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 Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
12
250mg oral tablet
250mg oral tablet
University of California, San Diego
San Diego, California, United States
RECRUITINGSaint Louis University
St Louis, Missouri, United States
RECRUITINGMedpace Clinical Pharmacology Unit
Cincinnati, Ohio, United States
RECRUITINGThe Medical University of South Carolina
Charleston, South Carolina, United States
RECRUITINGNHS Lothian
Edinburgh, UK, United Kingdom
RECRUITINGRoyal Free London NHS Foundation Trust
London, UK, United Kingdom
RECRUITINGNottingham University Hospitals
Nottingham, UK, United Kingdom
NOT_YET_RECRUITINGUniversity Hospital Southampton NHS Foundation Trust
Southampton, UK, United Kingdom
RECRUITINGParticipant Adverse Events, Serious Adverse Events, Dose Limit Toxicities, Adverse Event of Special Interests, abnormal laboratory tests, abnormal pulmonary function tests, and 12-lead ECG parameters
Number of participant AEs, SAEs, DLTs, AESIs per physician's assessment, abnormal laboratory tests through whole blood samples, abnormal pulmonary function spirometry tests, and 12-lead ECG parameters changes from baseline following a single oral dose of BMN 349
Time frame: 78 days
C max
Maximum observed plasma concentration
Time frame: 78 days
AUC 0-t
Area under the concentration-time curve from time 0 to the last measurable concentration
Time frame: 78 days
AUC 0-inf
Area under the concentration-time curve from time 0 to infinity
Time frame: 78 days
CL/F
Apparent total body clearance after oral dosing
Time frame: 78 days
T max
Time to reach maximum concentration
Time frame: 78 days
t 1/2
Terminal half-life in plasma
Time frame: 78 days
Vz/F
Apparent volume of distribution during terminal phase
Time frame: 78 days
Assess functional activity of circulating Alpha1 AntiTrypsin in participants
Changes in participant circulating AAT through whole blood samples following a single dose of BMN 349
Time frame: 78 days
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