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
verfasst von:
Shahram Attarian, Jean-Michel Vallat, Laurent Magy, Benoît Funalot, Pierre-Marie Gonnaud, Arnaud Lacour, Yann Péréon, Odile Dubourg, Jean Pouget, Joëlle Micallef, Jérôme Franques, Marie-Noëlle Lefebvre, Karima Ghorab, Mahmoud Al-Moussawi, Vincent Tiffreau, Marguerite Preudhomme, Armelle Magot, Laurène Leclair-Visonneau, Tanya Stojkovic, Laura Bossi, Philippe Lehert, Walter Gilbert, Viviane Bertrand, Jonas Mandel, Aude Milet, Rodolphe Hajj, Lamia Boudiaf, Catherine Scart-Grès, Serguei Nabirotchkin, Mickael Guedj, Ilya Chumakov, Daniel Cohen
Unfortunately, the original version of this article [1] contained an error due to a typographical error in the computer code used. This meant the percentage improvement over baseline of the endpoints CMTNS, ONLS, 9HPT and DML was slightly increased. Therefore some of the values in Tables 3, 4, Additional file 4: Table S4 and Fig. 4 are incorrect.
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
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
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*
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
Fig. 4
Response to PXT3003 on clinical scales (Full Analysis Set, n = 80). Mean % (s.e.m.) of improvement from baseline per group at 12 months for CMTNS (a) and ONLS (b). Sample sizes: Placebo (n = 19), LD (n = 21), ID (n = 21), HD (n = 19)
×
However this error has no bearing on statistical outcome of the study, the conclusions or text of the manuscript. Correct versions of Tables 3, 4 and Fig. 4 can be seen below and Additional file 4 accessed using the link below.
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Open AccessThis article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.
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
verfasst von
Shahram Attarian Jean-Michel Vallat Laurent Magy Benoît Funalot Pierre-Marie Gonnaud Arnaud Lacour Yann Péréon Odile Dubourg Jean Pouget Joëlle Micallef Jérôme Franques Marie-Noëlle Lefebvre Karima Ghorab Mahmoud Al-Moussawi Vincent Tiffreau Marguerite Preudhomme Armelle Magot Laurène Leclair-Visonneau Tanya Stojkovic Laura Bossi Philippe Lehert Walter Gilbert Viviane Bertrand Jonas Mandel Aude Milet Rodolphe Hajj Lamia Boudiaf Catherine Scart-Grès Serguei Nabirotchkin Mickael Guedj Ilya Chumakov Daniel Cohen
