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

Purpose

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

Condition

  • Alpha1-Antitrypsin Deficiency

Eligibility

Eligible Ages
Between 18 Years and 80 Years
Eligible 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

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Placebo Comparator
Placebo 		Participants received placebo matched to VX-864 in the treatment period for 28 days.
  • Drug: Placebo
    Placebo matched to VX-864 for oral administration.
Experimental
VX-864 100 mg 		Participants received VX-864 100 milligrams (mg) every 12 hours (q12h) in the treatment period for 28 days.
  • Drug: VX-864
    Tablets for oral administration.
Experimental
VX-864 300 mg 		Participants received VX-864 300 mg q12h in the treatment period for 28 days.
  • Drug: VX-864
    Tablets for oral administration.
Experimental
VX-864 500 mg 		Participants received VX-864 500 mg q12h in the treatment period for 28 days.
  • Drug: VX-864
    Tablets for oral administration.

More Details

Status
Completed
Sponsor
Vertex Pharmaceuticals Incorporated

Study Contact