Table 1.
Baseline characteristics of patients included in the PK cohort and the randomized IONIS-FXIRx and placebo groups (ITT population)
| Characteristic | PK cohort IONIS-FXIRx 300 mg (n = 6) |
Randomized cohorts |
||
|---|---|---|---|---|
| Pooled placebo (n = 13) | IONIS-FXIRx 200 mg (n = 15) |
IONIS-FXIRx 300 mg (n = 15) |
||
| Female, n (%) | 0 (0.0) | 4 (30.8) | 5 (33.3) | 12 (80.0) |
| Male, n (%) | 6 (100.0) | 9 (69.2) | 10 (66.7) | 3 (20.0) |
| Median age, yr (min, max) | 63 (50, 68) | 63 (40, 80) | 56 (31, 77) | 58 (29, 76) |
| Race, n (%) | ||||
| White | 5 (83.3) | 8 (61.5) | 10 (66.7) | 6 (40.0) |
| Black or African-American | 1 (16.7) | 3 (23.1) | 4 (26.7) | 5 (33.3) |
| Other | 0 (0) | 2 (15.4) | 1 (6.7) | 4 (26.7) |
| Vascular access, n (%) | ||||
| Central venous catheter | 3 (50.0) | 8 (61.5) | 8 (53.3) | 9 (60.0) |
| Arteriovenous fistula | 1 (16.7) | 5 (38.5) | 6 (40.0) | 5 (33.3) |
| Arteriovenous graft | 2 (33.3) | 0 (0.0) | 1 (6.7) | 1 (6.7) |
| Urea reduction ratio, mean (SD) | NA | 74 (10) | 65 (14) | 73 (7) |
| Cause of ESRD, n (%) | ||||
| Diabetes | 2 (33.3) | 5 (38.5) | 5 (33.3) | 8 (53.3) |
| Hypertension | 0 (0.0) | 2 (15.4) | 0 (0.0) | 1 (6.7) |
| Glomerulonephritis | 1 (16.7) | 2 (15.4) | 4 (26.7) | 2 (13.3) |
| Polycystic kidney disease | 0 (0.0) | 0 (0.0) | 2 (13.3) | 0 (0.0) |
| Other | 3 (50.0) | 4 (30.8) | 4 (26.7) | 4 (26.7) |
| HD circuit anticoagulation n (%) | ||||
| Unfractionated heparin | 6 (100) | 11 (84.6) | 12 (80.0) | 14 (93.3) |
| Saline flushes/no anticoagulation | 0 (0.0) | 0 (0.0) | 1 (6.7) | 1 (6.7) |
| Aspirin use, n (%) | 4 (66.7) | 6 (46.2) | 5 (33.3) | 7 (46.7) |
| Pre-HD | ||||
| FXI activity (U/ml), mean (SD) | 0.99 (0.17) | 0.93 (0.19) | 1.00 (0.22) | 0.94 (0.24) |
| FXI antigen (U/ml), mean (SD) | 1.00 (0.28) | 1.08 (0.26) | 1.03 (0.26) | 1.01 (0.29) |
| aPTT (s), mean (SD) | 27.0 (2.2) | 27.4 (2.5) | 29.0 (8.8) | 27.4 (2.4) |
| INR mean (SD) | 1.0 (0.1) | 1.0 (0.1) | 1.0 (0.1) | 1.0 (0.1) |
| Post-HD | ||||
| FXI activity (U/ml), mean (SD) | ND | 0.98 (0.23) | 1.08 (0.21) | 0.99 (0.36) |
| FXI antigen (U/ml), mean (SD) | ND | 1.12 (0.38) | 1.22 (0.21) | 1.06 (0.40) |
aPTT, activated partial thromboplastin time; ESRD, end-stage renal disease; FXI, factor IX; HD, hemodialysis; INR, international normalized ratio; ITT, intent-to-treat; max, maximum; min, minimum; NA, not applicable; ND, not done; PK, pharmacokinetics.