Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06738017 | Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants | PHASE1 | ACTIVE NOT_RECRUITING | 6 | — | — | Feb 21, 2025 | Aug 19, 2026 | May 6, 2026 | 7 | United States, United Kingdom |
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
| Arm | Type | Description |
|---|---|---|
| Group A (PiZZ) | OTHER | 5:1 (349:Placebo) |
| Group B (PiMZ) | OTHER | 5:1 (349:Placebo) |
| Name | Type | Description |
|---|---|---|
| BMN 349 | DRUG | 250mg oral tablet |
| Placebo | DRUG | 250mg oral tablet |
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 normal...