Trial Profile

A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 in PiZZ Subjects

Status: Completed

Phase of Trial: Phase II

Latest Information Update: 14 Sep 2023

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At a glance

Drugs VX 864 (Primary)
Indications Alpha 1-antitrypsin deficiency
Focus Adverse reactions; Proof of concept; Therapeutic Use
Sponsors Vertex Pharmaceuticals

Most Recent Events

15 Jun 2021 Status changed from active, no longer recruiting to completed.
10 Jun 2021 Results published in the Vertex Pharmaceuticals Media Release.
10 Jun 2021 Primary endpoint (Change in plasma functional alpha-1 antitrypsin (AAT) levels) has been met, according to a Vertex Pharmaceuticals media release.

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