Open Access 01-12-2016 | Erratum
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
Published in: Orphanet Journal of Rare Diseases | Issue 1/2016
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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*
|
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*
|
×
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