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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05643495
Recruitment Status : Active, not recruiting
First Posted : December 8, 2022
Last Update Posted : April 24, 2024
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Description
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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.

Condition or disease Intervention/treatment Phase
Alpha-1 Antitrypsin Deficiency Drug: VX-864 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : February 23, 2023
Estimated Primary Completion Date : November 2024
Estimated Study Completion Date : December 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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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.


Outcome Measures
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Primary Outcome Measures :
  1. Change in Blood Functional Alpha-1 Antitrypsin (AAT) Levels [ Time Frame: From Baseline at Week 48 ]

Secondary Outcome Measures :
  1. Change in Blood Functional AAT Levels [ Time Frame: From Baseline up to Week 48 ]
  2. Change in Blood Antigenic AAT Levels [ Time Frame: From Baseline up to Week 48 ]
  3. Change in Blood Z-polymer Levels [ Time Frame: From Baseline up to Week 48 ]
  4. Part B: Change in Z-polymer Accumulation in the Liver [ Time Frame: From Baseline up to Week 48 ]
  5. 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 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05643495


Locations
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United States, Florida
University of Florida
Gainesville, Florida, United States, 32610
Central Florida Pulmonary Group, P.A.
Orlando, Florida, United States, 32803
United States, Iowa
The University of Iowa Hospitals and Clinics: Adult Pulmonary Clinic
Iowa City, Iowa, United States, 52242
United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
United States, Missouri
Hannibal Regional Healthcare System
Hannibal, Missouri, United States, 63401
United States, New York
Columbia University Irving Medical Center
New York, New York, United States, 10032
United States, North Carolina
Marsico Clinical Research Center at UNC Pulmonary Clinic
Chapel Hill, North Carolina, United States, 27517
United States, Texas
Renovatio Clinical
Houston, Texas, United States, 77380
United States, Utah
University of Utah Health
Salt Lake City, Utah, United States, 84108
United States, Virginia
Inova Fairfax Medical Campus
Falls Church, Virginia, United States, 22042
Germany
University Hospital RWTH Aachen
Aachen, Germany
Ireland
Royal College of Surgeons in Ireland Clinical Research Centre, Beaumont Hospital
Beaumont, Ireland
United Kingdom
King's College Hospital
London, United Kingdom
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
More Information
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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT05643495    
Other Study ID Numbers: VX22-864-108
2022-002746-40 ( EudraCT Number )
First Posted: December 8, 2022    Key Record Dates
Last Update Posted: April 24, 2024
Last Verified: October 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Alpha 1-Antitrypsin Deficiency
Liver Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
 Genetic Diseases, Inborn
Subcutaneous Emphysema
Emphysema
Pathologic Processes