Prospective, Long Term, Observational Study (Patient Registry) of Paediatric Myotonic Disorders
ConditionMyotonic Disorders
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeUp to 6
SponsorLupin Ltd.
About this trial
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.
Eligibility criteria
Qualifiers
Male or female patients from birth to less than 6 years
A genetically confirmed diagnosis of NDM or DM (DM1or DM2), as per the treating clinician.
Presence of clinical symptoms of myotonia (hand grip myotonia, myotonia in the leg muscles, any other myotonia symptoms) to be confirmed by the treating clinician.
Patients already receiving mexiletine treatment or who are clinically considered for mexiletine treatment as per the treating physician judgement.
Disqualifiers
Any contraindication to mexiletine as listed in the Namuscla Summary of Product Characteristics (SmPC) (NaMuscla SmPC, 2023)
Hypersensitivity to the active substance, or to any of the excipients
Hypersensitivity to any local anaesthetic
Ventricular tachyarrhythmia
Trial design
Treatments tested in this trial
- Mexiletine
Treatment groups
10 Participants
are divided into 2 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov