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BMN 349 for Alpha 1-Antitrypsin Deficiency

This study is testing the safety of a single dose of BMN 349, an oral tablet, in adults with Alpha 1-Antitrypsin Deficiency (PiZZ or PiMZ/MASH types). You could be eligible if you are between 18 and 75 years old, a non-smoker, and have a confirmed PiZZ or PiMZ genotype. The main goal is to see if BMN 349 causes any side effects or abnormal lab or lung function tests over 78 days. Participants will receive either BMN 349 or a placebo (an inactive tablet) and be monitored closely. The study plans to enroll 6 participants, but its current recruitment status is unclear.

Study design
This is an interventional study where participants will receive either BMN 349 or a placebo. The study aims to enroll 6 participants.
What's involved
Participants will receive a single dose of either BMN 349 or placebo. They will then be monitored for safety and tolerability.
Compensation
Not stated in the trial record.
Follow-up
Participants will be monitored for safety and tolerability for 78 days after receiving the single dose.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06738017

Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants

Active, Not Recruiting
PHASE1Ages 18–75InterventionalTreatment
BioMarin Pharmaceutical
~6 participants
Updated 2026-05-06 on ClinicalTrials.gov
What's tested:BMN 349Placebo

At a glance

Recruiting sites
0 of 7 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Participant Adverse Events, Serious Adverse Events, Dose Limit Toxicities, Adverse Event of Special Interests, abnormal laboratory tests, abnormal pulmonary function tests, and 12-lead ECG parameters
Measured over 78 days

Conditions

Alpha 1-Antitrypsin Deficiency

Explore related trials

Alpha-1 Antitrypsin DeficiencyNonalcoholic SteatohepatitisAspartate Aminotransferase

Where it's being run

7 sites across 5 states
UK3
California1
Missouri1
Ohio1
South Carolina1

Study leadership

  • Medical Director, MD · STUDY_DIRECTOR · BioMarin Pharmaceutical

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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

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

What this trial measures

  • Participant Adverse Events, Serious Adverse Events, Dose Limit Toxicities, Adverse Event of Special Interests, abnormal laboratory tests, abnormal pulmonary function tests, and 12-lead ECG parameters78 days

    Number of participant AEs, SAEs, DLTs, AESIs per physician's assessment, abnormal laboratory tests through whole blood samples, abnormal pulmonary function spirometry tests, and 12-lead ECG parameters changes from baseline following a single oral dose of BMN 349