Publication of the results of the PLEO-CMT trial evaluating the PXT3003 in CMT1A

• PXT3003 is a low dose combination of baclofen, naltrexone and sorbitol which is believed to act synergistically to inhibit the excessive expression of PMP22 in Charcot-Marie-Tooth disease type 1A. 

• A phase III, double-blind trial evaluated two doses of PXT3003 in 323 people with CMT 1A for 15 months. It showed : 

• a decrease in the Overall Neuropathy Limitations Scale (ONLS) score of 0.37 in those treated with the highest dose compared to those on placebo, reflecting clinical improvement; 
• statistically improved performance on the 10-meter walk test in the high-dose group compared to the placebo group;
• no adverse effects related to treatment.

• A new phase III trial of PXT3003 is currently underway, with the participation of 6 centers in France (Angers, Bordeaux, Lille, Marseille, Paris – La Pitié Salpetrière- and Strasbourg).

 

A double-blind, placebo-controlled, randomized trial of PXT3003 for the treatment of Charcot-Marie-Tooth type 1A. Shahram Attarian, Peter Young, Thomas H Brannagan et al. Orphanet J Rare Dis. 2021 (Oct)