Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype

Alpha 1-Antitrypsin Deficiency Clinical Trial

Official title:

A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-814 in PiZZ Subjects

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT04167345
• Other study ID #: VX19-814-101
• Status: Terminated
• Phase: Phase 2
• 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
• 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

Related Conditions & MeSH terms

• Alpha 1-Antitrypsin Deficiency

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