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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: 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 ( n  = 61)

PXT3003 HD ( n  = 19)

Estimate

P -value

CMTNS

−0.1 (19.0)

7.7 (18.4)

8.0 (0.4;16.2)

0.042*

ONLS

−2.2 (21.2)

12.3 (28.4)

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)

3.1 (11.3)

7.8 (12.1)

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)

6.7 (25.6)

8.4 (21.7)

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; Boldface = 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.
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172