Open Label Study in Adolescents and Children With Myotonic Disorders

Myotonic Dystrophy Clinical Trial

Official title: 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 Recruiting

Clinical Trial 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.

Clinical Trial 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). ;

Related Conditions & MeSH terms

• Myotonic Disorders
• Myotonic Dystrophy

• NCT number: NCT04624750
• Study type: Interventional
• Source: Lupin Ltd.
• Contact: Nikki Adetoro
• Phone: 443-447-4534
• Email: NikkiAdetoro@lupin.com
• Status: Recruiting
• Phase: Phase 3
• Start date: September 3, 2021
• Completion date: August 8, 2024