NCT04167345

Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype

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

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 48 (actual)
• Sponsor: Vertex Pharmaceuticals Incorporated · Industry
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

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

Conditions

• Alpha 1-Antitrypsin Deficiency

Interventions

Type	Name	Description
DRUG	VX-814	Tablet for oral administration.
DRUG	Placebo	Placebo matched to VX-814 for oral administration.

Timeline

• Start date: 2020-01-13
• Primary completion: 2020-11-14
• Completion: 2020-11-14
• First posted: 2019-11-18
• Last updated: 2022-02-02
• Results posted: 2022-02-02

Locations

28 sites across 4 countries: United States, Canada, Germany, Ireland

Regulatory

• FDA-regulated drug study