NCT06862596

Clinical Trial of Mexiletine Hydrochloride for Spinal and Bulbar Muscular Atrophy

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

• Status: Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 68 (estimated)
• Sponsor: Masahisa Katsuno · Academic / Other
• Sex: Male
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

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

Interventions

Type	Name	Description
DRUG	Mexiletine hydrochloride	Mexiletine hydrochloride 300 mg is administered orally divided into three times a day after meals for 12 weeks.
OTHER	Placebo	Placebo is administered orally divided into three times a day after meals for 12 weeks.

Timeline

• Start date: 2025-02-28
• Primary completion: 2027-12-31
• Completion: 2027-12-31
• First posted: 2025-03-06
• Last updated: 2025-04-16

Locations

5 sites across 1 country: Japan