Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype

Phase 2TerminatedNCT04167345

Vertex Pharmaceuticals Incorporated48 enrolled

Overview

This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-814 in PiZZ subjects.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Alpha 1-Antitrypsin Deficiency

Interventions

VX-814DRUG

Tablet for oral administration.

PlaceboDRUG

Placebo matched to VX-814 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 (28)

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 Treatment Emergent 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 of VX-814

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

Data from ClinicalTrials.gov

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