Ascorbic Acid (Vit. C) Italian Trial Protocol

[Guest guest]

Pharmacol Res. 2006 Sep 9

A multicenter, randomized, double-blind, placebo-controlled trial of

long-term ascorbic acid treatment in Charcot-Marie-Tooth disease

type 1A (CMT-TRIAAL): The study protocol [EudraCT no.: 2006-000032-

27].

Pareyson D, Schenone A, Fabrizi GM, Santoro L, Padua L, Quattrone A,

Vita G, Gemignani F, Visioli F, Solari A; on behalf of the CMT-

TRIAAL Group.

Department of Biochemistry and Genetics, " C. Besta " National

Neurological Institute, Via Celoria 11, 20133 Milan, Italy.

There is no treatment for Charcot-Marie-Tooth disease 1A (CMT1A),

but ascorbic acid (AA) is efficacious in the transgenic mouse model.

Thus, a clinical trial of AA in CMT1A is warranted. The CMT-TRIAAL

is a phase III randomized, double-blind, placebo-controlled study

involving 222 CMT1A adults from eight Italian centers. Eligible for

the study are symptomatic adults with genetically confirmed CMT1A.

Treatment consists of 2-year oral AA (1500mg/day) or placebo. The

primary trial endpoint is an improvement in CMT Neuropathy Score.

Secondary efficacy endpoints are changes in distal arm and leg

maximum voluntary isometric contraction; 10m timed walking; 9-hole-

peg test; overall neuropathy limitations scale; pain and fatigue

visual analog scales; health-related quality of life (SF-36); and

electrophysiology. Clinical-electrophysiological assessments are

performed at baseline and every 6 months thereafter. In consenting

patients from three centers, skin biopsy is performed to evaluate

PMP22 expression. The study will last 34 months, starting from March

2006.