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

Alpha-1 Antitrypsin Deficiency Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05643495
• Other study ID #: VX22-864-108
• Secondary ID: 2022-002746-40
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: February 23, 2023
• Est. completion date: December 2024

Study information

• Verified date: October 2023
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 20
• Est. completion date: December 2024
• Est. primary completion date: November 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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.

Related Conditions & MeSH terms

• Alpha 1-Antitrypsin Deficiency
• Alpha-1 Antitrypsin Deficiency

Intervention

Drug:
• VX-864
Tablets for oral administration.

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

United States,  Germany,  Ireland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in Blood Functional Alpha-1 Antitrypsin (AAT) Levels		From Baseline at Week 48
Secondary	Change in Blood Functional AAT Levels		From Baseline up to Week 48
Secondary	Change in Blood Antigenic AAT Levels		From Baseline up to Week 48
Secondary	Change in Blood Z-polymer Levels		From Baseline up to Week 48
Secondary	Part B: Change in Z-polymer Accumulation in the Liver		From Baseline up to Week 48
Secondary	Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)		Day 1 up to Week 52