Oral high dose ascorbic acid treatment for one year in young CMT1A patients: a r

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BMC Med. 2009 Nov 12;7(1):70. [Epub ahead of print]

Oral high dose ascorbic acid treatment for one year in young CMT1A patients: a

randomised, double-blind, placebo-controlled phase II trial.

Verhamme C, de Haan RJ, Vermeulen M, Baas F, de Visser M, van Schaik IN.

ABSTRACT:

BACKGROUND: High dose oral ascorbic acid substantially improved myelination and

locomotor function in a Charcot-Marie-Tooth type 1A mouse model.

A phase II study was warranted to investigate whether high dose ascorbic acid

also has such a substantial effect on myelination in Charcot-Marie-Tooth type 1A

patients and whether this treatment is safe.

METHODS: Patients below age 25 years were randomly assigned to receive placebo

or ascorbic acid (one gram twice daily) in a double-blind fashion during one

year. The primary outcome measure was the change over time in motor nerve

conduction velocity of the median nerve. Secondary outcome measures included

changes in minimal F response latencies, compound muscle action potential

amplitude, muscle strength, sensory function, Charcot-Marie-Tooth neuropathy

score, and disability.

RESULTS: There were no significant differences between the six placebo-treated

(median age 16 years, range 13 to 24) and the five ascorbic acid-treated (19, 14

to 24) patients in change in motor nerve conduction velocity of the median nerve

(mean difference ascorbic acid as opposed to placebo treatment of 1.3 m/s,

confidence interval -0.3 to 3.0 m/s, P = 0.11) or in change of any of the

secondary outcome measures over time.

One patient in the ascorbic acid group developed a skin rash, which led to

discontinuation of the study medication.

CONCLUSIONS: Oral high dose ascorbic acid for one year did not improve

myelination of the median nerve in young Charcot-Marie-Tooth type 1A patients.

Treatment was relatively safe. Trial registration: Current Controlled Trials

ISRCTN56968278, ClinicalTrials.gov NCT00271635.