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

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ClinicalTrials.gov Identifier: NCT05643495
Recruitment Status : Recruiting
First Posted : December 8, 2022
Last Update Posted : December 23, 2022
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

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

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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
Estimated Study Start Date : December 2022
Estimated Primary Completion Date : November 2024
Estimated Study Completion Date : December 2024

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.

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 ]

Information from the National Library of Medicine

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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

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.

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

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Contact: Medical Information 617-341-6777 medicalinfo@vrtx.com

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United States, Missouri
Hannibal Regional Healthcare System Recruiting
Hannibal, Missouri, United States, 63401
United States, Texas
Renovatio Clinical Recruiting
Houston, Texas, United States, 77380
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
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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: December 23, 2022
Last Verified: November 2022
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
Pathologic Processes