Mexiletine in SBMA

JRCT ID: jRCTs041180106

Registered date:18/03/2019

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: Spinal and bulbar muscular atrophy
• Date of first enrollment: 17/08/2017
• Target sample size: 20
• Countries of recruitment: Not applicable,Japan
• Study type: Interventional
• Intervention(s): Medicine

Outcome(s)

• Primary Outcome: Longitudinal change in the difference in distal latencies of the ulnar nerve at the wrist in the dominant hand under room temperature and cold exposure conditions after 4 weeks

Secondary Outcome

(1)Quantitative muscle strength of upper limbs (grip power, pinch power, ten seconds test)
(2)Peripheral nerve conduction studies (distal latency, compound muscle action potential, motor nerve conduction velocity, sensory nerve conduction velocity, area, duration time, Harvey-Masland test, skin temperature)
(3)Motor function test (tongue pressure, timed walk test, modified QMG score)
(4)Pulmonary function test (vital capacity, forced vital capacity, forced expiratory volume, peak expiratory flow and V50/V25, flow-volume curve)
(5)ADL scales (ALSFRS-R, SBMAFRS)
(6)QOL scales (36-Item Short Form Health Survey, The Individualised Neuromuscular Quality of Life questionnaire, Interactive Voice Response System)
(7)Lean mass evaluation by Dual-energy X-ray Absorption
(8)Blood examinations (complete blood count, electrolytes, creatinine, creatine kinase, aldolase, lactate dehydrogenase, aspartate aminotransferase, alanine aminotransferase, and testosterone)

Key inclusion & exclusion criteria

• Age minimum: >= 20age old
• Age maximum: < 80age old
• Gender: Male

Include 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

Exclude 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

Related Information

• Primary Sponsor: Katsuno Masahisa
• Source(s) of Monetary Support: Nagoya University Hospital Funding for Clinical Research
• Secondary ID(s): UMIN000026150