Primary endpoints of Week 24 and Week 52: full analysis set (n = 22).
| Placebo, n = 12 | Pomalidomide 1 mg QD, n = 10 | |||||||
|---|---|---|---|---|---|---|---|---|
| Variables | Visit | Baseline | Value at Visit | Change from Baseline | Baseline | Value at Visit | Change fromBaseline | |
| Predicted FVC, % | Baselinea | n | — | 12 | — | — | 10 | — |
| Mean (± SD) | — | 60.9 (8.6) | — | — | 57.7 (7.3) | — | ||
| Min, Max | — | 47, 77 | — | — | 45, 67 | — | ||
| Week 24 | nb | 10 | 10 | 10 | 8 | 8 | 8 | |
| Mean (± SD) | 63.2 (7.3) | 61.9 (8.6) | −1.3 (3.3) | 53.6 (8.1) | 55.9 (13.6) | 2.3 (12.6) | ||
| Min, Max | 55, 77 | 52, 78 | −7, 4 | 45, 67 | 45, 86 | −8, 32 | ||
| Week 52a | nb | 11 | 11 | 11 | 8 | 8 | 8 | |
| Mean (± SD) | 60.7 (9.0) | 57.9 (10.0) | −2.8 (4.0) | 53.2 (8.2) | 48 (8.6) | −5.2 (5.3) | ||
| Min, Max | 47, 77 | 41, 47 | −8, 5 | 45, 67 | 40, 61 | −15, 4 | ||
| mRSS | Baseline | n | — | 11 | — | — | 10 | — |
| Mean (± SD) | — | 20.5 (10.0) | — | — | 17.1 (9.4) | — | ||
| Min, Max | — | 2, 32 | — | — | 4, 30 | — | ||
| Week 24 | na | 10 | 10 | 10 | 8 | 8 | 8 | |
| Mean (± SD) | 19.6 (10.1) | 16 (9.8) | −3.6 (5.7) | 15 (9.0) | 13 (10.5) | −2 (3.4) | ||
| Min, Max | 2, 32 | 3, 28 | −13, 3 | 4, 27 | 2, 29 | −6, 3 | ||
| Week 52 | nb | 11 | 11 | 11 | 10 | 10 | 10 | |
| Mean (± SD) | 20.5 (10.0) | 16.7 (10.9) | −3.7 (7.0) | 17.1 (9.4) | 14.4 (10.1) | −2.7 (5.7) | ||
| Min, Max | 2, 32 | 1, 31 | −15, 7 | 4, 30 | 2, 30 | −9, 10 | ||
| UCLA SCTC GIT 2.0 | Baseline | n | — | 12 | — | — | 10 | — |
| Mean (± SD) | — | 0.2 (0.18) | — | — | 0.5 (0.29) | — | ||
| Min, Max | — | 0, 1 | — | — | 0, 1 | — | ||
| Week 24 | nb | 10 | 10 | 10 | 8 | 8 | 8 | |
| Mean (± SD) | 0.2 (0.19) | 0.3 (0.32) | 0.1 (0.23) | 0.5 (0.28) | 0.4 (0.24) | −0.1 (0.2) | ||
| Min, Max | 0, 1 | 0, 1 | 0, 0 | 0, 1 | 0, 1 | 0, 0 | ||
| Week 52 | nb | 12 | 12 | 12 | 10 | 10 | 10 | |
| Mean (± SD) | 0.2 (0.18) | 0.3 (0.2) | 0 (0.18) | 0.5 (0.29) | 0.5 (0.39) | 0.1 (0.29) | ||
| Min, Max | 0, 1 | 0, 1 | 0, 0 | 0, 1 | 0, 1 | 0, 0 | ||
↵a FVC baseline: average of screening and baseline. FVC Week 52: average of Week 48 and Week 52.
↵b At a postbaseline timepoint for Baseline and Change from Baseline column, n = no. patients with a Baseline value and a postbaseline value at the timepoint. None of the primary endpoints achieved statistical significance. As a result, p values are not provided. QD: once daily; FVC: forced vital capacity; mRSS: modified Rodnan skin score; UCLA SCTC GIT: University of California, Los Angeles, Scleroderma Clinical Trial Consortium Gastrointestinal Tract score.