BRIEF—Green light for study to go in inherited neurological disorder

France’s neurodegenerative diseases specialist Pharnext (FR0011191287 - ALPHA) has announced successful intermediate analyses for its Phase III clinical trial, PLEO-CMT, testing PXT3003 in the inherited neurological disorder Charcot-Marie-Tooth Disease Type 1A (CMT1A).

The study started in December 2015, enrolling 323 patients with mild-to-moderate CMT1A in 30 sites across Europe, the USA and Canada. There have been two intermediate analyses, a blind variability analysis followed by a futility analysis, carried out as planned.

According to Professor Philippe Lehert, member of the Independent Data Safety Monitoring Board (DSMB), the variability of tests between patients is indeed within predefined limits.

In addition, the futility analysis concludes that PLEO-CMT is sufficiently powered to detect an effect of PXT3003 on the primary efficacy endpoint.

These two analyses follow two favorable intermediate safety analyses delivered by the DSMB in November 2016 and September 2017 and indicate therefore that PLEO-CMT can continue according to the original plan without having to increase the trial size.