Spinal and Bulbar Muscular AtrophyPhase 2/3Med-SBMANCT06862596
Clinical Trial of Mexiletine Hydrochloride for Spinal and Bulbar Muscular Atrophy
A Phase 2/3 study testing Mexiletine hydrochloride in people with Spinal and Bulbar Muscular Atrophy, run by Masahisa Katsuno at 5 sites in Japan.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 80 years, men only
- Conditions the study is for
- Spinal and Bulbar Muscular Atrophy
- What is being tested?
- Mexiletine hydrochloride
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 68 participants
- Where
- 5 sitesJapan
- Study dates
- 28 Feb 2025to 31 Dec 2027
- Phase
- Phase 2/3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
- Patients with muscle weakness (limb weakness and atrophy, or bulbar palsy) due to lower motor neuron lesion
- Patients with a total ALSFRS-R score of ≥ 24 and ≤ 42 at screening
- Patients who are at least 18 years old and less than 80 years old at the time of consent
- 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.)
Who cannot join
- Patients who have participated or are participating in a clinical trial within 12 weeks prior to enrollment
- Patients with a history of hypersensitivity to any component of this drug product
- Patients with a conduction disturbance (such as second- or third-degree atrioventricular block without a pacemaker, or left bundle branch block)
- Patients with Brugada-type ECG
- Patients with severe heart failure or heart disease (myocardial infarction, valvular disease, cardiomyopathy, etc.)
- Patients with sinus bradycardia (<50 beats/minute)
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- Patients with systolic blood pressure of 90 mmHg or less
- Patients with serum potassium level less than 3.5 mmol/L
- Patients on antiarrhythmic drugs
- Patients on antiepileptic drugs that affect to sodium channels
- Patients on theophylline
- Patients on narcotics
- Patients who used Mexiletine within 1 month prior to enrollment or used Mexiletine for expectations of improvement in symptoms of SBMA
- Patients with serious complications
- Patients who cannot agree to use contraception during the study period
- Other Patients deemed inappropriate by the investigator or subinvestigator
Trial locations and contacts
5 sites in 1 country. Contact the study team or a site near you to ask about taking part.
5 sites in 1 country
Japan5 sites, 5 recruiting
- Recruiting
Tokyo University Hospital
Bunkyō City
Tomotaka Yamamoto, PhD, MD, +81338155411, yamatyama@gmail.com
- Recruiting
Chiba University Hospital
Chiba
Kazumoto Shibuya, PhD, MD, +81432227171, kazumoto@net.email.ne.jp
- Recruiting
Hokkaido University Hospital
Sapporo
Ichiro Yabe, PhD, MD, +81117161161, yabe@med.hokudai.ac.jp
- Recruiting
Jichi Medical University Hospital
Shimotsuke
Mitsuya Morita, PhD, MD, +81285442111, morita@jichi.ac.jp
- Recruiting
Osaka University Hospital
Suita
Seiichi Nagano, PhD, MD, +81668795111, nagano@neurol.med.osaka-u.ac.jp
About this study
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
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
- Being tested
- Mexiletine hydrochloride, Placebo
- Medical subject headings
- Bulbo-Spinal Atrophy, X-Linked
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mexiletine hydrochlorideDrug
Mexiletine hydrochloride 300 mg is administered orally divided into three times a day after meals for 12 weeks.
PlaceboOther
Placebo is administered orally divided into three times a day after meals for 12 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Mexiletine group | Active comparator | Not described Receives: Drug: Mexiletine hydrochloride |
| Placebo group | Placebo comparator | Not described Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: at 4weeks
Secondary outcomes (8)
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.
Measured: 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.
Measured: up to 12 weeks
Grip strength
Grip strength (kg) is set to evaluate the upper limb motor function of patients with SBMA.
Measured: up to 12 weeks
Tongue pressure
Tongue pressure (kPa) is set to evaluate the bulbar function of patients with SBMA.
Measured: 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).
Measured: 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).
Measured: 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.
Measured: 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.
Measured: up to 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Mar 2025
- Study start28 Feb 2025
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinal and Bulbar Muscular AtrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 68 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Masahisa Katsuno
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Masahisa Katsuno, Nagoya University
- Investigators
- Masahisa Katsuno, PhD, MD, Principal investigator, Nagoya University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06862596
- Sponsor's protocol ID
- CAMCR-026
- Other IDs
- jRCT2041240174
- Acronym
- Med-SBMA
- Status last verified
- Mar 2025
- First submitted
- 26 Feb 2025
- Last updated
- 16 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- 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.
Study at a glance
From the registry record, last updated by the sponsor on 16 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06862596
- Acronym / sponsor ID
- Med-SBMA / CAMCR-026
- Conditions
- Spinal and Bulbar Muscular Atrophy
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 68 participants (estimated)
- Lead sponsor
- Masahisa KatsunoUniversity, hospital or other
- Study start
- 28 Feb 2025 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 6 Mar 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Mexiletine group | Active comparator | Drug: Mexiletine hydrochloride |
| Placebo group | Placebo comparator | Other: Placebo |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
- Patients with muscle weakness (limb weakness and atrophy, or bulbar palsy) due to lower motor neuron lesion
- Patients with a total ALSFRS-R score of ≥ 24 and ≤ 42 at screening
- Patients who are at least 18 years old and less than 80 years old at the time of consent
- 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.)
Key exclusion criteria (16 listed)
- Patients who have participated or are participating in a clinical trial within 12 weeks prior to enrollment
- Patients with a history of hypersensitivity to any component of this drug product
- Patients with a conduction disturbance (such as second- or third-degree atrioventricular block without a pacemaker, or left bundle branch block)
- Patients with Brugada-type ECG
- Patients with severe heart failure or heart disease (myocardial infarction, valvular disease, cardiomyopathy, etc.)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Mexiletine hydrochlorideMexiletine hydrochloride 300 mg is administered orally divided into three times a day after meals for 12 weeks. | Drug | Mexiletine group |
| PlaceboPlacebo is administered orally divided into three times a day after meals for 12 weeks. | Other | Placebo group |
Primary outcome
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score
Time frame: at 4weeks
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.
8 secondary outcomes
ALSFRS-R Score
Time frame: up to 12 weeks
Spinal and Bulbar Muscular Atrophy Functional Rating Scale (SBMAFRS) score
Time frame: up to 12 weeks
Grip strength
Time frame: up to 12 weeks
Tongue pressure
Time frame: up to 12 weeks
Timed walk test (4.6 meters)
Time frame: up to 12 weeks
6-minute walk test
Time frame: up to 12 weeks
Respiratory function test (Forced Vital Capacity (FVC))
Time frame: up to 12 weeks
Respiratory function test (Peak Expiratory Flow (PEF))
Time frame: up to 12 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Apr 2025.
- Individual participant dataSharing planned: 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Masahisa Katsuno, PhD, MD principal investigator
Nagoya University Hospital
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Mexiletine hydrochloride in people with Spinal and Bulbar Muscular Atrophy, run by Masahisa Katsuno at 5 sites in Japan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Masahisa Katsuno, PhD, MD (+81527442389, katsuno.masahisa.i1@f.mail.nagoya-u.ac.jp), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score (time frame: at 4weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Apr 2025.