Table 1 Demographics and baseline clinical characteristics (modified intent-to-treat population)a

From: AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial

Characteristic

Delandistrogene moxeparvovec (n = 63)

Placebo (n = 62)

All (N = 125)

Age, mean (s.d.), years

5.98 (1.06)

6.08 (1.05)

6.03 (1.05)

 4–5 years, n (%)

30 (47.6)

29 (46.8)

59 (47.2)

 6–7 years, n (%)

33 (52.4)

33 (53.2)

66 (52.8)

Race group, n (%)

 Asian

8 (12.7)

11 (17.7)

19 (15.2)

 Black or African American

0 (0)

2 (3.2)

2 (1.6)

 White

49 (77.8)

46 (74.2)

95 (76.0)

 Multiple

1 (1.6)

0 (0)

1 (0.8)

 Other

2 (3.2)

1 (1.6)

3 (2.4)

 Not reported

3 (4.8)

2 (3.2)

5 (4.0)

Ethnicity, n (%)

 Hispanic or Latino

15 (23.8)

8 (12.9)

23 (18.4)

 Not Hispanic or Latino

47 (74.6)

53 (85.5)

100 (80.0)

 Not reported/unknown

1 (1.6)

1 (1.6)

2 (1.6)

Dosing weight, mean (s.d.), kg

21.29 (4.62)

22.37 (6.42)

21.83 (5.59)

Time since corticosteroid treatment started, mean (s.d.), years

1.07 (0.92)

0.97 (0.83)

1.02 (0.88)

Steroid type, n (%), at baseline

 Any use of deflazacort

43 (68.3)

28 (45.2)

71 (56.8)

 Any use of prednisone/prednisolone

63 (100)

62 (100)

125 (100)

 Other

0 (0)

0 (0)

0 (0)

Pathogenic variant, n (%)b

 Large deletion

45 (71.4)

41 (66.1)

86 (68.8)

 Large duplication

3 (4.8)

3 (4.8)

6 (4.8)

 Small variant

15 (23.8)

18 (29.0)

33 (26.4)

  Nonsense variant

8 (12.7)

7 (11.3)

15 (12.0)

  Frameshift variant

4 (6.3)

5 (8.1)

9 (7.2)

  Intron variant

3 (4.8)

6 (9.7)

9 (7.2)

NSAA total score, mean (s.d.), points

23.10 (3.75)

22.82 (3.78)

22.96 (3.75)

TTR, mean (s.d.), seconds

3.52 (0.81)

3.60 (0.68)

3.56 (0.75)

10MWR, mean (s.d.), seconds

4.82 (0.79)

4.92 (0.73)

4.87 (0.76)

SV95C, mean (s.d.), meters per secondc

1.82 (0.30)

1.77 (0.29)

1.79 (0.30)

100MWR, mean (s.d.), secondsd

60.67 (15.55)

63.01 (17.01)

61.80 (16.25)

Time to ascend 4 steps, mean (s.d.), secondse

3.17 (1.01)

3.37 (1.09)

3.27 (1.05)

CK, mean (s.d.), U L−1 (f)

18,143.42 (8016.26)

18,188.89 (6521.12)

N/A

  1. aSex and racial and ethnic demographic answers were self-reported. Sex is not shown in this table; per inclusion criterion 1, patients must be male at birth to be eligible to participate in this study.
  2. bLarge deletions and large duplications are the two types of larger structural variants, which extend one or more exons and/or are 50 nucleotides or more in length, inclusive of any nucleotides affected in intronic regions. Small variants include single-nucleotide variants, small insertions and small deletions. One patient had two variants (a large duplication and a large inversion), and it is listed in the large duplication variant category.
  3. cSV95C: delandistrogene moxeparvovec n = 61, placebo n = 62, total N = 123.
  4. d100MWR: delandistrogene moxeparvovec n = 63, placebo n = 59, total N = 122.
  5. eTime to ascend 4 steps: delandistrogene moxeparvovec n = 63, placebo n = 61, total N = 124.
  6. fCK: delandistrogene moxeparvovec n = 62, placebo n = 62.
  7. Mean (s.d.) is provided for continuous variables. Frequency (%) is provided for categorical variables. N/A, not applicable.
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