Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype
NCT ID: NCT04167345
Last Updated: 2022-02-02
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
TERMINATED
PHASE2
48 participants
INTERVENTIONAL
2020-01-13
2020-11-14
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype
NCT04474197
A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype
NCT05643495
A Phase 1 Research Study to Evaluate Safety, Tolerability, and Pharmacokinetics of WVE-006 in Healthy Participants With Wild-type AAT Expression (RestorAATion-1)
NCT06186492
A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome
NCT05358886
Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis
NCT00409175
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Parts A1, A2 and B Combined: Placebo
Participants received placebo matched to VX-814 in the treatment period for 28 days.
Placebo
Placebo matched to VX-814 for oral administration.
Part A1: VX-814 100 milligrams (mg)
Participants received VX-814 100 mg every 12 hours (q12h) in the treatment period for 28 days.
VX-814
Tablet for oral administration.
Part A1: VX-814 200 mg
Participants received VX-814 200 mg q12h in the treatment period for 28 days.
VX-814
Tablet for oral administration.
Parts A1 and A2 Combined: VX-814 400 mg
Participants received VX-814 400 mg q12h in the treatment period for 28 days.
VX-814
Tablet for oral administration.
Part B: VX-814 600 mg
Participants received VX-814 600 mg q12h in the treatment period for 28 days.
VX-814
Tablet for oral administration.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
VX-814
Tablet for oral administration.
Placebo
Placebo matched to VX-814 for oral administration.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
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
Meet the organizations funding or collaborating on the study and learn about their roles.
Vertex Pharmaceuticals Incorporated
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
University of Alabama at Birmingham
Birmingham, Alabama, United States
University of California Davis Medical Center
Sacramento, California, United States
National Jewish Health
Denver, Colorado, United States
University of Florida, Shands Hospital
Gainesville, Florida, United States
University of Miami Miller School of Medicine
Miami, Florida, United States
Central Florida Pulmonary Group, PA
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
Columbia University Medical Center
New York, New York, 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
Cleveland Clinic Foundation
Cleveland, 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
Countries
Review the countries where the study has at least one active or historical site.
Provided Documents
Download supplemental materials such as informed consent forms, study protocols, or participant manuals.
Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
VX19-814-101
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.