Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants

Running, not enrolling · Phase 1 · Has a placebo group

Conditions studied: Alpha 1-Antitrypsin Deficiency

In brief

The goal of this clinical trial is to assess the safety and tolerability of a single oral dose of BMN 349 in participants with PiZZ or PiMZ/MASH. Primary outcome measures include incidence of any adverse events (including serious adverse events, dose limit toxicities, and adverse events of special interest), incidence of any laboratory test abnormalities, incidence of lung function test abnormalities and 12-lead ECG parameters. Participants will receive a single dose of either BMN 349 or placebo and then monitored for safety and tolerability.

Key facts

Study ID

NCT06738017

Run by

BioMarin Pharmaceutical

People needed

6

Starts

2025-02-21

Expected to finish

2026-08-19

Last updated by the study team

2026-05-06

Who can join

Age: 18 and older, up to 75. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Participants must have confirmation of PiZZ or PiMZ genotype
• Females and males, of any race, 18 to 75 years of age
• Nonsmokers, defined as not using tobacco or nicotine-containing products for at least 6 months prior to Screening

You may not qualify if…

• International normalized ratio (INR) > 1.2
• Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels > 125 U/L
• Current or recent use of AAT augmentation therapy
• Participants with recent (last 3 months) diagnosis of pneumonia

Where it is running

• University of California, San Diego — San Diego, California, United States
• Saint Louis University — St Louis, Missouri, United States
• Medpace Clinical Pharmacology Unit — Cincinnati, Ohio, United States
• The Medical University of South Carolina — Charleston, South Carolina, United States
• NHS Lothian — Edinburgh, UK, United Kingdom
• Royal Free London NHS Foundation Trust — London, UK, United Kingdom
• University Hospital Southampton NHS Foundation Trust — Southampton, UK, United Kingdom

Full record on ClinicalTrials.gov

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