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.
Alpha 1-Antitrypsin Deficiency
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.
Study of BMN 349 Single Dose in (PiZZ) and (PiMZ) Adult Participants
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University of California, San Diego, San Diego, California, United States, 92037
Saint Louis University, Saint Louis, Missouri, United States, 63110
Medpace Clinical Pharmacology Unit, Cincinnati, Ohio, United States, 45227
The Medical University of South Carolina, Charleston, South Carolina, United States, 29425
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
For general information about clinical research, read Learn About Studies.
18 Years to 64 Years
ALL
No
BioMarin Pharmaceutical,
Medical Director, MD, STUDY_DIRECTOR, BioMarin Pharmaceutical
2025-08