Guest guest Posted April 10, 2007 Report Share Posted April 10, 2007 I just found my info on the Italian trials. It is double-blind, placebo controlled, with over 200 people with confirmed CMT 1A. In Phase 3, going on now, participants are either getting 1500 mg daily or a placebo. There is a reason participants are given the DAILY DOSE. This trial goes on for 34 months. See below for Abstract (originally I think I posted this last December) ~ Gretchen Pharmacol Res. 2006 Dec;54(6):436-41. 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; 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. Quote Link to comment Share on other sites More sharing options...
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