NCT06862596招募中2 期
A Multicenter, Randomized, Placebo-controlled, Double-blind Clinical Trial: The Efficacy and Safety of Mexiletine Hydrochloride for Amelioration of Motor Dysfunction in Spinal and Bulbar Muscular Atrophy
Masahisa Katsuno5 个研究点 分布在 1 个国家目标入组 68 人开始时间: 2025年2月28日最近更新:
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 68
- 试验地点
- 5
- 主要终点
- Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •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 give their voluntary written consent after having received adequate information on this study (However, if the patient is unable to sign the consent form due to the condition of the disease, a person equivalent to a regal representative must be present to provide written explanation, the prospective candidate must verbally consent to participate in the study, and a person equivalent to a regal representative must sign the consent form on behalf of the patient. The person who is to be the regal representative may sign the document on his/her behalf, noting the circumstances and his/her relationship to the subject.)
排除标准
- •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
- •Patients with severe heart failure or heart disease (myocardial infarction, valvular disease, cardiomyopathy, etc.)
- •Patients with sinus bradycardia (<50 beats/minute)
- •Patients with systolic blood pressure of 90 mmHg or less
- •Patients with serum potassium level less than 3.5 mmol/L
- •Patients on antiarrhythmic drugs
- •Patients on antiepileptic drugs that affect to sodium channels
- •Patients on theophylline
- •Patients on narcotics
- •Patients who used Mexiletine within 1 month prior to enrollment or used Mexiletine for expectations of improvement in symptoms of SBMA
- •Patients with serious complications
- •Patients who cannot agree to use contraception during the study period
- •Other Patients deemed inappropriate by the investigator or subinvestigator
研究组 & 干预措施
Mexiletine group
Active Comparator
干预措施: Mexiletine hydrochloride (Drug)
Placebo group
Placebo Comparator
干预措施: Placebo (Other)
结局指标
主要结局
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score
时间窗: at 4weeks
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.
次要结局
- ALSFRS-R Score(up to 12 weeks)
- Spinal and Bulbar Muscular Atrophy Functional Rating Scale (SBMAFRS) score(up to 12 weeks)
- Grip strength(up to 12 weeks)
- Tongue pressure(up to 12 weeks)
- Timed walk test (4.6 meters)(up to 12 weeks)
- 6-minute walk test(up to 12 weeks)
- Respiratory function test (Forced Vital Capacity (FVC))(up to 12 weeks)
- Respiratory function test (Peak Expiratory Flow (PEF))(up to 12 weeks)
研究者
Masahisa Katsuno
Professor
Nagoya University
研究点 (5)
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