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

Autor: Philippe Lehert, Catherine Scart-Grès, Viviane Bertrand, Mickaël Guedj, Laurent Magy, Daniel Cohen, Jonas Mandel, Shahram Attarian, Joëlle Micallef, Ilya Chumakov
Přispěvatelé: Pharnext SAS, Faculty of Medicine, University of Melbourne, Faculty of Economics, UcLouvain - Louvain School of Management - Campus Mons Management department, Génétique Médicale et Génomique Fonctionnelle (GMGF), Aix Marseille Université (AMU)-Assistance Publique - Hôpitaux de Marseille (APHM)- Hôpital de la Timone [CHU - APHM] (TIMONE)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), CHU Limoges, Institut de Neurosciences de la Timone (INT), Aix Marseille Université (AMU)-Centre National de la Recherche Scientifique (CNRS), HAL AMU, Administrateur
Jazyk: angličtina
Rok vydání: 2015
Předmět:
2ND VERSION
NEUROPATHY SCORE
CMTNS
[SDV]Life Sciences [q-bio]
Antioxidants
0302 clinical medicine
Clinical trials
Charcot-Marie-Tooth Disease
Medicine
ONLS
Genetics(clinical)
Pharmacology (medical)
Letter to the Editor
Genetics (clinical)
Randomized Controlled Trials as Topic
Medicine(all)
0303 health sciences
[SDV.MHEP] Life Sciences [q-bio]/Human health and pathology
PXT3003
Inherited Peripheral Neuropathy
General Medicine
3. Good health
[SDV] Life Sciences [q-bio]
CMT1A
Meta-analysis
RELIABILITY
Ascorbic acid
[SDV.NEU]Life Sciences [q-bio]/Neurons and Cognition [q-bio.NC]
EXPRESSION
medicine.medical_specialty
Charcot-Marie-Tooth
Randomized
Placebo
Double blind
03 medical and health sciences
Internal medicine
Humans
In patient
[SDV.NEU] Life Sciences [q-bio]/Neurons and Cognition [q-bio.NC]
MARIE-TOOTH-DISEASE
COMBINATION
030304 developmental biology
ASCORBIC-ACID TREATMENT
business.industry
medicine.disease
Clinical trial
Peripheral neuropathy
business
1A
030217 neurology & neurosurgery
PERIPHERAL NEUROPATHY
[SDV.MHEP]Life Sciences [q-bio]/Human health and pathology
Zdroj: Orphanet Journal of Rare Diseases
Orphanet Journal of Rare Diseases, 2015
Orphanet Journal of Rare Diseases, BioMed Central, 2015
ISSN: 1750-1172
Popis: International audience; 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.
Databáze: OpenAIRE
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