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

• Published in: BMC medicine 2009-11, Vol.7 (1), p.70-70, Article 70
• Main Authors: Verhamme, Camiel, de Haan, Rob J, Vermeulen, Marinus, Baas, Frank, de Visser, Marianne, van Schaik, Ivo N
• Format: Article
• Language: English
• Subjects: Administration, Oral; Adolescent; Ascorbic Acid - administration & dosage; Ascorbic Acid - adverse effects; Ascorbic Acid - therapeutic use; Care and treatment; Charcot-Marie-Tooth disease; Charcot-Marie-Tooth Disease - drug therapy; Dosage and administration; Double-Blind Method; Female; Humans; Male; Median Nerve - physiology; Muscle Strength - drug effects; Myelination; Neural Conduction - physiology; Patient outcomes; Physiological aspects; Placebos - administration & dosage; Research article; Sensation - physiology; Severity of Illness Index; Treatment Outcome; Vitamin C; Vitamins - administration & dosage; Vitamins - adverse effects; Vitamins - therapeutic use; Young Adult
• ISSN: 1741-7015; 1741-7015
• DOI: 10.1186/1741-7015-7-70

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. 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. 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. 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. Current Controlled Trials ISRCTN56968278, ClinicalTrials.gov NCT00271635.