title: 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].
abstract: 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.
authors.affiliation: Department of Biochemistry and Genetics, C. Besta National Neurological Institute, Via Celoria 11, 20133 Milan, Italy. dpareys@istituto-besta.it
authors.forename: Davide
authors.initials: D
authors.lastname: Pareyson
authors.forename: Angelo
authors.initials: A
authors.lastname: Schenone
authors.forename: Gian Maria
authors.initials: GM
authors.lastname: Fabrizi
authors.forename: Lucio
authors.initials: L
authors.lastname: Santoro
authors.forename: Luca
authors.initials: L
authors.lastname: Padua
authors.forename: Aldo
authors.initials: A
authors.lastname: Quattrone
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Vita
authors.forename: Franco
authors.initials: F
authors.lastname: Gemignani
authors.forename: Francesco
authors.initials: F
authors.lastname: Visioli
authors.forename: Alessandra
authors.initials: A
authors.lastname: Solari
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000975:Antioxidants / Q000627:therapeutic use*; D001205:Ascorbic Acid / Q000627:therapeutic use*; D002607:Charcot-Marie-Tooth Disease / Q000188:drug therapy*; D004311:Double-Blind Method; D023381:Endpoint Determination; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007558:Italy; D008297:Male; D008875:Middle Aged; D018401:Sample Size
chemical_list: D000975:Antioxidants; D001205:Ascorbic Acid