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Clinical Trials

Start of the PLEO-CMT trial: phase III clinical trial on PXT3003 drug


A phase III clinical trial is currently ongoing in patients with type 1A Charcot-Marie-Tooth disease (CMT1A) to evaluate the efficacy and safety of the product PXT3003. The PXT3003 is a combination of three known and already marketed drugs, combined in a drinkable solution. The sponsor of this clinical trial is Pharnext.

This international clinical trial involves a total of 7 countries (France, Germany, United Kingdom, Belgium, Spain, Netherlands and USA).

In UK, PLEO-CMT has obtained the positive opinion of the ethics committee and the authorization of the Medicines and Healthcare Products Regulatory Agency. This clinical trial is coordinated by Professor Mark Roberts, from Salford Royal NHS Foundation Trust, Manchester. The participating centers are the following:

  • Department of Neurology, Salford Royal NHS Foundation Trust, Salford, Manchester (contact Pr Mark Roberts)
  • Ninewells Hospital and Medical School, Dundee, Scotland (contact Dr Cathy White)

Overall 300 patients with CMT1A, aged 16 to 65 years, will participate in the study, provided to meet a set of criteria. Among these criteria, the genetic diagnosis of CMT1A must have been completed (showing a duplication of the PMP22 gene on chromosome 17). A physician will perform clinical and biological tests to determine if you can participate in this clinical trial (screening visit).

Once this step validated, your participation in the study will take place over a period of 15 months, with 6 hospital visits. You will receive randomly either the treatment being tested (dose 1 and dose 2 of PXT3003) or the placebo. Neither the physician nor you will know which product will be given to you (this is a randomized, double-blind, placebo-controlled clinical trial).

At the end of this clinical trial, it will be offered to participants who completed the study (15 months treatment) the opportunity to extend the trial for 9 additional months, during which all participants will receive the active product PXT3003 (the participants who received the placebo will receive randomly either dose 1 or dose 2 of PXT3003).


For more information, you can consult the following website: https://clinicaltrials.gov/ct2/show/study/NCT02579759?term=pxt3003&rank=2


Thank you for your interest.

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