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