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Open-label Extension Study in Paediatric Patients Who Have Completed the MEX-NM-301 Study.

NCT04622553 · Status: ACTIVE_NOT_RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 14

Last updated 2025-07-25

No results posted yet for this study

Summary

Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Mexiletine in Paediatric Patients with Myotonic Disorders Who Have Completed the MEX-NM-301 study.

Conditions

  • Myotonic Disorders

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 pharmacokinetics (PK), safety and efficacy are confirmed in this population, of patients in Cohort 1

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
2021-11-05
Primary Completion
2026-01-26
Completion
2026-03-12

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 NCT04622553 on ClinicalTrials.gov