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

April 23, 2024 updated by: Vertex Pharmaceuticals Incorporated

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

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

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

20

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Germany
      • Aachen, Germany
        • University Hospital RWTH Aachen
  • Ireland
      • Beaumont, Ireland
        • Royal College of Surgeons in Ireland Clinical Research Centre, Beaumont Hospital
  • United Kingdom
      • London, United Kingdom
        • King's College Hospital
  • United States
    • Florida
      • Gainesville, Florida, United States, 32610
        • University of Florida
      • Orlando, Florida, United States, 32803
        • Central Florida Pulmonary Group, P.A.
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • The University of Iowa Hospitals and Clinics: Adult Pulmonary Clinic
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center
    • Missouri
      • Hannibal, Missouri, United States, 63401
        • Hannibal Regional Healthcare System
    • New York
      • New York, New York, United States, 10032
        • Columbia University Irving Medical Center
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27517
        • Marsico Clinical Research Center at UNC Pulmonary Clinic
    • Texas
      • Houston, Texas, United States, 77380
        • Renovatio Clinical
    • Utah
      • Salt Lake City, Utah, United States, 84108
        • University of Utah Health
    • Virginia
      • Falls Church, Virginia, United States, 22042
        • Inova Fairfax Medical Campus

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 80 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Group A
Participants will receive VX-864 every 12 hours (q12h) for 48 weeks.
Drug: VX-864
Tablets for oral administration.
Experimental: Group B
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.
Drug: VX-864
Tablets for oral administration.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Change in Blood Functional Alpha-1 Antitrypsin (AAT) Levels
Time Frame: From Baseline at Week 48
From Baseline at Week 48

Secondary Outcome Measures

Outcome Measure
Time Frame
Change in Blood Functional AAT Levels
Time Frame: From Baseline up to Week 48
From Baseline up to Week 48
Change in Blood Antigenic AAT Levels
Time Frame: From Baseline up to Week 48
From Baseline up to Week 48
Change in Blood Z-polymer Levels
Time Frame: From Baseline up to Week 48
From Baseline up to Week 48
Part B: Change in Z-polymer Accumulation in the Liver
Time Frame: From Baseline up to Week 48
From Baseline up to Week 48
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
Day 1 up to Week 52

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vertex Pharmaceuticals Incorporated

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 23, 2023

Primary Completion (Estimated)

November 1, 2024

Study Completion (Estimated)

December 1, 2024

Study Registration Dates

First Submitted

November 29, 2022

First Submitted That Met QC Criteria

November 29, 2022

First Posted (Actual)

December 8, 2022

Study Record Updates

Last Update Posted (Actual)

April 24, 2024

Last Update Submitted That Met QC Criteria

April 23, 2024

Last Verified

October 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX22-864-108
  • 2022-002746-40 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD 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

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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