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

Summary

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

Timeline

Start

2020-01-13

Primary completion

2020-11-14

Completion

2020-11-14

Outcome

Met primary endpoint

Stopped: “at Sponsor's discretion”

registry Placebo vs Parts A1 and A2 Combined: VX-814 400 mg; p <.0001; Mean Difference 1.7 (95% CI 1.1 to 2.3) reads as met primary endpoint NCT04167345 ↗

registry Placebo vs Part B: VX-814 600 mg; p <.0001; Mean Difference 2.0 (95% CI 1.1 to 2.9) reads as met primary endpoint NCT04167345 ↗

release “Phase 2 study of VX-814 in patients with alpha-1 antitrypsin deficiency discontinued based upon safety and pharmacokinetic data” reads as unknown vrtx.com ↗

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	VX-814	Unknown	100 mg	Oral
Subject	VX-814	Unknown	200 mg	Oral
Subject	VX-814	Unknown	400 mg	Oral
Subject	VX-814	Unknown	600 mg	Oral

Indications