- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04167345
Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype
January 7, 2022 updated by: Vertex Pharmaceuticals Incorporated
A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-814 in PiZZ Subjects
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-814 in PiZZ subjects.
Study Overview
Status
Terminated
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
48
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Halifax, Canada
- Queen Elizabeth II Health Sciences Center
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Toronto, Canada
- Inspiration Research Ltd
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Aachen, Germany
- University Hospital RWTH Aachen
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Essen, Germany
- Universitätsklinikum Essen
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Hannover, Germany
- Medizinische Hochschule Hannover
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Beaumont, Ireland
- Royal College of Surgeons in Ireland Clinical Research Centre, Beaumont Hospital
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Alabama
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Birmingham, Alabama, United States, 35233
- University of Alabama at Birmingham
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California
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Sacramento, California, United States, 95817
- University of California Davis Medical Center
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Colorado
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Denver, Colorado, United States, 80206
- National Jewish Health
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Florida
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Gainesville, Florida, United States, 32610
- University of Florida, Shands Hospital
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Miami, Florida, United States, 33136
- University of Miami Miller School of Medicine
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Orlando, Florida, United States, 32803
- Central Florida Pulmonary Group, PA
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Iowa
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Iowa City, Iowa, United States, 52242
- The University of Iowa Hospitals and Clinics
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Kansas
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Kansas City, Kansas, United States, 66160
- University of Kansas Medical Center
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Minnesota
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Minneapolis, Minnesota, United States, 55455
- University of Minnesota
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Missouri
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Hannibal, Missouri, United States, 63401
- Blessing Corporate Services, Inc., dba Blessing Health System
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New York
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New York, New York, United States, 10032
- Columbia University Medical Center
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North Carolina
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Chapel Hill, North Carolina, United States, 27514
- University of North Carolina Medical Center
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Winston-Salem, North Carolina, United States, 27157
- Wake Forest University Baptist Medical Center
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Ohio
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Cincinnati, Ohio, United States, 45267
- University of Cincinnati
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Cleveland, Ohio, United States, 44195
- Cleveland Clinic Foundation
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Oregon
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Portland, Oregon, United States, 97239
- Oregon Health & Science University
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19140
- Temple University Hospital
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South Carolina
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Charleston, South Carolina, United States, 29425
- Medical University of South Carolina
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Texas
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Houston, Texas, United States, 77380
- Renovatio Clinical
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Tyler, Texas, United States, 75708
- The University of Texas Health Science Center at Tyler
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Utah
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Salt Lake City, Utah, United States, 84108
- University of Utah Health
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Virginia
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Falls Church, Virginia, United States, 22042
- INOVA Fairfax Medical Campus
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years to 80 years (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Key Inclusion Criteria:
- Subjects must have a PiZZ genotype confirmed at screening
- Plasma AAT levels indicating severe deficiency at screening
Key Exclusion Criteria:
- 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 Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Placebo Comparator: Parts A1, A2 and B Combined: Placebo
Participants received placebo matched to VX-814 in the treatment period for 28 days.
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Placebo matched to VX-814 for oral administration.
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Experimental: Part A1: VX-814 100 milligrams (mg)
Participants received VX-814 100 mg every 12 hours (q12h) in the treatment period for 28 days.
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Tablet for oral administration.
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Experimental: Part A1: VX-814 200 mg
Participants received VX-814 200 mg q12h in the treatment period for 28 days.
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Tablet for oral administration.
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Experimental: Parts A1 and A2 Combined: VX-814 400 mg
Participants received VX-814 400 mg q12h in the treatment period for 28 days.
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Tablet for oral administration.
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Experimental: Part B: VX-814 600 mg
Participants received VX-814 600 mg q12h in the treatment period for 28 days.
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Tablet for oral administration.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels
Time Frame: From Baseline at Day 28
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From Baseline at Day 28
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Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: Day 1 up to Week 8
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Day 1 up to Week 8
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Secondary Outcome Measures
Outcome Measure |
Time Frame |
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Change in Plasma Antigenic AAT Levels
Time Frame: From Baseline at Day 28
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From Baseline at Day 28
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Observed Pre-dose Plasma Concentration of VX-814
Time Frame: Pre-dose at Day 7, Day 14, Day 21, and Day 28
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Pre-dose at Day 7, Day 14, Day 21, and Day 28
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
January 13, 2020
Primary Completion (Actual)
November 14, 2020
Study Completion (Actual)
November 14, 2020
Study Registration Dates
First Submitted
November 15, 2019
First Submitted That Met QC Criteria
November 15, 2019
First Posted (Actual)
November 18, 2019
Study Record Updates
Last Update Posted (Actual)
February 2, 2022
Last Update Submitted That Met QC Criteria
January 7, 2022
Last Verified
January 1, 2022
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- VX19-814-101
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
IPD Plan Description
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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