Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants (NCT06738017) | Clinical Trial Compass
Active — Not RecruitingPhase 1
Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants
United States, United Kingdom6 participantsStarted 2025-02-21
Plain-language summary
The goal of this clinical trial is to assess the safety and tolerability of a single oral dose of BMN 349 in participants with PiZZ or PiMZ/MASH.
Primary outcome measures include incidence of any adverse events (including serious adverse events, dose limit toxicities, and adverse events of special interest), incidence of any laboratory test abnormalities, incidence of lung function test abnormalities and 12-lead ECG parameters.
Participants will receive a single dose of either BMN 349 or placebo and then monitored for safety and tolerability.
Who can participate
Age range
18 Years – 75 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
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