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Table 1 Characteristics at baseline (Full Analysis Set, n  = 80)

From: An exploratory randomised double-blind and placebo-controlled phase 2 study of a combination of baclofen, naltrexone and sorbitol (PXT3003) in patients with Charcot-Marie-Tooth disease type 1A

 

Placebo ( n  = 19)

PXT3003 LD ( n  = 21)

PXT3003 ID ( n  = 21)

PXT3003 HD ( n  = 19)

Mild/Moderate

3 (16%)/16 (84%)

4 (19%)/17(81%)

5 (24%)/16 (76%)

3 (16%)/16 (84%)

Women/Men

11 (58%)/8 (42%)

14 (67%)/7 (33%)

13 (62%)/8 (38%)

10 (53%)/9 (47%)

Age (years)

43.2 (12.2)

47.9 (14.9)

44.3 (12.7)

44.6 (11.2)

Body Mass Index

25.0 (3.9)

24.5 (4.0)

23.5 (3.5)

23.6 (4.6)

Disease Duration (years)

7.1 (5.5)

10.2 (13.1)

6.6 (4.5)

8.9 (5.5)

CMTNS

14.3 (3.8)

14.2 (4.1)

13.0 (4.0)

13.8 (3.4)

ONLS

3.1 (1.1)

3.3 (1.0)

3.5 (0.9)

3.6 (0.8)

6MWT (m)

468.2 (99.9)

473.1 (70.9)

450.7 (71.1)

429.3 (83.7)

9HPT (s)

17.2 (2.5)

16.1 (3.9)

18.4 (4.7)

20.8 (7.8)

Ankle Dorsiflexion (Nm)

7.8 (6.6)

9.1 (5.0)

8.3 (5.6)

8.2 (6.1)

Grip (kg)

22.6 (10.7)

21.6 (6.1)

23.1 (9.2)

20.6 (10.4)

CMAP (milliV)

3.7 (2.0)

4.0 (1.8)

3.7 (2.1)

3.4 (2.3)

MCV (m/s)

21.5 (3.6)

22.7 (4.7)

20.8 (4.8)

20.5 (5.3)

DML (ms)

8.6 (2.2)

7.9 (2.1)

8.2 (1.8)

8.2 (1.9)

SNAP (microV)

2.6 (3.2)

2.3 (3.0)

2.6 (3.8)

2.2 (2.7)

SCV (m/s)

31.1 (14.8)

29.4 (8.2)

31.3 (9.4)

29.9 (7.7)

  1. Data are count (%) or mean (s.d.). CMTNS = Charcot-Marie-Tooth Neuropathy Score; ONLS = Overall Neuropathy Limitations Scale; 6MWT = 6-Minute Walk Test; 9HPT = 9-Hole Peg Test; CMAP = Amplitudes of Compound Muscle Action Potentials; MCV = Motor Conduction Velocity; DML = Distal Motor Latency; SNAP = Amplitudes of Sensory Nerve Action Potentials; SCV = Sensitive Conduction Velocity. Mild: CMTNS ≤ 10; Moderate: 11 ≤ CMTNS ≤ 20.