Open Access

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

  • Shahram Attarian1,
  • Jean-Michel Vallat3,
  • Laurent Magy3,
  • Benoît Funalot3,
  • Pierre-Marie Gonnaud4,
  • Arnaud Lacour5,
  • Yann Péréon6,
  • Odile Dubourg7,
  • Jean Pouget1,
  • Joëlle Micallef2,
  • Jérôme Franques1,
  • Marie-Noëlle Lefebvre2,
  • Karima Ghorab3,
  • Mahmoud Al-Moussawi4,
  • Vincent Tiffreau5,
  • Marguerite Preudhomme5,
  • Armelle Magot6,
  • Laurène Leclair-Visonneau5,
  • Tanya Stojkovic7,
  • Laura Bossi8,
  • Philippe Lehert9, 10,
  • Walter Gilbert11,
  • Viviane Bertrand12,
  • Jonas Mandel12,
  • Aude Milet12,
  • Rodolphe Hajj12,
  • Lamia Boudiaf12,
  • Catherine Scart-Grès12,
  • Serguei Nabirotchkin12,
  • Mickael Guedj12,
  • Ilya Chumakov12 and
  • Daniel Cohen12Email author
Contributed equally
Orphanet Journal of Rare Diseases201611:92

https://doi.org/10.1186/s13023-016-0463-6

Received: 16 May 2016

Accepted: 16 May 2016

Published: 7 July 2016

The original article was published in Orphanet Journal of Rare Diseases 2014 9:199

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.

Notes

Declarations

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.

Authors’ Affiliations

(1)
Centre de référence des maladies neuromusculaires et de la SLA, Pôle des neurosciences Cliniques, AP-HM et Aix Marseille Université
(2)
CIC-Centre de Pharmacologie Clinique et D’Evaluations Therapeutiques, AP-HM et Aix Marseille Université
(3)
CHU de Limoges - Hôpital Dupuytren
(4)
CHU Lyon Sud, 165 Chemin du Grand Revoyet
(5)
CHRU de Lille - Hôpital Roger Salengro, rue Emile Laine
(6)
CHU de Nantes - Hôtel Dieu, 1 place Alexis Ricordeau
(7)
CHU de Paris - Groupe Hospitalier Pitié-Salpétrière
(8)
Admissions
(9)
Faculty of Medicine, The University of Melbourne
(10)
Faculty of Economics, UCL Mons
(11)
Carl M. Loeb University Professor Emeritus, Harvard University
(12)
Pharnext, 11, rue des Peupliers

References

  1. Attarian S, Vallat JM, Magy L, Funalot B, Gonnaud PM, Lacour A, Péréon Y, Dubourg O, Pouget J, Micallef J, Franques J, Lefebvre MN, Ghorab K, Al-Moussawi M, Tiffreau V, Preudhomme M, Magot A, Leclair-Visonneau L, Stojkovic T, Bossi L, Lehert P, Gilbert W, Bertrand V, Mandel J, Milet A, Hajj R, Boudiaf L, Scart-Grès C, Nabirotchkin S, Guedj M, Chumakov I, Cohen D. 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. Orphanet J Rare Dis. 2014;9:199.View ArticlePubMedPubMed CentralGoogle Scholar

Copyright

© The Author(s). 2016

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