Prospective, Long Term, Observational Study (Patient Registry) of Paediatric Myotonic Disorders

Recruiting Observational Study
Myotonic Disorders
No Placebo Group Every participant receives an active treatment — no one gets a placebo. No Study Drug Researchers observe your health over time — no experimental treatment is given.
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At a Glance
Age
Up to 6
Sex
Any
Study type
Observational
Participants needed
10 (estimated)
Sponsor
Lupin Ltd. · Industry
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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.

Trial Locations
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Eligibility Criteria
Inclusion Criteria: 1. Male or female patients from birth to less than 6 years 2. A genetically confirmed diagnosis of NDM or DM (DM1or DM2), as per the treating clinician. 3. Presence of clinical symptoms of myotonia (hand grip myotonia, myotonia in the leg muscles, any other myotonia symptoms) to…
Contacts

Nikki Adetoro

4434474534

nikkiadetoro@lupin.com

CONTACT