PLEO-CMT

• Research type

  Research Study

• Full title

  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

• IRAS ID

  196861

• Contact name

  Mark Roberts

• Contact email

  mark.roberts@srft.nhs.uk

• Sponsor organisation

  PHARNEXT SA

• Eudract number

  2015-002378-19

• Duration of Study in the UK

  2 years, 0 months, 20 days

• Research summary

  A Phase III Double-blind, randomized, placebo-controlled, 3 parallel groups, international multi-center study for 15 months (65 weeks).

  The purpose of the study is to find out how effective PXT3003 is compared to Placebo on the disability measured by the ONLS score in patients with Charcot-Marie-Tooth disease type 1A (CMT1A) treated for 15 months.

  A total of 300 patients would participate in this study in sites throughout UK, Europe and US.
  100 participants would receive PXT3003 Dose 1, 100 participants would receive PXT3003 Dose 2 and another 100 would receive matching Placebo. Participants would be randomized after a screening period (up to 30 days) during which selection criteria are verified and baseline assessments such as electrophysiological testing, lab tests and ECG would be performed. Then, after randomization, participants would be included in the study under double blind study medication for 15 months. At the end of the study, if participants wish to continue, they would have the opportunity to participate in a second study for an additional period of 9 months during which they would receive one of both doses of the treatment. There would be no placebo in this follow up study so that all participants can be treated with the PXT3003.
  Participants would be asked to sign a consent form and would be screened to determine their eligibility to take part in the study. Only patients who have Charcot-Marie-Tooth type 1A (CMT1A) Disease and meet other inclusion criteria would be eligible to participate in this study.

  Some of the procedures/assessments that the participants would undergo include the following:
  • Questions about any current or previous illnesses
  • Diagnosis history (DNA) and treatments
  • Electophysiology (nerve conductions studies)
  • Blood sampling, ECG
  • Review of adverse events

  It is possible that participants taking part in this study may find no benefit to them, but the information obtained from this study will help in future to improve the treatment of people with Charcot-Marie-Tooth disease type 1A.

  Pharnext is sponsor of the study.

• REC name

  East Midlands - Leicester South Research Ethics Committee

• REC reference

  16/EM/0127

• Date of REC Opinion

  10 May 2016

• REC opinion

  Further Information Favourable Opinion