Clinical trial NCT06862596: Clinical Trial of Mexiletine Hydrochloride for Spinal and Bulbar Muscular Atrophy
Official title: 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
PHASE3
Conditions: Spinal and Bulbar Muscular Atrophy
Lead sponsor: Nagoya University
Sponsor class: OTHER
First posted: 2025-03-06
Enrollment: 68
Sex: MALE
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score
timeFrame: at 4weeks
Secondary outcomes:
measure: ALSFRS-R Score
timeFrame: up to 12 weeks
measure: Spinal and Bulbar Muscular Atrophy Functional Rating Scale (SBMAFRS) score
timeFrame: up to 12 weeks
measure: Grip strength
timeFrame: up to 12 weeks
measure: Tongue pressure
timeFrame: up to 12 weeks
measure: Timed walk test (4.6 meters)
timeFrame: up to 12 weeks
measure: 6-minute walk test
timeFrame: up to 12 weeks
measure: Respiratory function test (Forced Vital Capacity (FVC))
timeFrame: up to 12 weeks
measure: Respiratory function test (Peak Expiratory Flow (PEF))
timeFrame: up to 12 weeks
Other outcomes:
Locations:
city: Bunkyo
country: Japan
facility: Tokyo University Hospital
city: Chiba
country: Japan
facility: Chiba University Hospital
city: Sapporo
country: Japan
facility: Hokkaido University Hospital
city: Shimotsuke
country: Japan
facility: Jichi Medical University Hospital
city: Suita
country: Japan
facility: Osaka University Hospital
Study officials:
affiliation: Nagoya University Hospital
name: Masahisa Katsuno, PhD, MD
role: PRINCIPAL_INVESTIGATOR
References: