Clinical trial NCT05643495: A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Alpha-1 Antitrypsin Deficiency
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
First posted: 2022-12-08
Enrollment: 14
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility 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.

Primary outcomes:
measure: Change in Blood Functional Alpha-1 Antitrypsin (AAT) Levels
timeFrame: From Baseline at Week 48
Secondary outcomes:
measure: Change in Blood Functional AAT Levels
timeFrame: From Baseline up to Week 48
measure: Change in Blood Antigenic AAT Levels
timeFrame: From Baseline up to Week 48
measure: Change in Blood Z-polymer Levels
timeFrame: From Baseline up to Week 48
measure: Part B: Change in Z-polymer Accumulation in the Liver
timeFrame: From Baseline up to Week 48
measure: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Day 1 up to Week 52
Other outcomes:
Locations:
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Orlando
country: United States
facility: Central Florida Pulmonary Group, P.A.
state: Florida
city: Iowa City
country: United States
facility: The University of Iowa Hospitals and Clinics: Adult Pulmonary Clinic
state: Iowa
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Hannibal
country: United States
facility: Hannibal Regional Healthcare System
state: Missouri
city: New York
country: United States
facility: Columbia University Irving Medical Center
state: New York
city: Chapel Hill
country: United States
facility: Marsico Clinical Research Center at UNC Pulmonary Clinic
state: North Carolina
city: Houston
country: United States
facility: Renovatio Clinical
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah Health
state: Utah
city: Falls Church
country: United States
facility: Inova Fairfax Medical Campus
state: Virginia
city: Aachen
country: Germany
facility: University Hospital RWTH Aachen
city: Beaumont
country: Ireland
facility: Royal College of Surgeons in Ireland Clinical Research Centre, Beaumont Hospital
city: London
country: United Kingdom
facility: King's College Hospital
Study officials:
References: