At a glance
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International, Multi-center, Randomized, Double-blind, Placebo-controlled Phase III Study Assessing in Parallel Groups the Efficacy and Safety of 2 Doses of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A Treated 15 Months
In Brief
A Phase 3 clinical trial evaluating PXT3003 dose 1, PXT3003 dose 2, and 1 other intervention for Charcot-Marie-Tooth Disease Type 1A. Completed, enrolled 323 participants across 30 sites in 8 countries.
Detailed Summary
The purpose of this study is to determine whether PXT3003 is effective and safe in the treatment of Charcot-Marie-Tooth disease - Type 1 A (CMT1A). This double-blind study will assess in parallel groups 2 doses of PXT3003 compared to Placebo in CMT1A patients treated for 15 months.
Study Details
Timeline
Interventions
Liquid oral solution, 5 ml twice a day, morning and evening with food
Liquid oral solution, 5 ml twice a day, morning and evening with food
Liquid oral solution, 5 ml twice a day, morning and evening with food