Clinical Trial of Mexiletine Hydrochloride for Spinal and Bulbar Muscular Atrophy

NCT06862596 | Recruiting Phase 2

• 2 other identifiers: CAMCR-026jRCT2041240174
• Study Type: interventional
• Target: 68
• Locations: 1 country
• Sites: 5

Brief Summary

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.

Conditions

Spinal and Bulbar Muscular Atrophy

Outcome Measures

Primary Outcomes (1)

• 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.

  at 4weeks

Secondary Outcomes (8)

• 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

Study Arms (2)

Mexiletine group

ACTIVE COMPARATOR

Drug: Mexiletine hydrochloride

Placebo group

PLACEBO COMPARATOR

Other: Placebo

Interventions

Mexiletine hydrochloride DRUG

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

Mexiletine group

Placebo OTHER

Placebo is administered orally divided into three times a day after meals for 12 weeks.

Placebo group

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.)

You may not qualify if:

• 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

Sponsors & Collaborators

• Masahisa Katsuno: lead

Study Sites (5)

Tokyo University Hospital

Bunkyō City, Japan

RECRUITING

Chiba University Hospital

Chiba, Japan

RECRUITING

Hokkaido University Hospital

Sapporo, Japan

RECRUITING

Jichi Medical University Hospital

Shimotsuke, Japan

RECRUITING

Osaka University Hospital

Suita, Japan

RECRUITING

MeSH Terms

Conditions

Bulbo-Spinal Atrophy, X-Linked

Interventions

Mexiletine

Condition Hierarchy (Ancestors)

Muscular Atrophy, Spinal; Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Motor Neuron Disease; Neuromuscular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Propylamines; Amines; Organic Chemicals; Phenyl Ethers; Phenols; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons

Study Officials

• Masahisa Katsuno, PhD, MD

  Nagoya University Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Masahisa Katsuno, PhD, MD

CONTACT

+81527442389; katsuno.masahisa.i1@f.mail.nagoya-u.ac.jp

Shinobu Shimizu, PhD

CONTACT

+81527442942; shimizu.shinobu.w3@f.mail.nagoya-u.ac.jp

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: February 26, 2025
• First Posted: March 6, 2025
• Study Start: February 28, 2025
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: April 16, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will share

Locations

JP (5)