A Phase 2/3 interventional study of Mexiletine hydrochloride and Placebo in Spinal and Bulbar Muscular Atrophy, sponsored by Masahisa Katsuno. Recruiting at 5 sites in Japan. Open to male participants aged 18 Years to 80 Years. Per ClinicalTrials.gov, last updated 2025-04-16.
Sponsored by Masahisa Katsuno · Phase 2/3, Interventional, and Treatment
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.
57 studies on the registry are indexed under Bulbo-Spinal Atrophy, X-Linked; 12 are open to participants now.
This study's planned enrollment of 68 is above the median of 42 across 42 interventional studies indexed under Bulbo-Spinal Atrophy, X-Linked.
Browse Bulbo-Spinal Atrophy, X-Linked studies →This is the only study on the registry with Masahisa Katsuno as lead sponsor.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: Mexiletine hydrochloride
Other: Placebo
Mexiletine hydrochloride 300 mg is administered orally divided into three times a day after meals for 12 weeks.
Placebo is administered orally divided into three times a day after meals for 12 weeks.
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.
Time frame: at 4weeks
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.
Time frame: up to 12 weeks
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.
Time frame: up to 12 weeks
Grip strength
Grip strength (kg) is set to evaluate the upper limb motor function of patients with SBMA.
Time frame: up to 12 weeks
Tongue pressure
Tongue pressure (kPa) is set to evaluate the bulbar function of patients with SBMA.
Time frame: up to 12 weeks
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).
Time frame: up to 12 weeks
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).
Time frame: up to 12 weeks
Respiratory function test (Forced Vital Capacity (FVC))
FVC (actual value (L) and predicted rate (%) (%FVC)) is set to evaluate for respiratory function.
Time frame: up to 12 weeks
Respiratory function test (Peak Expiratory Flow (PEF))
PEF (actual value (L/sec) and predicted rate (%) (%PEF)) is set to evaluate for respiratory function.
Time frame: up to 12 weeks
Plan to share: Yes — If the principal investigator, clinical trial office, main stakeholder conclude that secondary use of individual data obtained in this clinical trial is beneficial for additional analysis, the secondary use of data excluding personal information will be acceptable after publication of results.
No publications or documents are linked to this record.
Started Feb 2025 (actual). Primary completion is estimated for Dec 2027, 34 months after the start.
How these figures are worked out, and which studies are left out of them.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Mexiletine
Bulbo-Spinal Atrophy, X-Linked →