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

Summary

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

Timeline

Start

2020-07-24

Primary completion

2021-05-04

Completion

2021-05-04

Outcome

Met primary endpoint

registry analysis (superiority test); Placebo vs VX-864 100 mg; p <0.0001; Least Squares (LS) Mean Difference 2.3 (95% CI 1.5 to 3.1) NCT04474197 ↗

registry analysis (superiority test); Placebo vs VX-864 300 mg; p <0.0001; LS Mean Difference 2.3 (95% CI 1.6 to 3.1) NCT04474197 ↗

release “The study met its primary endpoint, with all VX-864 dose groups demonstrating highly statistically significant increases in plasma fAAT levels” vrtx.com ↗

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	VX-864	Unknown	100 mg	Oral
Subject	VX-864	Unknown	300 mg	Oral
Subject	VX-864	Unknown	500 mg	Oral

Indications