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Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype

Primary Purpose

Alpha1-Antitrypsin Deficiency

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
VX-864
Placebo
Sponsored by
Vertex Pharmaceuticals Incorporated
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Alpha1-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
  • David Geffen School of Medicine at UCLA
  • University of California Davis Medical Center
  • National Jewish Health
  • University of Florida
  • University of Miami Miller School of Medicine
  • Central Florida Pulmonary
  • The University of Iowa Hospitals and Clinics
  • University of Kansas Medical Center
  • University of Minnesota
  • Blessing Corporate Services, Inc., dba Blessing Health System
  • Cardinal Glennon Children's Hospital - St. Louis University
  • University of North Carolina Medical Center
  • Wake Forest University Baptist Medical Center
  • University of Cincinnati
  • 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
  • Skanes Universitetssjukhus, Malmo
  • Staploe Medical Centre

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Placebo Comparator

Experimental

Experimental

Experimental

Arm Label

Placebo

VX-864 100 mg

VX-864 300 mg

VX-864 500 mg

Arm Description

Participants received placebo matched to VX-864 in the treatment period for 28 days.

Participants received VX-864 100 milligrams (mg) every 12 hours (q12h) in the treatment period for 28 days.

Participants received VX-864 300 mg q12h in the treatment period for 28 days.

Participants received VX-864 500 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 Adverse Events (AEs) and Serious Adverse Events (SAEs)

Secondary Outcome Measures

Change in Plasma Antigenic AAT Levels
Observed Pre-dose Plasma Concentration (Ctrough) of VX-864

Full Information

First Posted
July 13, 2020
Last Updated
May 3, 2022
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT04474197
Brief Title
Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype
Official Title
A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 in PiZZ Subjects
Study Type
Interventional

2. Study Status

Record Verification Date
May 2022
Overall Recruitment Status
Completed
Study Start Date
July 24, 2020 (Actual)
Primary Completion Date
May 4, 2021 (Actual)
Study Completion Date
May 4, 2021 (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-864 in PiZZ subjects.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Alpha1-Antitrypsin Deficiency

7. Study Design

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

8. Arms, Groups, and Interventions

Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Participants received placebo matched to VX-864 in the treatment period for 28 days.
Arm Title
VX-864 100 mg
Arm Type
Experimental
Arm Description
Participants received VX-864 100 milligrams (mg) every 12 hours (q12h) in the treatment period for 28 days.
Arm Title
VX-864 300 mg
Arm Type
Experimental
Arm Description
Participants received VX-864 300 mg q12h in the treatment period for 28 days.
Arm Title
VX-864 500 mg
Arm Type
Experimental
Arm Description
Participants received VX-864 500 mg q12h in the treatment period for 28 days.
Intervention Type
Drug
Intervention Name(s)
VX-864
Intervention Description
Tablets for oral administration.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo matched to VX-864 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 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 (Ctrough) of VX-864
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
David Geffen School of Medicine at UCLA
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
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
City
Gainesville
State/Province
Florida
ZIP/Postal Code
11735
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
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
Cardinal Glennon Children's Hospital - St. Louis University
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63104
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
45229
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
Facility Name
Skanes Universitetssjukhus, Malmo
City
Malmö
Country
Sweden
Facility Name
Staploe Medical Centre
City
Soham Ely
Country
United Kingdom

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-864 in Subjects With the PiZZ Genotype

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