New pill for rare lung and liver disease enters first human tests
NCT ID NCT06738017
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
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 check safety and how the drug moves through the body. Participants receive either BMN 349 or a placebo and are closely monitored for side effects.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
BMN 349 (oral tablet)
What this could lead to
If successful, this could point toward a new treatment option for alpha-1 antitrypsin deficiency, potentially slowing liver and lung damage.
What could go wrong
This is a very early Phase 1 trial with only 6 participants, so safety and dosing are still being explored. It may not lead to an effective treatment, and side effects are unknown.
Disclaimer
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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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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