A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-814 in PiZZ Subjects
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
Participants received placebo matched to VX-814 in the treatment period for 28 days.
Intervention: Placebo (Drug)
Part A1: VX-814 100 milligrams (mg)
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
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
Participants received VX-814 400 mg q12h in the treatment period for 28 days.
Intervention: VX-814 (Drug)
Part B: VX-814 600 mg
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)