title: 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.
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.
authors.affiliation: Pharnext SAS, Issy-les-Moulineaux, France. jmandel@pharnext.com.
authors.forename: Jonas
authors.initials: J
authors.lastname: Mandel
authors.affiliation: Pharnext SAS, Issy-les-Moulineaux, France. viviane.bertrand@pharnext.com.
authors.forename: Viviane
authors.initials: V
authors.lastname: Bertrand
authors.affiliation: Faculty of Medicine, University of Melbourne, Melbourne, Australia. philippe.lehert@gmail.com.|Faculty of Economics, UCL Mons, Louvain, Belgium. philippe.lehert@gmail.com.
authors.forename: Philippe
authors.initials: P
authors.lastname: Lehert
authors.affiliation: Centre de référence des maladies neuromusculaires et de la SLA, Pôle des neurosciences Cliniques, AP-HM et Aix Marseille Université, Marseille, France.
authors.forename: Shahram
authors.initials: S
authors.lastname: Attarian
authors.affiliation: CHU de Limoges Hôpital Dupuytren, Limoges, France.
authors.forename: Laurent
authors.initials: L
authors.lastname: Magy
authors.affiliation: CIC-Centre de Pharmacologie Clinique et D'Evaluations Thérapeutiques, AP-HM et Aix Marseille Université, Marseille, France.
authors.forename: Joëlle
authors.initials: J
authors.lastname: Micallef
authors.affiliation: Pharnext SAS, Issy-les-Moulineaux, France. ilya.chumakov@pharnext.com.
authors.forename: Ilya
authors.initials: I
authors.lastname: Chumakov
authors.affiliation: Pharnext SAS, Issy-les-Moulineaux, France. catherine.scart-gres@pharnext.com.
authors.forename: Catherine
authors.initials: C
authors.lastname: Scart-Grès
authors.affiliation: Pharnext SAS, Issy-les-Moulineaux, France. mickael.guedj@pharnext.com.
authors.forename: Mickael
authors.initials: M
authors.lastname: Guedj
authors.affiliation: Pharnext SAS, Issy-les-Moulineaux, France. jmandel@pharnext.com.
authors.forename: Daniel
authors.initials: D
authors.lastname: Cohen
mesh_terms: D000975:Antioxidants / Q000627:therapeutic use; D001205:Ascorbic Acid / Q000627:therapeutic use; D002607:Charcot-Marie-Tooth Disease / Q000188:drug therapy*; D006801:Humans; D016032:Randomized Controlled Trials as Topic
chemical_list: D000975:Antioxidants; D001205:Ascorbic Acid