University of Kansas Medical Center

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.	Drug: Placebo Placebo matched to VX-814 for oral administration.
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.	Drug: VX-814 Tablet for oral administration.
Experimental Part A1: VX-814 200 mg	Participants received VX-814 200 mg q12h in the treatment period for 28 days.	Drug: VX-814 Tablet for oral administration.
Experimental Parts A1 and A2 Combined: VX-814 400 mg	Participants received VX-814 400 mg q12h in the treatment period for 28 days.	Drug: VX-814 Tablet for oral administration.
Experimental Part B: VX-814 600 mg	Participants received VX-814 600 mg q12h in the treatment period for 28 days.	Drug: VX-814 Tablet for oral administration.

More Details

Status: Terminated
Sponsor: Vertex Pharmaceuticals Incorporated

Study Contact

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.