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
Verified date | October 2023 |
Source | Vertex Pharmaceuticals Incorporated |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
The purpose of this study is to evaluate the efficacy and safety of VX-864 in participants with the PiZZ genotype over 48 weeks.
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. |
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 |
Lead Sponsor | Collaborator |
---|---|
Vertex Pharmaceuticals Incorporated |
United States, Germany, Ireland, United Kingdom,
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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