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Open Label Study in Adolescents and Children With Myotonic Disorders

NCT04624750 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2024-11-25

No results posted yet for this study

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.

Conditions

  • Myotonic Dystrophy

Interventions

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.

Sponsors & Collaborators

  • Lupin Ltd.

    lead INDUSTRY

Principal Investigators

  • Christine Barnérias, MD · Hopital universitaire Necker-Enfants Malades

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-09-03
Primary Completion
2024-06-14
Completion
2024-08-08

Countries

  • France

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04624750 on ClinicalTrials.gov