A Phase II Randomised, Double-Blind, Placebo Controlled, Cross-Over Study to Investigate the Efficacy of Mexiletine in Patients with Non-Dystrophic Myotonia. - Mexiletine in Non-Dystrophic Myotonia

Phase 1

• Conditions: Non-Dystrophic Myotonia (NDM). Non-dystrophic myotonias are a group of rare neuromuscular disorders that cause episodes of muscle stiffness (known as myotonia) and paralysis. Predominantly the muscles of the face, hands and legs are affected. In addition to these episodes a permanent and debilitating muscle weakness can develop. The optimal treatment for these disorders is unknown.; MedDRA version: 9.1 Level: HLGT Classification code 10029317 Term: Neuromuscular disorders

• Registration Number: EUCTR2009-011184-36-GB
• Lead Sponsor: niversity College London-Joint UCLH/UCL Biomedical (R&D) Unit

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2009-05-29
• Last Update Posted: 25 November 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 60

Inclusion Criteria

1.Age 18 years or older
2.Clinical symptoms or signs suggestive of myotonic disorders
3.Presence of myotonic potentials on electromyography (EMG)
4. Participation in the Non-Dystrophic Myotonia Natural History study; or a new patient with genetically confirmed NDM; or a new patient with a first degree relative (parent, sibling or child) with genetically confirmed NDM; or a new patient with clinical features of NDM in whom the mutation has not been localized, who areis DM1 and DM2 negative.
5.Patients on a stable dose of the following medications for 30 days prior to enrollment. Medications are:
a.fibrate acid derivatives
b.hydroxymethylglutaryl CoA reductase inhibitors
6. Practising an acceptable method of birth control for the duration of the trial, if a women of child bearing potential.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1.Inability or unwillingness to provide informed consent
2.Other neurological conditions that might affect the assessment of the study measurements
3.Genetic confirme DM1 (CTG>100 repeats) or DM2, if patient does not have genetically confirmed NDM.
4.Patients with existing cardiac conduction defects, evidence on EKG including but not limited to the following conditions: malignant arrhythmia or cardiac conduction disturbances (such as second degree AV block, third degree AV block, or prolonged QT interval >500ms or QRS duration >150 msec).
5.Current use of the following medication for a cardiac disorder: flecainide acetate, encainide, disopyramide, procainamide, quinine, propafenone or mexiletine.
6.Women who are pregnant or lactating
7. Patients currently on medications for myotonia such as phenytoin and flecainide acetate within 5 days of enrollment, carbamazepine and mexiletine within 3 days of enrollment, or acetazolamide, propafenone, procainamide, disopyramide, quinidine and encainide within 2 days of enrollment.
8. Patients with an existing permanent pacemaker.
9. Patients with renal or hepatic disease, heart failure, or seizures disorders.
10. Patients on medications that produce myotonia. This includes one or more of the following:
a. chloroquine
b. colchicines

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified