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

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

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

Camiel Verhamme , Rob J de Haan , Marinus Vermeulen , Baas , nne de

Visser and Ivo N van Schaik

BMC Medicine Published: 12 November 2009

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.