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A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype

Primary Purpose

Alpha-1 Antitrypsin Deficiency

Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
VX-864
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: Participants 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 Ribonucleic acid interference (RNAi) therapy at any time previously Participants for whom discontinuation of augmentation therapy is not considered to be in their best interest, based on the clinical judgement of the treating physician Other protocol defined Inclusion/Exclusion criteria may apply.

Sites / Locations

  • University of FloridaRecruiting
  • Central Florida Pulmonary Group, P.A.
  • The University of Iowa Hospitals and Clinics: Adult Pulmonary ClinicRecruiting
  • University of Kansas Medical CenterRecruiting
  • Hannibal Regional Healthcare SystemRecruiting
  • Columbia University Irving Medical Center
  • Marsico Clinical Research Center at UNC Pulmonary Clinic
  • Renovatio ClinicalRecruiting
  • University of Utah HealthRecruiting
  • Inova Fairfax Medical CampusRecruiting
  • University Hospital RWTH AachenRecruiting
  • Royal College of Surgeons in Ireland Clinical Research Centre, Beaumont HospitalRecruiting
  • King's College HospitalRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Group A

Group B

Arm Description

Participants will receive VX-864 every 12 hours (q12h) for 48 weeks.

Participants will undergo a liver biopsy done before receiving VX-864 q12h for 48 weeks, and will undergo a second liver biopsy at either Week 24 or Week 48.

Outcomes

Primary Outcome Measures

Change in Blood Functional Alpha-1 Antitrypsin (AAT) Levels

Secondary Outcome Measures

Change in Blood Functional AAT Levels
Change in Blood Antigenic AAT Levels
Change in Blood Z-polymer Levels
Part B: Change in Z-polymer Accumulation in the Liver
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

Full Information

First Posted
November 29, 2022
Last Updated
September 11, 2023
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT05643495
Brief Title
A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype
Official Title
A Phase 2, Open-label Study Evaluating Efficacy and Safety of VX-864 in Subjects With Alpha-1 Antitrypsin Deficiency Who Have the PiZZ Genotype, Over 48 Weeks
Study Type
Interventional

2. Study Status

Record Verification Date
November 2022
Overall Recruitment Status
Recruiting
Study Start Date
February 23, 2023 (Actual)
Primary Completion Date
November 2024 (Anticipated)
Study Completion Date
December 2024 (Anticipated)

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
The purpose of this study is to evaluate the efficacy and safety of VX-864 in participants with the PiZZ genotype over 48 weeks.

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
None (Open Label)
Allocation
Non-Randomized
Enrollment
20 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Group A
Arm Type
Experimental
Arm Description
Participants will receive VX-864 every 12 hours (q12h) for 48 weeks.
Arm Title
Group B
Arm Type
Experimental
Arm Description
Participants will undergo a liver biopsy done before receiving VX-864 q12h for 48 weeks, and will undergo a second liver biopsy at either Week 24 or Week 48.
Intervention Type
Drug
Intervention Name(s)
VX-864
Intervention Description
Tablets for oral administration.
Primary Outcome Measure Information:
Title
Change in Blood Functional Alpha-1 Antitrypsin (AAT) Levels
Time Frame
From Baseline at Week 48
Secondary Outcome Measure Information:
Title
Change in Blood Functional AAT Levels
Time Frame
From Baseline up to Week 48
Title
Change in Blood Antigenic AAT Levels
Time Frame
From Baseline up to Week 48
Title
Change in Blood Z-polymer Levels
Time Frame
From Baseline up to Week 48
Title
Part B: Change in Z-polymer Accumulation in the Liver
Time Frame
From Baseline up to Week 48
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 52

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: Participants 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 Ribonucleic acid interference (RNAi) therapy at any time previously Participants for whom discontinuation of augmentation therapy is not considered to be in their best interest, based on the clinical judgement of the treating physician Other protocol defined Inclusion/Exclusion criteria may apply.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Medical Information
Phone
617-341-6777
Email
medicalinfo@vrtx.com
Facility Information:
Facility Name
University of Florida
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Individual Site Status
Recruiting
Facility Name
Central Florida Pulmonary Group, P.A.
City
Orlando
State/Province
Florida
ZIP/Postal Code
32803
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
The University of Iowa Hospitals and Clinics: Adult Pulmonary Clinic
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Individual Site Status
Recruiting
Facility Name
Hannibal Regional Healthcare System
City
Hannibal
State/Province
Missouri
ZIP/Postal Code
63401
Country
United States
Individual Site Status
Recruiting
Facility Name
Columbia University Irving Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Marsico Clinical Research Center at UNC Pulmonary Clinic
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27517
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Renovatio Clinical
City
Houston
State/Province
Texas
ZIP/Postal Code
77380
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Utah Health
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84108
Country
United States
Individual Site Status
Recruiting
Facility Name
Inova Fairfax Medical Campus
City
Falls Church
State/Province
Virginia
ZIP/Postal Code
22042
Country
United States
Individual Site Status
Recruiting
Facility Name
University Hospital RWTH Aachen
City
Aachen
Country
Germany
Individual Site Status
Recruiting
Facility Name
Royal College of Surgeons in Ireland Clinical Research Centre, Beaumont Hospital
City
Beaumont
Country
Ireland
Individual Site Status
Recruiting
Facility Name
King's College Hospital
City
London
Country
United Kingdom
Individual Site Status
Recruiting

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

A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype

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