Clinical Trial

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

Active, Not Recruiting Phase 1
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Summary
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.
Protocol Amendment History 9 amendments
This ClinicalTrials.gov record has been amended 9 times since 2024-12-12; most recent amendment 2026-05-04.
Status change: Recruiting → Active, Not Recruiting 2026-05-04
Trial Details
NCT Number NCT06738017
Lead Sponsor BioMarin Pharmaceutical
Conditions Alpha 1-Antitrypsin Deficiency
Enrollment 6 participants
Start Date 2025-02-21
Primary Completion 2026-08-19 (estimated)
Study Completion 2026-08-19 (estimated)
Updated on ClinicalTrials.gov 2026-05-06