NCT04624750

Open Label Study in Adolescents and Children With Myotonic Disorders

An Open-label, Non-Comparative Study to Evaluate the Steady-State Pharmacokinetics, Safety, and Efficacy of Mexiletine in Adolescents and Children With Myotonic Disorders

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Lupin Ltd. · Industry
• Sex: All
• Age: 6 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

This is an open-label, multi-centre, single arm, interventional study to describe the steady-state PK, safety, and efficacy of mexiletine in paediatric patients (6 to \<18 years of age) with myotonic disorders.

Detailed description

This is an open-label, multi-centre, single arm, interventional study to describe the steady-state PK, safety, and efficacy of mexiletine in paediatric patients (6 to \<18 years of age) with myotonic disorders. Patients who meet the eligibility criteria will be enrolled stepwise, sequentially in 2 cohorts by age groups. Cohort 1 - Adolescents aged 12 to \<18 years, will be enrolled first. If no safety concerns are observed (based on data evaluation by the Data Safety Monitoring Board \[DSMB\]), and the dose for the age group 6 to \<12 years is confirmed by PK model, enrolment for Cohort 2 will begin. Cohort 2 - Children aged 6 to \<12 years, will be enrolled. The overall treatment duration for each cohort will be approximately 56 days (8 weeks): a dose titration phase of 4 weeks and the maintenance phase of 4 weeks. The overall study duration would be approximately 22 months. Dose titration phase: In this phase, patients will receive mexiletine starting at an age appropriate dose (as evaluated by the investigator and based on body weight) at a frequency of once a day. Dose will be up-titrated every 14 days based on tolerability of mexiletine up to a maximum of three-times a day as assessed by investigator. Maintenance phase: During the maintenance phase, patients will continue to receive mexiletine at the best-tolerated dose from the titration phase for further 4 weeks. Following completion, all participants will be offered follow-up in PIP Study 7 (MEX-NM-303) (EudraCT: 2019-003758-97).

Conditions

• Myotonic Dystrophy

Interventions

Type	Name	Description
DRUG	Mexiletine	Patients will be enrolled sequentially into 2 cohorts. Cohort 1 - (patients aged 12 to \< 18 years): approximately 8 weeks - 4 weeks of dose titration period + 4 weeks of maintenance period. Cohort 2 - (patients aged 6 to \< 12 years,): approximately 8 weeks - 4 weeks of dose titration period + 4 weeks of maintenance period. Enrolment for Cohort 2 will begin after initial safety assessment of patients in Cohort 1 by the DSMB and no safety concerns are observed. The dose level for cohort 2 will be confirmed by PK modelling study.

Timeline

• Start date: 2021-09-03
• Primary completion: 2024-06-14
• Completion: 2024-08-08
• First posted: 2020-11-12
• Last updated: 2024-11-25

Locations

1 site across 1 country: France