New pill for rare lung and liver disease passes first safety check
NCT ID NCT06738017
First seen Nov 01, 2025 · Last updated May 07, 2026 · Updated 25 times
Summary
This early-stage trial tests a single oral dose of BMN 349 in 6 adults with a genetic form of alpha-1 antitrypsin deficiency (PiZZ or PiMZ/MASH). The main goal is to see if the drug is safe and how the body processes it. Participants receive either BMN 349 or a placebo and are closely monitored for side effects and changes in lab tests, lung function, and heart activity.
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Medpace Clinical Pharmacology Unit
Cincinnati, Ohio, 45227, United States
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NHS Lothian
Edinburgh, UK, United Kingdom
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Royal Free London NHS Foundation Trust
London, UK, United Kingdom
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Saint Louis University
St Louis, Missouri, 63110, United States
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The Medical University of South Carolina
Charleston, South Carolina, 29425, United States
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University Hospital Southampton NHS Foundation Trust
Southampton, UK, United Kingdom
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University of California, San Diego
San Diego, California, 92037, United States
Conditions
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