Mexiletine in SBMA

Phase 2

• Conditions: Spinal and bulbar muscular atrophy; G121

• Registration Number: JPRN-jRCTs041180106
• Lead Sponsor: Katsuno Masahisa

Brief Summary

ALSFRS-R (general motor function) in patients with SBMA tended to improve with mexiletine. The quantitative motor function tests (tongue pressure, grip strength, 10-second test and timed walk test) for the ALSFRS-R components of bulbar, upper, and lower limb symptoms also tended to improve with mexiletine, suggesting that mexiletine may improve overall motor function in patients with SBMA. There were no serious adverse events in participants.

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Completion: 2018-06-14
• Study Start: 2019-03-18
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: Complete
• Sex: Male
• Target Recruitment: 20

Inclusion Criteria

1. Male patients with 38 or more CAG repeats in the AR gene
2. Patients with muscle weakness due to lower motor neuron loss (bulbar palsy or extremity palsy)
3. Patients with paresis like sudden and relevant loss of force that affect ADL of themselves when exposed to cold temperatures
4. Patients between 20 and 80 years of age at the time of agreement to participate
5. Patients who are capable of ambulatory hospital visits
6. Patients whose hepatic and renal functions meet the following criteria:
Serum AST, ALT: < 4.0 * Upper Limit of Institutional Reference Value
Serum creatinine: < 1.5 * Upper Limit of Institutional Reference Value
7. Patients providing written informed consent

Exclusion Criteria

1. Patients who have hypersensitivity to mexiletine hydrochloride
2. Patients with second degree atrioventricular block, third degree atrioventricular block, or left bundle branch block
3. Patients with a Brugada-type electrocardiogram pattern
4. Patients with a past history of myocardial infarction, valvular disease, or cardiomyopathy
5. Patients whose heart rate is 50/minute or less
6. Patients with severe liver or renal dysfunction
7. Patients with heart failure
8. Patients whose systolic blood pressure is 90 mmHg or less
9. Patients with ulnar neuropathy at the dominant hand
10. Patients whose serum potassium level is 3.5 mEq/l or less
11. Patients who take antiarrhythmic drugs
12. Patients who take antiepileptic drugs
13. Patient with neurological disorders other than SBMA
14. Patients whose HbA1c (NGSP) level is 6.5% or more
15. Patients who take hypoglycaemic drugs
16. Patients with alcohol addiction
17. Patients with drug addiction
18. Patients who take cimetidine, rifampicin, or theophylline
19. Patients with severe complications such as serious infectious disease, post-operation, terminal cancer, or debilitation
20. Patients whose grip power in the dominant hand is less than 10 kg
21. Patients who are judged to be inappropriate to participate in the trial for any other reasons

Study & Design

• Study Type: Interventional
• Study Design: Not specified