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A Multicenter, Randomized, Placebo-controlled, Double-blind Clinical Trial of Mexiletine Hydrochloride to Improve Motor Function in Spinal and Bulbar Muscular Atrophy
Led by Masahisa Katsuno · Updated on 2025-04-16
68
Participants Needed
5
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the effects of mexiletine hydrochloride in men with spinal and bulbar muscular atrophy (SBMA), a condition that causes muscle weakness. This clinical trial aims to determine if mexiletine hydrochloride can improve motor function as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) score. The study is designed as a randomized, placebo-controlled, double-blind trial to assess both the efficacy and safety of the drug. Participants will be randomly assigned to receive either mexiletine hydrochloride or a placebo. The active treatment involves taking 300 mg of mexiletine hydrochloride orally, divided into three doses after meals each day for 12 weeks. The placebo group will follow the same schedule with an inactive substance. Hospital visits for evaluation will occur every four weeks during the treatment period. During the study, participants will undergo regular assessments including ALSFRS-R scoring at 4 weeks and up to 12 weeks, as well as other tests such as grip strength, tongue pressure, timed walking tests, and respiratory function tests measuring forced vital capacity and peak expiratory flow. These evaluations will help measure motor function and respiratory health. Safety and adherence will be closely monitored throughout the 3-month treatment period.
CONDITIONS
Brief Title
Clinical Trial of Mexiletine Hydrochloride for Spinal and Bulbar Muscular Atrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male patients with a CAG repeat count of 38 or more for the androgen receptor gene confirmed by genetic testing
- Diagnosed with spinal and bulbar muscular atrophy showing muscle weakness from lower motor neuron lesion
- ALS Functional Rating Scale-Revised (ALSFRS-R) score between 24 and 42 at screening
- Aged 18 years or older and younger than 80 years at the time of consent
- Able to provide voluntary written consent or have a legal representative provide consent if the patient is unable
You will not qualify if you...
- Participation in another clinical trial within 12 weeks prior to enrollment
- History of hypersensitivity to any component of mexiletine hydrochloride
- Presence of conduction disturbances like second- or third-degree atrioventricular block without pacemaker or left bundle branch block
- Brugada-type ECG
- Severe heart failure or heart disease such as myocardial infarction, valvular disease, or cardiomyopathy
- Sinus bradycardia below 50 beats per minute
- Systolic blood pressure 90 mmHg or less
- Serum potassium level below 3.5 mmol/L
- Use of antiarrhythmic drugs, antiepileptic drugs affecting sodium channels, theophylline, or narcotics
- Use of mexiletine within 1 month prior to enrollment or for symptom improvement expectations
- Serious complications
- Unable to agree to use contraception during the study period
- Other conditions deemed inappropriate by the investigator or subinvestigator
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - 12 weeks
Participants receive Mexiletine hydrochloride or placebo orally three times a day after meals.
Visits during the 12-week treatment period (visit frequency not specified)
Trial Site Locations
Total: 5 locations
1
Tokyo University Hospital
Bunkyō City, Japan
Actively Recruiting
2
Chiba University Hospital
Chiba, Japan
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3
Hokkaido University Hospital
Sapporo, Japan
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4
Jichi Medical University Hospital
Shimotsuke, Japan
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5
Osaka University Hospital
Suita, Japan
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Research Team
M
Masahisa Katsuno, PhD, MD
S
Shinobu Shimizu, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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