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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06389877 | A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD) | PHASE1 | RECRUITING | 106 | — | — | Jun 19, 2024 | May 1, 2030 | Mar 20, 2026 | 11 | United States, Australia +4 |
Numbers and percentages of patients reporting a given AE
Absolute Levels of AAT over time
| Arm | Type | Description |
|---|---|---|
| BEAM-302 Drug Product | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| BEAM-302 | DRUG | BEAM-302 is a lipid nanoparticle (LNP)-based therapy for the treatment of patients with AATD |
Part A: Inclusion Criteria: * Males or females 18 - 70 years of age inclusive at the time of consent. * Diagnosis of AATD and homozygous for the PiZZ mutation (confirmed by genetic testing). * Blood total AAT level \<11 μM or equivalent protein in mg/dL. * Patients receiving augmentation therapy i...