Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype

Phase 2CompletedNCT04474197

Vertex Pharmaceuticals Incorporated44 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Alpha1-Antitrypsin Deficiency

Interventions

VX-864DRUG

Tablets for oral administration.

PlaceboDRUG

Placebo matched to VX-864 for oral administration.

Eligibility

Sex: ALLMin age: 18 YearsMax age: 80 Years

Medical Language ↔ Plain English

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

Locations (30)

Outcomes

Primary Outcomes

Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels

Time frame: From Baseline at Day 28

Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

Time frame: Day 1 up to Week 8

Secondary Outcomes

Change in Plasma Antigenic AAT Levels

Time frame: From Baseline at Day 28

Observed Pre-dose Plasma Concentration (Ctrough) of VX-864

Time frame: Pre-dose at Day 7, Day 14, Day 21 and Day 28

Data from ClinicalTrials.gov

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University of Alabama at Birmingham

Birmingham, Alabama, United States

David Geffen School of Medicine at UCLA

Los Angeles, California, United States

University of California Davis Medical Center

Sacramento, California, United States

National Jewish Health

Denver, Colorado, United States

University of Florida

Gainesville, Florida, United States

University of Miami Miller School of Medicine

Miami, Florida, United States

Central Florida Pulmonary

Orlando, Florida, United States

The University of Iowa Hospitals and Clinics

Iowa City, Iowa, United States

University of Kansas Medical Center

Kansas City, Kansas, United States

University of Minnesota

Minneapolis, Minnesota, United States

...and 20 more locations