Clinical Trial of Mexiletine Hydrochloride for Spinal and Bulbar Muscular Atrophy

Официальное название

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

Обзор

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.

Вмешательства

• Препарат Mexiletine hydrochloride
  Mexiletine hydrochloride 300 mg is administered orally divided into three times a day after meals for 12 weeks.
• Другое Placebo
  Placebo is administered orally divided into three times a day after meals for 12 weeks.

Первичные конечные точки

• Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score [Срок оценки: at 4weeks]

Вторичные конечные точки (8)

• ALSFRS-R Score [Срок оценки: up to 12 weeks]
• Spinal and Bulbar Muscular Atrophy Functional Rating Scale (SBMAFRS) score [Срок оценки: up to 12 weeks]
• Grip strength [Срок оценки: up to 12 weeks]
• Tongue pressure [Срок оценки: up to 12 weeks]
• Timed walk test (4.6 meters) [Срок оценки: up to 12 weeks]
• 6-minute walk test [Срок оценки: up to 12 weeks]
• Respiratory function test (Forced Vital Capacity (FVC)) [Срок оценки: up to 12 weeks]
• Respiratory function test (Peak Expiratory Flow (PEF)) [Срок оценки: up to 12 weeks]

Критерии участия

Критерии включения

• 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.)

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Четверное слепое

Основная цель

Лечение

Центры проведения

Япония · 5 центров

• Tokyo University Hospital — Bunkyō City
• Chiba University Hospital — Chiba
• Hokkaido University Hospital — Sapporo
• Jichi Medical University Hospital — Shimotsuke
• Osaka University Hospital — Suita

Идентификаторы

NCT: NCT06862596 · CAMCR-026 · jRCT2041240174

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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