Table 4.
Response to PXT3003 on efficacy outcomes in HD and in PLI, with comparisons of HD versus PLI (Full Analysis Set, n = 80)
| Mean % of improvement | PXT3003 HD versus PLI | |||
|---|---|---|---|---|
| PLI ( n = 61) | PXT3003 HD ( n = 19) | Estimate | P -value | |
| CMTNS | −0.1 (19.0) | 7.7 (18.4) | 8.0 (0.4;16.2) | 0.042* |
| ONLS | −2.2 (21.2) | 12.3 (28.4) | 12.1 (2.0;23.2) | 0.024* |
| 6MWT (m) | 7.1 (8.6) | 9.9 (6.9) | 2.6 (−0.73;6.1) | 0.099 |
| 9HPT (s) | 3.1 (11.3) | 7.8 (12.1) | 1.2 (−3.4;6.0) | 0.33 |
| Ankle Dorsiflexion (Nm) | 33.1 (223.2) | 20.4 (64.1) | 5.5 (−12.8;27.7) | 0.32 |
| Grip (kg) | 5.1 (17.9) | 11.7 (18.1) | 6.0 (−1.2;13.7) | 0.088 |
| CMAP (milliV) | 19.6 (56.5) | 64.2 (208.5) | 6.6 (−15.8;35.1) | 0.33 |
| MCV (m/s) | 4.2 (10.9) | 9.0 (17.6) | 2.5 (−2.4;7.7) | 0.21 |
| DML (ms) | 6.7 (25.6) | 8.4 (21.7) | 2.2 (−5.1;10.0) | 0.31 |
| SNAP (microV) | 15.9 (110.2) | 5.2 (69.0) | 12.0 (−23.9;64.9) | 0.31 |
| SCV (m/s) | 12.7 (38.0) | 30.5 (10.0) | 20.1 (2.4;40.8) | 0.030* |
Data are mean % (s.d.) of improvement for HD and for PLI after 12 months. Differences between treatment groups were assessed by Analysis of Covariance (ANCOVA) on log-transformed values by adjusting for baseline values. Estimates were provided as mean percentage change over baseline (90% CI). P-values are one-tailed. *P < 0.05; Boldface = best improvement within groups. 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.