TABLE 1.
Baseline characteristics of the overall population a .
| Characteristic | Patients treated with delandistrogene moxeparvovec in Part 1 b (n = 20) | Patients treated with placebo in Part 1 c (n = 21) |
|---|---|---|
| Age (years) d | ||
| Mean (SD) | 6.29 (1.19) | 6.24 (1.13) |
| Range | 4.47–7.85 | 4.34–7.98 |
| Median | 6.52 | 6.03 |
| Years since corticosteroid treatment started | ||
| Mean (SD) | 0.99 (1.07) | 1.26 (1.22) |
| Range | 0.22, 3.80 | 0.23, 5.07 |
| Median | 0.56 | 0.63 |
| Corticosteroid type, deflazacort | ||
| n (%) | 7 (35.0) | 7 (33.3) |
| Dosing weight (kg) | ||
| Mean (SD) | 23.28 (4.37) | 21.60 (3.49) |
| Range | 18.0–34.5 | 15.0–30.0 |
| Median | 22.50 | 21.50 |
| NSAA total score e | ||
| Mean (SD) | 19.8 (3.3) | 22.6 (3.3) |
| Median | 20.0 | 22.0 |
| Time to Rise (seconds) | ||
| Mean (SD) | 5.10 (2.17) | 3.56 (0.65) |
| Median | 4.30 | 3.40 |
| 4-stair Climb (seconds) | ||
| Mean (SD) | 3.69 (1.46) | 3.10 (0.98) |
| Median | 3.30 | 3.00 |
| 100MWR (seconds) | ||
| Mean (SD) | 61.04 (12.71) | 53.86 (8.30) |
| Median | 57.10 | 55.60 |
| 10MWR (seconds) | ||
| Mean (SD) | 5.35 (1.14) | 4.83 (0.72) |
| Median | 5.00 | 4.70 |
Intent-to-treat population.
Patients who received delandistrogene moxeparvovec in Part 1 and placebo in Part 2.
Patients who received placebo in Part 1 and delandistrogene moxeparvovec in Part 2.
The majority of patients (61%) were ≥6 years of age at baseline, and age was the only stratification factor for randomization.
The 4- to 5-year-old subgroup was well matched at baseline; however, in the 6- to 7-year-old subgroup, NSAA scores were not well matched at baseline.
Mutation type in treated and placebo cohorts: whole-exon deletion, 15/20 and 16/21; whole-exon duplication, 1/20 and 2/21; premature stop codon, 1/20 and 2/21; small insertion/deletion, 0/20 and 1/21; other, 3/20 and 1/21, respectively.
10MWR, 10-m Walk/Run; 100MWR, 100-m Walk/Run; NSAA, North Star Ambulatory Assessment; SD, standard deviation.