Actively Recruiting
Prospective, Long Term, Observational Study (Patient Registry) of Paediatric Myotonic Disorders
Led by Lupin Ltd. · Updated on 2025-09-23
10
Participants Needed
1
Research Sites
125 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Prospective, Long Term, Observational Study (Patient Registry) of Paediatric Myotonic Disorders
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female patients from birth to less than 6 years
- Genetically confirmed diagnosis of non-dystrophic myotonia or dystrophic myotonia (DM1 or DM2) as diagnosed by the treating clinician
- Presence of clinical myotonia symptoms confirmed by the treating clinician
- Currently receiving or considered for mexiletine treatment per treating physician
- No history of significant cardiac abnormalities as assessed by cardiologist or treating physician
- No known history or signs of significant liver disorder
- No clinically relevant abnormal laboratory results in haematology, biochemistry, or urinalysis that affect study objectives
- Parent or legal guardian able to provide consent and perform study-related activities
You will not qualify if you...
- Contraindications to mexiletine as per Namuscla Summary of Product Characteristics including hypersensitivity, ventricular tachyarrhythmia, heart block, prolonged QT interval, myocardial infarction, symptomatic coronary artery disease, heart failure with ejection fraction <50%, atrial tachyarrhythmia, sinus node dysfunction, and co-administration with certain medications
- Other neurological or psychiatric conditions affecting study assessments
- Clinically significant illness or findings affecting study participation
- Use or planned use of strong CYP2D6 or CYP1A2 inducers or inhibitors during study
- Concurrent illnesses or medications affecting muscle function
- Seizure disorder or insulin-treated diabetes mellitus
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Centre Hospitalier Universitaire de Clermont-Ferrand
Clermont-Ferrand, France
Actively Recruiting
Research Team
N
Nikki Adetoro
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here