Evaluating mexiletine hydrochloride for spinal and bulbar muscular atrophy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• 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
• Email: katsuno.masahisa.i1@f.mail.nagoya-u.ac.jp
• Phone: +81527442389

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Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.