Demographic and baseline characteristics of the patients in the intention-to-treat population
| Statistic/category . | SC411 . | Placebo (n = 17) . | |||
|---|---|---|---|---|---|
| Dose level 1 (n=16) . | Dose level 2 (n = 18) . | Dose level 3 (n = 16) . | Pooled (n = 50) . | ||
| Age, mean (SD), y | 12.3 (2.3) | 11.3 (2.7) | 11.9 (2.8) | 11.8 (2.6) | 12.6 (3.7) |
| Age groups, y | |||||
| 5-11, n (%) | 5 (31.3) | 10 (55.6) | 8 (50) | 23 (46) | 7 (41.2) |
| 12-17, n (%) | 11 (68.8) | 8 (44.4) | 8 (50) | 27 (54) | 10 (58.8) |
| Sex, n (%) | |||||
| Male | 8 (50.0) | 9 (50.0) | 6 (37.5) | 23 (46.0) | 10 (58.8) |
| Female | 8 (50.0) | 9 (50.0) | 10 (62.5) | 27 (54.0) | 7 (41.2) |
| BMI, mean (SD) | 19 (4.5) | 18.5 (3.4) | 19.3 (3.6) | 18.9 (3.8) | 19.1 (4.6) |
| Race, n (%)* | |||||
| Black | 16 (100) | 18 (100) | 15 (93.8) | 49 (98) | 17 (100) |
| White | 0 (0) | 0 (0) | 1 (6.2) | 1 (2) | 0 (0) |
| Genotyping, n (%) | |||||
| Hemoglobin SS | 13 (81.3) | 14 (77.8) | 11 (68.8) | 38 (76) | 15 (88.2) |
| Hemoglobin SC | 2 (12.5) | 2 (11.1) | 4 (25) | 8 (16) | 2 (11.8) |
| Hemoglobin S/β0-thalassemia | 1 (6.3) | 2 (11.1) | 1 (6.3) | 4 (8) | 0 |
| Disease severity, n (%)† | |||||
| Moderate | 8 (50) | 11 (61.1) | 7 (43.8) | 26 (52) | 13 (76.5) |
| Severe | 8 (50) | 7 (38.9) | 9 (56.3) | 24 (48) | 4 (23.5) |
| HU use, n (%) | 13 (81.3) | 12 (66.7) | 12 (75.0) | 37 (74.0) | 14 (82.4) |
| Hematocrit, mean (SD), % | 28.6 (3.5) | 27.2 (4.8) | 29.7 (4.5) | 28.6 (4.3) | 27.5 (3.2) |
| Hemoglobin, mean (SD), g/dL | 9.19 (1.4) | 8.8 (1.4) | 9.7 (1.5) | 9.3 (1.4) | 9.0 (0.9) |
| Mean corpuscular hemoglobin, mean (SD), pg/cell | 29.0 (3.7) | 29.6 (4.4) | 30.4 (3.8) | 29.8 (3.9) | 30.8 (5.4) |
| Mean corpuscular volume, mean (SD), fL | 90.2 (9.7) | 90.3 (12.0) | 93.0 (11.1) | 91.3 (10.8) | 93.9 (14.1) |
| Platelets, mean (SD), ×109/L | 286.6 (143.2) | 334.9 (153.0) | 341.1 (112.1) | 325.7 (134.9) | 399.1 (178.9) |
| Erythrocytes, mean (SD), ×1012/L | 3.2 (0.6) | 3.1 (0.9) | 3.3 (0.8) | 3.2 (0.8) | 2.9 (0.8) |
| Reticulocytes, mean (SD), % | 6.6 (2.8) | 7.8 (4.3) | 6.3 (2.8) | 6.8 (3.3) | 6.7 (2.9) |
| Leukocytes, mean (SD), ×109/L | 8.2 (3.2) | 11.7 (5.4) | 8.8 (2.6) | 9.6 (4.0) | 10.6 (3.3) |
| Statistic/category . | SC411 . | Placebo (n = 17) . | |||
|---|---|---|---|---|---|
| Dose level 1 (n=16) . | Dose level 2 (n = 18) . | Dose level 3 (n = 16) . | Pooled (n = 50) . | ||
| Age, mean (SD), y | 12.3 (2.3) | 11.3 (2.7) | 11.9 (2.8) | 11.8 (2.6) | 12.6 (3.7) |
| Age groups, y | |||||
| 5-11, n (%) | 5 (31.3) | 10 (55.6) | 8 (50) | 23 (46) | 7 (41.2) |
| 12-17, n (%) | 11 (68.8) | 8 (44.4) | 8 (50) | 27 (54) | 10 (58.8) |
| Sex, n (%) | |||||
| Male | 8 (50.0) | 9 (50.0) | 6 (37.5) | 23 (46.0) | 10 (58.8) |
| Female | 8 (50.0) | 9 (50.0) | 10 (62.5) | 27 (54.0) | 7 (41.2) |
| BMI, mean (SD) | 19 (4.5) | 18.5 (3.4) | 19.3 (3.6) | 18.9 (3.8) | 19.1 (4.6) |
| Race, n (%)* | |||||
| Black | 16 (100) | 18 (100) | 15 (93.8) | 49 (98) | 17 (100) |
| White | 0 (0) | 0 (0) | 1 (6.2) | 1 (2) | 0 (0) |
| Genotyping, n (%) | |||||
| Hemoglobin SS | 13 (81.3) | 14 (77.8) | 11 (68.8) | 38 (76) | 15 (88.2) |
| Hemoglobin SC | 2 (12.5) | 2 (11.1) | 4 (25) | 8 (16) | 2 (11.8) |
| Hemoglobin S/β0-thalassemia | 1 (6.3) | 2 (11.1) | 1 (6.3) | 4 (8) | 0 |
| Disease severity, n (%)† | |||||
| Moderate | 8 (50) | 11 (61.1) | 7 (43.8) | 26 (52) | 13 (76.5) |
| Severe | 8 (50) | 7 (38.9) | 9 (56.3) | 24 (48) | 4 (23.5) |
| HU use, n (%) | 13 (81.3) | 12 (66.7) | 12 (75.0) | 37 (74.0) | 14 (82.4) |
| Hematocrit, mean (SD), % | 28.6 (3.5) | 27.2 (4.8) | 29.7 (4.5) | 28.6 (4.3) | 27.5 (3.2) |
| Hemoglobin, mean (SD), g/dL | 9.19 (1.4) | 8.8 (1.4) | 9.7 (1.5) | 9.3 (1.4) | 9.0 (0.9) |
| Mean corpuscular hemoglobin, mean (SD), pg/cell | 29.0 (3.7) | 29.6 (4.4) | 30.4 (3.8) | 29.8 (3.9) | 30.8 (5.4) |
| Mean corpuscular volume, mean (SD), fL | 90.2 (9.7) | 90.3 (12.0) | 93.0 (11.1) | 91.3 (10.8) | 93.9 (14.1) |
| Platelets, mean (SD), ×109/L | 286.6 (143.2) | 334.9 (153.0) | 341.1 (112.1) | 325.7 (134.9) | 399.1 (178.9) |
| Erythrocytes, mean (SD), ×1012/L | 3.2 (0.6) | 3.1 (0.9) | 3.3 (0.8) | 3.2 (0.8) | 2.9 (0.8) |
| Reticulocytes, mean (SD), % | 6.6 (2.8) | 7.8 (4.3) | 6.3 (2.8) | 6.8 (3.3) | 6.7 (2.9) |
| Leukocytes, mean (SD), ×109/L | 8.2 (3.2) | 11.7 (5.4) | 8.8 (2.6) | 9.6 (4.0) | 10.6 (3.3) |