische Hochschule Hannover
🇩🇪Hannover, Germany
Wake Forest University Baptist Medical Center
🇺🇸Winston-Salem, North Carolina, United States
Renovatio Clinical
🇺🇸Houston, Texas, United States
University Hospital RWTH Aachen
🇩🇪Aachen, Germany
David Geffen School of Medicine at UCLA
🇺🇸Los Angeles, California, United States
Central Florida Pulmonary
🇺🇸Orlando, Florida, United States
University of North Carolina Medical Center
🇺🇸Chapel Hill, North Carolina, United States
Inova Fairfax Medical Campus
🇺🇸Falls Church, Virginia, United States
University of California Davis Medical Center
🇺🇸Sacramento, California, United States
Queen Elizabeth II Health Sciences Center
🇨🇦Halifax, Canada
University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
University of Utah Health
🇺🇸Salt Lake City, Utah, United States
Staploe Medical Centre
🇬🇧Soham Ely, United Kingdom
Inspiration Research Ltd
🇨🇦Toronto, Canada
Skanes Universitetssjukhus, Malmo
🇸🇪Malmö, Sweden
The University of Iowa Hospitals and Clinics
🇺🇸Iowa City, Iowa, United States
Temple University Hospital
🇺🇸Philadelphia, Pennsylvania, United States
Oregon Health & Science University
🇺🇸Portland, Oregon, United States
National Jewish Health
🇺🇸Denver, Colorado, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Royal College of Surgeons in Ireland Clinical Research Centre, Beaumont Hospital
🇮🇪Beaumont, Ireland
Medical University of South Carolina
🇺🇸Charleston, South Carolina, United States
University of Florida
🇺🇸Gainesville, Florida, United States
University of Cincinnati
🇺🇸Cincinnati, Ohio, United States
The University of Texas Health Science Center at Tyler
🇺🇸Tyler, Texas, United States
University of Miami Miller School of Medicine
🇺🇸Miami, Florida, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States