Clinical trial NCT04474197: Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype
Official title: A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 in PiZZ Subjects
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Alpha1-Antitrypsin Deficiency
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
First posted: 2020-07-16
Enrollment: 44
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-864 in PiZZ subjects.


Eligibility criteria: Key Inclusion Criteria:

* Subjects 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 RNAi therapy at any time previously

Other protocol defined Inclusion/Exclusion criteria may apply

Primary outcomes:
measure: Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels
timeFrame: From Baseline at Day 28
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 8
Secondary outcomes:
measure: Change in Plasma Antigenic AAT Levels
timeFrame: From Baseline at Day 28
measure: Observed Pre-dose Plasma Concentration (Ctrough) of VX-864
timeFrame: Pre-dose at Day 7, Day 14, Day 21 and Day 28
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Los Angeles
country: United States
facility: David Geffen School of Medicine at UCLA
state: California
city: Sacramento
country: United States
facility: University of California Davis Medical Center
state: California
city: Denver
country: United States
facility: National Jewish Health
state: Colorado
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Miami
country: United States
facility: University of Miami Miller School of Medicine
state: Florida
city: Orlando
country: United States
facility: Central Florida Pulmonary
state: Florida
city: Iowa City
country: United States
facility: The University of Iowa Hospitals and Clinics
state: Iowa
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Hannibal
country: United States
facility: Blessing Corporate Services, Inc., dba Blessing Health System
state: Missouri
city: Saint Louis
country: United States
facility: Cardinal Glennon Children's Hospital - St. Louis University
state: Missouri
city: Chapel Hill
country: United States
facility: University of North Carolina Medical Center
state: North Carolina
city: Winston-Salem
country: United States
facility: Wake Forest University Baptist Medical Center
state: North Carolina
city: Cincinnati
country: United States
facility: University of Cincinnati
state: Ohio
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Philadelphia
country: United States
facility: Temple University Hospital
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Houston
country: United States
facility: Renovatio Clinical
state: Texas
city: Tyler
country: United States
facility: The University of Texas Health Science Center at Tyler
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: Halifax
country: Canada
facility: Queen Elizabeth II Health Sciences Center
city: Toronto
country: Canada
facility: Inspiration Research Ltd
city: Aachen
country: Germany
facility: University Hospital RWTH Aachen
city: Essen
country: Germany
facility: Universitätsklinikum Essen
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover
city: Beaumont
country: Ireland
facility: Royal College of Surgeons in Ireland Clinical Research Centre, Beaumont Hospital
city: Malmö
country: Sweden
facility: Skanes Universitetssjukhus, Malmo
city: Soham Ely
country: United Kingdom
facility: Staploe Medical Centre
Study officials:
References: