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Clinical Trial Details — Status: Terminated

Administrative data

NCT number NCT04167345
Other study ID # VX19-814-101
Secondary ID
Status Terminated
Phase Phase 2
First received
Last updated
Start date January 13, 2020
Est. completion date November 14, 2020

Study information

Verified date January 2022
Source Vertex Pharmaceuticals Incorporated
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-814 in PiZZ subjects.


Recruitment information / eligibility

Status Terminated
Enrollment 48
Est. completion date November 14, 2020
Est. primary completion date November 14, 2020
Accepts healthy volunteers No
Gender All
Age group 18 Years to 80 Years
Eligibility 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 Design


Related Conditions & MeSH terms


Intervention

Drug:
VX-814
Tablet for oral administration.
Placebo
Placebo matched to VX-814 for oral administration.

Locations

Country Name City State
Canada Queen Elizabeth II Health Sciences Center Halifax
Canada Inspiration Research Ltd Toronto
Germany University Hospital RWTH Aachen Aachen
Germany Universitätsklinikum Essen Essen
Germany Medizinische Hochschule Hannover Hannover
Ireland Royal College of Surgeons in Ireland Clinical Research Centre, Beaumont Hospital Beaumont
United States University of Alabama at Birmingham Birmingham Alabama
United States University of North Carolina Medical Center Chapel Hill North Carolina
United States Medical University of South Carolina Charleston South Carolina
United States University of Cincinnati Cincinnati Ohio
United States Cleveland Clinic Foundation Cleveland Ohio
United States National Jewish Health Denver Colorado
United States Inova Fairfax Medical Campus Falls Church Virginia
United States University of Florida, Shands Hospital Gainesville Florida
United States Blessing Corporate Services, Inc., dba Blessing Health System Hannibal Missouri
United States Renovatio Clinical Houston Texas
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 Miami Miller School of Medicine Miami Florida
United States University of Minnesota Minneapolis Minnesota
United States Columbia University Medical Center New York New York
United States Central Florida Pulmonary Group, PA Orlando Florida
United States Temple University Hospital Philadelphia Pennsylvania
United States Oregon Health & Science University Portland Oregon
United States University of California Davis Medical Center Sacramento California
United States University of Utah Health Salt Lake City Utah
United States The University of Texas Health Science Center at Tyler Tyler Texas
United States Wake Forest University Baptist Medical Center Winston-Salem North Carolina

Sponsors (1)

Lead Sponsor Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

United States,  Canada,  Germany,  Ireland, 

Outcome

Type Measure Description Time frame Safety issue
Primary Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels From Baseline at Day 28
Primary Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) Day 1 up to Week 8
Secondary Change in Plasma Antigenic AAT Levels From Baseline at Day 28
Secondary Observed Pre-dose Plasma Concentration of VX-814 Pre-dose at Day 7, Day 14, Day 21, and Day 28
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