Clinical Trial of Mexiletine Hydrochloride for Spinal and Bulbar Muscular Atrophy

Recruitment Status
RECRUITING
(See Contacts and Locations)Verified February 2025 by Masahisa Katsuno
Sponsor
Masahisa Katsuno
Information Provided by (Responsible Party)
Masahisa Katsuno
Clinicaltrials.gov Identifier
NCT06862596
Other Study ID Numbers:
CAMCR-026
First Submitted
February 25, 2025
First Posted
March 5, 2025
Last Update Posted
April 15, 2025
Last Verified
February 2025

ClinicalTrials.gov processed this data on April 2025. Link to the current ClinicalTrials.gov record .

History of Changes

Study Details

Study Description

Condition or DiseaseIntervention/Treatment
Spinal and Bulbar Muscular Atrophy
Drug: Mexiletine hydrochlorideOther: Placebo

Study Design

Study TypeInterventional
Actual Enrollment68 participants
Design AllocationRandomized
Interventional ModelParallel Assignment
MaskingQuadruple
Primary PurposeTreatment
Official TitleA 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 Start DateFebruary 27, 2025
Actual Primary Completion Date1yr 7mos from now
Actual Study Completion Date1yr 7mos from now

Groups and Cohorts

Group/CohortIntervention/Treatment
Mexiletine group
Drug: Mexiletine hydrochloride
Mexiletine hydrochloride 300 mg is administered orally divided into three times a day after meals for 12 weeks.
Placebo group
Other: Placebo
Placebo is administered orally divided into three times a day after meals for 12 weeks.

Outcome Measures

Primary Outcome Measures
  1. Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score
    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.
Secondary Outcome Measures
  1. ALSFRS-R Score
    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 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.
  2. Spinal and Bulbar Muscular Atrophy Functional Rating Scale (SBMAFRS) score
    The SBMAFRS is a disease-specific motor function assessment scale for Spinal and Bulbar Muscular Atrophy (SBMA).The SBMAFRS is a comprehensive severity index comprising 14 items covering bulbar, upper limb, lower limb, and respiratory symptoms to evaluate the ADLs of patients. Each item is rated on a five-point scale from 0 (worse) to 4 (better), and a total score (miniimum 0point and maximum 56 point) is calculated.
  3. Grip strength
    Grip strength (kg) is set to evaluate the upper limb motor function of patients with SBMA.
  4. Tongue pressure
    Tongue pressure (kPa) is set to evaluate the bulbar function of patients with SBMA.
  5. Timed walk test (4.6 meters)
    The timed walk test (4.6 meters) is an evaluation index for muscle weakness and atrophy of the lower limbs that measures the walking time (in seconds).
  6. 6-minute walk test
    The 6-minute walk test is an evaluation index for lower limb muscle weakness and muscle atrophy that measures the walking distance (in meters).
  7. Respiratory function test (Forced Vital Capacity (FVC))
    FVC (actual value (L) and predicted rate (%) (%FVC)) is set to evaluate for respiratory function.
  8. Respiratory function test (Peak Expiratory Flow (PEF))
    PEF (actual value (L/sec) and predicted rate (%) (%PEF)) is set to evaluate for respiratory function.

Eligibility Criteria

Ages Eligible for Study(Adult, Older Adult)
Sexes Eligible for StudyMale
Accepts Healthy VolunteersNo
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

Contacts and Locations

Sponsors and CollaboratorsMasahisa Katsuno
Locations
Tokyo University Hospital | Bunkyō City , Japan, Chiba University Hospital | Chiba , Japan, Hokkaido University Hospital | Sapporo , Japan, Jichi Medical University Hospital | Shimotsuke , Japan, Osaka University Hospital | Suita , Japan,
Investigators
Principal Investigator: Masahisa Katsuno, PhD, MD, Nagoya University Hospital