NCT06738017

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

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: Phase 1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Alpha 1-Antitrypsin Deficiency

Interventions

Type	Name	Description
DRUG	BMN 349	250mg oral tablet
DRUG	Placebo	250mg oral tablet

Timeline

• Start date: 2025-02-21
• Primary completion: 2025-09-01
• Completion: 2025-09-01
• First posted: 2024-12-17
• Last updated: 2025-09-08

Locations

8 sites across 2 countries: United States, United Kingdom

Regulatory

• FDA-regulated drug study