This is a prospective, open-label, multi-centre, single arm, registry study to collect standard relevant clinical and epidemiological data during routine medical evaluation and treatment in paediatric patients with myotonic disorders who are being treated with mexiletine therapy according to the physician.
This is a prospective, open-label, multi-centre, single arm, registry study to collect standard relevant clinical and epidemiological data during routine medical evaluation and treatment in paediatric patients with myotonic disorders who are being treated with mexiletine therapy according to the physician. Patients who meet the eligibility criteria will be enrolled in 2 cohorts by age groups although cohorts are not enrolled sequentially (cohort definition is to assure minimum requirements for meeting PIP agreements). Cohort 1 - Infants and children aged between 6 months to less than 6 years. Cohort 2 - Neonates and infants from birth to less than 6 months. The overall treatment duration follow-up for each cohort will be at least 2 years.
Study Type
OBSERVATIONAL
Enrollment
10
Non interventional
Centre Hospitalier Universitaire de Clermont-Ferrand
Clermont-Ferrand, France
RECRUITINGTo assess long-term safety by collection of SAEs, AEs, AESIs, changes in frequency, treatment interruptions
Number and frequency of adverse events (AEs), serious adverse events (SAEs) and AE leading to treatment discontinuation, throughout the study while on treatment with mexiletine; Incidence of adverse events of special interest (AESI), namely: Severe Cutaneous Adverse Reactions (SCARs) \& Cardiac arrhythmia. Increased frequency of seizure episodes in patients with epilepsy; Any changes in frequency, treatment interruptions of mexiletine
Time frame: Baseline to 24 months
To assess the suitability of mexiletine administration
To assess the suitability of mexiletine administration using available formulations including, where appropriate, capsules or opening and sprinkling contents in food or drink according to the child´s age (from birth to less than 6 years) and ability to swallow.
Time frame: Baseline to 24 months
To understand how mexiletine is used by clinicians
To understand how mexiletine is used by clinicians who have previously decided to use mexiletine for the treatment of myotonia in myotonic disorder patients, from birth to less than 6 years over a at least 2 years period, to establish risk:benefit conclusions.
Time frame: Baseline to 24 months
Description over time of outcomes of mexiletine use
Description over time of outcomes of mexiletine use including dosing, formulation used, method of administration, acceptability and palatability as per the data collected by the treating physician according to age and cohort.
Time frame: Baseline to 24 months
The treating physician with elaborate a brief text with risk-benefit conclusions
Description over time of any efficacy outcomes obtained from clinically indicated tests as per the treating physician typical clinical practice and according to patient's age and cohort.
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Time frame: Baseline to 24 months