A Phase 3 interventional study of Mexiletine and placebo in Non-dystrophic Myotonias, Paramyotonia Congenita and Myotonia Congenita, sponsored by Assistance Publique - Hôpitaux de Paris. Completed at 1 site in France. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2015-01-13.
Sponsored by Assistance Publique - Hôpitaux de Paris · Phase 3, Interventional, and Treatment
Treatment strategies in non-dystrophic myotonias are based on selective case reports, clinical experience and theoretical benefit. Presently, the most promising antimyotonic medication is mexiletine (MEX) but its manufacturing was stopped. The proposed randomized, double-blind, placebo-controlled, crossover trial is designed to:
A. Specific aims
Treatment strategies in non-dystrophic myotonias are based on selective case reports, clinical experience and theoretical benefit. Presently, the most promising antimyotonic medication is mexiletine (MEX) but its manufacturing was stopped. The proposed randomized, double-blind, placebo-controlled, crossover with wash-out trial is designed to:
B. Research design Because of their differing phenotypes, 12 Paramyotonia Congenita and 12 Myotonia Congenita subjects will be enrolled in a stratified trial
C. Outcome variables
secondary outcome measures:
of efficacy:
of safety:
D. Perspectives
It is anticipated that the trial will:
25 studies on the registry are indexed under Myotonia; 8 are open to participants now.
This study's enrollment of 24 is below the median of 35 across 17 interventional studies indexed under Myotonia.
Browse Myotonia studies →Assistance Publique - Hôpitaux de Paris is the lead sponsor of 3,505 studies on the registry; 1,006 are open to participants now.
Counted across the registry records on this site, refreshed daily.
The severity will be evaluated on:
Thus, patients who experience myotonic symptoms severe enough to justify treatment are those with myotonia that involves at least two segments and that have an impact on at least 3 daily activities.
Exclusion criteria :
Mexiletine / Placebo
Drug: Mexiletine · Drug: placebo
Placebo / Mexiletine
Drug: Mexiletine · Drug: placebo
* Blisters of 10 capsules of 200 mg mexiletine hydrochloride. * Patients will receive gradual dose of the treatment as it would be done in clinical practice. * Mexiletine will be started at 200 mg / day (1 capsule to be taken at the beginning of the meal) and will be increased by 200mg every 3 days to reach a maximum of 600mg / day in 3 taken in 1 week. * The duration of each treatment period is 18 days minimum (maximum 22 days).
score of stiffness severity on a self-assessment scale (100 mm VAS)
Time frame: 18 days
standardized EMG measures after repetitive short exercise test at cold and long exercise test
Time frame: 18 days
chair test: time needed to stand up from a chair, walk around it and sit down again
Time frame: 18 days
severity and disability scale of myotonia to be validated
Time frame: 18 days
quality of life scale (INQOL)
Time frame: 18 days
CGI efficacy (Clinical Global Impression- Efficacy index)
Time frame: 18 days
This study is completed, as verified in Jan 2015. You cannot join it, but the record below documents what was studied.
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Myotonia Congenita
Assistance Publique - Hôpitaux de Paris