A meta-analysis of randomized double-blind clinical trials in CMT1A to assess the change from baseline in CMTNS and ONLS scales after one year of treatment - Aix-Marseille Université Access content directly
Journal Articles Orphanet Journal of Rare Diseases Year : 2015

A meta-analysis of randomized double-blind clinical trials in CMT1A to assess the change from baseline in CMTNS and ONLS scales after one year of treatment

Jonas Mandel
  • Function : Author
Viviane Bertrand
  • Function : Author
Laurent Magy
  • Function : Author
Ilya Chumakov
  • Function : Author
Mickael Guedj
  • Function : Author
Daniel Cohen
  • Function : Author
  • PersonId : 760130
  • IdRef : 032639805

Abstract

CMT1A is the most common inherited peripheral neuropathy. There is currently no approved treatment. We performed a meta-analysis including four randomized, double-blind, Placebo-controlled clinical trials to assess the disease progression after one year under Placebo, Ascorbic Acid (AA) or PXT3003, a combination of three repurposed drugs. We observed a weak deterioration in patients under Placebo, well below the reported natural disease progression. Patients treated with AA were stable after one year but not significantly different from Placebo. Patients undergoing PXT3003 treatment showed an improvement in CMTNS and ONLS, statistically significant versus Placebo and potentially precursory of a meaningful change in the disease course.
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Dates and versions

hal-01203428 , version 1 (23-09-2015)

Identifiers

  • HAL Id : hal-01203428 , version 1

Cite

Jonas Mandel, Viviane Bertrand, Philippe Lehert, Shahram Attarian, Laurent Magy, et al.. A meta-analysis of randomized double-blind clinical trials in CMT1A to assess the change from baseline in CMTNS and ONLS scales after one year of treatment. Orphanet Journal of Rare Diseases, 2015. ⟨hal-01203428⟩
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