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Clinical Trials/NCT04167345
NCT04167345TerminatedPhase 2

A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-814 in PiZZ Subjects

Vertex Pharmaceuticals Incorporated28 sites in 4 countries48 target enrollmentStarted: January 13, 2020Last updated:
Conditions
Interventions
Drugs

Trial Snapshot

Phase
Phase 2
Status
Terminated
Enrollment
48
Locations
28
Primary Endpoint
Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels

Study Overview

Brief Summary

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

Study Design

Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Eligibility Criteria

Ages
18 Years to 80 Years (Adult, Older Adult)
Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Subjects must have a PiZZ genotype confirmed at screening
  • Plasma AAT levels indicating severe deficiency at screening

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

Arms & Interventions

Parts A1, A2 and B Combined: Placebo

Placebo Comparator

Participants received placebo matched to VX-814 in the treatment period for 28 days.

Intervention: Placebo (Drug)

Part A1: VX-814 100 milligrams (mg)

Experimental

Participants received VX-814 100 mg every 12 hours (q12h) in the treatment period for 28 days.

Intervention: VX-814 (Drug)

Part A1: VX-814 200 mg

Experimental

Participants received VX-814 200 mg q12h in the treatment period for 28 days.

Intervention: VX-814 (Drug)

Parts A1 and A2 Combined: VX-814 400 mg

Experimental

Participants received VX-814 400 mg q12h in the treatment period for 28 days.

Intervention: VX-814 (Drug)

Part B: VX-814 600 mg

Experimental

Participants received VX-814 600 mg q12h in the treatment period for 28 days.

Intervention: VX-814 (Drug)

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(From Baseline at Day 28)
  • Observed Pre-dose Plasma Concentration of VX-814(Pre-dose at Day 7, Day 14, Day 21, and Day 28)

Investigators

Sponsor Class
Industry
Responsible Party
Sponsor

Study Sites (28)

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