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.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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6 people
The number who actually took part.
- Started
-
Feb 2025
- Expected to finish
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Aug 2026
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 75 years
- Sex
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Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Participants must have confirmation of PiZZ or PiMZ genotype * 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: * International normalized ratio (INR) \> 1.2 * 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
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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
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Other studies related to the condition(s) this trial covers.
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