Oral high dose ascorbic acid treatment for one year in young CMT1A patients: a randomised, double-blind, placebo-controlled phase II trial

Table 2 Primary and secondary treatment outcomes according to assigned treatment.

	Placebo (n = 6)	Ascorbic acid (n = 5)
	Change	Change	Difference	Pvalue
Median nerve MNCV (m/s)^a	-0.8 (0.9)	0.5 (1.5)	1.3 (-0.3-3.0)	0.11
Median nerve minimal F response latency (ms)^a	3.5 (8.0)*	-5.6 (4.4)	-9.1 (-18.5-0.3)	0.06
CMAP amplitude abductor pollicis brevis muscle (mV)^a	-0.02 (0.9)	-0.2 (0.8)	-0.1 (-1.3 -1.1)	0.79
Three point grip (N)^a	-3.0 (9.2)	3.4 (9.0)	6.4 (-6.1-18.9)	0.28
Foot dorsiflexors (N)^a	-5.1 (17.6)	0.6 (15.6)	5.6 (-17.3-28.6)	0.59
ISS^b	0 (-4-1)	-2 (-2-1)	-2 (-2-2)	0.30
CMTNS^b	1 (-5-4)	-2 (-3-1)	-3 (-6-3)	0.14
ODSS^b	0 (0-1)	0 (-2-0)	0 (-2-0)	0.18
ALDS^a	1.0 (3.7)	0.3 (0.4)	-0.6 (-4.5-3.2)	0.30
Nine hole peg test (s)^a	-3.5 (2.8)	-2.0 (1.8)	1.5 (-1.8-4.8)	0.32
50 m walking (s)^a	-0.6 (1.9)	-0.9 (1.8)	-0.3 (-2.9-2.2)	0.77

Change = ^a mean (standard deviation) or ^b median (range) change scores from baseline to final follow-up. Difference = difference in ^a mean or ^b median change scores (95% confidence interval) of the patients receiving ascorbic acid and those receiving placebo. P-value = P-value for ^a the unpaired t test (equal variances assumed) or ^b the Mann-Whitney U test. * n = 5
Abbreviations: ALDS = Academic Medical Centre linear disability score; CMAP = compound muscle action potential; CMTNS = Charcot-Marie-Tooth neuropathy score; ISS = inflammatory neuropathy cause and treatment sensory sum score; MNCV = motor nerve conduction velocity; ODSS = overall disability sum score

ISSN: 1741-7015