Table 1. Demographics and Baseline Characteristics.
| Characteristic | No. (%) | ||
|---|---|---|---|
| Placebo (n = 15) | Zilucoplan | ||
| 0.1 mg/kg (n = 15) | 0.3 mg/kg (n = 14) | ||
| Age, mean (SD), y | 48.4 (15.7) | 45.5 (15.7) | 54.6 (15.5) |
| Male | 4 (26.7) | 7 (46.7) | 10 (71.4) |
| Weight, mean (SD), kg | 85.3 (21.44) | 93.7 (24.72) | 110.9 (30.79) |
| BMI, mean (SD) | 30.9 (7.39) | 32.8 (6.55) | 36.0 (8.24) |
| Race/ethnicity | |||
| White | 12 (80.0) | 13 (86.7) | 11 (78.6) |
| Asian | 1 (6.7) | 0 | 1 (7.1) |
| Black/African American | 2 (13.3) | 2 (13.3) | 2 (14.3) |
| MGFA class at screening | |||
| II | 7 (46.7) | 5 (33.3) | 5 (35.7) |
| III | 8 (53.3) | 10 (66.7) | 5 (35.7) |
| IV | 0 | 0 | 4 (28.6) |
| Age at disease onset, mean (SD), y | 40.3 (17.79) | 37.3 (16.04) | 46.9 (19.48) |
| Duration of disease, median (range), y | 6.3 (0.1-20.9) | 6.5 (1.6-24.1) | 5.3 (0.5-26.0) |
| Baseline score, mean (SD) | |||
| QMG | 18.7 (4.0) | 18.7 (4.0) | 19.1 (5.1) |
| MG-ADL | 8.8 (3.6) | 6.9 (3.3) | 7.6 (2.6) |
| MGC | 18.7 (5.7) | 14.5 (6.3) | 14.6 (6.3) |
| MG-QoL15r | 15.9 (7.4) | 19.1 (5.0) | 16.5 (7.3) |
| Prior MG therapies (standard of care) | |||
| Pyridostigmine | 14 (93.3) | 15 (100.0) | 14 (100.0) |
| Corticosteroids | 13 (86.7) | 13 (86.7) | 14 (100.0) |
| Immunosuppressants | 12 (80.0) | 12 (80.0) | 9 (64.3) |
| Prior IVIg | 9 (60.0) | 8 (53.3) | 10 (71.4) |
| Prior plasma exchange | 7 (46.7) | 9 (60.0) | 7 (50.0) |
| Diagnosis of thymoma | 4 (26.7) | 5 (33.3) | 4 (28.6) |
| Prior thymectomy | 5 (33.3) | 8 (53.3) | 7 (50.0) |
| Prior MG crisis requiring intubation | 3 (20.0) | 4 (26.7) | 2 (14.3) |
| MG therapy at baseline | |||
| Pyridostigmine | 14 (93.3) | 14 (93.3) | 13 (92.9) |
| Prednisone | 11 (73.3) | 9 (60.0) | 11 (78.6) |
| Azathioprine | 3 (20.0) | 3 (20.0) | 3 (21.4) |
| Mycophenolate mofetil | 5 (33.3) | 6 (40.0) | 1 (7.1) |
| Cyclosporine | 0 | 1 (6.7) | 0 |
| Tacrolimus | 1 (6.7) | 0 | 0 |
Abbreviations: BMI, body mass index (calculated as weight in kilograms divided by height in meters squared); IVIg, intravenous immunoglobulin; MG, myasthenia gravis; MG-ADL, Myasthenia Gravis Activities of Daily Living; MGC, Myasthenia Gravis Composite; MGFA, Myasthenia Gravis Foundation of America; MG-QoL15r, Myasthenia Gravis Quality-of-Life Revised Scale; QMG, Quantitative Myasthenia Gravis.