Subject disposition, demographics, and baseline clinical characteristics
| Parameter | Prior Trial Participation | Total | ||
|---|---|---|---|---|
| From Replicating Evidence of Preserved Renal Function: An Investigation of Tolvaptan Safety and Efficacy in Autosomal Dominant Polycystic Kidney Disease Tolvaptan | From Replicating Evidence of Preserved Renal Function: An Investigation of Tolvaptan Safety and Efficacy in Autosomal Dominant Polycystic Kidney Disease Placebo | From Tolvaptan Efficacy and Safety in Management of Autosomal Dominant Polycystic Kidney Disease and Its Outcomes 4:4 Tolvaptan | ||
| Disposition | ||||
| Enrolled | 506 | 570 | 718a | 1803b |
| Completed | 434 | 423 | 624 | 1488 |
| Discontinued | 72 | 147 | 95 | 316 |
| Demographics | ||||
| Age, yr | ||||
| Mean (SD) | 49 (8) | 49 (8) | 46 (8) | 47 (8) |
| Sex | ||||
| Men | 272 (54%) | 280 (49%) | 375 (52%) | 929 (52%) |
| Women | 234 (46%) | 290 (51%) | 343 (48%) | 874 (48%) |
| Height, cm | ||||
| Mean (SD) | 174 (11) | 173 (10) | 175 (11) | 174 (11) |
| Weight, kg | ||||
| Mean (SD) | 86 (19) | 84 (21) | 82 (19) | 84 (19) |
| Race | ||||
| White | 461 (91%) | 525 (92%) | 694 (97%) | 1689 (94%) |
| Baseline clinical characteristics | ||||
| CKD stage | ||||
| CKD 1 | 1 (0.2%) | 0 (0.0%) | 77 (11%) | 81 (4%) |
| CKD 2 | 35 (7%) | 33 (6%) | 264 (37%) | 334 (19%) |
| CKD 3a | 131 (26%) | 146 (26%) | 156 (22%) | 434 (24%) |
| CKD 3b | 189 (37%) | 230 (40%) | 132 (18%) | 554 (31%) |
| CKD 4 | 150 (30%) | 158 (28%) | 82 (11%) | 390 (22%) |
| CKD 5 | 0 (0.0%) | 3 (0.5%) | 7 (1.0%) | 10 (0.6%) |
| eGFR (CKD-EPI), ml/min per 1.73 m2 | ||||
| >45 | 167 (33%) | 179 (31%) | 497 (69%) | 849 (47%) |
| ≤45 | 339 (67%) | 391 (69%) | 221 (31%) | 954 (53%) |
| ≥25 | 429 (85%) | 486 (85%) | 662 (92%) | 1586 (88%) |
| <25 | 77 (15%) | 84 (15%) | 56 (8%) | 217 (12%) |
CKD stages were from the time of entry into this trial, and they were defined as follows: stage 1≥90 ml/min per 1.73 m2, stage 2=60–89 ml/min per 1.73 m2, stage 3a=45–59 ml/min per 1.73 m2, stage 3b=30–44 ml/min per 1.73 m2, stage 4=15–29 ml/min per 1.73 m2, and stage 5<15 ml/min per 1.73 m2. CKD-EPI, Chronic Kidney Disease Epidemiology Collaboration.
↵a The number of subjects enrolling from the Tolvaptan Efficacy and Safety in Management of Autosomal Dominant Polycystic Kidney Disease and Its Outcomes (TEMPO) 4:4 (n=718) trial is one less than the number who completed or discontinued (n=719) due to one subject with missing data that resulted in exclusion from the enrolled population.
↵b The table has no column for subjects who entered the extension directly from TEMPO 3:4 or NOCTURNE due to the small number of subjects (n=9; four from TEMPO 3:4 tolvaptan, two from NOCTURNE tolvaptan, and three from TEMPO 3:4 placebo) from those trials. Data from these subjects are included in the total column.