Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants
NCT ID: NCT06738017
Last Updated: 2025-09-08
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1
12 participants
INTERVENTIONAL
2025-02-21
2025-09-30
Brief Summary
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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.
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
TRIPLE
Study Groups
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Group A (PiZZ)
5:1 (349:Placebo)
BMN 349
250mg oral tablet
Placebo
250mg oral tablet
Group B (PiMZ)
5:1 (349:Placebo)
BMN 349
250mg oral tablet
Placebo
250mg oral tablet
Interventions
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BMN 349
250mg oral tablet
Placebo
250mg oral tablet
Eligibility Criteria
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Inclusion Criteria
* 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
Exclusion Criteria
* 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
18 Years
75 Years
ALL
No
Sponsors
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BioMarin Pharmaceutical
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director, MD
Role: STUDY_DIRECTOR
BioMarin Pharmaceutical
Locations
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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
Nottingham University Hospitals
Nottingham, UK, United Kingdom
University Hospital Southampton NHS Foundation Trust
Southampton, UK, United Kingdom
Countries
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Central Contacts
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Facility Contacts
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Role: primary
Other Identifiers
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349-102
Identifier Type: -
Identifier Source: org_study_id
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