Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants
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Sponsored by BioMarin Pharmaceutical (industry) · BMRN — their whole pipeline →.
Phase
Phase 1
Status
Active, not recruiting
Study type
Interventional
Design
Randomized · Triple · Treatment
Enrollment
6actual
Sites
7
Countries
United Kingdom, United States
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2025-02-21 | actual | When the study began enrolling |
| Primary completion | 2026-08-19 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2026-08-19 | estimated | The whole study's end, after follow-up |
| First posted | 2024-12-17 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-05-06 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Alpha 1-Antitrypsin Deficiency
Interventions
- Drug: BMN 349
- Drug: Placebo
Primary outcome
what the primary-completion date above is the date OFParticipant 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 78 days
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