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Table 4 Response to PXT3003 on efficacy outcomes in HD and in PLI, with comparisons of HD versus PLI (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 HD versus PLI
  PLI PXT3003 HD Estimate P-value
(n = 61) (n = 19)
CMTNS −3.4 (18.4) 5.2 (12.5) 8.0 (0.4;16.2) 0.042*
ONLS −7.7 (28.5) 6.8 (18.2) 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) 1.8 (11.1) 6.1 (10.6) 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) 3 (15.3) 5 (15.2) 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*
  1. 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; Shading = 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