Updated May 2026 · ClinicalTrials.gov

RECRUITINGPhase 2 / Phase 3INTERVENTIONAL

Clinical Trial of Mexiletine Hydrochloride for Spinal and Bulbar Muscular Atrophy

Q: Who can participate in NCT06862596?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

Q: How do I contact the trial site for NCT06862596?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Q: Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Q: Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

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

Clinical Trial of Mexiletine Hydrochloride for Spinal and Bulbar Muscular Atrophy (NCT06862596) is a Phase 2 / Phase 3 interventional studying Spinal and Bulbar Muscular Atrophy, sponsored by Masahisa Katsuno. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

About This Trial

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.

What Stage of Research Is This?

Phase 2 trials evaluate whether a treatment actually works against Spinal and Bulbar Muscular Atrophy and continue monitoring side effects. Phase 2 enrolls larger groups (typically 100–300 patients) and produces the first real efficacy signal. A successful Phase 2 readout is what unlocks the much larger Phase 3 confirmatory trials needed for FDA approval.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

Target enrollment of 68 participants puts this in the typical range for a Phase 2-style efficacy study or a moderate Phase 3 trial in a focused Spinal and Bulbar Muscular Atrophy subpopulation. At this scale, the study has enough statistical power to detect a clear treatment effect but is not the largest cohort in the field.

Who May Be Eligible (Plain English)

Who May Qualify: 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.) Who Should NOT Join This Trial: 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 ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language

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

Treatments Being Tested

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.

Locations (5)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

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

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT06862596), the sponsor (Masahisa Katsuno), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT06862596 clinical trial studying?

Who can participate in NCT06862596?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT06862596?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT06862596. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT06862596. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.