Trial Profile

A Randomized, Double-Blind, Placebo-Controlled, Single Oral Dose Study Evaluating the Safety and Pharmacokinetics of BMN 349 in Homozygous for the Z Mutation of Alpha 1 Antitrypsin Gene (PiZZ) and Heterozygous for the Z Mutation (PiMZ/MASH)

Status: Recruiting

Phase of Trial: Phase I

Latest Information Update: 06 Jun 2025

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At a glance

19 Feb 2025 According to a BioMarin Pharmaceutical media release,the single-ascending dose (SAD) phase of the first-in-human study is completed and dosing in the multiple-ascending dose (MAD) phase of the study began in December 2024.
01 Nov 2023 According to a BioMarin Pharmaceutical media release, the company plans to initiate a global clinical program with BMN 349 in 2024 and expects to have a determination of clinical proof of concept in 2025.
08 Sep 2023 New trial record

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