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]

Davide Pareyson, Angelo Schenone, Gian Maria Fabrizi, Lucio Santoro, Luca Padua, Aldo Quattrone, Giuseppe Vita, Franco Gemignani, Francesco Visioli, Alessandra Solari, E. Salsano, V. Scaioli, C. Ciano, M. Rimoldi, G. Lauria, E. Rizzetto, F. Camozzi, M. Grandis, E. Narciso, L. NobbioL. Benedetti, N. Rizzuto, T. Cavallaro, L. Bertolasi, A. Casano, F. Manganelli, M. Nolano, C. Pazzaglia, P. Valentino, R. Nisticò, 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, M. Rimoldi., V. Scaioli, G. Lauria

Research output: Contribution to journalArticlepeer-review

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 (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.

Original languageEnglish
Pages (from-to)436-441
Number of pages6
JournalPharmacological Research
Volume54
Issue number6
DOIs
Publication statusPublished - Dec 1 2006

Keywords

  • Ascorbic acid
  • Charcot-Marie-Tooth disease
  • Genetic disorders
  • Neuropathy
  • PMP22
  • Randomized controlled trial

ASJC Scopus subject areas

  • Pharmacology

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