Table 3.
Response to PXT3003 on efficacy outcomes in treatment groups, with comparisons of active doses versus Placebo (Full Analysis Set, n = 80)
| Mean % of improvement | PXT3003 LD versus Placebo | PXT3003 ID versus Placebo | PXT3003 HD versus Placebo | Dose-effect | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Placebo | PXT3003 LD | PXT3003 ID | PXT3003 HD | Estimate | P-value | Estimate | P-value | Estimate | P-value | Correlation | P-value | |
| (n = 19) | (n = 21) | (n = 21) | (n = 19) | |||||||||
| CMTNS | −0.25 (17.3) | −3.8 (20.4) | −5.8 (17.7) | 5.2 (12.5) | −2.6 (−11.9;7.6) | 0.67 | −3.1 (−11.0;5.4) | 0.74 | 5.5 (−3.4;15.2) | 0.16 | 0.54 | 0.30 |
| ONLS | −11.8 (33.7) | −12.7 (31.7) | 1.2 (16.7) | 6.8 (18.2) | −3.9 (−14.2;7.6) | 0.72 | 6.9 (−3.8;18.8) | 0.15 | 14.4 (0.55;30.2) | 0.043* | 0.28 | 0.006* |
| 6MWT (m) | 9.0 (8.3) | 6.2 (8.3) | 6.4 (9.4) | 9.9 (6.9) | −2.4 (−6.2;1.5) | 0.85 | −2.4 (−6.6;2.0) | 0.82 | 0.7 (−3.2;4.7) | 0.38 | 0.11 | 0.16 |
| 9HPT (s) | 3.6 (10.9) | −2.5 (12) | 4.4 (9.5) | 6.1 (10.6) | −4.6 (−10.3;1.5) | 0.89 | −0.2 (−5.3;5.2) | 0.52 | 0.3 (−5.7;6.6) | 0.47 | 0.15 | 0.092 |
| Ankle Dorsiflexion (Nm) | 20.2 (88.4) | −3.6 (43.0) | 81.5 (369.6) | 20.4 (64.1) | −4.0 (−21.7;17.8) | 0.63 | 11.4 (−15.4;46.8) | 0.26 | 8.2 (−13.8;35.9) | 0.28 | 0.11 | 0.16 |
| Grip (kg) | 9.9 (24.2) | 1.3 (15.6) | 4.7 (12.5) | 11.7 (18.1) | −7.1 (−15.6;2.1) | 0.90 | −3.6 (−11.8;5.4) | 0.75 | 1.6 (−7.7;11.9) | 0.39 | 0.12 | 0.15 |
| CMAP (milliV) | 34.4 (62.0) | 1.4 (38.7) | 22.9 (62.6) | 64.2 (208.5) | −25.1 (−44.8;1.5) | 0.94 | −9.2 (−27.3;13.5) | 0.77 | −5.1 (−27.1;23.6) | 0.63 | −0.001 | 0.50 |
| MCV (m/s) | 3.7 (8.5) | 3.0 (11.5) | 5.7 (12.3) | 9.0 (17.6) | −1.0 (−6.5;4.9) | 0.61 | 0.5 (−4.8;6.2) | 0.44 | 2.8 (−3.4;9.4) | 0.23 | 0.11 | 0.18 |
| DML (ms) | −0.33 (8.7) | 0.33 (16.1) | 8.3 (18.1) | 5 (15.2) | 3.4 (−4.3;11.7) | 0.24 | 13.8 (4.2;24.3) | 0.009* | 8.0 (0.59;16.0) | 0.038* | 0.21 | 0.035* |
| SNAP (microV) | 12.4 (121.7) | 11.5 (88.2) | 23.3 (128.4) | 5.2 (69.0) | −1.2 (−42.9;71.0) | 0.52 | 8.7 (−31.2;71.6) | 0.38 | 13.9 (−24.1;71.0) | 0.29 | 0.09 | 0.30 |
| SCV (m/s) | 3.4 (11.0) | 5.3 (11.2) | 29.5 (63.4) | 30.5 (10.0) | 1.5 (−5.8;9.4) | 0.36 | 17.5 (−5.5;46.2) | 0.11† | 26.6 (15.5;38.8) | 0.00037* | 0.42 | 0.01* |
Data are mean % (s.d.) of improvement for each treatment group. 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). Dose-effect was tested through Spearman’s rank correlation. P-values are one-tailed. *P < 0.05; Shading = best improvement within dosages. 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