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Recruiting Phase 2/3

Clinical Trial of Mexiletine Hydrochloride for Spinal and Bulbar Muscular Atrophy

NCT06862596 · tracked via the Priya Life Science Japan tracker
Sponsor
Masahisa Katsuno
Phase
Phase 2/3
Started
2025-02-28
Last updated
2025-04-16

Condition(s) studied

Spinal and Bulbar Muscular Atrophy

Investigational drug(s) / intervention(s)

Mexiletine hydrochloridePlacebo

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

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

Study 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.

Eligibility

Sex
MALE
Min age
18 Years
Max age
80 Years
Healthy volunteers
No
Inclusion Criteria: 1. 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 2. Patients with muscle weakness (limb weakness and atrophy, or bulbar palsy) due to lower motor neuron lesion 3. Patients with a total ALSFRS-R score of ≥ 24 and ≤ 42 at screening 4. Patients who are at least 18 years old and less than 80 years old at the time of consent 5. 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: 1. Patients who have participated or are participating in a clinical trial within 12 weeks prior to enrollment 2. Patients with a history of hypersensitivity to any component of this drug product 3. Patients with a conduction disturbance (such as second- or third-degree atrioventricular block without a pacemaker, or left bundle branch block) 4. Patients with Brugada-type ECG 5. Patients with severe heart failure or heart disease (myocardial infarction, valvular disease, cardiomyopathy, etc.) 6. Patients with sinus bradycardia (\<50 beats/minute) 7. Patients with systolic blood pressure of 90 mmHg or less 8. Patients with serum potassium level less than 3.5 mmol/L 9. Patients on antiarrhythmic drugs 10. Patients on antiepileptic drugs that affect to sodium channels 11. Patients on theophylline 12. Patients on narcotics 13. Patients who used Mexiletine within 1 month prior to enrollment or used Mexiletine for expectations of improvement in symptoms of SBMA 14. Patients with serious complications 15. Patients who cannot agree to use contraception during the study period 16. Other Patients deemed inappropriate by the investigator or subinvestigator

Primary outcome measure(s)

  • Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score — 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.

Trial sites (5)

FacilityCityRegionStatus
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
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06862596 on ClinicalTrials.gov ↗ ← All trials in Japan