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 (1500 mg/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; 10 m 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.
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] / D. Pareyson, A. Schenone, G.M. Fabrizi, L. Santoro, L. Padua, A. Quattrone, G. Vita, F. Gemignani, F. Visioli, A. Solari, E. Salsano, V. Scaioli, C. Ciano, M. Rimoldi, G. Lauria, E. Rizzetto, F. Camozzi, M. Grandis, E. Narciso, L. Nobbio, L. Benedetti, N. Rizzuto, T. Cavallaro, L. Bertolasi, A. Casano, F. Manganelli, M. Nolano, C. Pazzaglia, P. Valentino, R. Nistico, D. Pirritano, A. Lucisano, M. Canino, A. Mazzeo, M. Aguennouz, R. Di Leo, P. Girlanda, G. Majorana, A. Ciranni, N. Lanzano, F. Brindani, C. Lettieri, P. Bogani, R.A.C. Hughes, G.L. Mancardi, G. Cavaletti, S. Galimberti, D. Radice, D. Calabrese, G. Ferrari. - In: PHARMACOLOGICAL RESEARCH. - ISSN 1096-1186. - 54:6(2006), pp. 436-441.
|Titolo:||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]|
|Parole Chiave:||Charcot-Marie-Tooth disease; ascorbic acid; randomized controlled trial; neuropathy; genetic disorders; PMP22|
|Settore Scientifico Disciplinare:||Settore MED/26 - Neurologia|
|Data di pubblicazione:||2006|
|Digital Object Identifier (DOI):||http://dx.doi.org/10.1016/j.phrs.2006.09.001|
|Appare nelle tipologie:||01 - Articolo su periodico|