NCT07154654

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

Prospective, Long Term, Observational Study (Patient Registry) of Paediatric Myotonic Disorders From Birth to Less Than Six Years of Age Who Are Treated With Mexiletine (PEGASUS Study).

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 10 (estimated)
• Sponsor: Lupin Ltd. · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: —

Summary

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.

Detailed description

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.

Conditions

• Myotonic Disorders

Interventions

Type	Name	Description
DRUG	Mexiletine	Non interventional

Timeline

• Start date: 2025-09-30
• Primary completion: 2027-11-22
• Completion: 2028-02-22
• First posted: 2025-09-04
• Last updated: 2025-09-23

Locations

1 site across 1 country: France