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Study protocol for the MEXiletine hydrochloride administration trial: a placebo-controlled, randomised, double-blind, multicentre, crossover study of its efficacy and safety in spinal and bulbar muscular atrophy (MEXPRESS)

INTRODUCTION: Spinal and bulbar muscular atrophy (SBMA) is a slowly progressive neuromuscular disease. Cold exposure often leads to worsening of motor symptoms including paresis. Although mexiletine hydrochloride administration has been shown to be effective for the treatment of several muscular dis...

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Autores principales: Yamada, Shinichiro, Hashizume, Atsushi, Hijikata, Yasuhiro, Inagaki, Tomonori, Ito, Daisuke, Kinoshita, Fumie, Nakatochi, Masahiro, Kobayashi, Yumiko, Hirakawa, Akihiro, Nakamura, Tomohiko, Katsuno, Masahisa
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6144396/
https://www.ncbi.nlm.nih.gov/pubmed/30206090
http://dx.doi.org/10.1136/bmjopen-2018-023041
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author Yamada, Shinichiro
Hashizume, Atsushi
Hijikata, Yasuhiro
Inagaki, Tomonori
Ito, Daisuke
Kinoshita, Fumie
Nakatochi, Masahiro
Kobayashi, Yumiko
Hirakawa, Akihiro
Nakamura, Tomohiko
Katsuno, Masahisa
author_facet Yamada, Shinichiro
Hashizume, Atsushi
Hijikata, Yasuhiro
Inagaki, Tomonori
Ito, Daisuke
Kinoshita, Fumie
Nakatochi, Masahiro
Kobayashi, Yumiko
Hirakawa, Akihiro
Nakamura, Tomohiko
Katsuno, Masahisa
author_sort Yamada, Shinichiro
collection PubMed
description INTRODUCTION: Spinal and bulbar muscular atrophy (SBMA) is a slowly progressive neuromuscular disease. Cold exposure often leads to worsening of motor symptoms including paresis. Although mexiletine hydrochloride administration has been shown to be effective for the treatment of several muscular diseases, its effectiveness in SBMA has not been validated to date. The trial will test it as a symptomatic drug for cold paresis. This study is the first trial to evaluate the efficacy and safety of mexiletine hydrochloride administration in patients with SBMA. METHODS AND ANALYSIS: A placebo-controlled, randomised, double-blind, multicentre, crossover clinical trial will be conducted to assess the safety and efficacy of mexiletine hydrochloride in patients with SBMA. The eligible patients will be assigned randomly in a 1:1 ratio to two groups in a double-blind manner. Participants will take mexiletine hydrochloride (300 mg/day) or a placebo orally three times a day for 4 weeks (period 1). After a 1-week washout period, participants will take the other drug for 4 weeks (period 2). The primary endpoint is the difference in distal latencies between room temperature and cold exposure conditions. ETHICS AND DISSEMINATION: This study will be conducted in compliance with the Helsinki Declaration and the Ethical Guidelines for Medical and Health Research Involving Human Subjects by the Japanese government and has been approved by the ethics committee of Nagoya University Graduate School of Medicine, as a central institutional review board, and by each facility. The results will be disseminated in peer-reviewed journals and at scientific conferences. TRIAL REGISTRATION NUMBER: UMIN000026150; Pre-results.
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spelling pubmed-61443962018-09-21 Study protocol for the MEXiletine hydrochloride administration trial: a placebo-controlled, randomised, double-blind, multicentre, crossover study of its efficacy and safety in spinal and bulbar muscular atrophy (MEXPRESS) Yamada, Shinichiro Hashizume, Atsushi Hijikata, Yasuhiro Inagaki, Tomonori Ito, Daisuke Kinoshita, Fumie Nakatochi, Masahiro Kobayashi, Yumiko Hirakawa, Akihiro Nakamura, Tomohiko Katsuno, Masahisa BMJ Open Neurology INTRODUCTION: Spinal and bulbar muscular atrophy (SBMA) is a slowly progressive neuromuscular disease. Cold exposure often leads to worsening of motor symptoms including paresis. Although mexiletine hydrochloride administration has been shown to be effective for the treatment of several muscular diseases, its effectiveness in SBMA has not been validated to date. The trial will test it as a symptomatic drug for cold paresis. This study is the first trial to evaluate the efficacy and safety of mexiletine hydrochloride administration in patients with SBMA. METHODS AND ANALYSIS: A placebo-controlled, randomised, double-blind, multicentre, crossover clinical trial will be conducted to assess the safety and efficacy of mexiletine hydrochloride in patients with SBMA. The eligible patients will be assigned randomly in a 1:1 ratio to two groups in a double-blind manner. Participants will take mexiletine hydrochloride (300 mg/day) or a placebo orally three times a day for 4 weeks (period 1). After a 1-week washout period, participants will take the other drug for 4 weeks (period 2). The primary endpoint is the difference in distal latencies between room temperature and cold exposure conditions. ETHICS AND DISSEMINATION: This study will be conducted in compliance with the Helsinki Declaration and the Ethical Guidelines for Medical and Health Research Involving Human Subjects by the Japanese government and has been approved by the ethics committee of Nagoya University Graduate School of Medicine, as a central institutional review board, and by each facility. The results will be disseminated in peer-reviewed journals and at scientific conferences. TRIAL REGISTRATION NUMBER: UMIN000026150; Pre-results. BMJ Publishing Group 2018-09-11 /pmc/articles/PMC6144396/ /pubmed/30206090 http://dx.doi.org/10.1136/bmjopen-2018-023041 Text en © Author(s) (or their employer(s)) 2018. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ. This is an open access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited, appropriate credit is given, any changes made indicated, and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/.
spellingShingle Neurology
Yamada, Shinichiro
Hashizume, Atsushi
Hijikata, Yasuhiro
Inagaki, Tomonori
Ito, Daisuke
Kinoshita, Fumie
Nakatochi, Masahiro
Kobayashi, Yumiko
Hirakawa, Akihiro
Nakamura, Tomohiko
Katsuno, Masahisa
Study protocol for the MEXiletine hydrochloride administration trial: a placebo-controlled, randomised, double-blind, multicentre, crossover study of its efficacy and safety in spinal and bulbar muscular atrophy (MEXPRESS)
title Study protocol for the MEXiletine hydrochloride administration trial: a placebo-controlled, randomised, double-blind, multicentre, crossover study of its efficacy and safety in spinal and bulbar muscular atrophy (MEXPRESS)
title_full Study protocol for the MEXiletine hydrochloride administration trial: a placebo-controlled, randomised, double-blind, multicentre, crossover study of its efficacy and safety in spinal and bulbar muscular atrophy (MEXPRESS)
title_fullStr Study protocol for the MEXiletine hydrochloride administration trial: a placebo-controlled, randomised, double-blind, multicentre, crossover study of its efficacy and safety in spinal and bulbar muscular atrophy (MEXPRESS)
title_full_unstemmed Study protocol for the MEXiletine hydrochloride administration trial: a placebo-controlled, randomised, double-blind, multicentre, crossover study of its efficacy and safety in spinal and bulbar muscular atrophy (MEXPRESS)
title_short Study protocol for the MEXiletine hydrochloride administration trial: a placebo-controlled, randomised, double-blind, multicentre, crossover study of its efficacy and safety in spinal and bulbar muscular atrophy (MEXPRESS)
title_sort study protocol for the mexiletine hydrochloride administration trial: a placebo-controlled, randomised, double-blind, multicentre, crossover study of its efficacy and safety in spinal and bulbar muscular atrophy (mexpress)
topic Neurology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6144396/
https://www.ncbi.nlm.nih.gov/pubmed/30206090
http://dx.doi.org/10.1136/bmjopen-2018-023041
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