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Table 1 Subject demographics and baseline characteristics

From: A double-blind, placebo-controlled, randomized trial of PXT3003 for the treatment of Charcot–Marie–Tooth type 1A

 

High-dose PXT3003a

Low-dose PXT3003

Placebo

FAS

mFAS

FAS

mFAS

FAS

mFAS

(N = 113)

(N = 55)

(N = 109)

(N = 93)

(N = 101)

(N = 87)

CMTNS-v2b

13.68 (3.19)

13.02 (3.25)

14.13 (3.04)

14.04 (2.99)

13.94 (3.13)

14.01 (3.27)

ONLS score

3.18 (1.11)

3.05 (1.13)

3.39 (1.05)

3.33 (1.05)

3.20 (1.17)

3.23 (1.19)

Age (years)

39.6 (13.9)

41.2 (13.6)

41.0 (12.3)

40.7 (12.4)

42.1 (13.2)

42.3 (13.2)

BMI (kg/m2)

25.2 (4.52)

24.9 (4.53)

25.6 (4.72)

25.4 (4.68)

25.4 (4.94)

25.2 (4.90)

Sex (n, [%])

      

Female

68 (60.2)

34 (61.8)

60 (55.0)

51 (54.8)

62 (61.4)

51 (58.6)

Male

45 (39.8)

21 (38.2)

49 (45.0)

42 (45.2)

39 (38.6)

36 (41.4)

Race (n, [%])

      

White

110 (97.3)

53 (96.4)

107 (98.2)

91 (97.8)

100 (99.0)

86 (98.9)

Black

1 (0.9)

1 (1.8)

0 (0.0)

0 (0.0)

0 (0.0)

0 (0.0)

Asian

2 (1.8)

1 (1.8)

2 (1.8)

2 (2.2)

1 (1.0)

1 (1.1)

  1. Values other than race and sex are reported as mean (standard deviation)
  2. BMI, body mass index; CMTNS-v2, Charcot–Marie–Tooth Neuropathy Scale version 2; FAS, full analysis set; mFAS, modified full analysis set; ONLS, Overall Neuropathy Limitations Scale
  3. aHigh dose: 6 mg baclofen, 0.70 mg naltrexone, 210 mg D-sorbitol; low dose: 3 mg baclofen, 0.35 mg naltrexone, 105 mg D-sorbitol, administered twice-a-day
  4. bCMTNS-v2 total score; in these measurements, N = N − 1 for the high-dose and placebo groups in both the FAS and mFAS