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

Primary Purpose

Alpha 1-Antitrypsin Deficiency

Status
Terminated
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
VX-814
Placebo
Sponsored by
Vertex Pharmaceuticals Incorporated
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Alpha 1-Antitrypsin Deficiency

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • University of Alabama at Birmingham
  • University of California Davis Medical Center
  • National Jewish Health
  • University of Florida, Shands Hospital
  • University of Miami Miller School of Medicine
  • Central Florida Pulmonary Group, PA
  • The University of Iowa Hospitals and Clinics
  • University of Kansas Medical Center
  • University of Minnesota
  • Blessing Corporate Services, Inc., dba Blessing Health System
  • Columbia University Medical Center
  • University of North Carolina Medical Center
  • Wake Forest University Baptist Medical Center
  • University of Cincinnati
  • Cleveland Clinic Foundation
  • Oregon Health & Science University
  • Temple University Hospital
  • Medical University of South Carolina
  • Renovatio Clinical
  • The University of Texas Health Science Center at Tyler
  • University of Utah Health
  • Inova Fairfax Medical Campus
  • Queen Elizabeth II Health Sciences Center
  • Inspiration Research Ltd
  • University Hospital RWTH Aachen
  • Universitätsklinikum Essen
  • Medizinische Hochschule Hannover
  • Royal College of Surgeons in Ireland Clinical Research Centre, Beaumont Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm Type

Placebo Comparator

Experimental

Experimental

Experimental

Experimental

Arm Label

Parts A1, A2 and B Combined: Placebo

Part A1: VX-814 100 milligrams (mg)

Part A1: VX-814 200 mg

Parts A1 and A2 Combined: VX-814 400 mg

Part B: VX-814 600 mg

Arm Description

Participants received placebo matched to VX-814 in the treatment period for 28 days.

Participants received VX-814 100 mg every 12 hours (q12h) in the treatment period for 28 days.

Participants received VX-814 200 mg q12h in the treatment period for 28 days.

Participants received VX-814 400 mg q12h in the treatment period for 28 days.

Participants received VX-814 600 mg q12h in the treatment period for 28 days.

Outcomes

Primary Outcome Measures

Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels
Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

Secondary Outcome Measures

Change in Plasma Antigenic AAT Levels
Observed Pre-dose Plasma Concentration of VX-814

Full Information

First Posted
November 15, 2019
Last Updated
January 7, 2022
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT04167345
Brief Title
Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype
Official Title
A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-814 in PiZZ Subjects
Study Type
Interventional

2. Study Status

Record Verification Date
January 2022
Overall Recruitment Status
Terminated
Why Stopped
at Sponsor's discretion
Study Start Date
January 13, 2020 (Actual)
Primary Completion Date
November 14, 2020 (Actual)
Study Completion Date
November 14, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Alpha 1-Antitrypsin Deficiency

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
48 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Parts A1, A2 and B Combined: Placebo
Arm Type
Placebo Comparator
Arm Description
Participants received placebo matched to VX-814 in the treatment period for 28 days.
Arm Title
Part A1: VX-814 100 milligrams (mg)
Arm Type
Experimental
Arm Description
Participants received VX-814 100 mg every 12 hours (q12h) in the treatment period for 28 days.
Arm Title
Part A1: VX-814 200 mg
Arm Type
Experimental
Arm Description
Participants received VX-814 200 mg q12h in the treatment period for 28 days.
Arm Title
Parts A1 and A2 Combined: VX-814 400 mg
Arm Type
Experimental
Arm Description
Participants received VX-814 400 mg q12h in the treatment period for 28 days.
Arm Title
Part B: VX-814 600 mg
Arm Type
Experimental
Arm Description
Participants received VX-814 600 mg q12h in the treatment period for 28 days.
Intervention Type
Drug
Intervention Name(s)
VX-814
Intervention Description
Tablet for oral administration.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo matched to VX-814 for oral administration.
Primary Outcome Measure Information:
Title
Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels
Time Frame
From Baseline at Day 28
Title
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
Secondary Outcome Measure Information:
Title
Change in Plasma Antigenic AAT Levels
Time Frame
From Baseline at Day 28
Title
Observed Pre-dose Plasma Concentration of VX-814
Time Frame
Pre-dose at Day 7, Day 14, Day 21, and Day 28

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
Facility Name
University of Alabama at Birmingham
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Facility Name
University of California Davis Medical Center
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
National Jewish Health
City
Denver
State/Province
Colorado
ZIP/Postal Code
80206
Country
United States
Facility Name
University of Florida, Shands Hospital
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Facility Name
University of Miami Miller School of Medicine
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Central Florida Pulmonary Group, PA
City
Orlando
State/Province
Florida
ZIP/Postal Code
32803
Country
United States
Facility Name
The University of Iowa Hospitals and Clinics
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Facility Name
Blessing Corporate Services, Inc., dba Blessing Health System
City
Hannibal
State/Province
Missouri
ZIP/Postal Code
63401
Country
United States
Facility Name
Columbia University Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
University of North Carolina Medical Center
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27514
Country
United States
Facility Name
Wake Forest University Baptist Medical Center
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27157
Country
United States
Facility Name
University of Cincinnati
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45267
Country
United States
Facility Name
Cleveland Clinic Foundation
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
Oregon Health & Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Temple University Hospital
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19140
Country
United States
Facility Name
Medical University of South Carolina
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Facility Name
Renovatio Clinical
City
Houston
State/Province
Texas
ZIP/Postal Code
77380
Country
United States
Facility Name
The University of Texas Health Science Center at Tyler
City
Tyler
State/Province
Texas
ZIP/Postal Code
75708
Country
United States
Facility Name
University of Utah Health
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84108
Country
United States
Facility Name
Inova Fairfax Medical Campus
City
Falls Church
State/Province
Virginia
ZIP/Postal Code
22042
Country
United States
Facility Name
Queen Elizabeth II Health Sciences Center
City
Halifax
Country
Canada
Facility Name
Inspiration Research Ltd
City
Toronto
Country
Canada
Facility Name
University Hospital RWTH Aachen
City
Aachen
Country
Germany
Facility Name
Universitätsklinikum Essen
City
Essen
Country
Germany
Facility Name
Medizinische Hochschule Hannover
City
Hannover
Country
Germany
Facility Name
Royal College of Surgeons in Ireland Clinical Research Centre, Beaumont Hospital
City
Beaumont
Country
Ireland

12. IPD Sharing Statement

Plan to Share IPD
No
IPD Sharing 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

Learn more about this trial

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

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