Clinical Trial of Mexiletine Hydrochloride for Spinal and Bulbar Muscular Atrophy

NCT ID: NCT06862596

Last Updated: 2025-04-16

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 68 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-02-28
• Study Completion Date: 2027-12-31

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.

Conditions

Spinal and Bulbar Muscular Atrophy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Mexiletine group

Group Type: ACTIVE_COMPARATOR

Mexiletine hydrochloride

Intervention Type: DRUG

Mexiletine hydrochloride 300 mg is administered orally divided into three times a day after meals for 12 weeks.

Placebo group

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: OTHER

Placebo is administered orally divided into three times a day after meals for 12 weeks.

Interventions

Mexiletine hydrochloride

Mexiletine hydrochloride 300 mg is administered orally divided into three times a day after meals for 12 weeks.

Intervention Type: DRUG

Placebo

Placebo is administered orally divided into three times a day after meals for 12 weeks.

Intervention Type: OTHER

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Masahisa Katsuno

OTHER

Sponsor Role: lead

Responsible Party

Masahisa Katsuno

Professor

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Masahisa Katsuno, PhD, MD

Role: PRINCIPAL_INVESTIGATOR

Nagoya University Hospital

Locations

Tokyo University Hospital

Bunkyō City, , Japan

Site Status: RECRUITING

Chiba University Hospital

Chiba, , Japan

Site Status: RECRUITING

Hokkaido University Hospital

Sapporo, , Japan

Site Status: RECRUITING

Jichi Medical University Hospital

Shimotsuke, , Japan

Site Status: RECRUITING

Osaka University Hospital

Suita, , Japan

Site Status: RECRUITING

Countries

Japan

Central Contacts

Masahisa Katsuno, PhD, MD

Role: CONTACT

katsuno.masahisa.i1@f.mail.nagoya-u.ac.jp

+81527442389

Shinobu Shimizu, PhD

Role: CONTACT

shimizu.shinobu.w3@f.mail.nagoya-u.ac.jp

+81527442942

Facility Contacts

Tomotaka Yamamoto, PhD, MD

Role: primary

yamatyama@gmail.com

+81338155411

Kazumoto Shibuya, PhD, MD

Role: primary

kazumoto@net.email.ne.jp

+81432227171

Ichiro Yabe, PhD, MD

Role: primary

yabe@med.hokudai.ac.jp

+81117161161

Mitsuya Morita, PhD, MD

Role: primary

morita@jichi.ac.jp

+81285442111

Seiichi Nagano, PhD, MD

Role: primary

nagano@neurol.med.osaka-u.ac.jp

+81668795111

Other Identifiers

jRCT2041240174

Identifier Type: OTHER

Identifier Source: secondary_id

CAMCR-026

Identifier Type: -

Identifier Source: org_study_id