Table 2.
Overview of Treatment-Emergent Adverse Events by Treatment Phase
| Double-Blind Phase* | Open-Label Phase† | ||||
|---|---|---|---|---|---|
| LAI (n = 44) | Placebo (n = 45) | LAI‡ (n = 35) | Placebo‡ (n = 43) | ||
| Patients with TEAEs, n (%) | 41 (93.2) | 40 (88.9) | 31 (88.6) | 42 (97.7) | |
| Number of TEAEs, n | 240 | 140 | 107 | 160 | |
| Patients with TEAEs by maximum severity, n (%) | |||||
| Grade 1: mild | 12 (27.3) | 25 (55.6) | 16 (45.7) | 10 (23.3) | |
| Grade 2: moderate | 24 (54.5) | 10 (22.2) | 10 (28.6) | 24 (55.8) | |
| Grade 3: severe | 4 (9.1) | 5 (11.1) | 4 (11.4) | 8 (18.6) | |
| Grade 4: life-threatening or disabling | 0 | 0 | 0 | 0 | |
| Grade 5: death§ | 1 (2.3) | 0 | 1 (2.9) | 0 | |
| Patients with TEAEs by seriousness, n (%) | |||||
| Serious | 8 (18.2) | 4 (8.9) | 5 (14.3) | 5 (11.6) | |
| Not serious | 33 (75.0) | 36 (80.0) | 26 (74.3) | 37 (86.0) | |
| Relationship to study drug, n (%) | |||||
| Related | 32 (72.7) | 17 (37.8) | 17 (48.6) | 26 (60.5) | |
| Not related | 9 (20.5) | 23 (51.1) | 14 (40.0) | 16 (37.2) | |
| Patients with audiovestibular TEAEs (e.g., tinnitus and dizziness), n (%) | 5 (11.4) | 5 (11.1) | 2 (5.7) | 2 (4.7) | |
| Patients with renal TEAEs, n (%) | 1 (2.3) | 0 | 1 (2.9) | 0 | |
| Patients with TEAEs leading to study drug discontinuation, n (%) | 7 (15.9) | 0 | 6 (17.1) | 12 (27.9) | |
Definition of abbreviations: LAI = liposomal amikacin for inhalation; TEAEs = treatment-emergent adverse events.
*
TEAEs during the double-blind phase are those with onset from the date of first dose of double-blind study drug.
†
TEAEs during the open-label phase are those with onset from the date of first dose of open-label study drug.
‡
All patients in the open-label phase received LAI. “LAI” and “placebo” here refer to treatment assignment in the double-blind phase.
§
Deaths were caused by acute respiratory distress syndrome and urosepsis, and both TEAEs were unrelated to study drug.