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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Gene-Editing shot aims to fix lung and liver damage in rare disease
- New drug AIR-001 enters first human tests for rare lung condition