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Table 2 Baseline demographic and clinical characteristics in the modified intent-to-treat population of COMET

From: Applying the win ratio method in clinical trials of orphan drugs: an analysis of data from the COMET trial of avalglucosidase alfa in patients with late-onset Pompe disease

Characteristic

Overall (N = 100)

AVA (N = 51)

ALG (N = 49)

Mean age (SD), years

48 (14)

46 (14)

50 (14)

Male, n (%)

52 (52)

27 (53)

25 (51)

Race, n (%)

 Asian

3 (3)

3 (6)

0 (0)

 Black or African American

3 (3)

1 (2)

2 (4)

 White

94 (94)

47 (92)

47 (96)

Mean age at symptom onset (SD), years

35 (16)a

33 (17)a

38 (16)

Mean age at diagnosis (SD), years

46 (15)

45 (15)

48 (15)

Mean FVC (SD), % predicted

62.1 (13.4)

62.6 (14.4)

61.6 (12.4)

Mean 6MWT (SD), m

388.9 (113.5)

399.3 (110.9)

378.1 (116.2)

  1. The modified intent-to-treat population included all randomized participants who received at least one partial or full infusion of study drug. Full baseline demographics and clinical characteristics were published in Diaz-Manera et al. 2021 [15]
  2. 6MWT six-minute walk test, ALG alglucosidase alfa, AVA avalglucosidase alfa, FVC forced vital capacity, SD standard deviation
  3. aOverall based on n = 99; AVA based on n = 50
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172