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Table 5 Proportion of non-deteriorated patients (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

 

Placebo ( n  = 19)

PXT3003 LD ( n  = 21)

PXT3003 ID ( n  = 21)

PXT3003 HD ( n  = 19)

HD versus Placebo

HD versus PLI

Non-deteriorated

10 (52%)

10 (48%)

9 (43%)

15 (79%)

Relative Risk

P-value

Relative Risk

P-value

1.5 (1.03;1.77)

0.047*

1.66 (1.24;1.93)

0.010*

  1. Data are count (%) of non-deteriorated patients per group and Relative Risks (90% CI) for the HD group versus Placebo and versus PLI assessed with a logistic regression model. P-values are one-tailed. *P < 0.05.
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172