Table 1.
Baseline demographics, disease characteristics, and pharmacodynamic measures
| Placebo (n = 26) |
Reldesemtiv 150 mg bid (n = 24) |
Reldesemtiv 450 mg bid (n = 20) |
Overall (n = 70) | |
|---|---|---|---|---|
| Age, years, mean (SD) | 28.5 (16.0) | 27.8 (12.0) | 32.6 (17.9) | 29.4 (15.3) |
| Age < 18 years, n (%) | 8 (30.8) | 7 (29.2) | 5 (25.0) | 20 (28.6) |
| Male, n (%) | 15 (57.7) | 14 (58.3) | 12 (60.0) | 41 (58.6) |
| White, n (%) | 22 (84.6) | 23 (95.8) | 18 (90.0) | 63 (90.0) |
| BMI, mean (SD) | 24.3 (7.4) | 25.4 (9.2) | 25.1 (5.5) | 24.9 (7.5) |
| SMA type II, n (%) | 2 (7.7) | 3 (12.5) | 1 (5.0) | 6 (8.6) |
| SMA type III, n (%) | 24 (92.3) | 21 (87.5) | 19 (95.0) | 64 (91.4) |
| Age at SMA onset, years, mean (SD) | 3.8 (4.6) | 7.8 (6.9) | 8.1 (9.4) | 6.4 (7.2) |
| Ambulatory, n (%) | 11 (42.3) | 12 (50.0) | 8 (40.0) | 31 (44.3) |
| % predicted FVC, mean (SD) | 84.4 (22.4) | 83.1 (22.0) | 85.9 (21.2) | 84.4 (21.7) |
| MEP, cm H2O, mean (SD) | 86.5 (36.9) | 94.0 (43.4) | 88.9 (47.7) | 89.8 (41.8) |
| MIP, cm H2O, mean (SD) | − 105.7 (38.5) | − 109.1 (44.2) | − 101.0 (43.2) | − 105.6 (41.3) |
| HFMSE score, mean (SD) | 30.6 (16.6) | 36.0 (17.2) | 30.4 (16.3) | 32.4 (16.7) |
| RULM total score, mean (SD) | 31.0 (8.7) | 34.8 (7.9) | 33.7 (8.0) | 33.1 (8.3) |
| TUG, s, mean (SD) | 21.5 (11.0) | 15.7 (6.5) | 22.8 (16.1) | 19.2 (10.3) |
| 6MWD, m, mean (SD) | 240.1 (111.8) | 316.6 (69.0) | 311.0 (107.3) | 287.3 (99.2) |
| SMA-HI total score, mean (SD)a | 33.1 (19.9) | NA | 39.8 (17.1) | 37.5 (18.1) |
aSMA-HI was only performed in participants in cohort 2
6MWD 6-minute walking distance, BMI body mass index, bid twice daily, FVC forced vital capacity, HFMS-E Hammersmith Functional Motor Score-Expanded, MEP maximum expiratory pressure, MIP maximum inspiratory pressure, NA not assessed, RULM revised upper limb module, SD standard deviation, SMA-HI Spinal Muscular Atrophy-Health Index, TUG timed up and go