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Table 2 Treatment effect of high and low-dose PXT3003 vs placebo in exploratory efficacy endpoints (mFAS)

From: A double-blind, placebo-controlled, randomized trial of PXT3003 for the treatment of Charcot–Marie–Tooth type 1A

 

Test

High-dose PXT3003

Low-dose PXT3003

ONLS

Arm score (↓)

 − 0.19 (− 0.43, 0.04)

0.01 (− 0.19, 0.22)

 

Leg score (↓)

 − 0.18 (− 0.36, 0.01)

 − 0.13 (− 0.29, 0.02)

QMT

Grip strength (↑) (kg)

 − 1.49 (− 3.92, 0.93)

0.37 (− 1.64, 2.37)

 

Dorsiflexion strength (↑) (Newton)

7.48 (− 3.60, 18.57)

4.09 (− 5.01, 13.20)

Electrophysiology

CMAP (↑) (mV)

 − 0.33 (− 1.05, 0.40)

0.14 (− 0.46, 0.74)

 

Motor nerve conduction velocity (↑) (m/s)

0.08 (− 1.52, 1.67)

 − 0.09 (− 1.42, 1.25)

 

Distal motor latency (↓) (ms)

 − 0.20 (− 1.58, 1.18)

0.08 (− 1.07, 1.24)

 

Radial SNAP (↑) (µV)

 − 0.11 (− 1.20, 0.99)

 − 0.04 (− 0.96, 0.88)

Quality of life

Mobility (↓)

 − 0.07 (− 0.35, 0.20)

 − 0.11 (− 0.34, 0.12)

 

Self-care (↓)

 − 0.12 (− 0.31, 0.06)

 − 0.18 (− 0.34, − 0.03)

 

Usual activities (↓)

 − 0.02 (− 0.27, 0.23)

 − 0.11 (− 0.32, 0.10)

 

Pain/discomfort (↓)

0.00 (− 0.25, 0.26)

 − 0.12 (− 0.33, 0.09)

 

Anxiety/depression (↓)

 − 0.04 (− 0.29, 0.22)

 − 0.06 (− 0.28, 0.15)

 

Visual analog scale (↑) (mm)

2.57 (− 2.36, 7.50)

4.67 (0.59, 8.75)

  1. Arrows indicate whether a higher (↑) or a lower (↓) value indicates a better clinical condition. Values in parentheses are 97.5% confidence intervals. Bolded values were statistically significant—the change in the ONLS leg score in the high-dose group had p = 0.030, the change in the self-care quality of life score in the low-dose group had p = 0.007, and the change in the visual analog scale in the low-dose group had p = 0.010. No other items were statistically significant. The change from baseline to end of treatment of exploratory endpoints are shown for each treatment group in Additional file 2: Table S2
  2. CMAP, Compound muscle action potential; mFAS, modified full analysis set; SNAP, radial sensory nerve action potential; QMT, quantified muscular testing