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Recruiting NCT06862596

Clinical Trial of Mexiletine Hydrochloride for Spinal and Bulbar Muscular Atrophy

Phase II / Phase III Interventional Spinal and Bulbar Muscular Atrophy

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Mexiletine hydrochloride, Placebo.
Who it may be relevant to
Registry conditions: Spinal and Bulbar Muscular Atrophy. Basic parameters: 18 years — 80 years · Male.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Japan
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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.

Interventions

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

Primary outcome measures

  • Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score [Time frame: at 4weeks]
Secondary outcome measures (8)
  • ALSFRS-R Score [Time frame: up to 12 weeks]
  • Spinal and Bulbar Muscular Atrophy Functional Rating Scale (SBMAFRS) score [Time frame: up to 12 weeks]
  • Grip strength [Time frame: up to 12 weeks]
  • Tongue pressure [Time frame: up to 12 weeks]
  • Timed walk test (4.6 meters) [Time frame: up to 12 weeks]
  • 6-minute walk test [Time frame: up to 12 weeks]
  • Respiratory function test (Forced Vital Capacity (FVC)) [Time frame: up to 12 weeks]
  • Respiratory function test (Peak Expiratory Flow (PEF)) [Time frame: up to 12 weeks]

Eligibility criteria

Inclusion criteria

  • 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.)

Exclusion criteria

  • 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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Quadruple blind
Primary purpose
Treatment

Study locations

Japan · 5 centers
  • Tokyo University Hospital — Bunkyō City
  • Chiba University Hospital — Chiba
  • Hokkaido University Hospital — Sapporo
  • Jichi Medical University Hospital — Shimotsuke
  • Osaka University Hospital — Suita

Identifiers

NCT: NCT06862596 · CAMCR-026 · jRCT2041240174

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗