Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype
Purpose
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-814 in PiZZ subjects.
Condition
- Alpha 1-Antitrypsin Deficiency
Eligibility
- Eligible Ages
- Between 18 Years and 80 Years
- Eligible Genders
- 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
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Placebo Comparator Parts A1, A2 and B Combined: Placebo |
Participants received placebo matched to VX-814 in the treatment period for 28 days. |
|
|
Experimental Part A1: VX-814 100 milligrams (mg) |
Participants received VX-814 100 mg every 12 hours (q12h) in the treatment period for 28 days. |
|
|
Experimental Part A1: VX-814 200 mg |
Participants received VX-814 200 mg q12h in the treatment period for 28 days. |
|
|
Experimental Parts A1 and A2 Combined: VX-814 400 mg |
Participants received VX-814 400 mg q12h in the treatment period for 28 days. |
|
|
Experimental Part B: VX-814 600 mg |
Participants received VX-814 600 mg q12h in the treatment period for 28 days. |
|
More Details
- Status
- Terminated
- Sponsor
- Vertex Pharmaceuticals Incorporated