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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
First received
Last updated
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
Health authority
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.

Study Design


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
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