Difference between riociguat group and placebo group in change from baseline to week 52 in secondary endpoints
| Endpoint | Riociguat (n=60) | Placebo (n=61) | Estimate of difference (95% CI) | Nominal p value* |
| ACR CRISS | ||||
| No improvement, n (%) | 1 (1.7) | 4 (6.6) | 0.20% (–13.68 to 14.09)† | 0.977 |
| ≥3 missing criteria, n (%) | 6 (10.0) | 7 (11.5) | ||
| CRISS probability ≥60%, n (%) | 11 (18.3) | 11 (18.0) | ||
| CRISS probability <60%, n (%) | 49 (81.7) | 50 (82.0) | ||
| Mean HAQ-DI (SD), units | ||||
| Baseline | 0.89 (0.67) | 0.69 (0.69) | –0.07 (–0.23 to 0.08)‡ | 0.3529 |
| Change at week 52 | 0.05 (0.38) (n=56) | 0.13 (0.42) (n=52) | ||
| Mean patient global assessment (SD), units | ||||
| Baseline | 3.93 (2.50) | 3.77 (2.34) | 0.79 (–0.12 to 1.69)‡ | 0.0887 |
| Change at week 52 | 0.69 (2.75) (n=45) | –0.02 (2.23) (n=46) | ||
| Mean physician global assessment (SD), units | ||||
| Baseline | 4.33 (2.11) | 4.02 (2.00) | 0.83 (0.11 to 1.54)‡ | 0.0241 |
| Change at week 52 | –0.07 (2.16) (n=45) | –0.75 (2.09) (n=47) | ||
| Mean % predicted FVC (SD), % | ||||
| Baseline | 90.74 (18.52) | 94.82 (17.03) | –0.20 (–3.40 to 3.00)‡ | 0.901 |
| Change at week 52 | –2.38 (7.52) (n=55) | –2.95 (9.73) (n=51) |
*Since the primary endpoint was not met, all other p values cannot be considered statistically significant and are presented for information only.
†Mantel-Haenszel estimate.
‡Mixed model repeated measures applied with baseline value, treatment group, region, visit and treatment by visit interaction as fixed effects, and subject as a random effect.
ACR, American College of Rheumatology; CI, confidence interval; CRISS, Composite Response Index for Systemic Sclerosis; FVC, forced vital capacity; HAQ-DI, Health Assessment Questionnaire Disability Index.