The purpose of this clinical trial is to evaluate the efficacy and safety of mexiletine hydrochloride in patients with spinal and bulbar muscular atrophy.
The main questions it aims to answer are:
Does mexiletine hydrochloride improve the ALSFRS-R score in spinal and bulbar muscular atrophy patients?
Participants will:
Take mexiletine hydrochloride or a placebo every day for 3 months Visit the hospital once every 4 weeks for evaluations.
Male patients with a CAG repeat count of 38 or more for the androgen receptor gene in genetic testing and a confirmed diagnosis of SBMA
Patients with muscle weakness (limb weakness and atrophy, or bulbar palsy) due to lower motor neuron lesion
Patients with a total ALSFRS-R score of ≥ 24 and ≤ 42 at screening
Patients who are at least 18 years old and less than 80 years old at the time of consent
Patients who have participated or are participating in a clinical trial within 12 weeks prior to enrollment
Patients with a history of hypersensitivity to any component of this drug product
Patients with a conduction disturbance (such as second- or third-degree atrioventricular block without a pacemaker, or left bundle branch block)
Patients with Brugada-type ECG
Parallel
Mexiletine hydrochloride
DrugMexiletine hydrochloride 300 mg is administered orally divided into three times a day after meals for 12 weeks.
Placebo
Other interventionPlacebo is administered orally divided into three times a day after meals for 12 weeks.
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score
The ALSFRS-R is a comprehensive severity index comprising 12 items covering bulbar, upper limb, lower limb, and respiratory symptoms to evaluate the ADLs of patients with amyotrophic lateral sclerosis (ALS). Each item is rated on a five-point scale from 0 (worse) to 4 (better), and a total score (miniimum 0point and maximum 48 point) is calculated.
ALSFRS-R Score
The ALSFRS-R is a comprehensive severity index comprising 12 items covering bulbar, upper limb, lower limb, and respiratory symptoms to evaluate the ADLs of patients with ALS. Each item is rated on a five-point scale from 0 (worse) to 4 (better), and a total score (miniimum 0point and maximum 48 point) is calculated.
Spinal and Bulbar Muscular Atrophy Functional Rating Scale (SBMAFRS) score
The SBMAFRS is a disease-specific motor function assessment scale for Spinal and Bulbar Muscular Atrophy (SBMA).The SBMAFRS is a comprehensive severity index comprising 14 items covering bulbar, upper limb, lower limb, and respiratory symptoms to evaluate the ADLs of patients. Each item is rated on a five-point scale from 0 (worse) to 4 (better), and a total score (miniimum 0point and maximum 56 point) is calculated.
Grip strength
Grip strength (kg) is set to evaluate the upper limb motor function of patients with SBMA.
Tongue pressure
Tongue pressure (kPa) is set to evaluate the bulbar function of patients with SBMA.
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Masahisa Katsuno
Lead sponsor
Nagoya University
Sponsor institution
