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Table 3 Response to PXT3003 on efficacy outcomes in treatment groups, with comparisons of active doses versus Placebo (Full Analysis Set, n = 80)

From: Erratum to: 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

  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*
  1. 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