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

Daniel Cohen⁵  Mickael Guedj⁵  Catherine Scart-Grès⁵  Ilya Chumakov⁵  Joëlle Micallef²  Laurent Magy⁴  Shahram Attarian³  Philippe Lehert¹  Viviane Bertrand⁵  Jonas Mandel⁵ 

[1] Faculty of Economics, UCL Mons, Louvain, Belgium;CIC-Centre de Pharmacologie Clinique et D’Evaluations Thérapeutiques, AP-HM et Aix Marseille Université, Marseille, France;Centre de référence des maladies neuromusculaires et de la SLA, Pôle des neurosciences Cliniques, AP-HM et Aix Marseille Université, Marseille, France;CHU de Limoges Hôpital Dupuytren, Limoges, France;Pharnext SAS, Issy-les-Moulineaux, France

关键词: Double blind;    Randomized;    Clinical trials;    Ascorbic acid;    Meta-analysis;    PXT3003;    ONLS;    CMTNS;    CMT1A;    Charcot-Marie-Tooth;   

Others  :  1219127
DOI  :  10.1186/s13023-015-0293-y

 received in 2015-04-24, accepted in 2015-06-04,  发布年份 2015

【 摘 要 】

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.

【 授权许可】

   
2015 Mandel et al.

【 图 表 】

Fig. 1.

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