Evaluating mexiletine hydrochloride for spinal and bulbar muscular atrophy

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

This study is testing if a medication called mexiletine can help improve the daily functioning of people with spinal and bulbar muscular atrophy over three months.

Quick facts

• Phase: Phase2; Phase3
• Study type: Interventional
• Enrollment: 68 (estimated)
• Ages: 18 Years to 80 Years
• Sex: Male
• Sponsor: Nagoya University (Academic / other)
• Locations: 5 sites (Bunkyō City and 4 other locations)
• Trial ID: NCT06862596 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to assess the efficacy and safety of mexiletine hydrochloride in patients diagnosed with spinal and bulbar muscular atrophy (SBMA). Participants will receive either mexiletine or a placebo daily for three months, with evaluations conducted at the hospital every four weeks. The primary outcome measure is the improvement in the ALSFRS-R score, which assesses functional abilities in patients with SBMA.

Who should consider this trial

Good fit: Ideal candidates include male patients aged 18 to 80 with a confirmed diagnosis of SBMA and specific genetic markers.

Not a fit: Patients with a CAG repeat count below 38 or those with severe disease progression may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could improve muscle function and quality of life for patients with spinal and bulbar muscular atrophy.

How similar studies have performed: While there have been studies on treatments for SBMA, the specific use of mexiletine hydrochloride in this context is novel and has not been extensively tested.

Eligibility criteria

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

Where this trial is running

Bunkyō City and 4 other locations

• 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)

Study contacts

• Principal investigator: Masahisa Katsuno, PhD, MD — Nagoya University Hospital
• Study coordinator: Masahisa Katsuno, PhD, MD

Conditions Spinal and Bulbar Muscular Atrophy